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‘Limb vs. Tail’ lecture articulates variations of axolotl regeneration during research development The Maine Campus – The Maine Campus

Axolotls are a part of the salamander family, which makes them one of very few animals in the world that are able to fully regenerate their limbs and tails when injured or lost. During a talk on Oct. 29, Dr. Prayag Murawala discussed the differences between cell regeneration of axolotl limbs and tails and what that means for the future of biological research.

If there were no regeneration, there would be no life. If everything regenerated, there would be no death, Richard J. Goss said in his 1969 work about regeneration. Dr. Murawala used this definition to start the lecture, along with showing the wide array of animals that are capable of regeneration, ranging from earthworms to starfish. Murawala defined the axolotl as the champion of regeneration because of its effective and quick acting abilities.

Regeneration occurs in two methods; the expansion of stem cells that already existed in the body part being regenerated and the dedifferentiation of a cell intended for another use into a cell intended for regeneration. In axolotls, the regeneration method depends on which part of the body you are looking at: on the primary body axis, like a tail, or on the secondary body axis, like limbs.

Murawalas teams research, which took place as a part of his post doctoral research, found that limb regeneration, like all regeneration in axolotls, requires the formation of blastema cells, a group of undifferentiated progenitors that carries the code for limb regeneration. Through a series of experiments and tracings, they were able to discover that uninjured limbs had no pre-existing blastema cells, meaning that the cells in the limbs differentiate to form those blastema. They also discovered that fibroblasts, a cell specialized in creating structural frames, are progenitors that create cells of multiple lineages.

The research team found that tail regeneration, however, takes on the other method of regeneration. Through the same tracing methods they used on axolotl limbs, they discovered that through the process of somitogenesis, an evolutionary process that all vertebrates have undergone, progenitors are formed from preexisting stem cells. They then differentiate into the lineages needed to regenerate a fully functioning tail.

The main differences between the two regenerative processes are the methods and the amount of heterogeneity of the cells post regeneration. In axolotl limbs, the new connective tissue cells formed post-regeneration homogenize a lot more than those post-regeneration in tails, and it takes a good amount of time for those connective tissue cells to be fully heterogeneous again, if ever.

In humans, we are rarely able to fully regenerate lost appendages, like fingers and toes, let alone limbs. Being able to regenerate something on the primary body axis, like a tail, is unique to salamanders and other lizards, which is what makes this research so groundbreaking.

Murawalas research team hopes to tackle the question of how different the cells found in axolotl limbs and tails really are in further research. For more information about Murawalas team and where his research has taken him, you can visit https://calendar.umaine.edu/event/community-engagement-to-enhance-research-in-maine-2/.

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'Limb vs. Tail' lecture articulates variations of axolotl regeneration during research development The Maine Campus - The Maine Campus

Clade Therapeutics Raises $87 Million Series A Financing to – GlobeNewswire

CAMBRIDGE, Mass., Nov. 03, 2021 (GLOBE NEWSWIRE) -- Clade Therapeutics, a biopharmaceutical company focused on discovering and delivering scalable, off-the-shelf, next-generation stem cell-based medicines, today announced it has secured an $87 million Series A financing led by Syncona Ltd. with participation from LifeSci Venture Partners, Emerson Collective and Bristol Myers Squibb. Proceeds from this financing will support the development of the Companys proprietary platform, which enables the immune cloaking of induced pluripotent stem cells (iPSCs) and the differentiation of cloaked stem cells into therapeutic cells.

We have reached an era in medicine where insights across genetic engineering, regenerative medicine and immunology have enabled a revolution of cell medicines, said Chad Cowan, Ph.D., Chief Executive Officer of Clade Therapeutics. Clade was founded to overcome the clinical limitations of current cell therapies by addressing durability, patient compatibility, reproducibility and scalability to deliver on the transformative potential of this increasingly important therapeutic modality.

Jim Glasheen, Ph.D., President and Chief Business Officer of Clade Therapeutics, said, We feel very fortunate to partner with a world-class group of investors. The syndicates combination of industry insight, technical expertise, entrepreneurial zeal, and focus on patient impact brings incredible value to the Company.

Martin Murphy, Ph.D., Chief Executive Officer of Syncona Ltd., said, Clades inherent focus on developing therapies derived from a single engineered cell source has the potential to shift the paradigm of cell medicine with unprecedented scalability and standardization. We are thrilled to support Clades aggressive development of broadly accessible, off-the-shelf products with consistent pharmaceutical criteria to expand the reach of cell therapies across patients and indications.

Ryan Cinalli, Ph.D., Chief Scientific Officer of LifeSci Venture Partners, said, Clade has assembled a world-class team of scientific pioneers whose foundational discoveries are integral to the Companys immune cloaking technology platform. We are confident that Clades leadership will innovate the next generation of cell therapies that harness cloaking technology to overcome the immune barriers that have limited durability and redosing in the field.

Neil White, Investment Manager of Emerson Collective, said, The unparalleled expertise and novel approach to generating stem cell-derived adult T, NK and B cells positions Clade as leaders in developing widely accessible cell medicines. With differentiation and cloaking technologies in place, this funding round will accelerate the development of Clades immune cell-focused, cancer therapeutics.

About Clade Therapeutics Clade Therapeutics mission is to discover and deliver next generation cell medicines to improve the lives of patients in need. Our platform technology cloaks human pluripotent stem cells and their adult derivatives enabling the development of immune compatible cell transplantation therapies. Led by a world-class team of company builders and scientific innovators with an unparalleled expertise in generating stem cell-derived adult T, NK and B cells, Clade promises to become a leading innovator in developing widely accessible cell medicines. The company is initially focused on harnessing the potential of cloaked immune cells for cancer treatment. For further information, please visit the company's website athttps://www.cladetx.com/.

About SynconaSyncona's purpose is to invest to extend and enhance human life. We do this by founding and building a portfolio of global leaders in life science to deliver transformational treatments to patients in areas of high unmet need.

Our strategy is to found, build and fund companies around exceptional science to create a diversified portfolio of 15-20 globally leading healthcare businesses for the benefit of all our stakeholders. We focus on developing treatments for patients by working in close partnership with world-class academic founders and management teams. Our balance sheet underpins our strategy enabling us to take a long-term view as we look to improve the lives of patients with no or poor treatment options, build sustainable life science companies and deliver strong risk-adjusted returns to shareholders.

About LifeSci Venture Partners Formed in 2017, LifeSci Venture Partners is the early stage investing arm of LifeSci Partners, a unique life sciences and healthcare consultancy. We focus on private institutional financing rounds of transformational healthcare companies managed by exceptional founder/entrepreneurs. Our most recent fund, LifeSci Venture Partners II, LP was launched in 2020 and has invested in more than 25 breakthrough biotechnology and healthcare technology companies. For further information, please visit the company's website athttps://www.lifesciventure.com/.

About Emerson Collective Emerson Collective deploys a wide range of tools from impact investing to philanthropy to advocacy in pursuit of a more equal and just society. We focus on creating systemic change in education, immigration, climate, and cancer research and treatment.

Forward Looking Statements

This press release contains forward-looking statements including, but not limited to, statements related to Clades iPSC immune cloaking and differentiation platform technology to address compatibility, durability, reproducibility and scalability of cell therapies, Clades ability to develop broadly accessible, off-the-shelf products with consistent pharmaceutical criteria and expand the reach of cell therapies across patients and indications, the funding round resulting in the acceleration of the development of Clades immune cell-focused, cancer therapeutics and the value that the investor syndicate adds to the Company. These forward-looking statements are based on our current expectations and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks that Clades actual future financial and operating results may differ from its expectations or goals, Clades ability to commercialize and successfully launch its products, risks relating to Clades ability to successfully implement its business strategies, including potential competition, the ability to protect intellectual property and defend patents, regulatory obligations and oversight, including any changes in the legal and regulatory environment in which Clade operates and the effects of the COVID-19 pandemic on the business. We undertake no duty or obligation to update any forward-looking statements contained in this press release as a result of new information.

