Category Archives: Gene Medicine

-- Ten abstracts, including four podium presentations, reflect Sareptas ongoing commitment to advancing genetic medicine for rare neuromuscular disease and facilitating greater understanding of these devastating conditions --

CAMBRIDGE, Mass., March 15, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the Muscular Dystrophy Association (MDA) Annual Clinical and Scientific Conference, which will take place virtually March 15-18, 2021. Among the research that will be presented:

All posters are available on-demand throughout the Congress beginning on Monday, March 15, 2021 at 6:00 a.m. ET. Podium presentations will take place on Thursday, March 18, 2021. The full MDA 2021 Virtual Congress program is available here: https://mdaconference.org.

Podium Presentations:

Poster Presentations:

Presentations will be archived on the events and presentations page in the Investor Relations section of http://www.sarepta.com for one year following their presentation at MDA.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052, iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com

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Sarepta Therapeutics to Present Results from its Gene Therapy and RNA Platforms at the 2021 Annual MDA Clinical and Scientific Conference -...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Be Biopharma (Be Bio), whose mission is to pioneer the emerging new category of engineered B cells as medicines, today announced that Joanne Smith-Farrell, Ph.D., has been appointed Chief Executive Officer and Director. Dr. Smith-Farrell will be joined by Chief Scientific Officer, Richard Morgan, Ph.D., a leading expert in cell and gene therapies.

Be Bios rapidly growing team of scientists, drug developers, manufacturing experts, and business builders is leading the creation of a new category of cellular therapies, engineered B cell medicines. B cells are exquisitely designed by nature to embody a unique mix of functionalities, including prolific protein production, tissue targeting, and durable engraftment in cellular niches. Be Bio was founded by Longwood Fund in October 2020 with a $52 million Series A investment led by Atlas Ventures and RA Capital, and joined by Alta Partners and Takeda Ventures to unlock this rich biology by precisely engineering B cells as therapies to develop a broad pipeline of potent and potentially curative cellular medicines.

Prior to joining Be Bio as Chief Executive Officer, Dr. Smith-Farrell was Chief Operating Officer and Business Unit Head, Oncology, at bluebird bio, where she led the growth of bluebird Oncology from an early single-candidate effort into a leading oncology cell therapy business. Prior to this, she held executive leadership roles as Chief Business Officer of bluebird bio, Vice President of Transactions at Merck, and Vice President of Business Development at Pfizer, as well as executive positions in public and private biotechs. Prior to entering the biopharmaceutical industry, she worked in the healthcare practice at The Boston Consulting Group. Dr. Smith-Farrell did her postdoctoral research in Biomedical Engineering in Bob Langers lab at the Harvard-MIT Division for Health Science and Technology and holds a Ph.D. in Physics from The Catholic University of America and a B.S. in Physics and Mathematics from Vanderbilt University.

Be Bios mission - to unleash the power of B cells, natures protein factories, on many of humanitys most challenging diseases is an inspiring and humbling journey to be joining, said Dr. Smith-Farrell. It has been a great privilege to participate in the birth of the first generation of cell therapies to come to market, and to witness, first-hand, cell therapys power to transform the lives of patients with devastating diseases. Be Bios aspiration, fueled by the broad utility of engineered B cell medicines to offer previously impossible solutions across a wide array of therapeutic areas, takes the potential of cell therapy to an entirely new level.

Rick Morgan, Ph.D., joins Be Bio as Chief Scientific Officer, and brings decades of experience as one of the pioneers of cell and gene therapy. Most recently, Dr. Morgan was Senior Vice President of Immunogenetics at Editas Medicine, where he focused on genome engineering to produce off-the-shelf cell medicines for cancer. Prior to that, he was Vice President of Immunotherapy at bluebird bio in 2013, where he led pre-clinical activities for bluebirds first oncology medicine, the anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cell therapy idecabtagene vicleucel (ide-cel)the first CAR-T for the treatment of multiple myeloma filed in the U.S. and Europe. He started his career at the National Institutes of Health, where he conducted groundbreaking research in the development of gene therapy for genetic diseases such as hemophilia, HIV/AIDS, and cancer immunotherapy. He was a member of the team that published the first approved human gene transfer experiment in 1990, and was also the first to report the successful use of T-cell receptor gene therapy for the treatment of cancer in 2006.

By exploiting the intrinsic drug-like properties of B cells, we can make redosable medicines with superior pharmacokinetic profiles that can be administered without toxic conditioning regimens, said Dr. Morgan. Be Bios ability to engineer B cells is a true paradigm shift in gene therapy that creates major opportunities to treat diseases such as cancer, autoimmune conditions, infectious disease, and protein deficiencies. As CSO, I am excited to have the rare opportunity to shape the development of a new class of medicine from the very start.

