Stem Cell Clinic

Comparison of the efficiency, safety, and survival outcomes in two stem cell mobilization regimens with cyclophosphamide plus G-CSF or G-CSF alone in…

This article was originally published here

Ann Hematol. 2021 Jan 6. doi: 10.1007/s00277-020-04376-w. Online ahead of print.

ABSTRACT

Autologous stem cell transplantation as a frontline treatment for patients with multiple myeloma (MM) requires an adequate peripheral blood stem cell (PBSC) collection before processing. Granulocyte-colony stimulating factor (G-CSF) with or without cyclophosphamide (CTX) is a common regimen for PBSC mobilization; their benefits and risks are controversial. To compare the efficiency, safety, and survival outcomes between the two regimens, we conducted a meta-analysis including 18 studies with 4 prospective and 14 retrospective studies; a total of 2770 patients with MM were analyzed. The CTX plus G-CSF regimen had higher yields of total CD34+ cells (SMD = 0.39, 95% CI (0.30, 0.49)), and higher mobilization rates of the target 2 106/kg (OR = 3.34, 95% CI (1.82, 6.11)) and 4 106/kg (OR = 2.16, 95% CI (1.69, 2.76)) cells. A favorable event-free survival (EFS) (HR = 0.73, 95% CI (0.58, 0.93), p = 0.01) and better 3-year EFS rate (OR = 1.65, 95% CI (1.1, 2.47), p = 0.02) were also reached in the patients with CTX plus G-CSF mobilization, although the risks of admission (OR = 26.49, 95% CI (7.31, 95.97)) and fever (OR = 13.66, 95% CI (6.21, 30.03)) during mobilization were increased, the treatment-related mortality was consistent (p = 0.26). The CTX plus G-CSF regimen was superior to the G-CSF-alone regimen for PBSC mobilization in patients with MM.

PMID:33404694 | DOI:10.1007/s00277-020-04376-w

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Comparison of the efficiency, safety, and survival outcomes in two stem cell mobilization regimens with cyclophosphamide plus G-CSF or G-CSF alone in...

Top 10 ALS Stories of 2020 – ALS News Today

ALS News Today brought you daily coverage of key findings, treatment developments, clinical trials, and other important events related to amyotrophic lateral sclerosis (ALS) throughout 2020, a year marked by the COVID-19 pandemic.

As a reminder of what mattered most to you in 2020, here are the top 10 most-read articles of last year with a brief description of what made them interesting and relevant to the ALS community.

We look forward to reporting more relevant news to patients, family members, and caregivers dealing with ALS throughout 2021.

A team of researchers in Germany found that caffeine and nicotinamide adenine dinucleotide in its oxidized form (NAD+) two powerful antioxidants improved the health of lab-grown motor neurons derived from a mouse model of sporadic ALS.

These benefits, seen in cells derived from mice either in a progressive or a stable disease state, were likely associated with a reduction in oxidative stress, a known contributor to sporadic ALS.

Of note, motor neurons, the specialized nerve cells that control voluntary movement, are progressively lost in people with ALS. Oxidative stress is an imbalance between the natural production of potentially harmful reactive oxygen species and the ability of cells to detoxify them with antioxidant agents.

In an April story, we reported AB Sciences plans to launch a Phase 3 clinical trial (NCT03127267) testing its experimental oral therapy masitinib as an add-on treatment for people with ALS, after the U.S. Food and Drug Administration (FDA) cleared its request for this study.

Masitinib is designed to block the activity of multiple cell types involved in the inflammatory and neurodegenerative processes marking ALS.

The study aims to assess whether add-on treatment with masitinib is superior to placebo at slowing functional decline in up to 495 ALS patients diagnosed in the past two years. Participants functional abilities will be assessed through the ALS functional rating scale-revised (ALSFRS-R). Both masitinib and placebo will be given in combination with Sanofis Rilutek (riluzole), an approved ALS medication.

The trial is currently recruiting patients at a single U.S. clinical site(Johns Hopkins in Maryland), but another site in Ulm, Germany, is expected to open shortly. Should study findings be positive, they are expected to support future requests for regulatory approval of masitinib as an ALS treatment.

Using different mouse models of ALS, a team of researchers in the U.S. discovered a self-destructive mechanism in mitochondria the cells powerhouses that may be one of the first triggers of motor neuron degeneration in ALS.

This mitochondrial suicide was found only in the upper motor neurons those that send messages from the brain to the spinal cord, and whose degeneration is thought to be an early disease event of ALS mice, and before any signs or symptoms of the disease were evident.

These findings suggest that currently available therapies targeting mitochondrial degeneration may help to stop neurodegeneration in ALS, supporting further research in this area.

In July, BrainStorm Cell Therapeutics announced that all ALS patients enrolled in a pivotal Phase 3 clinical trial (NCT03280056) testing NurOwn, its investigational cell-based therapy, had completed dosing.

NurOwn involves expanding and maturing mesenchymal stem cells (MSCs) collected from a patients own bone marrow into cells that produce high levels of molecules promoting nerve cell growth and survival. MSCs are stem cells that can generate a variety of other cell types.

The mature cells called MSC-NTF cells are then injected into the patients spinal canal to promote and support nerve cell repair.

In the U.S.-based trial, 189 patients with rapidly progressing ALS were randomly assigned to either a total of three injections of either NurOwn, or a placebo, given directly into the spinal canal every other month.

The studys main goal was to assess the therapys safety, and whether treatment was superior to placebo at slowing disease progression as measured by the ALSFRS-R at seven months following the first dose.

A couple of months earlier, we reported the results of a preclinical study suggesting that NurOwn may not only boost nerve cell protection and repair, but also suppress the damaging immune responses that contribute to ALS progression by promoting a shift toward an anti-inflammatory state.

BrainStorm researchers found that growing healthy B-cells and T-cells immune cells known to be involved in ALS in the lab with NurOwn suppressed the growth of pro-inflammatory cell subsets, and lowered the levels of pro-inflammatory molecules. At the same time, the therapy increased the numbers of immunosuppressive cell subsets and the levels of a major anti-inflammatory molecule.

BrainStorm announced in June that patient dosing in its Phase 3 trial evaluating NurOwn in people with ALS remained on track, despite occasional treatment scheduling changes due to the COVID-19 pandemic.

The company attributed the trials successful advancement during the pandemic to coordination among its six U.S. clinical sites, support and guidance from the FDA, and the fact that its main goal based on the ALSFRS-R could be assessed by phone.

Top-line data were shared before the years end, as anticipated by BrainStorm, and are under review by the FDA.

In April, ALS News Today reported onSeneca Biopharmas plans to launch a Phase 3 clinical trial to assess the safety and effectiveness of NSI-566, its leading stem cell treatment candidate, in adults with ALS.

The decision was supported by previous positive data from a Phase 1 (NCT01348451) and Phase 2 (NCT01730716) clinical trial and a meeting with the FDA that provided guidance on how to best design and conduct the upcoming late-stage trial.

NSI-566 treatment involves the injection of fetal spinal cord stem cells into a patients spinal cord, where they mature into nerve cells that surround and support motor neurons. These mature cells also produce certain molecules that promote motor neuron growth and survival.