Contact Ligia Vela Reid LifeSci Advisors Tel: +4407413825310 lvela-reid@lifesciadvisors.com

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Clade Therapeutics Raises $87 Million Series A Financing to - GlobeNewswire

NextCure Reports Third Quarter 2021 Financial Results and Provides Business Update – Yahoo Finance

BELTSVILLE, Md., Nov. 04, 2021 (GLOBE NEWSWIRE) -- NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class immunomedicines to treat cancer and other immune-related diseases, today reported third quarter 2021 financial results and provided a business update.

We are pleased to present clinical and biomarker data for our NC318 and NC410 programs at the upcoming SITC annual meeting, said Michael Richman, NextCures president and chief executive officer. In addition, we will host a virtual research and development update event following the conference on November 15th. The event will also feature Dr. Roy Herbst who will highlight the significant remaining unmet need in the treatment of lung cancer. Dr Herbst is currently a collaborator in the Phase 2 investigator-initiated clinical trial of NC318 in combination with pembrolizumab in patients with advanced non-small cell lung cancer.

Business Highlights and Upcoming Milestones

Published preclinical data pertaining to the NC410 program in the open access journal, Frontiers in Immunology.

Appointed Ellen G. Feigal, M.D., a Partner and Head of the Biologics Practice at NDA Partners LLC, and Anne Borgman, M.D., former Vice President and Global Therapeutic Area Lead, Hematology-Oncology, at Jazz Pharmaceuticals, to the Board of Directors.

Appointed Elizabeth Jaffee, M.D., Ursula Matulonis, M.D., and Weiping Zou, M.D., Ph.D., to its Scientific Advisory Board.

Clinical and biomarker data for NC318 and NC410 programs to be presented at the upcoming Society for Immunotherapy of Cancer (SITC) annual meeting on November 10-14, 2021.

Hosting a virtual research and development update event on November 15, 2021, at 4:30 pm EST.

Announced presentations by collaborators at the American Society of Hematology Annual Meeting (ASH) on December 11-14, 2021, two research studies evaluating the role of Siglec-15 as a therapeutic target in childhood leukemia and the impact of a LAIR-1 antibody that selectively targets AML stem cells.

Financial Guidance Based on its current research and development plans, NextCure expects its existing cash, cash equivalents and marketable securities will enable it to fund operating expenses and capital expenditure requirements into the second half of 2023.

Story continues

Financial Results for Quarter Ended September 30, 2021

Cash, cash equivalents and marketable securities as of September 30, 2021, were $235.3 million, as compared to $283.4 million as of December 31, 2020. The decrease of $48.1 million primarily reflects cash used to fund operations, to purchase fixed assets, and to repay a term loan.

Research and development expenses were $13.6 million for the quarter ended September 30, 2021, as compared to $12.7 million for the quarter ended September 30, 2020. The increase was driven primarily by clinical-related costs, partially offset by timing of research and manufacturing supply costs.

General and administrative expenses were $4.9 million for the quarter ended September 30, 2020, as compared to $4.7 million for the quarter ended September 30, 2020. The increase was primarily related to personnel-related costs.

Net loss was $17.9 million for the quarter ended September 30, 2021, as compared to $16.4 million for the quarter ended September 30, 2020. The increase in net loss for the quarter was primarily due to increased research and development expenses and increased general and administrative expenses from an increase in headcount.

About NextCure, Inc. NextCure is a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class immunomedicines to treat cancer and other immune-related diseases. Through our proprietary FIND-IO platform, we study various immune cells to discover and understand targets and structural components of immune cells and their functional impact in order to develop immunomedicines. Our initial focus is to bring hope and new treatments to patients who do not respond to current cancer therapies, patients whose cancer progresses despite treatment and patients with cancer types not adequately addressed by available therapies. http://www.nextcure.com

Forward-Looking Statements This press release contains forward-looking statements, including statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are based on current expectations, forecasts, assumptions and other information available to NextCure as of the date hereof. Forward-looking statements include statements regarding NextCures expectations, beliefs, intentions or strategies regarding the future and can be identified by forward-looking words such as may, will, potential, expects, believes, intends, hope, towards, forward, later and similar expressions. Examples of forward-looking statements in this press release include, among others, statements about the development plans for NC410 and expected upcoming milestones, the potential benefits of NC410, and NextCures plans, objectives and intentions with respect to the discovery and development of immunomedicines. Forward-looking statements involve substantial risks and uncertainties that could cause actual results to differ materially from those projected in any forward-looking statement. Such risks and uncertainties include, among others: the impacts of the COVID-19 pandemic on NextCures business, including NextCures clinical trials, third parties on which NextCure relies and NextCures operations; positive results in preclinical studies may not be predictive of the results of clinical trials; NextCures limited operating history and no products approved for commercial sale; NextCures history of significant losses; NextCures need to obtain additional financing; risks related to clinical development, marketing approval and commercialization; the unproven approach to the discovery and development of product candidates based on NextCures FIND-IO platform; and dependence on key personnel. More detailed information on these and additional factors that could affect NextCures actual results are described in NextCures filings with the Securities and Exchange Commission (the SEC), including in Item 1A of NextCures most recent Form 10-K and elsewhere in the Companys filings with the SEC. You should not place undue reliance on any forward-looking statements. Forward-looking statements speak only as of the date of this press release, and NextCure assumes no obligation to update any forward-looking statements, even if expectations change.

NEXTCURE, INC. CONDENSED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (unaudited, in thousands, except share and per share amounts)

Three Months Ended

Nine Months Ended

September 30,

September 30,

2021

2020

2021

2020

Revenue:

Revenue from former research and development arrangement

$

$

$

$

22,378

Operating expenses:

Research and development

13,597

12,740

37,928

34,448

General and administrative

4,911

4,659

15,766

12,918

Total operating expenses

18,508

17,399

53,694

47,366

Loss from operations

(18,508

)

(17,399

)

(53,694

)

(24,988

)

Other income, net

578

1,032

1,244

3,846

Net loss

$

(17,930

)

$

(16,367

)

$

(52,450

)

$

(21,142

)

Loss per share - basic and diluted

$

(0.65

)

$

(0.59

)

$

(1.90

)

$

(0.77

)

Weighted-average shares outstanding - basic and diluted

27,615,038

27,547,737

27,607,685

27,524,350

Comprehensive loss:

Net loss

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NextCure Reports Third Quarter 2021 Financial Results and Provides Business Update - Yahoo Finance

Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent … – The Bakersfield Californian

Updates from OTL-201 Clinical Proof-of-Concept Study in MPS-IIIA and OTL-204 Preclinical Study for GRN-FTD at ESGCT Showcase Potential for HSC Gene Therapy in Multiple Neurodegenerative Disorders

Launch Activities for Libmeldy Across Key European Countries, including Reimbursement Discussions, Progressing in Anticipation of Treating Commercial Patients

Frank Thomas, President and Chief Operating Officer, to Step Down Following Transition in 2022; Search for a Chief Financial Officer Initiated

Cash and Investments of Approximately $254M Provide Runway into First Half 2023

BOSTONandLONDON, Nov. 04, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the quarter ended September 30, 2021, as well as recent business updates and upcoming milestones.

This quarter, we are pleased by the progress demonstrated by our investigational neurometabolic HSC gene therapy programs with promising preclinical and clinical updates at ESGCT, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. With follow-up in OTL-201 for MPS-IIIA patients now ranging between 6 and 12 months, biomarker data remain highly encouraging, showing supraphysiological enzyme activity and corresponding substrate reductions in the CSF and urine. The launch strategy for Libmeldy is also advancing in Europe with momentum building on reimbursement discussions and patient finding activities.