Extraordinary science attracts extraordinary leaders, said David Steinberg, Longwood General Partner, Director and and outgoing Chief Executive Officer, Be Bio. For over 25 years, Dr. Smith-Farrell has been leading teams that are committed to conquering cancer and rare diseases, most recently at bluebird bio where she built a 400 person oncology cell therapy business unit. We are very fortunate to have her joining alongside Dr. Morgan, an internationally recognized trailblazer in cell and gene therapy and a member of an elite group of scientists who have successfully developed these groundbreaking medicines. Together, Joanne and Rick will be invaluable to Be Bio as they lead our efforts to unlock the potential of B cell medicines, bringing transformational therapies to patients in need.

About Be Biopharma

Be Biopharma is a leader in developing B cells as medicines, treating disease with the human bodys native protein factories. We precisely engineer B cells to harness their intrinsic drug-like properties remarkable protein production, selective tissue targeting, and fine control of their cellular environment to forge a new category of cell therapy. These medicines are designed to be durable, re-dosable and administered without toxic conditioning, creating new avenues to halt or reverse severe diseases like cancer, autoimmune conditions, and enzyme deficiency. Founded by Longwood Fund and B cell engineering pioneers David Rawlings, M.D., and Richard James, Ph.D., Be Biopharma is re-imagining medicine based on the power of B cell therapy. For more information, please visit Be Biopharma.

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Be Bio Announces Appointment of Cell and Gene Therapy Veterans Joanne Smith-Farrell, Ph.D., as Chief Executive Officer and Rick Morgan, Ph.D., as...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--ElevateBio, a cell and gene therapy technology company focused on powering transformative cell and gene therapies, today announced that the companys Chief Scientific Officer of Regenerative Medicine, Dr. Melissa Carpenter, has been appointed to the International Society for Stem Cell Research (ISSCR) Board of Directors. In this role, Dr. Carpenter will work with the ISSCR officers and board to advance the organizations mission of bringing together researchers, clinicians, academics, and industry to promote excellence in stem cell science and applications to human health.

I am honored to have been elected to, and serve on the Board of, the ISSCR and foster the continued progress in advancing stem cell science alongside this impressive leadership and fellow board members, said Melissa Carpenter, PhD, Chief Scientific Officer of Regenerative Medicine at ElevateBio. Collaboration across the stem cell professional community is critical to our ability to translate promising stem cell research and regenerative medicine science into treatments that can have dramatic benefit for global human health globally.

Dr. Carpenter served on the ISSCR Task Force to revise the Guidelines for Stem Cell Research and Clinical Translation that will be released in May and advocated in support of the value of stem cell research as part of the Societys 2019 Advocacy Day, meeting with members of the U.S. Congress. She has also served on the Clinical Translation Committee.

We are delighted to welcome Melissa Carpenter to the ISSCR Board of Directors, said Christine Mummery, ISSCR President. Melissas dedication to supporting the translation of stem cell discoveries into therapeutics and her leadership has been crucial for advancing the clinical development of multiple therapies. Her experience will be an asset to the Board as the field of stem cell science continues to rapidly evolve.

The International Society for Stem Cell Research is the preeminent global, cross-disciplinary, science-based organization dedicated to stem cell research and its translation to the clinic. With nearly 4,000 members from more than 60 countries, the ISSCR mission is to promote excellence in stem cell science and applications to human health.

About ElevateBio:

ElevateBio is a cell and gene therapy technology company built to power the development of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built world-class facilities, and integrated diverse technology platforms necessary for rapid innovation and commercialization of cell, gene, and regenerative therapies. The company has built an initial technology stack, including gene editing, induced pluripotent stem cells, and protein, viral, and cellular engineering. At the center of the business model is ElevateBio BaseCamp, a centralized R&D and manufacturing company that offers research and development (R&D), process development (PD), and Current Good Manufacturing Practice (CGMP) manufacturing capabilities. The company is focused on increasing long-term collaborations with industry partners while also continuing to develop its own highly innovative cell and gene therapies. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.

ElevateBio is headquartered in Cambridge, Mass, with ElevateBio BaseCamp located in Waltham, Mass. For more information, visit us at http://www.elevate.bio, or follow Elevate on LinkedIn, Twitter, or Instagram.