Results from the previous studies confirmed NSI-566s safety, and suggested that the therapy may help to prevent further functional decline in ALS patients, when compared with data from other ALS trials.

A small study in Italy suggested that creatinine kinase a marker of muscle damage could be used as a biomarker to predict the rate of disease progression in people with ALS.

By analyzing this enzyme in 126 ALS patients, the researchers found that creatinine kinase levels were significantly higher in people with slow progressing disease compared with those with fast progressing disease, and that these differences were sustained over time.

Further analyses in mouse models of ALS confirmed these findings, and suggested that the slow progression was associated with greater muscle mass and a better ability to counter disease mechanisms for longer periods.

Elevated creatinine kinase blood levels also seemed to be specific to ALS among neurodegenerative diseases, suggesting that the muscle may be a therapeutic target in ALS.

In January, we reported that a Phase 1/2a clinical trial (NCT03482050) testing AstroRx, Kadimastems investigational cell therapy, had completed dosing a second group ofALS patients.

AstroRx delivers healthy, mature astrocytes derived from human embryonic stem cells to a patients spinal cord to compensate for diseased astrocytes and to prevent motor neuron loss. Astrocytes are star-shaped cells that normally support and protect nerve cells, but are abnormal in ALS.

Data from the first group of patients given the lowest therapy dose showed that the treatment was safe and slowed the rate of disease progression over the first three to four months following dosing. Results from the second group (given a higher dose) went on toconfirm these promising three-month findings of a single treatment.

Our most-read article of 2020 concerned the discovery that an abnormal uptake of metals from chromium to zinc during childhood is associated with ALS in adults.

By analyzing teeth samples from 36 ALS patients and 31 unaffected people with a powerful technology, the researchers were able to establish and assess differences in temporal profiles of metal exposure. They found that ALS patients had greater exposure to several metals at various developmental stages, starting as early as birth.

These findings were confirmed in mouse models of ALS, both in their teeth and in their brains, suggesting that abnormal metal metabolism may contribute to several molecular changes that could increase the susceptibility of motor neurons to premature damage.

While deficiencies and excess of essential elements and toxic metals are known to contribute to ALS, researchers were now able to provide an idea of when these metabolic abnormalities start. The results also suggested that metal metabolism could be a viable therapeutic target to prevent or halt ALS.

***

At ALS News Today, we hope these stories and our reporting throughout 2021 help to better inform and improve the lives of everyone affected by ALS.

We wish all our readers a happy 2021.

Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.

Total Posts: 45

Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia. Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.

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Top 10 ALS Stories of 2020 - ALS News Today

Celularity and GX Acquisition Corp. Announce Merger Agreement to Create a Publicly Listed Leader in Allogeneic Cellular Therapy – BioSpace

FLORHAM PARK, N.J. and NEW YORK, Jan. 8, 2021 /PRNewswire/ -- Celularity Inc. ("Celularity"), a clinical-stage biotechnology company, leading the next evolution in cellular medicine with the development of off-the-shelf allogeneic therapies derived from the postpartum human placenta, and GX Acquisition Corp. (Nasdaq: GXGX), a special purpose acquisition company, today announced they have entered into a definitive merger agreement pursuant to which GX Acquisition Corp. will combine with Celularity. Upon the closing of the transaction, which is expected to occur in the second quarter of 2021, GX Acquisition Corp. will be renamed Celularity Inc., and its common stock and warrants are expected to remain listed on Nasdaq under the new ticker symbols "CELU" and "CELUW."

In addition to the approximately $292 million held in GX Acquisition Corp.'s trust account (assuming no stockholder redemptions are effected), a group of existing and other institutional investors have committed to participate in a concurrent equity financing through the sale of approximately $80 million of GX Acquisition Corp. Class A common stock at $10.00 per share. Investors in the PIPE include existing Celularity stockholders or their affiliates including Starr Insurance Companies, Dragasac Limited, Sorrento Therapeutics, as well as unaffiliated institutional investors.

Approximately $372 million of total expected proceeds from the PIPE and cash held in GX Acquisition Corp.'s trust account will be added to the combined company's balance sheet (assuming no stockholder redemptions are effected). The company will operate under the Celularity management team, led by Founder, Chairperson and Chief Executive Officer, Robert J. Hariri, M.D., Ph.D. The boards of directors of both GX Acquisition Corp. and Celularity have unanimously approved the proposed transaction. Completion of the transaction is subject to approval of both GX Acquisition Corp.'s and Celularity's stockholders and the satisfaction or waiver of certain other customary closing conditions.

"I would like to thank the team at Celularity, existing Celularity and GX Acquisition Corp. stockholders, the PIPE investors, and all our advisors for their dedication in preparing for this transaction. We anticipate that the proceeds will provide us added runway and enable us to accelerate the development of innovative, off the shelf allogeneic cell therapies, including genetically modified natural killer (NK) cell therapies and CAR T cell therapies derived from the postpartum placenta. We expect to leverage this transaction and our new state-of-the-art manufacturing facility to advance the delivery of best-in-class cell therapies to patients with unmet medical needs," said Dr. Hariri.

"At Celularity we believe the next evolution in allogeneic cell therapy entails the delivery of rapidly scalable, high quality and economical solutions. It is in the continued spirit of evolution that today we announce our plans for becoming a public company," Dr. Hariri added.

John Sculley, Vice Chairman of the Board of Celularity, former CEO of Apple Inc., and former President of Pepsi Cola, further speaks to the Company's dynamic footprint: "Bob is creating systemic change with Celularity he started with his insight of the incredible power of the placenta, something that is being discarded, as the source material to come up with immuno-oncology therapies that would touch many types of cancer, and be scalable to reach millions of people."

Dean C. Kehler, Co-Chairman and CEO at GX Acquisition Corp., added, "We are excited to partner with the management of Celularity to create a new publicly-traded cell therapy company. Most importantly, this transaction will help to continue the decades of innovation by Dr. Hariri and his seasoned team, with the goal of developing new immunotherapies to treat cancer and other diseases."

Celularity is a clinical-stage biotechnology company leading the next evolution in cellular medicine with the development of allogeneic placental-derived cell therapy products, including genetically engineered placental-derived natural killer ("NK") cells and unmodified NK cells; placental-derived T cells engineered with a chimeric antigen receptor ("CAR -T cells"); and mesenchymal-like adherent stromal cells ("ASCs"). The cell therapy products are being developed to target indications across cancer, infectious and degenerative diseases.

Celularity believes that by harnessing the placenta's unique biology and ready availability, it will be able to develop therapeutic solutions that address a significant unmet global need for effective, accessible, and affordable therapeutics.

Proceeds of the business combination and PIPE are expected to be used, among other things, to support Celularity's research and clinical development programs, including:

Celularity also plans to use the funding from the transaction to bolster the continued build-out of internal discovery capabilities, enhance business development activities and support general corporate activities.

Celularity's current science is the product of over two decades of discovery, research, and development. Celularity has its roots in Anthrogenesis Corporation ("Anthrogenesis"), a company founded in 1998 by Dr. Hariri and acquired in 2002 by Celgene Corporation ("Celgene"). The team continued to hone its discoveries and expertise in the field of placental-derived cells at Celgene through August 2017, when Celularity, led by Dr. Hariri, acquired Anthrogenesis from Celgene.