Recent Presentations and Business Updates

Data presentations at ESGCT

Clinical and pre-clinical data from across the companys investigational hematopoietic stem cell (HSC) gene therapy portfolio were featured in two oral and seven poster presentations at the European Society of Gene & Cell Therapy Congress (ESGCT) on October 19-22. Highlights from key presentations are summarized below:

OTL-201 for Mucopolysaccharidosis type IIIA (MPS-IIIA): A poster presentation featured supportive biomarker data from the first four patients with evaluable results, with duration of follow-up ranging from 6 to 12 months. The treatment has been generally well-tolerated in all enrolled patients (n=5) with no treatment-related serious adverse events (SAEs). Supraphysiological N-sulphoglucosamine sulphohydrolase ( SGSH) enzyme activity above the normal range was seen in leukocytes and plasma within one to three months in all evaluable patients (n=4).A greater than 90% reduction in urinary glycosaminoglycans (GAGs) was seen within three months in all evaluable patients (n=4).SGSH activity in the cerebrospinal fluid (CSF) increased from undetectable at baseline to within or above the normal range by six months in all patients with available data (n=3).CSF GAGs decreased from baseline in patients with available data (n=3).OTL-204 for Progranulin-mutated Frontotemporal Dementia (GRN-FTD): Preliminary in vivo data from the preclinical proof-of-concept study showed that murine GRN -/- HSPCs, transduced with an LV expressing progranulin under the control of a novel promoter, are able to engraft and repopulate the brain myeloid compartment of FTD mice and to locally deliver the GRN enzyme.

R&D Investor Event Summary

In September, Orchard hosted an R&D investor event highlighting its discovery and research engine in HSC gene therapy, including an update on the OTL-104 program in development for NOD2 Crohns disease (NOD2-CD) and potential new applications in HSC-generated antigen-specific regulatory T-cells (Tregs) and HSC-vectorization of monoclonal antibodies (mAbs).

The discussion also covered the differentiated profile of Orchards HSC gene therapy approach, which has exhibited favorable safety, long-term durability and broad treatment applicability.

In particular, Orchards lentiviral vector-based HSC gene therapy programs have shown no indication of insertional oncogenesis and no evidence of clonal dominance due to integration into oncogenes. Importantly, the promoters and regulatory elements of Orchard vectors are derived from human (not viral) sequences and are specifically designed to have limited enhancer activity on neighboring genes thereby mitigating the potential for safety concerns.In addition, because of the fundamental biological differences between the HSC and adeno-associated virus (AAV) gene therapy approaches, Orchards programs have not, to date, seen the safety and durability concerns experienced by the AAV gene therapy field.

Libmeldy (atidarsagene autotemcel) launch in Europe

Orchard is providing an update on the following key launch activities for Libmeldy in Europe:

Discussions with health authorities and payors are underway across Europe in key markets including Germany, the UK, France and Italy.Qualification of treatment centers is progressing with The University of Tbingen in Germany ready to treat commercial patients and other centers in the final stages of qualification and treatment readiness.Disease awareness and patient identification activities continue and have supported patient referrals in major European centers. Orchards partnerships in the Middle East and Turkey allow for opportunities to treat eligible patients from these territories at qualified European centers.Orchard is providing sponsorship for an ongoing newborn screening pilot in Germany and is working with laboratories to implement pilots in Italy, the UK, France and Spain.

Executive organizational update

The company also announced that Frank Thomas will step down from his role as president and chief operating officer, following a transition in 2022. A search for a chief financial officer is underway. Mr. Thomas other responsibilities will be assumed by existing members of the leadership team in commercial and corporate affairs. Orchard recently strengthened the executive team with the appointments of Nicoletta Loggia as chief technical officer and Fulvio Mavilio as chief scientific officer and the promotion of Leslie Meltzer to chief medical officer.

I want to extend my gratitude to Frank Thomas for his immense contributions to Orchard, said Gaspar. During his tenure, Frank oversaw the transition of the organization to a publicly traded company and has managed operations with a focus on cross-company innovation, including his role as a key architect in creating and executing the focused business plan we rolled out in 2020. Along with the entire board of directors and leadership team, I appreciate Franks commitment to facilitate a smooth transition during this time.

Gaspar continued, Our search is focused on a CFO to lead the broad strategic planning efforts necessary to capitalize on the full potential of our hematopoietic stem cell gene therapy platform. We have a strong team in place to aid Orchards success in this next phase of growth and are well capitalized through the anticipated completion of several value-creating milestones.

Upcoming Milestones

In June 2021, Orchard announced several portfolio updates following recent regulatory interactions for the companys investigational programs in metachromatic leukodystrophy (MLD), Mucopolysaccharidosis type I Hurler syndrome (MPS-IH) and Wiskott-Aldrich syndrome (WAS).

OTL-200 for MLD in the U.S: Based on feedback received from the U.S. Food and Drug Administration (FDA), the company is preparing for a Biologics License Application (BLA) filing for OTL-200 in pre-symptomatic, early-onset MLD in late 2022 or early 2023, using data from existing OTL-200 patients. This approach and timeline are subject to the successful completion of activities remaining in advance of an expected pre-BLA meeting with FDA, including future CMC regulatory interactions and demonstration of the natural history data as a representative comparator for the treated population.OTL-203 for MPS-IH: Orchard is incorporating feedback from FDA and the European Medicines Agency (EMA) into a revised global registrational study protocol, with study initiation expected to occur in 2022.OTL-201 for MPS-IIIA: Additional interim data from this proof-of-concept study are expected to be presented at medical meetings in 2022, including early clinical outcomes of cognitive function.OTL-103 for WAS: The company expects a MAA submission with EMA for OTL-103 in WAS in 2022, subject to the completion of work remaining on potency assay validation and further dialogue with EMA. The company will provide updated guidance for a BLA submission in the U.S. following additional FDA regulatory interactions.

Third Quarter 2021 Financial Results

Revenue from product sales of Strimvelis were $0.7 million for the third quarter of 2021 compared to $2.0 million in the same period in 2020, and cost of product sales were $0.2 million for the third quarter of 2021 compared to $0.7 million in the same period in 2020. Collaboration revenue was $0.5 million for the third quarter of 2021, resulting from the collaboration with Pharming Group N.V. entered into in July 2021. This revenue represents expected reimbursements for preclinical studies and a portion of the $17.5 million upfront consideration received by Orchard under the collaboration, which will be amortized over the expected duration of the agreement.

Research and development (R&D) expenses were $20.8 million for the third quarter of 2021, compared to $14.7 million in the same period in 2020. The increase was primarily due to higher manufacturing and process development costs for the companys neurometabolic programs and lower R&D tax credits as compared to the same period in 2020. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and development milestone payments under the companys agreements with third parties, and personnel costs to support these activities.

Selling, general and administrative (SG&A) expenses were $13.0 million for the third quarter of 2021, compared to $13.0 million in the same period in 2020. SG&A expenses are expected to increase in future periods as the company builds out its commercial infrastructure globally to support additional product launches following regulatory approvals.

Net loss was $36.4 million for the third quarter of 2021, compared to $20.3 million in the same period in 2020. The increase in net loss as compared to the prior year was primarily due to higher R&D expenses as well as the impact of foreign currency transaction gains and losses. The company had approximately 125.5 million ordinary shares outstanding as of September 30, 2021.

Cash, cash equivalents and investments as of September 30, 2021, were $254.1 million compared to $191.9 million as of December 31, 2020. The increase was primarily driven by net proceeds of $143.6 million from the February 2021 private placement and $17.5 million in upfront payments from the July 2021 collaboration with Pharming Group N.V., offset by cash used for operating activities and capital expenditures. The company expects that its cash, cash equivalents and investments as of September 30, 2021 will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes an additional $67 million that could become available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future U.S. approvals.

About Libmeldy / OTL-200 Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability. For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website. Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. About Orchard

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website ( http://www.orchard-tx.com ), the investor relations website ( ir.orchard-tx.com ), and on social media ( Twitter and LinkedIn ), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2023. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2021, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Reports Third Quarter 2021 Financial Results and Highlights Recent ... - The Bakersfield Californian

Bristol Myers Squibb to Highlight More than 80 Abstracts at ASH 2021 Demonstrating Strength of Innovative Therapeutic Platforms Improving Outcomes for…

PRINCETON, N.J.--(BUSINESS WIRE)--Bristol Myers Squibb (NYSE: BMY) today announced the presentation of research across a wide range of hematologic diseases at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition, which will take place in Atlanta, Georgia, and virtually, from December 11 to 14, 2021. Data from more than 80 company-sponsored studies will be featured, including 23 oral presentations, highlighting key research and development programs in lymphomas, leukemias, multiple myeloma and myeloid diseases, and showcasing our commitment to delivering transformative medicines across major hematologic diseases.