*As of the date of this press release, SoftBank Group Corp. has made capital contributions to allow investments by SoftBank Vision Fund 2 ("SVF 2") in certain portfolio companies. The information included herein is made for informational purposes only and does not constitute an offer to sell or a solicitation of an offer to buy limited partnership interests in any fund, including SVF 2. SVF 2 has yet to have an external close, and any potential third-party investors shall receive additional information related to any SVF 2 investments prior to closing.

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ElevateBio Announces Chief Scientific Officer of Regenerative Medicine, Melissa Carpenter, PhD, Elected to the International Society for Stem Cell...

A pioneering gene therapy innovation centre at the University of Sheffield is set to advance scientific discoveries into promising treatment options for millions of patients with life-threatening diseases.

The Gene Therapy Innovation and Manufacturing Centre (GTIMC), led by Professor Mimoun Azzouz, is one of three pioneering hubs announced today (18 March 2021) in a new 18 million network funded by LifeArc and the Medical Research Council (MRC), with support from the Biotechnology and Biological Sciences Research Council (BBSRC).

Gene therapy is a promising treatment option for more than 7,000 rare diseases that currently have no cure. It aims to treat these conditions, by engineering another gene to replace, silence or manipulate the faulty one.

The UK has a world-class genetics research base however, to date, academics have found it difficult to get access to the clinical materials, facilities and expertise required to progress gene therapy research into clinical trials.

Professor Mimoun Azzouz, Director of the GTIMC and Chair of Translational Neuroscience at the University of Sheffield, said: The Gene Therapy Innovation and Manufacturing Centre will tackle major challenges in gene therapy development for some of the most devastating diseases.

Gene therapies are pioneering medical advances that have the potential to offer much-needed, novel, effective treatments for many rare and incurable diseases that cannot be treated by conventional drug compounds.

This is a momentous milestone for revolutionary medical advances not only for Sheffield and South Yorkshire, but also for the UK.

Sheffield has emerged as one of the leading players in the cell and gene therapy and this national network of partners, facilities and training programmes will allow us to keep pace with translational discoveries for new and potentially life changing treatments

Professor Mimoun Azzouz

Director of the GTIMC and Chair of Translational Neuroscience at the University of Sheffield

The new centre, which will be the first in the north of England, builds on the University of Sheffields strong history of translational research and its reputation as an international centre of excellence for gene therapeutics.

Professor Dame Pam Shaw, Director of the NIHR Sheffield Biomedical Research Centre and co-applicant on the GTIMC application said: This exciting development and partnership will speed up the pull through of new gene therapies into early phase clinical trials and offer hope to patients with neurological and other rare diseases that can be addressed in this way.

The support given to this initiative will greatly accelerate the translational potential of genetic therapies in the UK and bring benefits in key areas of unmet medical need.

The state-of-the-art centre will bring together academic institutions, NHS trusts, non-profit and industry partners across the north of England, Midlands and Wales enabling academic-led clinical trials of novel gene therapies. The GTIMC will deliver essential translational and regulatory support alongside extensive training and skills programmes to enable upskilling and address shortage of skills in Good Manufacturing Practice (GMP) manufacturing.

Professor Koen Lamberts, President and Vice-Chancellor of the University of Sheffield, said: At the University of Sheffield we focus our research on finding real-world solutions to some of the biggest global challenges. The Gene Therapy Innovation and Manufacturing Centre will unlock development pathways for new treatments for people affected by devastating genetic disorders, many of which have no cure.

We are delighted that our University is at the forefront of research in this pioneering field of medicine and that this new centre will build on our reputation as an international centre of excellence for gene therapeutics.

This is fantastic news for the City Region and the North of England and we look forward to working collaboratively to share technical skills and resources across the coordinated network.

Alongside the national network funding from LifeArc, the MRC and BBSRC, the GTIMC was made possible thanks to a 3 million donation from The Law Family Charitable Foundation, established by Andrew Law and his wife Zo. This funding was part of a record 5 million donation from the University of Sheffield alumnus, which will also see the launch of a new student support programme.

Andrew Law, who is Chairman and CEO of Caxton Associates, said: The University of Sheffield is rapidly developing a global reputation in gene therapy. The new Gene Therapy Innovation and Manufacturing Centre will drive innovation and world-class research, while presenting a real opportunity to catalyse the creation of new start-up companies to facilitate commercialisation in the North.

This investment will enhance vital partnerships with biotechnology and pharmaceutical companies to help accelerate gene therapy programmes and clinical trials, at the same time as supporting regional economic growth and job creation.

The GTIMC is planned for a site on the University of Sheffields Innovation District close to existing translational research facilities and will contribute to an ongoing programme of regional investment and regeneration.