Celularity benefits from Celgene's twenty-plus years' investment in developing technologies and capabilities to enable the manufacture cellular products at scale, with consistent and reliable quality.

Celularity has a robust global intellectual property portfolio comprised of over 1,500 patents and patent applications around the Celularity IMPACT platform, covering its processes, technologies, and key cell therapy programs. In 2020, Celularity completed construction of its 150,000 square foot purpose-built manufacturing and research facility located in Florham Park, New Jersey. This facility incorporates a world-class cGMP-ready manufacturing center, research and product development laboratories and biorepository, along with dedicated office space and space for shared services. Celularity's facility includes nine Grade C/ISO-7 and six Grade D/ISO-8 manufacturing suites designed for the parallel commercial production of multiple cellular therapy products and advanced biomaterials.

Summary of Transaction

The transaction will be effected pursuant to a merger of Celularity with a wholly owned subsidiary of GX Acquisition Corp. In the merger, outstanding shares of Celularity capital stock, options and warrants will be converted into shares of common stock, options and warrants, respectively, of the combined company at an implied Celularity equity value of $1.25 billion.

Advisors

Ardea Partners LP is serving as lead financial advisor to Celularity. Morgan Stanley & Co. is also serving as a financial advisor. Truist Securities and Oppenheimer & Co. Inc. are acting as capital markets advisors to Celularity. Cooley LLP is serving as legal counsel to Celularity.

Credit Suisse is serving as lead capital markets advisor and lead private placement agent on the PIPE to GX Acquisition Corp. Cantor Fitzgerald is also serving as capital markets advisor to GX Acquisition Corp. Skadden, Arps, Slate, Meagher & Flom LLP is serving as legal counsel to GX Acquisition Corp.

Conference Call Information

January 8, 2021 at 8:00 a.m. EDT Stream Recording: https://celularity.com/joint-investor-conference-call/

About GX Acquisition Corp.

GX Acquisition Corp. is a blank check company incorporated in Delaware for the purpose of effecting a merger, share exchange, asset acquisition, share purchase, reorganization, or similar business combination with one or more businesses or entities. GX Acquisition Corp. is led by Jay R. Bloom and Dean C. Kehler, who serve as Managing Partners of Trimaran Capital Partners.

About Celularity

Celularity is a clinical stage biotechnology company leading the next evolution in cellular medicine by developing off-the-shelf placenta-derived allogeneic cell therapies, including genetically-modified NK cells, T cells engineered with a CAR (CAR T-cells), and ASCs, targeting indications across cancer, infectious and degenerative diseases. Celularity believes that by harnessing the placenta's unique biology and ready availability, it will be able to develop therapeutic solutions that address a significant unmet global need for effective, accessible, and affordable therapeutics. Celularity currently has four active and enrolling clinical trials and plans to submit three additional investigational new drug ("IND") applications in 2021. The Celularity IMPACT platform capitalizes on the benefits of placental-derived cells to target multiple diseases, and provides seamless integration, from bio-sourcing through manufacturing cryopreserved and packaged allogeneic cells, which Celularity handles at its purpose-built U.S.-based 150,000 square foot facility in Florham Park, NJ. Celularity believes the use of placental-derived cells sourced from full-term healthy informed consent donors have potential inherent advantages, both from an economic and a scientific perspective. Relative to adult-derived cells, placental-derived cells demonstrate greater stemness, which means the ability to expand and persist. Further, their immunological navet, meaning having an immune system that has never been exposed to a specific antigen, may allow for an improved safety profile. Celularity's placental-derived cells are allogeneic, meaning they are intended for use in any patient, as compared to autologous cells, which are derived from an individual patient for that patient's sole use. Celularity believes this a key difference that will enable readily available off-the-shelf treatments that can be delivered faster, more reliably, at greater scale and to more patients.

Additional Information about the Business Combination and Where to Find It

GX Acquisition Corp. intends to file the Registration Statement with the SEC, which will include a preliminary proxy statement to be distributed to holders of GX Acquisition Corp.'s common stock in connection with GX Acquisition Corp.'s solicitation of proxies for the vote by GX Acquisition Corp.'s stockholders with respect to the business combination and other matters as described in the Registration Statement, and a prospectus relating to the offer of the securities to be issued to Celularity's stockholders in connection with the business combination. After the Registration Statement has been filed and declared effective, GX Acquisition Corp. will mail a definitive proxy statement and other relevant documents to its stockholders as of the record date established for voting on the business combination and the other proposals regarding the business combination set forth in the Registration Statement.GX Acquisition Corp.'s stockholders and other interested persons are advised to read, once available, the Registration Statement, including the preliminary proxy statement / prospectus contained therein, and any amendments thereto and, once available, the definitive proxy statement / prospectus, in connection with GX Acquisition Corp.'s solicitation of proxies for its special meeting of stockholders to be held to approve, among other things, the business combination, because these documents will contain important information about GX Acquisition Corp., Celularity and the business combination.Stockholders may also obtain a copy of the preliminary or definitive proxy statement/prospectus, once available, as well as other documents filed with the SEC regarding the business combination and other documents filed with the SEC by GX Acquisition Corp., without charge, at the SEC's website located at http://www.sec.gov or by directing a request to GX Acquisition Corp., 1325 Avenue of the Americas, 25th Floor, New York, NY 10019.

Participants in the Solicitation

GXAcquisition Corp., Celularity and their respective directors and officers may be deemed participants in the solicitation of proxies of GXAcquisition Corp.'s stockholders in connection with the business combination. GXAcquisition Corp.'s stockholders and other interested persons may obtain, without charge, more detailed information regarding the directors and officers of GXAcquisition Corp. in GXAcquisition Corp.'s Annual Report onForm10-Kfor the fiscal year ended December31, 2019, which was filed with the SEC on March24, 2020, and GXAcquisition Corp.'s Definitive Proxy Statement on Schedule 14A, which was filed with the SEC on December 4, 2020. Information regarding Celularity's directors and officers will be set forth in the Registration Statement for the business combination.

Information regarding the persons who may, under SEC rules, be deemed participants in the solicitation of proxies of GXAcquisition Corp.'s stockholders in connection with the business combinationand other matters to be voted upon at the special meeting will be set forth in the Registration Statement for the business combination. Additional information regarding the interests of participants in the solicitation of proxies in connection with the business combinationwill be included in the Registration Statement for the business combination.

Non-Solicitation

This press release is not a proxy statement or solicitation of a proxy, consent or authorization with respect to any securities or in respect of the potential transaction and shall not constitute an offer to sell or a solicitation of an offer to buy the securities of Celularity, the combined company or GX Acquisition Corp., nor shall there be any sale of any such securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of such state or jurisdiction. No offer of securities shall be made except by means of a prospectus meeting the requirements of the Securities Act.