Key data being presented by Bristol Myers Squibb and its partners at the 2021 ASH Annual Meeting and Exposition include:

Our presence at ASH continues our longstanding commitment to hematology and underscores the potential of our innovative research platforms to deliver meaningful, new treatment options for people with unmet needs living with hematologic diseases, said Samit Hirawat, M.D., executive vice president, chief medical officer, global drug development, Bristol Myers Squibb. These data reinforce our progress in advancing transformative research across a wide range of hematologic malignancies including multiple myeloma, lymphoma, and myeloid diseases.

SelectedBristol Myers Squibb studies at the 63rd ASH Annual Meeting and Exposition include:

Abstract Title

Author

Presentation Type/#

Session Title

Session Date/Time

Acute Myeloid Leukemia

Prognostic Impact of NPM1 and FLT3 Mutations at Diagnosis and Presence of Measurable Residual Disease (MRD) after Intensive Chemotherapy (IC) for Patients with Acute Myeloid Leukemia (AML) in Remission: Outcomes from the QUAZAR AML-001 Trial of Oral Azacitidine (Oral-AZA) Maintenance

Hartmut Dhner

Oral

Abstract #804

617. Acute Myeloid Leukemia: Biomarkers, Molecular Markers and Minimal Residual Disease in Diagnosis and Prognosis: New options of risk assessment and prediction of therapy response in AML

Monday, December 13,

5:45 PM

Long-term Overall Survival (OS) with Oral Azacitidine (Oral-AZA) in Patients with Acute Myeloid Leukemia (AML) in First Remission after Intensive Chemotherapy (IC): Updated Results from the Phase 3 QUAZAR AML-001 Trial

Andrew Wei

Oral

Abstract #871

615. Acute Myeloid Leukemias: Commercially Available Therapies, Excluding Transplantation and Cellular Immunotherapies: Updates in treatment for high-risk AML

Monday, December 13,

6:15 PM

Beta Thalassemia

Luspatercept Redistributes Body Iron to the Liver in Transfusion-Dependent-Thalassemia (TDT) During Erythropoietic Response

Maciej Garbowski

Oral Abstract

#761

102. Iron Homeostasis and Biology: Disorders of Iron and Heme and Novel Treatments

Monday, December 13,

5:30 PM

Luspatercept Improves Health-Related Quality of Life (HRQoL) Symptoms and RBC Transfusion Burden in Patients with Non-Transfusion-Dependent -thalassemia (NTDT) in the BEYOND Trial

Antonis Kattamis

Poster Abstract #3081

112. Thalassemia and Globin Gene Regulation: Poster III

Monday, December 13,

6:00 - 8:00 PM

Graft vs. Host Disease

Overall Survival of Patients Treated with Abatacept in Combination with a Calcineurin Inhibitor and Methotrexate After Allogeneic Hematopoietic Stem Cell Transplantation - Analysis of the Center for International Blood and Marrow Transplant Research Database

Leslie Kean

Poster Abstract #3912

722. Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Poster III

Monday, December 13, 6:00 8:00 PM

Lymphoma

Lisocabtagene Maraleucel (liso-cel), a CD19-Directed Chimeric Antigen Receptor (CAR) T Cell Therapy, Versus Standard of Care (SOC) with Salvage Chemotherapy (CT) Followed by Autologous Stem Cell Transplantation (ASCT) as Second-Line (2L) Treatment in Patients (Pts) with Relapsed or Refractory (R/R) Large B-Cell Lymphoma (LBCL): Results from the Randomized Phase 3 TRANSFORM Study

Manali Kamdar

Oral Abstract

#91

704. Cellular Immunotherapies: Cellular Therapies for Lymphomas

Saturday, December 11,

9:30 AM

Ruxolitinib Plus Nivolumab in Patients with R/R Hodgkin Lymphoma after Failure of Check-Point Inhibitors: Preliminary Report on Safety and Efficacy

Veronika

Bachanova

Oral Abstract

#230

624. Hodgkin Lymphomas and T/NK cell Lymphomas: Hodgkin Lymphoma Clinical Trials

Hematology Disease Topics & Pathways:

Clinical Trials

Saturday, December 11, 2:15 PM

Nivolumab First-Line Therapy for Elderly Hodgkin Lymphoma Patients: a LYSA Phase II Study

Julien Lazarovici

Oral Abstract

#232

624. Hodgkin Lymphomas and T/NK cell Lymphomas: Hodgkin Lymphoma Clinical Trials

Saturday, December 11, 2:45 PM

OUTREACH: Results from a Phase 2 Study of Lisocabtagene Maraleucel (liso-cel) Administered as Inpatient (Inpt) or Outpatient (Outpt) Treatment in the Nonuniversity Setting in Patients (Pts) with R/R Large B-Cell Lymphoma (LBCL)

John Godwin

Poster Abstract

#1762

704. Cellular Immunotherapies: Clinical: Poster I

Saturday, December 11,

5:30 7:30 PM

Six-Year Results from the Phase 3 Randomized Study Relevance Show Similar Outcomes for Previously Untreated Follicular Lymphoma Patients Receiving Lenalidomide Plus Rituximab (R2) Versus Rituximab-Chemotherapy Followed By Rituximab Maintenance

Franck Morschhauser

Poster Abstract

#2417

623. Mantle Cell, Follicular, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster II

Sunday, December 12,

6:00 - 8:00 PM

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Bristol Myers Squibb to Highlight More than 80 Abstracts at ASH 2021 Demonstrating Strength of Innovative Therapeutic Platforms Improving Outcomes for...

Atara Biotherapeutics to Present Eight Abstracts at the 63rd American Society of Hematology (ASH) Annual Meeting, Including First Presentation of…

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, today announced the upcoming first release of efficacy and safety results from its Phase 3 ALLELE study. The pivotal trial is investigating tabelecleucel (tab-cel) for the treatment of Epstein-Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD) following solid organ transplant (SOT) or hematopoietic cell transplant (HCT). Detailed findings, along with combined data from investigator-sponsored Phase 2 and multicenter Expanded Access Program (EAP) studies, will be featured among eight abstracts, including two oral presentations, at the 63rd American Society of Hematology (ASH) Annual Meeting taking place December 11-14, 2021, in Atlanta.

There is significant unmet need in patients with EBV+ PTLD, with poor overall survival measured in weeks to a few months after first-line treatment failure, said Jakob Dupont, MD, Head of Global Research & Development at Atara. Tab-cel demonstrated a clinically meaningful objective response rate and striking overall survival in a patient population with no approved treatment options, representing a first-in-kind allogeneic therapy with transformative potential. At ASH, we will share data from our Phase 3 ALLELE study, which further supports tab-cel as a potentially safe and effective treatment option for patients with EBV-driven diseases.

As reported in the full abstract available today on the ASH website, top-line data with additional patients and extended follow up confirm a strong ORR in line with prior Phase 2 and multicenter EAP results and demonstrate durability of response with no new safety signals.

In this ongoing Phase 3 study, 38 evaluable patients as of May 2021 24 EBV+ PTLD following SOT patients after failure of rituximab chemotherapy and 14 EBV+ PTLD following HCT patients after failure of rituximab monotherapy were treated with tab-cel and had the opportunity for a six-month follow-up after response. An ORR, as measured by independent oncologic response adjudication (IORA) assessment, of 50% (19/38, 95% CI: 33.4, 66.6) was observed, with an ORR of 50.0% (12/24, 95% CI: 29.1, 70.9) in PTLD following SOT and 50.0% (7/14, CI: 23.0, 77.0) in PTLD following HCT, with a best overall response of Complete Response (CR; n=5, SOT; n=5, HCT) or Partial Response (PR; n=7, SOT; n=2, HCT).

Overall, the median time to response (TTR) was 1.1 months (0.7-4.7). Of 19 responders, 11 had a duration of response (DOR) lasting more than six months and median DOR has not been reached yet. Those who responded had a longer survival compared to the non-responders, with a median OS not evaluable (NE) (95% CI: 16.4, NE) and a 1-year survival rate of 89.2% (95% CI: 63.1, 97.2).

