Stem Cell Clinic

Prognostic Significance Of Platelet-To-Lymphocyte Ratio (PLR) And Mean | CMAR – Dove Medical Press

Xia-Bo Shen,1,2,* Yong Wang,2,* Ben-Jie Shan,2 Lin Lin,2 Li Hao,2 Yu Liu,1,2 Wei Wang,2 Yue-Yin Pan1,2

1Department of Medical Oncology, Anhui Provincial Hospital Affiliated to Anhui Medical University, Hefei 230001, Peoples Republic of China; 2Department of Medical Oncology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei 230001, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Yue-Yin PanDepartment of Medical Oncology, Anhui Provincial Hospital Affiliated to Anhui Medical University, Hefei 230001, Peoples Republic of ChinaEmail yueyinpan1965@163.comWei WangDepartment of Medical Oncology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei 230001, Peoples Republic of ChinaEmail docweiwang@fsyy.ustc.edu.cn

Background: Small cell lung cancer (SCLC) is a special type of lung cancer and it is responsive to chemotherapy. Blood parameters have been proved to be associated with survival for many types of malignancies. This study aimed to investigate the prognostic significance of platelet-to-lymphocyte ratio (PLR) and mean platelet volume (MPV) for SCLC patients with etoposide-based first-line treatment.Methods: We retrospectively identified 138 patients diagnosed as SCLC who underwent etoposide-based first-line chemotherapy. The patients baseline clinical characteristics and blood parameters were collected. KaplanMeier analysis and Cox regression methods were used to determine the factors associated with progression-free survival (PFS).Results: The optimal cut-off value of diagnosis was depended on the ROC curve, the cut-off value of pretreatment PLR was 190 (sensitivity 39.0%, specificity 88.5%), and the cut-off value of pretreatment MPV was 10.0 (sensitivity 60.7%, specificity 61%). KaplanMeier analysis showed patients with high PLR levels in baseline had worse PFS than those with low PLR levels (P <0.001). Multivariate analysis revealed pretreatment MPV was an independent prognostic factor for PFS (HR: 0.815, 95% CI: 0.7110.933, P =0.003). Further research suggested continuous high PLR indicated a poor therapy outcome (P =0.002).Conclusion: Pretreatment MPV can be an independent predictor for first-line treatment outcome and a continuously high level of PLR suggested inferior PFS in etoposide-treated SCLC patients.

Keywords: small cell lung cancer, SCLC, first-line chemotherapy, mean platelet volume, MPV, platelet-to-lymphocyte ratio, PLR, prediction

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Prognostic Significance Of Platelet-To-Lymphocyte Ratio (PLR) And Mean | CMAR - Dove Medical Press

Global Regenerative Medicine Market: Analysis By Type (Cell Therapy, Tissue Engineered, Gene Therapy), By Application, By Region, By Country (2019…

arcognizance.com has added latest research report on Global Regenerative Medicine Market, this report helps to analyze top Key Players, regions, revenue, price, and also covers Industry sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

As stated by the research report in August 2019, the Global Regenerative Medicine Market was valued at USD 22,814.45 million the year 2018. Driven by a number of differentiated fundamental factors including rise in the prevalence of chronic diseases and increase in medical research investments, the global market for regenerative medicine has been advancing at an augmented pace. The growth has been primarily driven by the search to find permanent cure of large number of incurable diseases such as various autoimmune and metabolic ailments, cancer, neurodegenerative disorders among others.

Over the recent years, regenerative medicine market has been witnessing considerable growth on the back of rising incidence of chronic diseases, rapidly growing medical research facilities, increasing investment by pharmaceutical manufacturers, and growing government initiatives. In addition, expanding product pipeline of companies and growing number of partnerships and collaborative agreements in this industry is anticipated to fuel the market growth in forecast period. However, high cost associated with the manufacturers and use of regenerative therapy has been hindering market growth.

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A comprehensive research report created through extensive primary research (inputs from industry experts, companies, stakeholders) and secondary research, the report aims to present the analysis of regenerative medicine market. The report analyses the Global Regenerative Medicine Market By Type (Cell Therapy, Gene Therapy, Tissue Engineered) and By Application (Orthopaedic and Dental, Cardiology, Wound Healing, Metabolism and Inflammation, Immunology & Oncology, Others). The global regenerative medicine market has been analyzed By Region (North America, Europe, Asia Pacific, and ROW) and By Country (U.S., Canada, U.K., Germany, France, Italy, China, Japan, India, Brazil) for the historical period of 2014-2018 and the forecast period of 2019-2024.