The centre will include a cutting-edge GMP (good manufacturing practice) facility that will support gene therapy projects emerging from universities across the UK.

The facility will utilise highly efficient processes to manufacture clinical grade adeno-associated viruses (AAV) and provide all the necessary quality assurance, regulatory certification and governance for human trials at Advanced Therapies Treatment Centres and NHS trusts within the GTIMC and the national network.

The three national hubs, located at the University of Sheffield, Kings College London, and NHS Blood and Transplant in Bristol will operate as a coordinated network, sharing technical skills and resources to enable innovative gene therapy research.

Dr Melanie Lee, CEO of LifeArc, said: Recent innovations in gene therapies hold enormous potential for treating conditions such as rare diseases, but often promising ideas particularly in academia are not making it through to patients. Through our collaboration, we aim to meet the need for researchers to have access to the essential facilities and translational advice to progress promising research.

The GTIMC will manufacture commonly used vectors including both lentivirus and adeno-associated virus that are needed for genetic therapy trials, while positioning the UK for significant bioprocessing innovation work with the potential to radically increase yields and reduce productivity barriers in future years. The hub network will also design and share commercially ready platforms, using common cell-lines, plasmids and reagents to reduce costs, facilitate simplified licensing agreements and streamline regulatory reviews. A key aim is to smooth the transition between small-scale supply for early clinical trials through to larger-scale manufacture for patient trials, and beyond.

Professor Fiona Watt, MRC's Executive Chair, said: The new network of Innovation Hubs for gene therapies will build on the UKs great strengths in this area, providing targeted investment in vital infrastructure to accelerate academic research programmes down the path to patient benefit, supporting the delivery of a new wave of genetic medicines.

Notes for editors

Sheffield GTIMC Lead Institution

The University of Sheffield

Sheffield GTIMC Co-Lead Institutions

Cell & Gene Therapy Catapult

Centre of Process Innovation (CPI)

University of Birmingham

University of Liverpool

The Midlands-Wales Advanced Therapies Treatment Centre [MW-ATTC]

Sheffield GTIMC Partners

University of Leeds

University of Leicester

University of York

Cardiff University

University of Bradford

Northern Health Science Alliance (NHSA)

NHS Hospital Trusts of Sheffield, Leeds, Birmingham Womens and Children Hospital

Leeds Teaching Hospitals NHS Foundation Trust

Genomic Laboratory Hubs

Lonza AG

Cobra Biologics

Advanced Manufacturing Research Centre (AMRC)

National Horizons Centre (NHC)

National Institute for Biological Standards

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New gene therapy innovation centre to advance scientific discoveries into life-changing treatments - University of Sheffield News

LEXINGTON, Mass., March 15, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio or the Company), a clinical- stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood, today reported financial results for the year ended December 31, 2020 and provided a business update.

"The fourth quarter of 2020 marked a continuation of strong momentum for LogicBio which has also extended into 2021," said Frederic Chereau, President and CEO. "Most notably, we continue to anticipate enrolling the first patient in our SUNRISE Trial of LB-001 for the treatment of MMA in the very near future. We expect this event will be followed by additional enrollments soon after and allow us to begin to generate data regarding the safety, and clinical impact of treatment with LB-001 for these children. In addition, we also recently announced that we have extended our collaboration with the Children's Medical Research Institute of Australia to continue to develop sAAVy, our next generation capsids platform, which has already yielded novel liver-tropic capsids that we believe are superior to ones that are currently used in the clinic. We believe these capsids will allow LogicBio to continue to expand its gene editing platform, GeneRide, and potentially initiate gene therapy programs, either alone or in collaboration with strategic partners. We look forward to providing further updates on these programs throughout 2021." Mr. Chereau concluded by saying, "I am also very excited by the new members we have added to our team. Both Mariana Nacht, our new CSO, and Cecilia Jones, our new CFO, bring a wealth of biotech industry experience, and I am delighted they have chosen to join LogicBio as we move into this exciting next phase of progress."

Anticipated LogicBio Milestones for 2021:

LB-001 for MMA

Pipeline

Full Year 2020 Financial Results

Twelve Months Ended December 31, 2020 and 2019

About LogicBio Therapeutics

LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood. The company's proprietary GeneRide platform is a new approach to precise gene insertion that harnesses a cell's natural DNA repair process leading to durable therapeutic protein expression levels. LogicBio's cutting-edge sAAVy capsid development platform is designed to support development of treatments in a broad range of indications and tissues. The company is based in Lexington, MA. For more information, visit http://www.logicbio.com.