Safety findings were consistent with previously published data, with no new signals or concerns reported. There were no reports of tumor flare reaction, and no confirmed evidence of graft versus host disease (GvHD), organ rejection, infusion reactions, or cytokine release syndrome related to tab-cel.

Further detail on baseline demographics and disease characteristics, and additional safety data including tab-cel exposure details, will be presented on December 11 in the oral presentation.

Atara will present additional data on tab-cel and PTLD through several abstracts, including a second oral presentation on long term OS from Phase 2 and multicenter EAP studies with tab-cel in relapsed/refractory EBV+ PTLD showing median OS of 54.6 months in all patients and OS at two years reaching over 86% in responders whether patients experienced CR or PR. Treatment was well tolerated with no confirmed evidence for graft versus host disease, cytokine release syndrome, SOT rejection, or neurologic events attributable to tab-cel.

In total, five abstracts will be presented at the 63rd ASH Annual Meeting. An additional three accepted abstracts will be published online in the November supplemental issue of Blood.

Oral Presentation Details: Title: Multicenter, Open-Label, Phase 3 Study of Tabelecleucel for Solid Organ or Allogeneic Hematopoietic Cell Transplant Recipients with Epstein-Barr Virus-Driven Post Transplant Lymphoproliferative Disease after Failure of Rituximab or Rituximab and Chemotherapy (ALLELE)

Title: Overall Survival by Best Overall Response with Tabelecleucel in Patients with Epstein-Barr Virus-Driven Post-Transplant Lymphoproliferative Disease Following Solid Organ or Allogeneic Hematopoietic Cell Transplant

Poster Presentation Details: Title: Clinical Outcomes of Patients with Epstein-Barr Virus-Driven Post-Transplant Lymphoproliferative Disease Following Hematopoietic Stem Cell Transplantation Who Fail Rituximab: A Multinational, Retrospective Chart Review Study

Title: Clinical Outcomes of Solid Organ Transplant Patients with Epstein-Barr Virus-Driven (EBV+) Post-Transplant Lymphoproliferative Disorder (PTLD) Who Fail Rituximab Plus Chemotherapy: A Multinational, Retrospective Chart Review Study

Title: Comprehensive Activation Profiling of the Tabelecleucel Library, an Off-the-Shelf, Allogeneic EBV-Specific T-Cell Therapy

About Tabelecleucel Tabelecleucel (tab-cel) is an off-the-shelf, allogeneic T-cell immunotherapy in development for the treatment of Epstein-Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD). EBV+ PTLD is a type of lymphoma (cancer) that may occur after a solid organ transplant (SOT) or allogeneic hematopoietic cell transplant (HCT). There are currently no approved treatments indicated to treat PTLD and if left untreated, PTLD can have life-threatening consequences.

Tab-cel is currently being investigated in the Phase 3 ALLELE study to assess efficacy and safety for the treatment of EBV+ PTLD in SOT and HCT after failure of standard of care.

Tab-cel has been granted Breakthrough Therapy Designation for EBV+ PTLD following allogeneic HCT by the U.S. Food and Drug Administration (FDA) and PRIME designation by the European Medicines Agency (EMA) for the same indication. Tab-cel has orphan drug designation in the U.S. and EU.

About Atara Biotherapeutics, Inc. Atara Biotherapeutics, Inc. (@Atarabio) is a pioneer in T-cell immunotherapy leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with serious diseases including solid tumors, hematologic cancers and autoimmune disease. With our lead program in Phase 3 clinical development, Atara is the most advanced allogeneic T-cell immunotherapy company and intends to rapidly deliver off-the-shelf treatments to patients with high unmet medical need. Our platform leverages the unique biology of EBV T cells and has the capability to treat a wide range of EBV-associated diseases, or other serious diseases through incorporation of engineered CARs (chimeric antigen receptors) or TCRs (T-cell receptors). Atara is applying this one platform, which does not require TCR or HLA gene editing, to create a robust pipeline including: tab-cel in Phase 3 development for Epstein-Barr virus-driven post-transplant lymphoproliferative disease (EBV+ PTLD) and other EBV-driven diseases; ATA188, a T-cell immunotherapy targeting EBV antigens as a potential treatment for multiple sclerosis; and multiple next-generation chimeric antigen receptor T-cell (CAR-T) immunotherapies for both solid tumors and hematologic malignancies. Improving patients lives is our mission and we will never stop working to bring transformative therapies to those in need. Atara is headquartered in South San Francisco and our leading-edge research, development and manufacturing facility is based in Thousand Oaks, California.

For additional information about the company, please visit atarabio.com and follow us on Twitter and LinkedIn.

Forward-Looking Statements This press release contains or may imply "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. For example, forward-looking statements include statements regarding: the potential benefits, safety and efficacy of tab-cel; the timing and progress of tab-cel, including (i) data and analyses from ALLELE study, the investigator-initiated Phase 2 study, and the EAP; (ii) tab-cel clinical trials, and (iii) Ataras ability to successfully advance the development of tab-cel. Because such statements deal with future events and are based on Ataras current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Atara could differ materially from those described in or implied by the statements in this press release. These forward-looking statements are subject to risks and uncertainties, including, without limitation, risks and uncertainties associated with the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success; the ongoing COVID-19 pandemic, which may significantly impact (i) our business, research, clinical development plans and operations, including our operations in South San Francisco and Southern California and at our clinical trial sites, as well as the business or operations of our third-party manufacturer, contract research organizations or other third parties with whom we conduct business, (ii) our ability to access capital, and (iii) the value of our common stock; the sufficiency of Ataras cash resources and need for additional capital; and other risks and uncertainties affecting Ataras and its development programs, including those discussed in Ataras filings with the Securities and Exchange Commission (SEC), including in the Risk Factors and Managements Discussion and Analysis of Financial Condition and Results of Operations sections of the Companys most recently filed periodic reports on Form 10-K and Form 10-Q and subsequent filings and in the documents incorporated by reference therein. Except as otherwise required by law, Atara disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

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Atara Biotherapeutics to Present Eight Abstracts at the 63rd American Society of Hematology (ASH) Annual Meeting, Including First Presentation of...

CRISPR Therapeutics Provides Business Update and Reports Third Quarter 2021 Financial Results – GlobeNewswire

-Achieved target enrollment in CTX001 clinical trials for beta thalassemia (TDT) and sickle cell disease (SCD); regulatory submissions planned for late 2022-

-Reported positive results from the ongoing Phase 1 CARBON clinical trial evaluating the safety and efficacy of CTX110 for CD19+ B-cell malignancies; enrollment continues, with potential registrational trial incorporating consolidation dosing expected to initiate in Q1 2022-

-Implementing consolidation dosing protocols for CTX120 and CTX130 clinical trials; enrollment continues, with top-line data expected to report in 1H 2022-

-Regenerative medicine and in vivo programs continue to progress and remain on track-

ZUG, Switzerland and CAMBRIDGE, Mass., Nov. 03, 2021 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the third quarter ended September 30, 2021.

The third quarter marked significant progress across our portfolio, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. With our partner Vertex, we achieved target enrollment for the CTX001 clinical trials in patients with beta thalassemia and sickle cell disease, which can support regulatory submissions in late 2022. Additionally, we demonstrated proof of concept for our allogeneic CAR-T platform with positive data from our CARBON trial of CTX110, which showed that immediately available off-the-shelf cell therapies can offer efficacy similar to autologous CAR-T with a differentiated safety profile for patients with large B-cell lymphomas. Based on these encouraging results, we plan to expand the CARBON trial into a potentially registrational trial in the first quarter of 2022. Furthermore, we hope to bring these transformative allogeneic CAR-T therapies to patients in outpatient and community oncology settings, enabling broad access."

Recent Highlights and Outlook

Third Quarter 2021 Financial Results

About CTX001CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patients hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate or eliminate transfusion requirements for patients with TDT and reduce or eliminate painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published as a Brief Report in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About the CRISPR-Vertex Collaboration Vertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. Under a recently amended collaboration agreement, Vertex will lead global development, manufacturing and commercialization of CTX001 and split program costs and profits worldwide 60/40 with CRISPR Therapeutics.