Scope of the Report

Global Regenerative Medicine Market (Actual Period: 2014-2018, Forecast Period: 2019-2024)

Global Regenerative Medicine Market- Size, Growth, Forecast

Analysis By Type: Cell Therapy, Gene Therapy, Tissue Engineered.

Analysis By Application: Orthopaedic & Dental, Cardiology, Wound Healing, Metabolism & Inflammation, Immunology & Oncology, Others

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Regional Regenerative Medicine Market North America, Europe, Asia Pacific, ROW (Actual Period: 2014-2018, Forecast Period: 2019-2024)

Global Regenerative Medicine Market- Size, Growth, Forecast

Analysis By Type: Cell Therapy, Gene Therapy, Tissue Engineered.

Analysis By Application: Orthopaedic & Dental, Cardiology, Wound Healing, Metabolism & Inflammation, Immunology & Oncology, Others

Country Regenerative Medicine Market U.S., Canada, Germany, U.K, France, Italy, China, Japan, India, and Brazil (Actual Period: 2014-2018, Forecast Period: 2019-2024)

Global Regenerative Medicine Market- Size, Growth, Forecast

Analysis By Type: Cell Therapy, Gene Therapy, Tissue Engineered.

Analysis By Application: Orthopaedic & Dental, Cardiology, Wound Healing, Metabolism & Inflammation, Immunology & Oncology, Others

Other Report Highlights

Competitive Landscape:

Company Share Analysis

Collaborations, Partnerships and Alliances between Key Industry Players

Approved Product Analysis

Product Pipeline Analysis

Market Dynamics Drivers and Restraints.

Market Trends

Porter Five Forces Analysis.

SWOT Analysis.

Company Analysis Vericel, Gilead Sciences, Novartis, Spark Therapeutics, Orchard Therapeutics, MolMed, Celgene, Sanofi, Amgen, Shanghai Sunway Biotech.

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Chapter One: Research Methodology

Chapter Two: Executive Summary

Chapter Three: Strategic Recommendations

Chapter Four: Regenerative Medicine Market: Product Outlook

Chapter Five: Global Regenerative Medicine Market : Growth and Forecast

Chapter Six: Global Regenerative Medicine Market : Segment Analysis

Chapter Seven: Global Regenerative Medicine Market : Regional Analysis

Chapter Eight: Global Regenerative Medicine Market: Competitive Landscape

Chapter Nine: Global Regenerative Medicine Market Dynamicscontinues

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Global Regenerative Medicine Market: Analysis By Type (Cell Therapy, Tissue Engineered, Gene Therapy), By Application, By Region, By Country (2019...

NCAPG Promotes The Proliferation Of Hepatocellular Carcinoma Through P | OTT – Dove Medical Press

Chengwu Gong,1,* Jiyuan Ai,1,* Yun Fan,2 Jun Gao,1 Weiwei Liu,1 Qian Feng,3 Wenjun Liao,1 Linquan Wu1

1Department of General Surgery, The Second Affiliated Hospital of Nanchang University, Nanchang 330006, Peoples Republic of China; 2Department of Neurology, Tongji Hospital, Huazhong University of Science and Technology, Wuhan 430000, Peoples Republic of China; 3Department of Emergency Medicine, The Second Affiliated Hospital of Nanchang University, Nanchang 330006, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Linquan Wu; Wenjun LiaoDepartment of General Surgery, The Second Affiliated Hospital of Nanchang University, No. 1, Minde Road, Nanchang 330006, Peoples Republic of ChinaTel +86 791 86311529Fax +86 791 86262262Email wulqnc@163.com; liaowenjun120@163.com