Forward Looking Statements

This press release contains "forward-looking" statements within the meaning of the federal securities laws, including those related to the Company's plans to enroll patients in, advance and complete its planned Phase 1/2 SUNRISE clinical trial of LB-001 in MMA and the potential benefits to patients of LB-001; the timing, progress and results of the Company's research and development activities, including those related to the GeneRide technology platform and sAAVy, our next generation capsids program; and the sufficiency of our cash on hand to fund our operating expenses and capital expenditures. These are not statements of historical facts and are based on management's 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 Company's plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Company's current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Company's ability to progress with its research, development, manufacturing and regulatory efforts, including the Company's plans to enroll patients in, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Company's common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the United States and in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Company's filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Company's Annual Report on Form 10-K filed on March 16, 2020 with the SEC, the Company's Quarterly Report on Form 10-Q filed on May 11, 2020, and the Company's 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.

LogicBio Therapeutics, Inc.

Condensed Consolidated Statements of Operations

(In thousands, except share and per share data)

Year Ended December 31,

2020

2019

REVENUE

Service revenue

$ 3,454

$

Total revenue

3,454

OPERATING EXPENSES

Research and development

22,753

30,656

General and administrative

12,212

10,385

Total operating expenses

34,965

41,041

LOSS FROM OPERATIONS

(31,511)

(41,041)

OTHER (EXPENSE) INCOME, NET:

Interest income

181

1,500

Interest expense

(1,098)

(546)

Other expense, net

(5)

(19)

Total other (expense) income, net

(922)

935

Loss before income taxes

(32,433)

(40,106)

Income tax provision

(188)

(22)

Net loss

$ (32,621)

$ (40,128)

Net loss per sharebasic and diluted

$ (1.29)

$ (1.78)

Weighted-average common stock outstandingbasic and diluted

25,364,453

22,602,954

LogicBio Therapeutics, Inc.

Condensed Consolidated Balance Sheets

(In thousands)

As of

December 31, 2020

December 31, 2019

Cash, cash equivalents and investments

$ 70,075

$ 50,647

Other assets

10,565

5,013

TOTAL ASSETS

$ 80,640

$ 55,660

Accounts payable, accrued expenses and other liabilities

$ 19,213

$ 13,373

Stockholders' equity

61,427

42,287

TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY

$ 80,640

$ 55,660

SOURCE LogicBio Therapeutics, Inc.

http://www.logicbio.com

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LogicBio Therapeutics Reports Full Year 2020 Financial Results and Provides Business Update - PRNewswire

$262.3 million cash balance at the end of 2020 bolstered by additional net proceeds of $211.3 million from follow-on offering, funding key milestones into 2024

Ron Cooper appointed to Board of Directors

Company remains on track to begin IND-enabling studies for hemophilia A in 2021

CAMBRIDGE, Mass., March 18, 2021 (GLOBE NEWSWIRE) --Generation BioCo. (Nasdaq: GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, reported recent business highlights and fourth quarter and full year 2020 financial results.

2020 was a foundational year for Generation Bio in which we strengthened the leadership team, advanced our platform science, and added substantially to our balance sheet to fund our lead programs to human data, said Geoff McDonough, M.D., president and chief executive officer of Generation Bio. We entered 2021 with preclinical data in mice and non-human primates that we believe demonstrate our platforms potential to go beyond what is currently possible with gene therapy for hemophilia A. We expect to announce additional data throughout the remainder of this year that will support the rapid expansion of our pipeline and showcase our differentiated approach to creating a new class of genetic medicine.

Recent Business Highlights

Selected Anticipated Company Milestones in 2021

Fourth Quarter and Full Year 2020 Financial Results

About Generation Bio

Generation Bio is an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is engineered to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The companys efficient, scalable manufacturing process supports Generation Bios mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.

Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for the Company, including statements about our strategic plans or objectives, our technology platforms, our research and clinical development plans, and other statements containing the words believes, anticipates, plans, expects, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the Companys product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; expectations for regulatory approvals to conduct trials or to market products; challenges in the manufacture of genetic medicine products; whether the Companys cash resources are sufficient to fund the Companys operating expenses and capital expenditure requirements for the period anticipated; the impact of the COVID-19 pandemic on the Companys business and operations; as well as the other risks and uncertainties set forth in the Risk Factors section of our most recent quarterly report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in subsequent filings the Company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Companys views as of the date hereof. The Company anticipates that subsequent events and developments will cause the Companys views to change.However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.These forward-looking statements should not be relied upon as representing the Companys views as of any date subsequent to the date on which they were made.