About CLIMB-111 The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121 The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-131 This is a long-term, open-label trial to evaluate the safety and efficacy of CTX001 in patients who received CTX001 in CLIMB-111 or CLIMB-121. The trial is designed to follow participants for up to 15 years after CTX001 infusion.

About CTX110 CTX110, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the ongoing CARBON trial.

About CARBON The ongoing Phase 1 single-arm, multi-center, open label clinical trial, CARBON, is designed to assess the safety and efficacy of several dose levels of CTX110 for the treatment of relapsed or refractory B-cell malignancies.

About CTX120 CTX120, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting B-cell maturation antigen, or BCMA. CTX120 is being investigated in an ongoing Phase 1 single-arm, multi-center, open-label clinical trial designed to assess the safety and efficacy of several dose levels of CTX120 for the treatment of relapsed or refractory multiple myeloma. CTX120 has been granted Orphan Drug designation from the FDA.

About CTX130 CTX130, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies. CTX130 is being investigated in two ongoing independent Phase 1, single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 for the treatment of relapsed or refractory renal cell carcinoma and various subtypes of lymphoma, respectively.

About CRISPR Therapeutics CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR THERAPEUTICS word mark and design logo, CTX001, CTX110, CTX120, and CTX130 are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

CRISPR Therapeutics Forward-Looking Statement This press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni in this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the safety, efficacy, data and clinical progress of CRISPR Therapeutics various clinical programs, including CTX001, CTX110, CTX120 and CTX130; (ii) the status of clinical trials and preclinical studies (including, without limitation, the expected timing of data releases and development, as well as initiation and completion of clinical trials) and development timelines for CRISPR Therapeutics product candidates; (iii) expectations regarding the data that has been presented from our various clinical trials (including our CARBON trial) as well as data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials or to support regulatory filings; (iv) the actual or potential benefits of regulatory designations; (v) the potential benefits of CRISPR Therapeutics collaborations and strategic partnerships; (vi) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties as well as the status and potential outcome of proceedings involving any such intellectual property; (vii) the sufficiency of CRISPR Therapeutics cash resources; and (viii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies including as compared to other therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients not to be indicative of final trial results; the potential that clinical trial results may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates (including, without limitation, availability and timing of results and whether such results will be predictive of future results of the future trials); uncertainties about regulatory approvals to conduct trials or to market products; the potential impacts due to the coronavirus pandemic such as (x) delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; (y) the timing and progress of clinical trials, preclinical studies and other research and development activities; and (z) the overall impact of the coronavirus pandemic on its business, financial condition and results of operations; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

Investor Contact: Susan Kim +1-617-307-7503 susan.kim@crisprtx.com

Media Contact: Rachel Eides +1-617-315-4493 rachel.eides@crisprtx.com

CRISPR Therapeutics AG Condensed Consolidated Statements of Operations (Unaudited, In thousands except share data and per share data)

CRISPR Therapeutics AG Condensed Consolidated Balance Sheets Data (Unaudited, in thousands)

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CRISPR Therapeutics Provides Business Update and Reports Third Quarter 2021 Financial Results - GlobeNewswire

UVA Science and Engineering Faculty Win 12 NSF Career Awards – University of Virginia

From stopping deadly diseases to developing futuristic materials, from making self-driving vehicles smarter to studying global inequalities in pollution exposure, the University of Virginias early career faculty are more deeply involved than ever in making peoples lives safer, healthier and more efficient.

In 2021 so far, 12 UVA assistant professors have earned National Science Foundation Early Career Development Awards, among the most competitive and prestigious grants for science and engineering faculty in the first stages of their careers. Thats up from eight CAREER Awards in 2020, and four to five awards per year before then.

The CAREER Award is given to early career researchers who have the potential to make a significant impact through their careers as academic researchers and educators, Melur Ram Ramasubramanian, UVAs vice president for research, said. Getting 12 of these prestigious awards for our faculty so far this year is impressive, and really shows the great talent we have across the University.

Meet the most recent UVA CAREER Award winners, whom NSF expects to become the next great leaders and role models in research and education.

In May 2020, Partners for Automated Vehicle Education shared results from a poll of 1,200 Americans about attitudes around autonomous vehicle technology. Three in four believed the technology was not ready for primetime; almost half indicated they would never ride in a self-driving car; and a fifth do not believe that autonomous vehicles will ever be safe.

The poll outlines the deep skepticism surrounding self-driving vehicles. Methods to improve and prove safety will be needed for broad-based acceptance. Behls pioneering research at UVA is accelerating safety for autonomous vehicles.

Using auto racing as a platform, Behl has invented artificial intelligence methods to agilely maneuver an autonomous vehicle while pushing the limits of its steering, throttle and braking capabilities. His novel racing research is creating advanced algorithms that hold the key to safer autonomous vehicles, enabling them to avoid collisions even when they encounter unexpected challenges at high speeds while close to obstacles or other vehicles.

Demonstrating their skills in programming a full-sized, fully autonomous race car, Behl and his student Cavalier Autonomous Racing team clocked the fastest laps from a U.S. university team in the historic Indy Autonomous Challenge, held Oct. 23 at the Indianapolis Motor Speedway.

Fibrosis, the stiffening of normally soft or pliant living tissue, contributes significantly to about 40% of the overall deaths in the developed world.

Yeah, its a hell of a stat, Caliari said. But thats because fibrosis, or chronic scarring, itself isnt a disease; its an outcome of many different diseases.

The list includes some cancers, viral infections such as hepatitis, and idiopathic pulmonary fibrosis, a cruel condition in which scar tissue grows in the lungs, restricting the flow of oxygen. Idiopathic means the disease has no known cause. It has no cure, either.

Researchers like Caliari believe stopping or even reversing the progression of fibrosis is possible, but they need to know a lot more about what is happening in the body to make cells go from normal to a diseased state. Caliari is using biomaterials developed in his lab to open a window on that process.

Caliaris plans include partnering with the UVA chapter of the Society of Hispanic Professional Engineers to develop teaching modules on biomaterials concepts for elementary school students and initiating a high school summer research program involving labs in UVAs Fibrosis Initiative.

Holographic displays, color-changing frames and pliable screens are just a few of the innovations the next generation of smartphones may offer. And while engineers and coders will be responsible for making much of that technology possible, theres a good chance well also need to thank Gilliard.

Gilliard explores strategies for incorporating boron into chemical compounds to help him understand how to harness the elements unique capacity to carry and transfer electric charge and to produce the colors displayed by our cellphones and electronic devices.

In collaboration with the chemical engineering department here at UVA, weve already started to explore the applications of some of these boron-based materials, and were seeing that the utility is probably going to be pretty important going forward, Gilliard said.

His research may also make components in those devices more stable over time, less expensive to produce and less harmful to the environment.

This is the technology that has resulted in your lights in your home lasting much longer than they did even five years ago, Gilliard said.

We have a unique potential to be one of the leaders in this area of boron chemistry, he added. There are not many people in the United States exploring these areas of chemistry, and increasing our ability to compete globally in this area of science is extremely important.

As we struggle to come to terms with the fact that more than half a million lives have been lost to COVID-19 in the United States, it can be easy to forget that nearly as many people die from malaria worldwide every single year.

Malaria is caused by a single-celled, mosquito-borne parasite, but like a virus, it can adapt to survive a variety of challenges that could wipe it out completely. Gler studies how the malaria parasite responds to changes in its environment that are hostile to its survival.

Over the course of its life cycle, the malaria parasite must be able to adapt to the conditions that allow it to survive in the body of a mosquito, in the liver of an infected host or in a hosts bloodstream before it infects another mosquito. Evolution has also equipped it with the capacity to develop a resistance to the drugs that researchers develop to defeat it. Gler uses a powerful combination of laboratory studies and computational modeling to understanding the complexities of the cellular behaviors that make the malaria parasite so resilient.