Purpose: Studies show that high expression of non-SMC condensin I complex subunit G (NCAPG) is associated with many tumors. In this study, we explore the mechanism by which NCAPG promotes proliferation in hepatocellular carcinoma (HCC).Patients and methods: Liver cancer and paracancerous tissue specimens of 90 HCC patients were collected, and expression levels of NCAPG in these tissues and cell lines were evaluated by Western blotting and immunohistochemistry. HCC cells were transfected with siRNAs and plasmids, and pathway activators or inhibitors were added. The 5-ethynyl-2-deoxyuridine (EdU) proliferation assay was used to measure cell proliferation. Flow cytometry was used to evaluate cell apoptosis. Western blot assays were performed as a standard procedure to detect total protein expression. Treated HCC cells were subcutaneously injected into nude mice.Results: Analysis using the Oncomine database showed that NCAPG was upregulated in HCC and immunohistochemistry and Western blot assays showed it was upregulated in both HCC tissues and HCC cell lines. The overexpression of NCAPG could promote HCC cell proliferation and reduce HCC cell apoptosis. More importantly, RNA-sequencing analysis predicted that NCAPG plays a role in the HCC via PI3K-AKT signaling pathway. The PI3K/AKT/FOXO4 pathway was aberrantly activated, and the expressions of apoptosis-related protein were altered when NCAPG was overexpressed or silenced both in vitro and in vivo. LY294002, a PI3K inhibitor, could eliminate the NCAPG role of promoting HCC cell proliferation and reducing HCC cell apoptosis, while 740Y-P, a PI3K activator, contributed to the opposite effect.Conclusion: NCAPG functions as an oncogene in HCC and plays a role in promoting cell proliferation and antiapoptosis through activating the PI3K/AKT/FOXO4 pathway.

Keywords: NCAPG, hepatocellular carcinoma, PI3K/AKT, FOXO4, proliferation

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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NCAPG Promotes The Proliferation Of Hepatocellular Carcinoma Through P | OTT - Dove Medical Press

Community Oncologist: A Key Player in CAR-T Cell Therapy – Cancer Therapy Advisor

Although administration of anti-CD19 chimeric antigen receptor (CAR)-T cell therapy takes place at authorized treatment centers, community oncologists still play an important role, particularly in the recognition of eligible patients and the management of adverse effects of the treatment.

Arecent piece in The Oncologist detailed this crucial element of CAR-Tadministration and highlighted key aspects of CAR-T cell indications andeligibility for community oncology providers.1

Tomaximize the chances of a patient receiving CAR-T cell therapy, communityoncologists should refer patients early and broadly, as the time of referralto CAR-T cell infusion can take 4 to 6 weeks.

Mostbroadly, patients with relapsed or refractory large B-cell lymphoma who havefailed on 2 or more prior therapies can be referred. Patients who have failedor relapsed after first-line immunochemotherapy may also be eligible.

Patientswho progress on first-line therapy should be referred directly to academiccenters whenever possible for management because high rates of relapse areobserved with second-line treatments, the authors wrote. Academic centers areequipped to facilitate a smooth and rapid transition to the next line oftherapy, especially CAR-T cell therapy, if patients are already receivingtreatment there, which may be particularly important for patients with rapidlyprogressing disease.

Aspart of this process, community oncologists should be aware of which centers intheir state offer CAR-T cell therapy.

Communityoncologists also play an important role in postinfusion care. Patients treatedwith CAR-T cell therapy are advised to carry a wallet card with them at alltimes that defines symptoms that could indicate a serious adverse event forwhich to seek medical attention. Any patient in response that does notexperience a serious adverse event after a 4- to 8-week stay returns home.

Thesepatients can experience prolonged hypogammaglobulinemia and B-cell aplasia, andsome patients may require supportive care with IVIG. Prolonged cytopenias canalso occur. Because the treatment causes immunosuppression, patients are atongoing risk for serious infections after discharge as well.

Coordinationand communication between the local oncologist and CAR-T cell treatment oncologistare important during the months after patients return home from their minimum4-week stay near the treatment center, the authors wrote. After this period,the authorized treatment center, in coordination with the local oncologist, mayhave patient follow-ups every 2 weeks until month 3, then decreasing infrequency to 6 months and 12 months after CAR T-cell infusion, then yearlyuntil 5 years after CAR T-cell infusion, the authors wrote.

Reference

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Community Oncologist: A Key Player in CAR-T Cell Therapy - Cancer Therapy Advisor

Cell therapy GammaDelta spins off Adaptive to direct body’s surveillance system – Endpoints News

If alpha beta T-cells the foundation of CAR-T cell therapy are killer cells, trained in the biologically ancient art of executing intruders, think of their gamma delta cousins as armed guards, capable of engaging an interloper but also of sounding an alarm to kick the rest of the bodys defenses into action.

The key role they play is to conduct immune surveillance, Natalie Mount told Endpoints News. And once they find an intruder they can stimulate a whole immune response as well as be cytotoxic [cell-killing].