Special Note Regarding Forward-Looking Statements

This press release contains, or incorporates by reference, "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. Forward-looking statements may include, but are not limited to, statements regarding GX Acquisition Corp.'s, GX Acquisition Corp.'s management team's, Celularity's and Celularity's management team's expectations, hopes, beliefs, intentions, or strategies regarding the future. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words "anticipate," "believe," "can," "contemplate," "continue," "could," "estimate," "expect," "forecast," "intends," "may," "might," "outlook," "plan," "possible," "potential," "predict," "project," "seek," "should," "strive," "target," "will," "would" and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Forward-looking statements in this press release may include, for example: (i) the ability to consummate the business combination, (ii) the expected benefits of the business combination; (iii) the financial and business performance of Celularity, (iv) the inability to complete the PIPE Investment; (v) the success and timing of Celularity's cellular therapeutic development activities and initiating clinical trials; (vi) the success and timing of Celularity's planned clinical trials; (vii) Celularity's ability to obtain and maintain regulatory approval of any of Celularity's therapeutic candidates; (viii) Celularity's plans to research, discover and develop additional therapeutic candidates, including by leveraging genetic engineering and other technologies and expanding into additional indications; (ix) Celularity's ability to expand its manufacturing capabilities, and to manufacture Celularity's therapeutic candidates and scale production; (x) Celularity's ability to meet certain milestones; (xi) changes in Celularity's strategy, future operations, financial position, estimated revenues and losses, projected costs, prospects and plans; (xii) the implementation, market acceptance and success of Celularity's business model; (xiii) developments and projections relating to Celularity's competitors and industry; (xiv) the impact of health epidemics, including the COVID-19 pandemic, on Celularity's business and the actions Celularity may take in response thereto; (xv) Celularity's expectations regarding its ability to obtain and maintain intellectual property protection and not infringe on the rights of others; (xvi) expectations regarding the time during which GX Acquisition Corp. will be an emerging growth company under the JOBS Act; (xvii) Celularity's future capital requirements and sources and uses of cash; (xviii) Celularity's ability to obtain funding for its operations; (xix) Celularity's business, expansion plans and opportunities; and (xx) the outcome of any known and unknown litigation and regulatory proceedings. These forward-looking statements are based on information available as of the date of this press release, and current expectations, forecasts and assumptions, and involve a number of judgments, risks and uncertainties. These risks and uncertainties may be amplified by the COVID- 19 pandemic, which has caused significant economic uncertainty. If any of these risks materialize or underlying assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. There may be additional risks that neither GX Acquisition Corp. nor Celularity presently know, or that GX Acquisition Corp. or Celularity currently believe are immaterial, that could also cause actual results to differ from those contained in the forward-looking statements. In addition, forward-looking statements reflect GX Acquisition Corp.'s and Celularity's expectations, plans, or forecasts of future events and views as of the date of this press release. GX Acquisition Corp. and Celularity anticipate that subsequent events and developments will cause GX Acquisition Corp.'s and Celularity's assessments to change. Accordingly, forward-looking statements should not be relied upon as representing GX Acquisition Corp.'s or Celularity's views as of any subsequent date, and GX Acquisition Corp. does not undertake any obligation to update forward-looking statements to reflect events or circumstances after the date they were made, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws. Additional risks and uncertainties are identified and discussed in GX Acquisition Corp.'s reports filed with the SEC and available at the SEC's website at http://www.sec.gov.

GX Acquisition Corp. Contact:

Caroline Luz Lambert & Co. cluz@lambert.com

Celularity Investor Contacts:

Carlos Ramirez Celularity carlos.ramirez@celularity.com

Alexandra Roy Solebury Trout aroy@troutgroup.com

Celularity Media Contact:

media@celularity.com

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Celularity and GX Acquisition Corp. Announce Merger Agreement to Create a Publicly Listed Leader in Allogeneic Cellular Therapy - BioSpace

Results Show Promising Efficacy in MCL With Next-Generation BTK Inhibitor LOXO-305 – Cancer Network

Results of a phase 1/2 trial that were presented at the 2020 American Society of Hematology Annual Meeting and Exposition showed that patients with mantle cell lymphoma (MCL) and other B-cell malignancies had promising response rates when treated with LOXO-305.

A highly selective Bruton tyrosine kinase (BTK) inhibitor that targets both wild-type and C481-mutant BTK, LOXO-305 could be a viable treatment option for patients with difficult-to-treat hematologic malignancies, such as MCL and Waldenstrm macroglobulinemia (WM).

LOXO-305 demonstrates promising efficacy in [patients with MCL and other non-Hodgkin lymphomas (NHLs)] previously treated with all classes of available therapy, Michael Wang, MD, professor in the Department of Lymphoma & Myeloma at The University of Texas MD Anderson Cancer Center, said during a presentation of the data. Responses were observed across all dose levels and efficacy was independent of prior therapy.

The agents use in patients with various B-cell malignancies, including those with chronic lymphocytic leukemia/small lymphocytic lymphoma, was examined in the dose-escalation, dose-expansion BRUIN trial (NCT03740529). Patients had to have received at least 2 prior regimens, given as a combination or sequentially, or 1 prior BTK inhibitorcontaining regimen. LOXO-305 was administered orally in cycles of 28 days according to a 3 + 3 design, with a starting dose of 25 mg daily.

The primary end points of the trial were determining the maximum tolerated dose in the phase 1 portion and preliminary antitumor activity and safety in phase 2. In total, 323 patients were enrolled as of the September 2020 cutoff, 61 of whom had MCL, 26 with WM, and 66 with other NHLs.

The median number of prior therapies for patients with MCL and WM was 3, of whom 93% and 69%, respectively, previously received a BTK inhibitor. Those with other NHLs received an average of 4 prior therapies. Interestingly, progressive disease following chimeric antigen receptor (CAR) T-cell therapy was seen in 5% of patients with MCL and 14% of those with other NHLs.

Overall response rates (ORR) varied by disease cohort. Of note, those with MCL who were evaluable for response (n = 56) had an ORR of 52%, and this remained true when the cohort of patients was reduced to just those who had prior BTK inhibitor therapy (n = 52). Complete responses accounted for 25% of both groups, as well. In those with prior stem cell transplant (n = 14) or prior CAR T-cell therapy (n = 2), ORRs were 64% and 100%, respectively. At a median follow-up of 6 months, most of those with MCL who responded to therapy (83%; 24 of 29 patients) had an ongoing response.

In response-evaluable patients with WM (n = 19), the ORR was 68% and was consistent when only those with prior BTK inhibitor therapy were examined (n = 13; 69%). Responses in both groups were comprised exclusively of partial responses and 77% of patients overall had an ongoing response at data cutoff. ORRs in other NHL subtypes included 75% for Richter transformation (RT), 50% for follicular lymphoma, 24% for diffuse large B-cell lymphoma, and 22% for marginal zone lymphoma. A large majority of patients with RT (83%) had an ongoing response at the median follow-up of 6 months.

Of note, there were no dose-limiting toxicities noted in the study and the maximum-tolerated dose was not reached. Only 5 patients (1.5%) discontinued treatment due to treatment-related adverse events and 200 mg was selected as the recommended phase 2 dose.

Longer follow-up is needed to better understand the LOXO-305 safety profile associated with chronic administration, Wang said.

