Governor Roy Cooper announced today that Beam Therapeutics (Nasdaq; BEAM), a biotechnology company developing precision medicines through DNA base editing, plans to build a manufacturing facility in North Carolinas Research Triangle Park, creating 201 jobs. Over a period of 5 years, the company expects to invest $83 million in the facility, which will support clinical and commercial manufacturing for the companys novel base editing programs.

"North Carolina is a leader in biotechnology, from the research in our labs to the states biomanufacturers, said Governor Cooper. Companies like Beam Therapeutics work in developing precision medicines will help keep North Carolina on the cutting edge of this industry.

Beam Therapeutics, with headquarters in Cambridge, Massachusetts, develops precision genetic medicines through base editing. The foundational level of genetic information is a single base letter in DNA, and an error to a single letter, known as a point mutation, can cause disease. Base editors have the ability to rewrite just a single letter, and thereby intervene at the most foundational level. Beams proprietary base editors create precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs.

We believe investment in strategic manufacturing capabilities is an important component of fully realizing the power of our base editing technology and achieving our vision to provide life-long cures to patients suffering from serious diseases, said John Evans, CEO of Beam Therapeutics. Research Triangle Park is a thriving biopharmaceutical hub, providing significant access to the broad range of talent we will need to make this vision a reality.

Although wages will vary depending on position, the average salary for the new positions will be $102,654. The average wage in Durham County is $71,756. The state and local area will see a yearly economic impact of more than $20.6 million from this companys new payroll.

"North Carolina has been a world leader in biotechnology for many years, but were not resting on our past accomplishments, said North Carolina Commerce Secretary Anthony M. Copeland. Beam Therapeutics joins a host of gene therapy companies that are keeping North Carolina at the forefront of this new frontier of medicine.

Beam Therapeutics project in North Carolina will be facilitated, in part, by a Job Development Investment Grant (JDIG) approved by the states Economic Investment Committee earlier today. Over the course of 12 years, the project is estimated to grow the states economy by $1.36 billion. Using a formula that takes into account the new tax revenues generated by the new jobs, the agreement authorizes the potential reimbursement to the company of up to $3,237,750, spread over 12 years. Payments for all JDIGs only occur following performance verification by the departments of Commerce and Revenue that the company has met its incremental job creation and investment targets. JDIG projects result in positive net tax revenue to the state treasury, even after taking into consideration the grants reimbursement payments to a given company.

Because Beam Therapeutics chose a site in Durham County, classified by the states economic tier system as Tier 3, the companys JDIG agreement also calls for moving as much as $1,079,250 into the states Industrial Development Fund Utility Account. The Utility Account helps rural communities finance necessary infrastructure upgrades to attract future business. Even when new jobs are created in a Tier 3 county such as Durham, the new tax revenue generated through JDIG grants helps more economically challenged communities elsewhere in the state. More information on the states economic tier designations is available here.

In addition to the North Carolina Department of Commerce and the Economic Development Partnership of N.C., other key partners on this project were the the North Carolina Community College System, the North Carolina Biotechnology Center, Durham County, and the Greater Durham Chamber of Commerce.

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Governor Cooper Announces Genetic Medicine Company Will Create 201 Jobs in Durham County - NC Dept of Commerce

CAMBRIDGE, Mass. and GAINESVILLE, Fla., Aug. 11, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and the University of Florida today announced a strategic collaboration to enable cutting-edge research for novel genetic medicines. Through the agreement, Sarepta will fund multiple research programs at the University, and will have an exclusive option to further develop any new therapeutic compounds that result from the funded research programs.

We have developed a productive incubator approach to our pipeline development, partnering with the best and brightest in genetic medicine, including leading academic researchers like those at the University of Florida, to discover and translate into meaningful therapies genetic medicine for rare diseases, said Sarepta President and CEO Doug Ingram. Weare excited topartner with andsupportUF research that has the potentialto profoundlyimproveand extend the lives of patients with rare genetic-based diseases.

Through the collaboration, currently unique to UF, funding has been allocated for four innovative projects. These projects include exploratory research in novel gene therapy vectors, next-generation capsids and gene editing technologies as well as work in new therapeutic areas in degenerative genetic diseases. The goal is to foster early relationships with experts and accelerate the scientific advancements that lead to the development of transformational precision genetic medicines for patients in need.

Our researchers intend to find solutions for diseases that have no cure or limited therapeutic options. Their goal is to move these solutions from their labs to patients who need them to see their discoveries change lives. Because Sarepta has a focus and expertise in disease areas that coincide with the work of some of our scientists, its a match and collaboration that make sense and, we hope, will save lives, said Jim OConnell, assistant vice president of UF Innovate, the technology commercialization arm of the university. Sarepta has a bold vision for transforming genetic disease because the company, ultimately, serves patients. That end goal drives its willingness and ability to translate research into a medical reality. We want to be part of that.