Only discovered in 1985, these gamma delta T-cells () have already become a popular target in cancer immunotherapy. This morning, a top British biotech exploring potential applications founded a new company to help reach that goal as GammaDelta Therapeutics spun off Adaptate Biotherapeutics with Mount at its helm.

While GammaDelta will continue to focus on a CAR-T-like cell therapy approach, the new company will develop antibodies that guide the cells as they patrol a patient, Mount said.Both have the same aim: getting these cellular guards to notice and effectively strike cancers.

We are developing antibodies that are able to recognize the gamma delta cells and target those and modulate their activities, Mount said. Weve discovered a range of substrates and what we can do now is take that forward in non-clinical development.

Gamma delta cells have risen in popularity in oncology research largely because they show potential to bring cell therapys effectiveness in blood cancers to solid tumors, although they present other potential advantages, including broader targeting and faster response.

GammaDelta Therapeutics got in on the ground floor, opening its doors in 2016. Since then, new and bigger players have entered the game. Last week, Regeneron dropped $25 million as part of an $80 million funding round for Adicet Bio, another company looking to use antibodies to guide gamma delta T cells.

Just in the last 2 to 3 years theres been a real solid interest, Mount said.

The broad idea of targeting these cells for oncology is not new. Clinical trials have been conducted evaluating gamma delta T cell treatments on several cancers, including leukemia and sarcoma. They were safe but with highly limited efficacy, although some appeared for not-fully-understood reasons to actually fuel tumors.

But GammaDelta and Adaptate say they work on a different subset of cells than these earlier trials did, one called 2. 2 is found in the tissues, making it an intuitive weapon for attacking solid tumors.

The antibody concept behind Adaptate is an increasingly popular form of therapy. AbbVie, Eli Lilly, Regeneron, and Sanofi, among a long list of others, are developing a form of antibody therapy called bispecific. Theyre still in the early stage, but last year Bairds Brian Skorney argued that the class of drugs has huge potential.

Our bias is that bispecifics pose an existential risk to the cellular therapies, he wrote. If a regularly administered therapeutic can keep anti-tumor pressure on by consistently engaging and activating T-cells, we think the much more expensive CART would become an even harder sell than it already is.

The exhaustive CAR-T process is like a specialized masterclass in tumor-killing, with doctors withdrawing cells, equipping them with a specific antigen receptor to identify malignancies and re-injecting them. The antibody approach for Adaptate can be thought of more as directives from a command center guiding the surveilling immune cells.

GammaDelta will focus on a cell therapy process similar to CAR-T, while Adaptate focuses on antibodies. But Mount argued one of the big advantages for GammaDelta Therapeutics and gamma delta cells is that they can identify cancer cells based on patterns as opposed to the specific antigens that CAR-T (chimeric antigen receptor T-cells) therapies use, opening up the potential for a range of targets.

GammaDelta is much closer to the clinic than the Adaptate spinoff, although they have yet to reveal exactly when they will begin trials, and for what indications. Mount said they were at least 12 months from evaluating whether they were ready to enter the clinic.

Social image: Adaptive CEO Natalie Mount via GammaDelta

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Cell therapy GammaDelta spins off Adaptive to direct body's surveillance system - Endpoints News

Intellia Therapeutics Announces Presentations at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) – BioSpace

CAMBRIDGE, Mass., Oct. 16, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, announced one oral presentation and four poster presentations were accepted for the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) taking place October 22-25, 2019, in Barcelona, Spain.

Intellias data includes important updates about the companys programs and platform development activities:

Oral Presentation:

In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression

Intellia will present data on its alpha-1 antitrypsin deficiency (AATD) program, which uses a modular hybrid delivery system combining lipid nanoparticle (LNP) encapsulated CRISPR/Cas9 with an adeno-associated virus (AAV) donor DNA template. Intellias gene knockout approach eliminates the production of the faulty PiZ variant of the protein, while targeted insertion of a wild-type gene copy facilitates production of a functional circulating protein. This builds on Intellias similar approach for targeted gene insertion of Factor 9, which achieved increased levels of circulating human Factor IX protein through two months in non-human primates and sustained through 12 months in mice.

Presenter: Anthony Forget, Ph.D.Abstract number: OR48Session 5b: New delivery systems and technologiesPresentation date/time: Friday, October 25, 2019, 11:30 a.m. 1:30 p.m. CETLocation: Room 113-115

Poster Presentations:

In Silico, Biochemical and Cell-Based Integrative Genomics Identifies Precise CRISPR/Cas9 Targets for Human Therapeutics

This poster presentation will highlight Intellias approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Researchers demonstrated that potential off-target editing profiles discovered through empirical data from biochemical approaches were the most sensitive and accurate.