Reference:

1. Wang M, Shah NN, Alencar AJ, et al. LOXO-305, a next generation, highly selective, non-covalent BTK inhibitor in previously treated mantle cell lymphoma, Waldenstrm's macroglobulinemia, and other non-Hodgkin lymphomas: results from the phase 1/2 BRUIN study. Blood. 2020;136(suppl 1):8-10. Abstract 117. doi:10.1182/blood-2020-134314

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Results Show Promising Efficacy in MCL With Next-Generation BTK Inhibitor LOXO-305 - Cancer Network

BlueRock Therapeutics in Collaboration with Memorial Sloan Kettering Cancer Center Receives IND Clearance for DA01 in Parkinson’s Disease – BioSpace

CAMBRIDGE, Mass., Jan. 7, 2021 /PRNewswire/ -- BlueRock Therapeutics, a preclinical stage biopharmaceutical company and wholly-owned subsidiary of BayerAG,in collaboration withMemorialSloan Kettering Cancer Center(MSK), announcedtodaythat the U.S. Food and Drug Administration (FDA) has clearedtheirInvestigational New Drug (IND) application toproceedwith aPhase 1(Ph1)study in patients with advanced Parkinson's disease (PD).This is the first trial in the United States to study pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson's disease.Under the IND, BlueRockand MSK willexecute aPh1 clinical trial to evaluate the safety, tolerability andpreliminaryefficacyof DA01 in patients with PD.

"This is a big step for the stem cell field to finally test a truly "off-the-shelf" dopamine neuron product in human PD patients," said Lorenz Studer, MD, scientific co-founder of BlueRock and Director, Center for Stem Cell Biology at MSK. "We are also grateful for the visionary support by NYSTEM, the NY state-sponsored stem cell program that supported the earlier stages of this project."

"This trial is the culmination of a decade of arduous collaborative work that is based on very rigorous science. It is an important milestone on the road towards regenerative brain repair," said Viviane Tabar, MD, founding investigator of BlueRock and Chair of MSK's Department of Neurosurgery. "It is a real privilege and very exciting to be able to participate in both the bench science and the actual surgical intervention, here at MSK. Our collaborators at Weill Cornell Neurology will also be an integral part of the trial."

"Today, thereisno disease-modifying treatment for Parkinson's. Through this trial and those to follow, we hope to change that,"stated EmileNuwaysir, Ph.D., President and Chief Executive Officer of BlueRock. "Our therapy is intended to replace the midbrain dopaminergic neurons lost in the degenerative condition to rebuild the neural circuit, and thereby restore motor controlto Parkinson's patients. This could shift the treatment paradigm for millions ofPD patients, as well as demonstrate for the first time that degenerative disease is, in principle, reversible. We believe this would represent an enormous step forthe PD community worldwide, and formedicine."

The trial plans to enroll ten patients starting with a first clinical site at Weill Cornell Medicinein the initial open-label study. The primary objective of thePh1study is to assess the safety and tolerability of DA01 cell transplantation at one-year post-transplant. The secondary objectives of the study are to assess the evidence of transplanted cell survival and motor effects at one- and two-years post-transplant, to evaluate continued safety and tolerability at two years, and to assess feasibility of transplantation.

About Parkinson's Disease Parkinson's disease is a progressive neurodegenerative disorder caused by nerve cell damage in the brain, leading to decreased dopamine levels. The worsening of motor and non-motor symptoms is caused by the loss of dopamine-producing neurons. At diagnosis, it is estimated that patients have already lost 60-80% of their dopaminergic neurons.Parkinson's disease often starts with a tremor in one hand. Other symptoms are rigidity, cramping and dyskinesias. Parkinson's disease is the second most common neurodegenerative disorder, impacting more than 7.5 million people, including 1.3 million people in North America.

About BlueRock Therapeutics BlueRock Therapeutics is a leading engineered cell therapy company with a mission to develop regenerative medicines for intractable diseases. BlueRock Therapeutics' cell+gene platform harnesses the power of cells to create new medicines for neurology, cardiology and immunology indications. BlueRock Therapeutics' cell differentiation technology recapitulates the cell's developmental biology to produce authentic cell therapies, which are further engineered for additional function. Utilizing these cell therapies to replace damaged or degenerated tissue brings the potential to restore or regenerate lost function. BlueRock Therapeutics was founded in 2016 by Versant Ventures and Bayer AG and capitalized with one of the largest-ever Series A financings in biotech history by Bayer AG (through its Leaps by Bayer unit) and Versant Ventures. The company was fully acquired by Bayer in 2019. BlueRock Therapeutics' culture is defined by scientific innovation, the highest ethical standards and an urgency to bring transformative treatments to all who would benefit. For more information, visit bluerocktx.com.

About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, the Group aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2019, the Group employed around 104,000 people and had sales of 43.5 billion euros. Capital expenditures amounted to 2.9 billion euros, R&D expenses to 5.3 billion euros. For more information, visit bayer.com.

Disclosures Dr. Studer has intellectual property rights and interests and financial interests related to BlueRock. Dr. Tabar has financial interests related to BlueRock. Researchers at Memorial Sloan Kettering Cancer Center, including Dr. Studer, developed stem cell-derived dopaminergic neurons for the treatment of neurodegenerative diseases, and MSK licensed this intellectual property to BlueRock. MSK has institutional financial interests related to this intellectual property and BlueRock.

Forward-Looking Statements Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as "anticipate," "believe," "forecast," "estimate" and "intend," among others. These forward-looking statements are based on BlueRock's current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the timing of our clinical trial for DA01; our results regarding the safety, tolerance and efficacy of DA01 cell transplantation for patients with Parkinson's disease; and ongoing FDA and other regulatory requirements regarding the development of DA01. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Except as expressly required by law, BlueRock does not undertake an obligation to update or revise any forward-looking statement. All of the Company's forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date hereof.

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Stem Cell Therapy Market Size, Share & Trends Analysis Report By Product Types, And Applications Forecast To 2026 – Farming Sector

A newly researched report on global Stem Cell Therapy market added recently concludes that the growth prospects are likely to remain robust in the coming years.

Systematic research to unravel historical developments suggest that the market has been recurrently displaying high end growth in the past and is therefore assumed to continue the same in the forecast tenure. In-depth research opines that CAGR valuation in percentage is likely to remain highly plush allowing impressive growth outlook through 2021-25.

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The primary focus of this highly versatile research presentation is to aid decision making potential of top stalwarts besides the new aspirants and novice investors striving to carve a suitable business stance despite high intensity competition as well as sudden and unprecedented odds and challenges.

For utmost reader discretion and subsequent profit-oriented business discretion, this section of the report also includes a dedicated section on pre-and post COVID-19 analysis and beyond, thus encouraging futuristic planning.

Report Deliverable: Major Highlights

On the back of thorough and detailed market research practices aligning with international research standards, this intensive Stem Cell Therapy market synopsis is a thorough summation and collated business information depot, meticulously compiled from multiple sources to meet highest accuracy in report development.