Contacts:

InvestorsMaren KillackeyGeneration Bio541-646-2420mkillackey@generationbio.com

MediaAlicia WebbGeneration Bio847-254-4275awebb@generationbio.com

Stephanie SimonTen Bridge Communications617-581-9333stephanie@tenbridgecommunications.com

GENERATION BIO CO.CONSOLIDATED BALANCE SHEET DATA (Unaudited)(In thousands)

GENERATION BIO CO.CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited)(in thousands, except share and per share data)

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Generation Bio Reports Fourth Quarter and Full Year 2020 Financial Results - BioSpace

AUniversity of Virginia School of Medicinescientist is developing an innovative gene therapy she hopes will slow disease progression and improve movement, coordination and communication in children with Rett syndrome. The approach also may be useful for battling other genetic disorders involving the X chromosome.

UVA researcher Sanchita Bhatnagar discovered that tiny bits of RNA, called microRNAs, play an important role in Rett, a rare genetic disorder that can impair childrens ability to speak, move and even breathe. Based on that finding, she is seeking to sop up those RNA bits, called microRNA, using absorbent particles called microRNA sponges.

Early work in lab models has produced promising results, and she hopes the approach could lead to a better quality of life for children with Rett.

We are seeing that lab animals treated with this gene therapy are more mobile. Theyre moving faster, theyre smarter, Bhatnagar said. And if that translates into even modest improvements for children, it could make a big difference, she said: If we can help a child to move more independently, or improve their ability to communicate, I think for a parent, thats a big win.

Rett syndrome affects approximately 1 in 10,000 children, almost exclusively girls. Thats because its caused by a mutation in theMECP2gene found on the X chromosome. Girls have two X chromosomes, while boys have an X and a Y.

Bhatnagars discovery of the role of microRNA in Rett came as a surprise, because it was the first time microRNA had been linked to X chromosome biology. That finding dovetailed perfectly with her labs expertise in creating microRNA sponges that can target microRNA, absorbing it and then breaking it down.

We are not altering the genomic DNA, Bhatnagar emphasized. We are just using them [the microRNA sponges] as inhibitors that are delivered through [adeno-associated viral] vectors. These vectors do not go and integrate into the genome, so we hope for minimal secondary effects.

Bhatnagar hopes her new approach could be useful both for treating Rett and other genetic disorders that involve the X chromosome, such as Fragile X syndrome, a condition that causes learning disabilities, developmental delays and behavioral problems. To advance her research toward patient impact, she works with the UVA Licensing & Ventures Group to navigate patent protection and industry partnerships. After disclosing her discovery to the Licensing & Ventures Group in 2019, Bhatnagar presented her research and attracted a top expert in developing new gene therapies, Kathrin Meyer of the Abigail Wexner Research Institute at Nationwide Childrens Hospital, as a research collaborator. Massachusetts-based Alcyone Therapeutics Inc. is sponsoring the research at both UVA and the Wexner Research Institute to support its transition to clinic.

The research collaborators hope to begin clinical trials in the near future. If the initial trials prove safe and successful, that would lead to larger trials to evaluate the effectiveness of the approach. The federal government would then consider the trial results to determine if the approach should be approved as a treatment.

We think what Dr. Bhatnagar has come up with is very elegant, and we are excited about its potential to improve the lives of many patients, PJ Anand, CEO of Alcyone Therapeutics, said. The beautiful part is that this gene therapy is what we call in the industry a pipeline in a product. The exact same gene therapy product can be used to potentially address diseases that are caused by other genes on the X chromosome. Once we have the initial proof of concept in the clinic for the first disease, we can use much of that same information to move toward clinical trials in other X-linked diseases as well, so it will really be an efficient path forward.

Bhatnagar, of UVAs Department of Biochemistry and Molecular Genetics, said she feels very emotional about the potential clinical implications of her work, especially knowing the dramatic impact Rett has on the affected girls and their families.

Rett syndromeleads to severe impairments, affecting nearly every aspect of a childs life.Its often a full-time job as a parent, because these girls need assistance with all their living skills, Bhatnagar said. I dont know if this therapy is going to fix all of that. But it may be able to help them, and any help will be a big step forward.

The early-stage studies for the project in Bhatnagar lab were supported by the Hartwell Foundation and the UVA Brain Center.

To keep up with the latest medical research news from UVA, subscribe to theMaking of Medicineblog.

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UVA Scientist Developing Gene Therapy to Help Girls With Rett Syndrome - University of Virginia

This article is part of a Technology and Innovation Insights series paid for by Samsung.