Presenter: Daniel OConnell, Ph.D.Poster ID Number: P655Date: Wednesday, October 23, 2019

Generation of a Library of WT1-Specific T Cell Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute Leukemia

This poster presentation focuses on Intellias ongoing research collaboration with IRCCS Ospedale San Raffaele to develop CRISPR/Cas9-edited T cell therapies to address intractable cancers, such as acute myeloid leukemia (AML). Researchers have successfully established a protocol enabling consistent and efficient tumor-specific TCR isolation and characterization from healthy donors. Based on these results, Intellia has selected multiple lead TCRs, which are undergoing development candidate evaluation.

Presenter: Erica Carnevale, Ph.D., Ospedale San RaffaelePoster ID Number: P111Date: Wednesday, October 23, 2019

Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In-Locus Insertion Combined with Endogenous TCR Knockout

This poster presentation focuses on the companys T cell engineering technology, which is being applied in its Wilms Tumor 1 (WT1) lead ex vivo program. Intellia has identified an efficient CRISPR/Cas9-mediated process that inserts tumor-specific TCRs with high yield into the TRAC locus. Simultaneous knockout of the TRBC1 and TRBC2 loci substantially eliminates production of the endogenous T cell receptors.

Presenter: Birgit Schultes, Ph.D.Poster ID Number: P162Date: Thursday, October 24, 2019

CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria

This poster presentation will demonstrate the effects of independent CRISPR/Cas9-mediated knockout of each of two target genes involved in oxalate formation, lactate dehydrogenase A (LDHA) and hydroxyacid oxidase 1 (HAO1), to address primary hyperoxaluria type 1 (PH1).

Presenter: Sean Burns, M.D.Poster ID Number: P552Date: Thursday, October 24, 2019

About Intellia Therapeutics

Intellia Therapeuticsis a leading genome editing company focused on developing curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more aboutIntellia Therapeuticsand CRISPR/Cas9 atintelliatx.comand follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements ofIntellia Therapeutics, Inc.(Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an IND application for NTLA-2001 in mid-2020; its plans to generate preclinical and other data necessary to nominate a first engineered cell therapy development candidate for its AML program by the end of 2019; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program and otherin vivoandex vivoprograms; develop our proprietary LNP/AAV hybrid delivery system to advance our complex genome editing capabilities, such as gene insertion; its presentation of additional data at upcoming scientific conferences regarding CRISPR-mediated, targeted transgene insertion in the liver of NHPs, using F9 as a model gene, via the Companys proprietary LNP-AAV delivery technology, and other preclinical data by the end of 2019; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR and AML programs, in any future studies, including human clinical trials; its ability to develop otherin vivoorex vivocell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc. or Ospedale San Raffaele; statements regarding the timing of regulatory filings regarding its development programs; and the ability to fund operations into the second half of 2021.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain our intellectual property position, including through our arbitration proceedings against Caribou; risks related to Intellias relationship with third parties, including our licensors; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations withNovartisor Regeneron or its otherex vivocollaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with theSecurities and Exchange Commission. All information in this press release is as of the date of the release, andIntellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Media:Jennifer Mound SmoterSenior Vice PresidentExternal Affairs & Communications+1 857-706-1071jenn.smoter@intelliatx.com

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

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Intellia Therapeutics Announces Presentations at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) - BioSpace

Adaptate Biotherapeutics Formed To Develop T-cell Modulating Antibody-based Therapies – Technology Networks

Product News Oct 16, 2019

GammaDelta Therapeutics, a company focused on harnessing the unique properties of gamma delta (d) T-cells to develop transformational immunotherapies, has announced the formation of a spin-out company: Adaptate Biotherapeutics. While GammaDelta Therapeutics primary goal is to develop d T-cell based cell therapy products, the new spin-out will build on GammaDeltas knowledge to modulate d T-cell activity using therapeutic antibodies, with the potential to trigger an immune response against cancer.

d T-cells are a distinct T-cell sub-type that respond to molecular patterns of distress and have been shown to have tremendous potential in treating cancer and other immunological disorders. GammaDelta Therapeutics was formed in 2016 to harness these properties, and since then has gained knowledge of d T-cell biology developing a portfolio of investigational cell therapies poised to enter clinical development. In addition to gaining insight into cell growth and isolation, the companys scientists have also discovered a number of potential drug targets and antibodies that have potential to modulate the activity of d T-cells in situ.