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Some of the most prominent information presented in the report include the following:

1. A detailed and systematic estimation of market size and dimensions across regional, global and local levels to unravel offbeat growth opportunities 2. A thorough synopsis of dominant as well as emerging trends in Stem Cell Therapy market 3. Complete geographical detailing in terms of most popular manufacturing practices, growth highlights and high potential end-user engagement 4. Futuristic milestones such as future-ready opportunities and disruptions based on technological leaps, product and service diversification have been thoroughly highlighted in this report.

Stem Cell Therapy Market Segmentation

Type Analysis of Stem Cell Therapy Market:

Based on cell source, the market has been segmented into,

Adipose Tissue-Derived Mesenchymal SCs Bone Marrow-Derived Mesenchymal SCs Embryonic SCs Other Sources

Applications Analysis of Stem Cell Therapy Market:

Based on therapeutic application, the market has been segmented into,

Musculoskeletal Disorders Wounds & Injuries Cardiovascular Diseases Gastrointestinal Diseases Immune System Diseases Other Applications

Reader Queries Addressed in Brief:

1. Besides the global perspective, the report includes discernible information on growth estimations defined in both volume and value-based indices 2. A close review of all growth catalysts as well as systematic understanding of major deterrents that stun growth 3. The report is based on high precision research practices to make optimum forecast predictions and CAGR valuations for the growth span, 2021-25 4. The report entails crucial market specific information encapsulation detailed vendor profiles, region-wise updates as well as a thorough market overview and executive summary section to entice lucrative business outcome.

Our in-house research professionals have deployed multiple information scavenging practices to decode classified information from multiple data sources such as international journals, corporate houses as well as annual reports of various companies.

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Stem Cell Therapy Market Size, Share & Trends Analysis Report By Product Types, And Applications Forecast To 2026 - Farming Sector

Stem Cell Therapy Market by Treatment,Application,End Users and Geography Forecast To 2027 – LionLowdown

Stem Cell Therapy Market is expected to reach 202.77 billion by 2027 from XX billion in 2019 at CAGR of XX %.

The report includes the analysis of impact of COVID-19 lock-down on the revenue of market leaders, followers, and disrupters. Since lock down was implemented differently in different regions and countries, impact of same is also different by regions and segments. The report has covered the current short term and long term impact on the market, same will help decision makers to prepare the outline for short term and long term strategies for companies by region.

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Stands for use of stem cells to treat or prevent disease or condition.Bone marrow transplant and some therapies derived from umbilical cord blood are mainly used in stem cell therapy. Advancement, in order to establish new sources for stem cells, and to apply stem-cell treatments for neurodegenerative diseases and conditions such as diabetes, heart disease, and other conditions, are increased in recent years.

Stem Cell Therapy Market Researchers are making efforts to discover novel methods to create human stem cells. This will increase the demand as well as supply for stem cell production and potential investigation in disease management. Increasing investment & research grants for developing safe and effective stem cell therapy products, the growing patient base for target diseases, concentrated product pipelines, increasing approval of the new clinical trials, rapid technological advancement in genomics, and the rising awareness about the stem cell are expected to drive the growth of the Stem Cell Therapy solutions market during the forecast period.

However, improper infrastructure, insufficient storage systems, nascent technology in underdeveloped economies, Ethical issues related to an embryonic stem cell, low patient acceptance rate, Difficulty in the preservation of stem cell are expected to restrain the market growth. North America is expected to be the largest growing region by 2026; the reason behind that is extensive funding by Government. However, Emerging countries like India, china, Korea have low growth rate as compared to Developed regions in 2017 but increase in awareness about stem cell therapy will lead the Asia Pacific to generate a significant level of revenue by 2026.

Key Highlights of Stem Cell Therapy Market report

Detailed quantitative analysis of the current and future trends from 2017 to 2026, which helps to identify the prevailing market opportunities. Comprehensive analysis of factors instrumental in changing the market scenario, rising prospective opportunities, market shares, core competencies in terms of market development, growth strategies and identification of key companies that can influence this market on a global and regional scale. Assessment of Market definition along with the identification of key drivers, restraints opportunities and challenges for this market during the forecast period. Complete analysis of micro-markets with respect to individual growth trends, prospects, and contributions to the overall Stem Cell Therapy Solutions market. Stem Cell Therapy market analysis and comprehensive segmentation with respect to the Application, End users, Treatment, and geography to assist in strategic business planning. Stem Cell Therapy market analysis and forecast for five major geographies-North America, Europe, Asia Pacific, Middle East & Africa, Latin America, and their key regions. For company profiles, 2017 has been considered as the base year. In cases, wherein information was unavailable for the base year, the years prior to it have been considered.

Research Methodology:

The market is estimated by triangulation of data points obtained from various sources and feeding them into a simulation model created individually for each market. The data points are obtained from paid and unpaid sources along with paid primary interviews with key opinion leaders (KOLs) in the market. KOLs from both, demand and supply side were considered while conducting interviews to get an unbiased idea of the market. This exercise was done at a country level to get a fair idea of the market in countries considered for this study. Later this country-specific data was accumulated to come up with regional numbers and then arrive at a global market value for the stem cell therapy market. Key Players in the Stem Cell Therapy Market are:

Chiesi Farmaceutici S.P.A Are: Gamida Cell ReNeuron Group, plc Osiris Therapeutics, Inc. Stem Cells, Inc. Vericel Corporation. Mesoblast, Ltd.

Key Target Audience:

Stem Cell Associations and Organizations Government Research Boards and Organizations Research and consulting firms Stem Cell Therapy Market Investors Healthcare Service Providers (including Hospitals and Diagnostic Centers) Stem Cell Therapeutic Product Manufacturing Organizations Research Labs Clinical research organizations (CROs) Stem Cell Therapy Marketing Players Pharmaceutical Product Manufacturing Companies

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Scope of the Stem Cell Therapy Market Report:

Stem Cell Therapy market research report categorizes the Stem Cell Therapy market based on Application, End users, Treatment, and geography (region wise). Market size by value is estimated and forecasted with the revenues of leading companies operating in the Stem Cell Therapy market with key developments in companies and market trends. Stem Cell Therapy Market, By Treatments:

Allogeneic Stem Cell Therapy Autologous Stem Cell Therapy

Stem Cell Therapy Market, By End Users:

Hospitals Ambulatory Surgical Centers

Stem Cell Therapy Market, By Application:

Oncology Central Nervous System Diseases Eye Diseases Musculoskeletal Diseases Wound & Injuries Metabolic Disorders Cardiovascular Disorders Immune System Disorders Stem Cell Therapy Market, By Geography:

North America Europe Asia Pacific Middle East & Africa Latin America

Available Customization:

With the given market data, Maximize Market Research offers customization of report and scope of the report as per the requirement

Regional Analysis:

Breakdown of the North America stem cell therapy market Breakdown of the Europe stem cell therapy market Breakdown of the Asia Pacific stem cell therapy market Breakdown of the Middle East & Africa stem cell therapy market Breakdown of the Latin America stem cell therapy market

MAJOR TOC OF THE REPORT

Chapter One: Stem Cell Therapy Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Stem Cell Therapy Market Competition, by Players

Chapter Four: Global Stem Cell Therapy Market Size by Regions

Chapter Five: North America Stem Cell Therapy Revenue by Countries

Chapter Six: Europe Stem Cell Therapy Revenue by Countries

Chapter Seven: Asia-Pacific Stem Cell Therapy Revenue by Countries

Chapter Eight: South America Stem Cell Therapy Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Stem Cell Therapy by Countries

Chapter Ten: Global Stem Cell Therapy Market Segment by Type

Chapter Eleven: Global Stem Cell Therapy Market Segment by Application

Chapter Twelve: Global Stem Cell Therapy Market Size Forecast (2019-2026)

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Maximize Market Research provides B2B and B2C market research on 20,000 high growth emerging technologies & opportunities in Chemical, Healthcare, Pharmaceuticals, Electronics & Communications, Internet of Things, Food and Beverages, Aerospace and Defense and other manufacturing sectors.