In this episode of The Next Wave, Young Sohn sits down with Lisa Alderson, CEO of Genome Medical, an innovative healthcare firm that is working hard to ensure that genomic medicine is made accessible to everyone.

Alderson shares her views on a range of issues around genomics. She discusses the vast promise of the technology and her companys efforts to bring its game-changing potential to all patients by providing clinical support tools to non-genetics professionals. She also addresses the ethical questions raised by genomics, particularly its use in prenatal diagnostics. And she highlights the critical role genomics played in developing COVID vaccines in record time.

Genomics is swiftly moving to the forefront of medicine. This potentially limitless technology is transforming health care by making it more targeted, personalized, and proactive.

Genomics can not only help with preventive care but also with determining treatment options for chronic ailments such as heart disease. It is becoming essential in reproductive health counseling, as well as in screening for infertility and newborn genetic disorders. And it is proving to be instrumental in the development of advanced therapeutics for diseases such as cancer and even for treating global outbreaks like COVID-19.

But, to date, there has been one big problem with genomic medicine: access. Lisa Alderson, CEO of Genome Medical, explains that even though the technology is now advanced, the vast majority of patients are still not getting access to genomics, even when it can be medically beneficial. This is mostly due to insurance barriers, a broad lack of clinical understanding, and the sheer volume and complexity of new tests being brought to market.

Her company is on a mission to change the dynamic by bringing genome-enabled health care to everyone through Genome Medicals extensive network of genetic specialists and a technology platform delivering genomics as a service.

Alderson points out that virtually everybody can benefit from access to genetic insights. Not only does genetics offer the promise of getting the right therapy to the right patient at the right time, it can also uncover new information to better inform clinical care.

And as genomics advances, its ability to detect and treat diseases like cancer vastly improves. As a result, the technology is increasingly important not just for individuals with rare genetic conditions but for everyone from birth to old age.

Historically, for instance, only one or two genes were used to test for hereditary breast cancer or ovarian cancer. But because the science has evolved, we now know there are 11 genes that can help detect hereditary breast and ovarian cancer. In fact, says Alderson, there are over a hundred genes that can increase our risk of cancer. So as genetic testing becomes more precise and accurate, it has the potential to save exponentially more lives.

One way to help genomics reach its full potential is by providing clinical support tools to non-genetics professionals. This will allow them to better understand which patients would benefit from what tests, and how to interpret and use the resulting information to guide clinical care.

One quandary, of course, is that most local health care systems dont have full-time metabolic geneticists on staff. As a result, they end up referring their patients out to leading academic centers, which is where the bulk of genomic health care is carried out today. And that means local health systems could lose those patients for the full continuum of their care.

To address this, Genome Medicals genomics as a service SaaS platform provides rapid patient education and engagement and also supports providers with the necessary clinical tools and knowledge they need to utilize genomics, as appropriate. Alderson says she wants every hospital and health system to have access to genomics in its community setting.

Alderson is increasingly heartened by the fact that genetic testing is expanding beyond its origins in prenatal medicine and oncology to new areas such cardiology, endocrinology, nephrology, and urology.

In the past, for example, most urologists did not have strong use cases for ordering genetic testing. But now, genetic testing is recommended for nearly every metastatic prostate cancer patient. Ultimately, genomics will touch all medical professionals, whether they are pediatricians, oncologists, cardiologists, neurologists, urologists the list goes on and on.

Were sitting at the precipice of what is a huge inflection point in the use of genetics and genomics, says Alderson. Were going to see it having a profound impact on human health, not just for prevention but also to get a deeper understanding of what is causing the disease and thus a richer opportunity to improve how we treat disease. Were getting to the root cause rather than just observing symptoms and then trying to treat those symptoms.

It needs to be mentioned that genomics is not without controversy. With new genetic capabilities comes much debate about how they should be used. For instance, can genetic information be used by insurance companies to discriminate against those with risk factors for certain disorders?

Or what about the use of genetic testing to determine whether youre having a healthy child? And then there is the issue of gene editing and giving scientists the ability to alter the DNA of many organisms. These are areas fraught with significant moral and ethical questions.

Whats powerful about the technology is it can actually cure disease, says Alderson. What is scary about the technology is, do we understand the full downstream effects in editing the genome? We dont know everything about the genome and its role and implications in human health. So there is a lot of complexity here.

There is also much hope. After all, genomics recently played a starring role in the development of COVID-19 vaccines in record time. The vaccines wouldnt be here without genomics and its power to sequence the virus rapidly and correctly.