Adaptate Biotherapeutics has been formed to further develop these targets and antibodies for therapeutic purposes and advance them into clinical studies. The two companies will continue sharing their insights into d T-cell biology as they work towards developing different therapeutic modalities.

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Adaptate Biotherapeutics Formed To Develop T-cell Modulating Antibody-based Therapies - Technology Networks

Mogrify Raises Additional $16M To Advance Its Mission To Transform The Development Of Life-Saving Cell Therapies – Clinical Leader

Mogrify Ltd (Mogrify), a UK company aiming to transform the development of life-saving cell therapies, recently announced the initial close of its Series A funding. The Company raised $16M USD in this round, bringing the total investment to over $20M USD to date. The funding will support internal cell therapy programs, and the development and out-license of novel IP relating to cell conversions of broad therapeutic interest. Mogrify is also actively recruiting, and will increase headcount to 60 scientific, operational and commercial staff located at its state-of-the-art facility on Cambridge Science Park.

The funding round was led by existing investor Ahren Innovation Capital (Ahren), an investment fund co-founded by leading UK scientific entrepreneurs, supporting transformational companies at the cutting edge of deep science and deep tech. Parkwalk, the largest EIS growth fund manager, backing businesses with IP-protected innovations creating solutions to real-world challenges, 24Haymarket, an early investor in Mogrify and a prolific early-stage investment syndicate in deep technology and the life sciences, and the University of Bristol Enterprise Fund III, also contributed to the fundraise.

Mogrify has developed a proprietary direct cellular conversion technology, which makes it possible to transform (transmogrify) any mature human cell type into any other without going through a pluripotent stem cell- or progenitor cell-state. The Company is deploying this platform to develop novel cell therapies addressing musculoskeletal, auto-immune, cancer immunotherapy, ocular and respiratory diseases as well as generating a broad IP position relating to cell conversions that exhibit safety, efficacy and scalable manufacturing profiles suitable for development as cell therapies.

Mogrify is commercializing its technology platform via a model that includes development and out-license of internally developed cell therapy assets, development and license of novel cell conversion IP, and the formation of joint-ventures to exploit the platform and/or novel cell conversion IP in non-core areas.

Mogrify launched in February 2019, announcing $3.7M USD seed funding from Ahren, 24Haymarket and Dr. Darrin M. Disley, OBE and went on to secure grants from Innovate UK and SBRI Healthcare.

Mogrify also strengthened its management and scientific teams and relocated a 20-strong workforce to its new headquarters at TusParks Cambridge Bio-Innovation Centre on Cambridge Science Park in May. It has now begun recruiting up to 40 additional commercial, operational and scientific roles to support its expanding pipeline of internal programs, as well as supporting numerous biotech and pharma collaborators in developing novel IP to underpin existing and new cell therapy programs.

Dr. Darrin M. Disley, OBE, CEO, Mogrify, said: Following the recent announcement of Dr. Jane Osbourn, OBE, as Chair of Mogrify, I am delighted we have been able to make an initial close of this fundraising round, with the backing of both existing and new investors. Due to the significant interest, we have been able to secure this growth-funding without engaging in a protracted and distracting fund-raising process. Having now raised over $20M, we can focus on delivery of our business strategy with the support of an aligned investor group. We will continue to engage with high-caliber investors with computational biology and cell therapy domain expertise as part of our on-going investor relations and capital markets strategy.

Alice Newcombe-Ellis, Founder and Managing Partner, Ahren Innovation Capital, said: Mogrifys technology is well positioned to disrupt the global cell therapy market. The Company has grown rapidly since February, appointing a world-class management team and delivering strongly against its business plans. We look forward to supporting Mogrify as it continues to go from strength to strength.

Alastair Kilgour, Chief Investment Officer, Parkwalk, said: We are delighted to be supporting the team at Mogrify, many of whom have been involved successfully with companies we have previously invested in, in this investment round. The science and technology base Mogrify are building is truly unique and disruptive. If successful, the positive effect on patient outcomes across a wide range of diseases will be staggering.

Alice Newcombe-Ellis, Founder and Managing Partner of Ahren, and Alastair Kilgour, Chief Investment Officer, Parkwalk, both join Mogrifys board of directors along with Dr. Karin Schmitt, the Companys Chief Business Officer.