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Stem Cell Therapy Market by Treatment,Application,End Users and Geography Forecast To 2027 - LionLowdown

Stem Cell Therapy Market Analysis, COVID-19 Impact,Outlook, Opportunities, Size, Share Forecast and Supply Demand 2021-2025 – Farming Sector

Global Stem Cell Therapy market research report is an exceptional guide to encourage accurate market-based understanding, thorough trend assessment as well as`product utilization to ensure optimum competitive advantage for delivering accurate customer behavior and user preferences. The report is in place to allow market players plan and deliver novel business strategies and tactical business output to sustain gradual evolution into a better business entity. The report is a highly reliable and unbiased data presentation which has been prepared following the highest standards of research endeavors by our in-house research activities undertaken by seasoned research practitioners. Further, to induce logical decision making, data sets are presented in the form of charts, graphs and tables to incur maximum benefits.

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Vendor Landscape

The report has been meticulously drafted to ensure intensive business decision making, strategy building as well as inventory management and delivery of thought provoking business decisions. The report sheds ample light on specific market developments and participant competencies, comprising specific data on vendor growth objectives, followed by an in-depth SWOT assessment to highlight core market strengths and player competencies. Details about their threat probabilities as well as weaknesses have also been highlighted, besides identifying their strengths and opportunities based on which market players in global Stem Cell Therapy market may deliver growth proficient business discretion.

The complete value chain and downstream and upstream essentials are scrutinized in this report. Essential trends like globalization, growth progress boost fragmentation regulation & ecological concerns. This Market report covers technical data, manufacturing plants analysis, and raw material sources analysis of Stem Cell Therapy Industry as well as explains which product has the highest penetration, their profit margins, and R&D status. The report makes future projections based on the analysis of the subdivision of the market which includes the global market size by product category, end-user application, and various regions.

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Regional Diversification

Regional growth developments and identification of growth potential hotbeds have been specifically identified to ensure thoughtful business decisions across these areas in particular. The growth stimulation mettle of the growth hotbeds have been adjudged on the basis of several core parameters such as vendor activities, manufacturing preferences, customer preferences and eventual investments in promotional activities and advertising, based on which futuristic business decisions are well designed and deployed.

To ensure high ROI based business decisions, this report highlights core areas such as Canada, Mexico, and the US as top North American with promising growth mettle. Based on European growth proficient developments, Netherlands, Russia, Italy and the UK are spotted as chief growth hotbeds.

Across APAC, Japan, China, India, South Korea and other Southeast Asian countries are flagged as top investment areas. Additionally, the report also suggests that MEA and South America also showcases ample growth mettle, thus unleashing novel growth opportunities in the regions.

Stem Cell Therapy Market Segmentation

Type Analysis of Stem Cell Therapy Market:

Based on cell source, the market has been segmented into,

Adipose Tissue-Derived Mesenchymal SCs Bone Marrow-Derived Mesenchymal SCs Embryonic SCs Other Sources

Applications Analysis of Stem Cell Therapy Market:

Based on therapeutic application, the market has been segmented into,

Musculoskeletal Disorders Wounds & Injuries Cardiovascular Diseases Gastrointestinal Diseases Immune System Diseases Other Applications

Reasons for Report Investment:

1. This global Stem Cell Therapy market research report analyzed on the basis of regional. product-based and end-use applications have been presented with thorough value-based and volume centric elements 2. The report categorically highlights prominent growth steering factors as well as major limitations containing revenue maximization and growth stability 3. The report also renders a clear perspective of prominent market opportunities and growth conducive stimuli 4. The commercial landscape of global Stem Cell Therapy market has been innately discussed at length to identify market players and their proficiencies.

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Contact Us :

Ryan Johnson Account Manager Global 3131 McKinney Ave Ste 600, Dallas, TX75204, U.S.A. Phone No.: USA: +1 972-362 -8199/ +91 9665341414

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AGC Biologics Confirms Cell and Gene Therapy Commercial Expertise as Manufacturer of Orchard Therapeutics’ Newly Approved Libmeldy – PRNewswire

SEATTLE, Jan. 4, 2021 /PRNewswire/ -- AGC Biologics, a leading global Biopharmaceutical Contract Development and Manufacturing Organization (CDMO), is the first manufacturer of Orchard Therapeutics' Libmeldy, which was recently approved by the European Commission (EC) as a one-time therapy for eligible patients with early-onset Metachromatic Leukodystrophy (MLD).

The EC has granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy manufactured at AGC Biologics' Milan facility. Orchard Therapeutics is already underway with EU launch preparations to support commercial-scale drug manufacturing at the AGC Biologics Milan facility.

"AGC Biologics is delighted to have successfully partnered with Orchard Therapeutics on this great milestone," says AGC Biologics Chief Business Officer, Mark Womack. "We're very proud to be one of few Cell and Gene Therapy CDMOs with commercial experience Libmeldy being the third commercial product we've manufactured and we look forward to continuing our partnership with Orchard to provide this life-changing therapy to those affected by MLD."

"We are very honored to have been a part of the entire clinical journey of this product, from the very first treated patient, all the way to market approval. As manufacturer of both lentiviral vector and drug product, we are proud to support Orchard in treating this devastating disease," says AGC Biologics General Manager of Milan, Luca Alberici.

"The EC approval of Libmeldy opens up tremendous possibilities for eligible MLD children faced with this devastating disease," saysBobby Gaspar, M.D., Ph.D., Chief Executive Officer of Orchard. "We are humbled by the opportunity to bring this remarkable innovation to young eligible patients in the EU, and confident in the capabilities of our partners at AGC Biologics to help us achieve this goal."

MLD is a rare neurodegenerative disorder caused by mutations in the ARSA gene. While there are several forms of MLD, the disorder primarily affects young children and disease progression and life expectancy varies based on age of symptom onset. Libmeldy is designed to correct the genetic cause of MLD by inserting functional copies of the ARSA gene into the genome of a patient's own hematopoietic stem cells (HSCs) using a self-inactivating (SIN) lentiviral vector. It is approved in the EU for 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.