The CAREER Award will help us look, specifically, at how the parasites respond to stress, so if theyre in one of these new environments and its stressful for them maybe theres a limiting nutrient or a drug present and its causing stress what sort of programs are going on inside that cell that allow it to survive? Gler said. Ultimately, what we learn could help us find a better way to treat this disease.

The National Science Foundation places a priority on inventing new computing and networking technologies. The need is urgent, because such technologies will help researchers use big data sets to find solutions for complex global challenges.

The problem is that the amount of data available globally has outpaced the processing power needed to analyze it. International Data Corporation predicts that the collective sum of the worlds data will grow to 175 zettabytes 175 trillion gigabytes by 2025, a massive data explosion compared to 4.4 zettabytes available in 2015.

Khan is developing revolutionary computer architectures that will make problem-solving with big data possible.

Datasets are so large they must be broken up into bundles across multiple computers in a data center, Khan said. Computations get bottlenecked as larger and larger data packets get moved from computer to computer in progression to a single processor.

Khans research aims to redesign programmable switches and smart network interface cards to allow data to be processed in transit instead, a fundamental redesign of outdated computer infrastructure. Her research team has built the first protype network that uses the revolutionary architecture, making data requests four times faster.

In the real world, this would mean people could update their social media or make online transactions, like purchasing tickets, lightning-fast compared to today.

Reducing the amount of data that needs to be moved to that single point of processing dramatically speeds things up and fuels the entire systems capacity, Khan said. We are expecting that processing in the reconfigured network will achieve more than 10-fold increases in processing speeds for scientific and machine-learning workloads.

Despite the fact that the STEM workforce has shown considerable growth in recent years, Black workers, and especially Black women, remain underrepresented in the fields of science, technology, engineering and math. And according to a study of trends in STEM degrees by the Pew Research Center, the gap is unlikely to narrow any time soon. For Seanna Leath, an assistant professor of psychology, universities have a critical part to play in addressing Black womens retention in STEM fields.

Leaths CAREER award will allow her to explore how improving the academic, social and psychological wellbeing of Black college women will help attract them to the study of STEM disciplines and allow them to thrive as students. Funding from the award will allow Leath to develop longitudinal surveys and interview tools to assess Black undergraduate womens experiences over a four-year period, and using the data she collects, she hopes to identify the most important factors affecting the motivation and retention of Black women in STEM degrees.

You might think that air pollution is an equal-opportunity threat, but there is a solid body of evidence suggesting that not everyone who lives in urban areas experiences the same level of exposure, which means that some communities are faced with a lower quality of life and a lower life expectancy.

Using a variety of airborne and ground-based data-collection methods, Pusede, an atmospheric chemist, is interested in advancing sciences understanding of how variations in exposure to airborne pollutants occur in urban areas and why.

With the help of the CAREER award, Pusede will conduct field work in Dakar, Senegal, which will lead to the training of U.S. and Senegalese students in an international collaboration of physical and social scientists that will involve collecting and integrating scientific data and demographic information from a wide range of sources to shed light on inequalities in pollutant exposure and their consequences.

Pusedes project will also include the development of educational and public-outreach activities based on her research, including the development of a middle-school curriculum aimed at encouraging students interest in the STEM fields and demonstrating how those fields can help advance the cause of environmental justice.

Did you know that a tuna is a super swimmer?

Theyre really fast, theyre really strong, theyre big, theyre at the top of their food chain without any natural predators. Theyre a model organism for roboticists because theyre phenomenal swimmers, Quinn said. Besides being fast, tuna dart back and forth very quickly complex, high-speed maneuvers and were not sure how they do it.

Quinn is using his CAREER Award to find out. His Smart Fluids Systems Lab is using a tuna model rigged up to swim inside a tank to try to discover how liquids flow past the fish a process called fluid dynamics and govern high-speed, irregular or asymmetric swimming.

By mapping out these flows, bio-inspired roboticists who have to rely on models of low-speed, regular or symmetric movements when designing and testing robots will have the information they need to start modeling and designing fast, highly maneuverable water and aerial drones. Even though Quinn is studying swimming, the principles of fluid dynamics apply to water and air propulsion, so his research will inform both.

Well be creating the first-ever flow visualizations of bio-inspired robots darting side-to-side, Quinn said. Our measurements could lay the groundwork for a new generation of intelligent swimming and flying machines.

According to the U.S. Energy Information Administration, approximately 5% of the energy that is generated by power plants in the country is lost to resistance in the power lines used to transmit energy to homes and businesses, and the complexity of the problem at the atomic level makes it difficult for researchers to understand exactly what properties of the electrons involved might lead to the ability to conduct current without resistance.

Direct imaging of electrons is almost impossible, but quantum simulation can shed light on the microscopic properties of these complex quantum systems. CAREER winner Peter Schauss, an assistant professor of physics who specializes in experimental atomic, molecular and optical physics, will use funding from the award to develop quantum simulations using atoms cooled to a few billionths of a degree and trapped in an artificial crystal of light. Using a quantum gas microscope with high-resolution imaging capabilities that will allow him to capture images of individual atoms, hell search for answers that could lead to the next generation of superconductors.

In this modern age of materials, complex alloys lighten the weight of cars and planes to save fuel and help the environment, biomaterials replace human joints so we can remain active into our elder years, and graphene-coated smart screens put us in touch literally with individual creativity and global commerce.

These breakthroughs demonstrate the power of nanotechnology, a term introduced in 1974 to describe the precision machining of materials to the atomic scale. While experimentation, development and commercialization of nanomaterials has evolved, the textbook model describing how and when a material changes its form remains stuck in the 1970s.

Zhou has a plan to bring this model into the modern age and democratize materials design. He will use his CAREER Award to innovate a valuable tool in alloy development called the CALPHAD method, which stands for CALculation of PHAse Diagrams.

My grand vision is to make computational tools easy to use and valuable for all materials scientists, Zhou said.

Editors note: Two additional faculty members from UVA earned CAREER Awards, but are no longer with the University.

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UVA Science and Engineering Faculty Win 12 NSF Career Awards - University of Virginia

MAGENTA THERAPEUTICS, INC. Management’s Discussion and Analysis of Financial Condition and Results of Operations. (form 10-Q) – marketscreener.com

FORWARD LOOKING STATEMENTSThis Quarterly Report on Form10-Qof Magenta Therapeutics, Inc. (the "Company") contains or incorporatesstatements that constitute forward-looking statements within the meaning of thefederal securities laws. Any express or implied statements that do not relate tohistorical or current facts or matters are forward-looking statements. In somecases, you can identify forward-looking statements by terminology such as "may,""will," "could," "should," "expects," "intends," "plans," "anticipates,""believes," "estimates," "predicts," "projects," "seeks," "endeavor,""potential," "continue" or the negative of these terms or other comparableterminology. Forward-looking statements appear in a number of places in thisQuarterly Report on Form10-Qand include, but are not limited to, statements about: the initiation, timing and success of clinical trials of MGTA-145, MGTA-117 and any other product candidates; the outcomes of our preclinical studies; our ability to commence and enroll patients in our clinical trials at the pace that we project; whether the results of our trials will be sufficient to support domestic or foreign regulatory approvals for MGTA-145, MGTA-117 or any other product candidates we may develop; our ability to establish clinical programs moving forward in multiple indications, with a rapidly advancing portfolio and sustainable platform; regulatory actions with respect to our product candidates or our competitors' products and product candidates our ability to obtain, including on an expedited basis, and maintain regulatory approval of MGTA-145, MGTA-117 or any other product candidates we may develop; the level of expenses related to any of our product candidates or clinical development programs; our expectation that our existing capital resources will be sufficient to enable us to fund our planned development of MGTA-145, MGTA-117 and any other product candidates we may identify and pursue; the benefits of the use of MGTA-145, MGTA-117 or any other product candidate, if approved; our ability to successfully commercialize MGTA-145, MGTA-117 or any other product candidates we may identify and pursue, if approved; our ability to successfully find collaborators for E478 or any of our current and future programs and product candidates; the rate and degree of market acceptance of MGTA-145, MGTA-117 or any other product candidates we may identify and pursue; our ability to obtain orphan drug designation for any of our product candidates we may identify and pursue; our expectations regarding government and third-party payor coverage and reimbursement; our ability to manufacture MGTA-145, MGTA-117 or any other product candidate in conformity with the U.S. Food and Drug Administration's requirements and to scale up manufacturing of our product candidates to commercial scale, if approved; our ability to successfully build a specialty sales force and commercial infrastructure; our ability to compete with companies currently producing or engaged in the clinical development of treatments for the disease indications that we pursue and treatment modalities that we develop; our reliance on third parties to conduct our clinical trials; our reliance on third-party contract manufacturers to manufacture and supply our product candidates for us; our ability to retain and recruit key personnel; our ability to obtain and maintain intellectual property protection for MGTA-145, MGTA-117 or any other product candidates we may identify and pursue; 18