For more information, visit https://mogrify.co.uk/investors/

About MogrifyMogrify has developed a proprietary direct cellular conversion technology, which makes it possible to transform (transmogrify) any mature human cell type into any other without going through a pluripotent stem cell- or progenitor cell-state.

The platform takes a systematic big-data approach to identify, from next-generation sequencing and gene-regulatory networks, the transcription factors (in vitro) or small molecules (in vivo), needed to convert a cell. By bypassing the stem cell-stage of cell transformation, Mogrify simultaneously addresses challenges associated with efficacy, safety and scalability.

Mogrify is deploying this platform to develop novel cell therapies addressing musculoskeletal, auto-immune, cancer immunotherapy, ocular and respiratory diseases as well as generating a broad IP position relating to cell conversions that exhibit safety, efficacy and scalable manufacturing profiles suitable for development as cell therapies.

Uniquely positioned to address a cell therapy market estimated to be $35B USD by 2023, Mogrify is commercializing its technology via IP licensing, product development, and drug development. Based in Cambridge, UK, the Company has raised over $20M USD funding from Ahren Innovation Capital, Parkwalk, 24Haymarket, Dr. Darrin M. Disley, OBE and the University of Bristol Enterprise Fund III. For more information, visit http://www.mogrify.co.uk

About Ahren Innovation CapitalAhren LP is an investment fund that supports transformational companies at the cutting edge of deep science and deep tech. The technologies of its Founding Partners are today valued more than $100B combined.

A group of highly diverse, creative and original thinkers leading their domains, Ahren believes in taking considered risk that will deliver superior rewards capturing a generational opportunity to provide smart capital to deep technology pioneers.

With a philosophy espousing the importance of relationships and trust, Ahren provides long-term capital and support to exceptional founders and teams, empowering them to achieve the unimaginable.

Ahren Innovation Capital was founded by Alice Newcombe-Ellis, together with Science Partners Sir Shankar Balasubramanian, Professor John Daugman, Professor Zoubin Ghahramani, Professor Steve Jackson, Professor Andy Parker, Sir Venki Ramakrishnan, Lord Martin Rees and Sir Gregory Winter. For more information, visit http://www.ahreninnovationcapital.com

About ParkwalkParkwalk is the largest growth EIS fund manager, backing world-changing technologies emerging from the UKs leading universities and research institutions. With 250M of assets under management, it has invested in over 100 companies across its flagship Parkwalk Opportunities EIS Fund as well as the award-winning enterprise and innovation funds Parkwalk manages for the Universities of Cambridge, Oxford and Bristol.

Parkwalk invests in businesses creating solutions to real-world challenges, with IP-protected innovations, across a range of sectors including life sciences, AI, quantum computing, advanced materials, genomics, cleantech, future of mobility, MedTech and big data.

For more information, visit http://parkwalkadvisors.com

About 24Haymarket24Haymarket is a premium deal-by-deal investment platform focused on high-growth businesses, investing up to 5M in any company. 24Haymarkets Investor Network includes several highly experienced private equity and venture capital investors, seasoned entrepreneurs and senior operators. We invest our own capital in direct alignment with entrepreneurs and typically seek Board representation to actively support their growth agenda. Since its inception in 2011, 24Haymarket has invested in more than 50 high-growth businesses. For more information, visit http://www.24haymarket.com

The University of Bristol Enterprise Fund III (Managed By Parkwalk)The University of Bristol Enterprise Fund is an early stage investment fund backing scientific and technological companies spun out of the University of Bristol or being supported by the Universitys SETsquared incubator. For more information, visit http://parkwalkadvisors.com/fund/university-of-bristol-enterprise-fund.

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Mogrify Raises Additional $16M To Advance Its Mission To Transform The Development Of Life-Saving Cell Therapies - Clinical Leader

LogicBio Therapeutics to Present New Data on Next Generation Capsid Development Program and GeneRide Platform Program at the European Society of Gene…

CAMBRIDGE, Mass., Oct. 16, 2019 (GLOBE NEWSWIRE) -- LogicBio Therapeutics Inc. (Nasdaq:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients, today announced upcoming presentations at the European Society of Gene and Cell Therapy (ESGCT) 27th Annual Congress, held in Barcelona, Spain, October 22-25, 2019.