About AGC Biologics: AGC Biologics is a leading global biopharmaceutical Contract Development and Manufacturing Organization (CDMO) with a strong commitment to deliver the highest standard of service as we work side-by-side with our clients and partners, every step of the way. We provide world-class development and manufacture of mammalian and microbial-based therapeutic proteins, plasmid DNA (pDNA), viral vectors and genetically engineered cells. Our global network spans the U.S., Europe and Asia, with cGMP-compliant facilities in Seattle, Washington; Boulder, Colorado; Copenhagen, Denmark; Heidelberg, Germany; Milan, Italy; and Chiba, Japan and we currently employ more than 1,600 employees worldwide. Our commitment to continuous innovation fosters the technical creativity to solve our clients' most complex challenges, including specialization in fast-track projects and rare diseases. AGC Biologics is the partner of choice. To learn more, visit http://www.agcbio.com.

About Orchard Therapeutics:Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSK's 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. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us onTwitterandLinkedIn.

About OTL-200/Libmeldy:Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, is approved in the European Union (as appropriate may add: "UK, Iceland, Liechtenstein and Norway" as an alternative can rewrite as "was approved by the European Commission" or something similar) 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 (AAA). 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).

Libmeldy is not approved outside of the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy, which has not been approved by the U.S. Food and Drug Administration or any other health authority.

OTL-200 was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

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http://www.agcbio.com

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AGC Biologics Confirms Cell and Gene Therapy Commercial Expertise as Manufacturer of Orchard Therapeutics' Newly Approved Libmeldy - PRNewswire

Forge Biologics Announces FDA Clearance of Investigational New Drug Application for Phase 1/2 Clinical Trial (RESKUE) of FBX-101 Gene Therapy for…

COLUMBUS, Ohio, Jan. 4, 2021 /PRNewswire/ --Forge Biologics Inc., a gene therapy manufacturing and development company, today announced that the company has received FDA clearance of the Investigational New Drug (IND) to initiate a Phase 1/2 clinical trial evaluating its novel, first-in-human AAV gene therapy, FBX-101, for patients with Krabbe disease. FBX-101 is the company's first therapeutic program to receive an IND which also received Institutional Biosafety Committee (IBC) and Institutional Review Board (IRB) approvals required prior to any patient enrollment. This marks a major step forward in building out the company's hybrid model as a gene therapy manufacturing and development engine.

Krabbe disease is a rare and fatal pediatric leukodystrophy affecting about 1-2.6 in 100,000 people in the United States. Patients are born with mutations in the galactosylceramidase(GALC) gene, which encodes an enzyme that helps break down lipid molecules inside cells. This results in the toxic buildup of psychosine, a lipid molecule that can't be degraded in cells, particularly in cells in the brain and peripheral nerves, and leads to toxic levels that cause cell death and myelin loss. The disease initially presents as physical delays in development, muscle tightness and irritability, and rapidly advances to difficulty swallowing and breathing, loss of vision and hearing, and increasing cognitive degeneration. Early onset, or "Infantile", Krabbe disease cases usually results in death by age 2-4 years, while later onset or "Late Infantile" cases have a more variable course of progressive decline. There is currently no approved treatment for either form of Krabbe disease.

FBX-101 is an adeno-associated viral (AAV) gene therapy administered after hematopoietic stem cell transplant (HSCT), that delivers a functioning copy of the GALC gene, the enzyme needed to prevent the buildup of psychosine in myelinated cells of both the central and peripheral nervous system. FBX-101 has been shown to correct the central and peripheral myelination deficits, significantly improve the behavioral impairments associated with Krabbe disease in animal models, and drastically improve the lifespan of treated animals. The use of transplant and intravenous AAV gene therapy infusion has the potential to overcome some of the immunological safety challenges of traditional AAV gene therapies.

"The ground-breaking treatment approach using HSCT and AAV gene therapy, initially developed by Dr. Escolar, has safely demonstrated superior benefits in preclinical animal studies of Krabbe disease than either treatment method alone," said Timothy J. Miller, Ph.D., Forge's CEO, President, and Co-Founder. "We are grateful for the FDA's engaged review and allowance of the IND, and look forward to enrolling patients very soon."

FBX-101 is the culmination of nearly 20 years of Krabbe disease research, led by Maria Escolar, M.D., M.S., Chief Medical Officer at Forge Biologics and the pioneer for evaluating the natural history and new treatment approaches for patients with Krabbe disease. "This combination approach is extremely exciting because the preclinical data demonstrate significant correction of survival, behavior and neuromuscular function in animal models compared to either transplant or AAV treatment alone. This is a significant milestone for Krabbe disease families suffering from this deadly disease," said Dr. Escolar.

The initiation of the RESKUE trial in Forge's gene therapy pipeline continues Forge's momentum within the biotechnology industry in Columbus, Ohio, bringing positive impact to both Ohio and the global rare disease community.

Patients and families can learn more about clinical trials for FBX-101 by visiting https://www.forgebiologics.com/science/#krabbe.

About Krabbe diseaseKrabbe disease is a rare, pediatric leukodystrophy affecting about 1-2.6 in 100,000 people in the U.S. and is inherited in an autosomal recessive manner. Krabbe disease is caused by loss-of-function mutations in the galactosylceramidase (GALC) gene, a lysosomal enzyme responsible for the breakdown of certain types of lipids such as psychosine. Without functional GALC, psychosine accumulates to toxic levels in cells. The psychosine toxicity is most severe in the myelin cells surrounding the nerves in the brain and in the peripheral nervous system, eventually leading to the death of these cells. The disease initially manifests as physical delays in development, muscle weakness and irritability and advances rapidly to difficulty swallowing, breathing problems, cognitive, vision and hearing loss. Early onset or "Infantile", Krabbe disease cases usually results in death by age 2-4 years, while later onset or "Late Infantile" cases have a more variable course of progressive decline. There is currently no approved treatment for Krabbe disease.

About FBX-101Forge is developing FBX-101 to treat patients with infantile Krabbe disease. FBX-101 is an adeno-associated viral (AAV) gene therapy that is delivered after a hematopoietic stem cell transplant. FBX-101 delivers a functional copy of the GALC gene to cells in both the central and peripheral nervous system. FBX-101 has been shown to functionally correct the central and peripheral neuropathy and correct the behavioral impairments associated with Krabbe disease in animal models, and to drastically improve the lifespan of treated animals. This approach has the potential to overcome some of the immunological safety challenges observed in traditional AAV gene therapies.

About Forge BiologicsForge Biologics is a hybrid gene therapy contract manufacturing and therapeutic development company. Forge's mission is to enable access to life changing gene therapies and help bring them from idea into reality. Forge has a 175,000 ft2 facility in Columbus, Ohio, "The Hearth", to serve as their headquarters. The Hearth is the home of a custom-designed cGMP facility dedicated to AAV viral vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing.By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.

For more information, please visit https://www.forgebiologics.com.

Patient, Pediatrician, Genetic Counselors & Family Inquiries

Dr. Maria Escolar Chief Medical Officer Forge Biologics Inc. [emailprotected]

Media Inquiries:

Dan Salvo Director of Communications and Community Development Forge Biologics Inc. [emailprotected]

Investor Relations and Business Development

Christina Perry Vice President, Finance and Operations Forge Biologics Inc. [emailprotected]

SOURCE Forge Biologics

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Forge Biologics Announces FDA Clearance of Investigational New Drug Application for Phase 1/2 Clinical Trial (RESKUE) of FBX-101 Gene Therapy for...