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We expense research and development costs to operations as incurred. Advance payments for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed. Our direct research and development expenses are tracked on a program-by-program basis and consist primarily of external costs, such as fees paid to consultants, central laboratories, contractors, CMOs and CROs in connection with our preclinical and clinical development activities. We do not allocate employee costs, costs associated with our platform technology or facility expenses, including depreciation or other indirect costs, to specific product development programs because these costs are deployed across multiple product development programs and, as such, are not separately classified. The successful development and commercialization of our product candidates is highly uncertain. This is due to the numerous risks and uncertainties, including the following:

A change in the outcome of any of these variables with respect to the development of any of our product candidates would significantly change the costs and timing associated with the development of that product candidate. We may never succeed in obtaining regulatory approval for any of our product candidates. Research and development activities are central to our business model. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. We expect research and development costs to increase significantly for the foreseeable future as our product candidate development programs progress. However, we do not believe that it is possible at this time to accurately project total program-specific expenses through commercialization. There are numerous factors associated with the successful commercialization of any of our product candidates, including future trial design and various regulatory requirements, many of which cannot be determined with accuracy at this time based on our stage of development. Additionally, future commercial and regulatory factors beyond our control will impact our clinical development programs and plans.

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Accordingly, we believe the policies set forth above are critical to fully understanding and evaluating our financial condition and results of operations. If actual results or events differ materially from the estimates, judgments and assumptions used by us in applying these policies, our reported financial condition and results of operations could be materially affected. Results of Operations Comparison of the Three Months Ended September 30, 2021 and 2020 The following table summarizes our results of operations for the three months ended September 30, 2021 and 2020:

Operating expenses: Research and development $ 10,795 $ 11,786 $ (991 ) General and administrative

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Direct research and development expenses by program: Conditioning

Unallocated expenses: Personnel related (including stock-based compensation) 4,886 3,704 1,182 Consultant (including stock-based compensation)

Expenses related to our conditioning program decreased primarily due to a decrease in manufacturing costs as we completed our GMP manufacturing activities to support the submission of an IND and future clinical trials. Expenses related to our cell therapy program decreased primarily due to the discontinuance of enrollment in our Phase 2 trial in inherited metabolic diseases in June 2020. The increase in personnel related costs was due primarily to an increase in headcount in our research and development function and an increase in stock-based compensation. Personnel related costs for the three months ended September 30, 2021 and 2020 included stock-based compensation expense of $1.3 million and $0.7 million, respectively. The increase in facility related and other was primarily due to higher operating costs related to our Cambridge, Massachusetts facility. General and Administrative Expenses

Personnel related (including stock-based compensation) $ 4,213 $ 3,539 $ 674 Professional and consultant

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Table of Contents Comparison of the Nine Months Ended September 30, 2021 and 2020 The following table summarizes our results of operations for the nine months ended September 30, 2021 and 2020:

Operating expenses: Research and development $ 33,652 $ 38,359 $ (4,707 ) General and administrative 20,900 21,278 (378 )

Interest and other income, net 2,708 2,869 (161 )

Research and Development Expenses

Direct research and development expenses by program: Conditioning

Unallocated expenses: Personnel related (including stock-based compensation) 14,193 11,120 3,073 Consultant (including stock-based compensation)

Expenses related to our conditioning program decreased primarily due to a decrease in manufacturing costs as we completed our GMP manufacturing activities to support the submission of an IND and future clinical trials. Expenses related to our cell therapy program decreased primarily due to the discontinuance of enrollment in our Phase 2 trial in inherited metabolic diseases in June 2020. The increase in personnel related costs was due primarily to an increase in headcount in our research and development function and an increase in stock-based compensation. Personnel related costs for the nine months ended September 30, 2021 and 2020 included stock-based compensation expense of $3.2 million and $2.2 million, respectively. The increase in facility related and other was primarily due to higher operating costs related to our Cambridge, Massachusetts facility. General and Administrative Expenses

Personnel related (including stock-based compensation) $ 10,508 $ 11,097 $ (589 ) Professional and consultant

The decrease in professional and consultant costs was primarily due to lower patent related costs. The increase in facility related and other was primarily due to higher operating costs related to our Cambridge, Massachusetts facility and higher directors and officers' insurance.

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Net increase in cash, cash equivalents and restricted cash $ 99,387 $ 76,609

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Edgar Online, source Glimpses

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MAGENTA THERAPEUTICS, INC. Management's Discussion and Analysis of Financial Condition and Results of Operations. (form 10-Q) - marketscreener.com

Researcher awarded $12 million for a stem cell trial to improve outcomes of young blood cancer patients – Stanford Medical Center Report

The California Institute for Regenerative Medicine has awarded nearly $12 million to support a clinical trial of a new cell-based treatment to improve outcomes and survival rates among children and young adults with blood cancer who receive a stem cell transplant.

The treatment, named T-allo10, aims to improve immune responses to pathogens and cancer without increasing the likelihood of graft-versus-host disease in patients who must receive a transplant from an imperfectly matched donor.

The trial will be led by Maria Grazia Roncarolo, MD, professor of pediatrics and of medicine. Roncarolo is the George D. Smith Professor of Stem Cell and Regenerative Medicine, director of the Stanford Center for Definitive and Curative Medicine and co-director of the Institute for Stem Cell Biology and Regenerative Medicine.

Every year around 500 children receive stem cell transplants in California, and while many children do well, too many experience a rejection of the transplant or a relapse of the cancer, Maria Millan, MD, president and CEO of the institute said in a press release. Finding an improved therapy for these children means a shorter stay in the hospital, less risk of the need for a second transplant, and a greater quality of life for the child and the whole family.

The current standard of care for many blood cancers is a two-part treatment of chemotherapy to destroy a patients cancer cells followed by a transplant of blood and immune stem cells from an immunologically matched donor. However, patients sometimes only have the option of receiving a transplant thats a partial immunological match, increasing the risk of graft-versus-host disease, in which the donor immune cells attack the recipients tissues. To reduce this risk, a subset of the donated cells is removed prior to transplant, which in turn increases the chance of a cancer relapse or dangerous infection.

Roncarolo and her team will test T-allo10, in which mature immune cells are concurrently administered with cells called type 1 regulatory T cells, or Tr1 cells, from the donor after a stem cell transplant. The Tr1 cells, which were originally discovered by Roncarolos team, reduce the likelihood that the donors immune cells will perceive the recipients tissue as foreign.

T-allo10 is intended to improve transplant outcomes by reducing cancer recurrence and infection rates, as well as the likelihood of graft-versus-host disease.

My team and I are thrilled to receive CIRMs support for our immunotherapy clinical trial, which may help patients with leukemia receiving a blood stem and progenitor cell transplant fromnonperfectly matched donors a population that continues to suffer poor outcomes and that has high unmet need, Roncarolo said. T-allo10 is unique, as it contains both Tr1 cells, which prevent graft-versus-host disease and rejection, and cells that can fight infections and eliminate residual cancer cells in a single cell product. The development from the bench to the bedside of a Tr1 cell-based product to improve the outcomes of stem cell transplant and induce tolerance is a shining example of the cutting-edge translational work conducted at the Stanford Center for Definitive and Curative Medicine.

Original post:
Researcher awarded $12 million for a stem cell trial to improve outcomes of young blood cancer patients - Stanford Medical Center Report