We are thrilled to be presenting positive data on our Next Generation Capsid Development Program on the anniversary of our collaboration with Childrens Medical Research Institute of Australia, a leader in gene therapy, childhood cancer, embryology and neurological diseases. The goal of the collaboration is to develop novel, synthetic adeno-associated virus (AAV) capsids which are highly tropic for human tissues and optimized for manufacturing. These data give us further confidence that we can improve the performance of current AAV vectors, expanding our pipeline and strengthening our GeneRide platform, said Fred Chereau, CEO of LogicBio. Further, we are pleased to present additional preclinical data further supporting the durability of expression, compared to canonical gene therapy, in one of our GeneRide platform programs and to have been invited to speak on AAV manufacturing.

Panel PresentationTitle: AAV manufacturing: critical parameters influencing vector quality attributesPresenter: Matthias Hebben, Ph.D., VP, Technology Development, LogicBio Therapeutics (INV36)Session: 1d ATMP manufacturingSession date/time: October 23, 2019, 8:30-10:30 a.m. CEST

Poster PresentationsTitle: AAV development program: towards next generation of livertropic AAV variants (P025)Session date/time: October 23rd, 2019, 1:00-3:00 p.m. CEST

Title: Durability of factor IX expression in mice treated neonatally with a nuclease-free, promoterless, AAV-based gene therapy, GeneRide (P423)Session date/time: October 23rd, 2019, 1:00-3:00 p.m. CEST

Additional information on the meeting can be found on the ESGCT website: https://www.esgct.eu/home.aspx

About LogicBio TherapeuticsLogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical needs using GeneRide, its proprietary technology platform. GeneRide enables the site-specific integration of a therapeutic transgene in a nuclease-free and promoterless approach by relying on the native process of homologous recombination to drive potential lifelong expression. Headquartered in Cambridge, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.

For more information, please visit http://www.logicbio.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on April 1, 2019 with the SEC, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts

Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200

Stephanie SimonTen Bridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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LogicBio Therapeutics to Present New Data on Next Generation Capsid Development Program and GeneRide Platform Program at the European Society of Gene...

Dunbar CAR T-Cell Program brings advanced immunotherapy to cancer patients – WHAS11.com

LOUISVILLE, Ky. Cancer patients in Louisville and throughout the region soon will have access to some of the most advanced immunotherapy treatments available.

Louisville resident Thomas E. Dunbar has pledged $1 million to the University of Louisville to create a specialized center to provide chimeric antigen receptor positive T (CAR T) cell therapies to patients at the U of L James Graham Brown Cancer Center and other centers in the Midwest.

The new program will be named the Dunbar CAR T-Cell Program.

This gift will allow both kids and adults to be treated right here in Kentucky with the most innovative cell-based immunotherapy being developed, said Jason Chesney, M.D., Ph.D., director of the U of L Brown Cancer Center.

In CAR T-cell therapies, immune cells are extracted from the patients own blood and then are genetically modified to fight cancer. The modified cells are infused back into the patient where they fight the cancer and create long-term immunity to its recurrence.

In addition to dramatic treatment results, CAR T-cell immunotherapy leads to fewer toxic side effects than traditional chemotherapy.

Patients who have been treated with all the conventional therapies who then underwent treatment in clinical trials with CAR T cells had dramatic response rates. Eighty-three percent of kids in the original trial who had lethal, terminal B-cell acute lymphoblastic leukemia responded to this therapy, Chesney said.

The Dunbar CAR T-Cell Program will include laboratories for manufacturing the CAR T cells and will administer both FDA-approved and clinical-trial therapies to adult and pediatric cancer patients.

The goal is for the facilities to be fully functional and receiving patients by Sept. 30, 2020.

Tom Dunbars son, Evan, lost his battle to cancer with neuroblastoma in 2001 at the age of 6. In 2009, Wally Dunbar, Tom Dunbars father, lost his battle with melanoma.

Donor Tom Dunbar with his son, Evan

U of L Brown Cancer Center

This year, Toms physician wife, Stephanie Altobellis, M.D., helped identify his own cancer.

Kentucky is at ground zero, with the nations highest rates of cancer diagnosis and death, Tom Dunbar said. Its completely unacceptable. We have to lead the charge right here where the need is the greatest and we can do the most good. We need treatments that are not toxic. Watching our loved ones miserable with pain, often just from the treatments, and yet still die in front of us simply cant be the best that we can do.

To learn more about how CAR T-cell treatment works visit: uoflbrowncancercenter.org

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Dunbar CAR T-Cell Program brings advanced immunotherapy to cancer patients - WHAS11.com