Alderson says that genomics helped scientists gain a deep understanding of the virus and gave them the ability to create the vaccine in less than a year a previously unheard-of accomplishment. She believes the use of genomics in immunology will accelerate the advancement of treatment options in other disease areas and help address any new viruses that could emerge.

Genomics is obviously one of the most exciting advancements in our lifetime in health care, Alderson concludes. I couldnt be more excited about how far weve come over the last couple of decades but also for what lies ahead.

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Most genetic tests focus on your odds of developing certain diseases or health conditions. But some known as pharmacogenomic (or pharmacogenetic) tests can reveal how your body may respond and react to different medications. To date, researchers have identified more than 400 genetic variations known to affect the metabolism of numerous drugs, including some that help lower cholesterol or prevent blood clots (see "Pharmacogenomics of common heart drugs").

In theory, knowing how people metabolize specific drugs could help doctors choose the safest, most effective treatment for their patients. But in practice, it's not that straightforward, says Dr. Jason Vassy, assistant professor of medicine at Harvard Medical School and a primary care physician at the VA Boston Healthcare System.

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Increasing Demand for Gene Mutation and Cancer Research to Drive Market

Over the past decade, organoids are being increasingly used in a range of applications, including disease modeling, pathogenesis, drug screening, and regenerative medicine, and the trend is set to continue during the forecast period. Organoid technologies have witnessed considerable developments in recent years, and continue to pave the way for the development of in vitro physiologic systems that model the origin tissues with high degree of accuracy compared to conventional approaches. The increasing focus on research and development activities is expected to play a key role in the development of the global organoids market during the assessment period.

The capability of 3D-organoid cultures to mimic organ functionality to some degree is one of the key factors likely to establish organoids as an ideal model for applications such as stem cell research to precision medicine. Furthermore, the increasing adoption of the organoid technology for use in a range of applications, including genetic mutation, gene assessment, and tumor modelling is expected to accelerate the overall development of the global organoids market during the assessment period. The assessment of different diseases by leveraging the organoid technology is anticipated to remain one of the most prominent applications of organoids in the upcoming years.

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At the back of these factors coupled with the consistent rise in the investment for research and development, the global orgnoids market is expected to cross the market value of US$ 12.8 Bn by the end of 2030.

Increasing Demand for Tumor Modelling and Biobanking to Propel Market Growth

The consistent progress in the organoid technology has gradually paved the way for tumouroids and patient-centric 3D cultures of cells that are isolated from tumor biopsies. Over the past few decades, tumouroid lines have been established from a plethora of cancer types such as liver, breast, prostate, brain, bladder, and more from metastasis as well as primary tumors. Due to consistent advancements, in the current scenario, tumouroid lines are increasingly being passed in vitro, which opens up new avenues for a range of downstream applications.

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Moreover, 3D-based cultures offer additional advantages over the conventional 2D cancer-derived cell lines. Moreover, research and development activities have indicated that tumouroid culturing is highly efficient in enabling the collection of various cancer subtypes from a large pool of patients. As organoids can be easily derived and expanded from single cancer cells, the adoption of organoids to mimic intratumour diversification in culture is on the rise a factor that is projected to propel the global organoids market during the assessment period.

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New Discoveries Driven by Research & Development to Boost Prospects

Research and development activities are set to play a key role in the overall development of the global organoids market during the assessment period. In addition, increasing collaborations between researchers and medical experts are anticipated to accelerate the development of complex organoids. For instance, two recent discoveries are expected to boost the overall prospects of next-generation organoid development. The new research collaboration between a team of scientists from the Cincinnati Childrens and Japan was announced in August 2020.

As per the research team, discoveries from the new research collaboration are likely to be one of the most critical aspects for the development of a new wave of complex organoids. Although the organoid technology has progressed at a consistent pace over the past few years, the application of the same continues to remain sluggish in the current scenario. However, increasing focus on the commercialization of organoids and consistent progress in genome editing techniques are expected to bolster the growth of the global organoids market during the assessment period.

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Demand for Human and Animal Organoids on Rise amid COVID-19 Pandemic

The advent of the novel COVID-19 pandemic is anticipated to open up new opportunities for the players involved in the current organoids market landscape. While researchers continue to assess the biology of the novel coronavirus, human and animal organoids are gradually anticipated to prove their value as an experimental virology platform. Furthermore, several researchers from various regions of the world have turned toward organoid technologies to assess the tissue tropism of the novel SARS-COV-2 virus.

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Organoids Market: Increasing Demand for Gene Mutation and Cancer Research to Drive Market - BioSpace