University of Florida is a gene therapy powerhouse. UF researchers were the first to discover the life cycle of the adeno-associated virus (AAV), the smallest human virus. Using AAV as a benign delivery vehicle to carry therapeutics to a target, UF was first to reverse blindness in dogs with genetic disease, and UF researchers were integral in the first gene therapy approved by the FDA to treat an inherited genetic disease that can cause blindness. Today, UF is developing technologies in manufacturing, capsid design and therapies to address neuromuscular, cardiovascular, inflammatory, metabolic, pulmonary, skeletal, ophthalmic, and other disorders.

About SareptaAt 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.comor follow us onTwitter,LinkedIn,InstagramandFacebook.

Sarepta Forward-Looking Statements This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the ability of the collaboration between Sarepta and UF to engage in cutting-edge research for novel genetic medicines; Sareptas commitment to fund multiple research programs at UF; Sareptas option to further develop any new therapeutic compounds that result from the funded research programs; Sareptas incubator approach to discover and translate into meaningful therapies genetic medicine for rare diseases; the collaborations potential to profoundly improve and extend the lives of patients with rare genetic-based diseases; the collaborations ability to foster early relationships with experts to accelerate the scientific advancements that lead to the development of transformational precision genetic medicines; and Sareptas vision to transform genetic disease and translate research into a medical reality.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and UF may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in the discovery of any new therapeutic compounds or any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Contacts:

Sarepta Therapeutics Investors: Ian Estepan, 617-274-4052, iestepan@sarepta.comMedia: Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com

UF Innovate: Sara Dagen, 352-294-0998, saradagen@ufl.edu

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Sarepta Therapeutics and University of Florida Announce Collaboration to Accelerate the Discovery and Development of Therapies for Rare Genetic...

LEXINGTON, Mass., Aug. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio or the Company), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, today reported financial results for the quarter ended June 30, 2020, provided a business update and announced the U.S. Food and Drug Administration (FDA) has cleared the Companys Investigational New Drug (IND) application for LB-001 for the treatment of methylmalonic acidemia in pediatric patients. LogicBio released a separate press release this morning providing further details on the planned Phase 1/2 clinical design for LB-001.

We are thrilled to have received clearance to move forward with this first-in-human clinical trial with our lead product candidate, LB-001, for the treatment of methylmalonic acidemia, a life-threatening congenital genetic disease with no current therapeutic treatment options. This represents a significant milestone in our goal of bringing a treatment to MMA patients as well as for our GeneRide platform. We have maintained continuous dialogue with the centers of excellence that are planned to participate in the Phase 1/2 clinical trial, and we look forward to activating these sites as quickly as possible, said Fred Chereau, CEO of LogicBio. We have instituted systems attempting to mitigate COVID-19 dynamics on our study start-up process and, based on our best estimates, we plan to enroll our first patient in early 2021.

Commenting on the Next Generation Capsid Program, Mr. Chereau said, We are very excited about the recent advances in our novel capsid program, which has generated liver-tropic capsids intended for use in gene editing technologies such as GeneRide and other gene therapy approaches. We are focused on executing across all of our programs and look forward to sharing further details on our novel capsids in early 2021.

Appointment of Daniel Gruskin, M.D. to SVP, Head of Clinical Development

Daniel Gruskin, M.D. was appointed as SVP, head of clinical development in August 2020. Dr. Gruskin has served as interim head of clinical development of LogicBio since June 2020. In April 2020, Dr. Gruskin started consulting with the Company as a special advisor. Previously, Dr. Gruskin served in roles of increasing responsibility at Sanofi Genzyme, most recently as vice president, head of global medical affairs, rare disease, in which capacity he oversaw medical affairs, life cycle management, scientific affairs and other medical and development activities related to metabolic, rare and/or genetic diseases. Prior to his role at Sanofi Genzyme, Dr. Gruskin served as assistant professor, human genetics and pediatrics at Emory University School of Medicine, where he was also the chief of the genetics section at Childrens Healthcare of Atlanta.

Daniel has been instrumental in leading LB-001 clinical development efforts including getting the IND cleared. His deep experience in genetic medicines and metabolic diseases will serve LogicBio well as we look to execute on our goals for both the GeneRide and Next Generation Capsid platforms in search of transformative medicines, said Mr. Chereau.

Anticipated Milestones for 2020 and 2021:

Second Quarter 2020 Financial Results

Three Months Ended June 30, 2020 and 2019

About LogicBio Therapeutics

LogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms.

LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration with Takeda to research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.

LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.

LogicBio is headquartered inLexington, Mass. For more information, please visitwww.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 Companys plans to initiate, advance and complete its planned SUNRISE Phase 1/2 clinical trial of LB-001 in MMA; the timing, progress and results of the Companys research and development activities, including those related to the GeneRide technology platform and Next Generation Capsid Program; its plans for LB-301 in Crigler-Najjar; and the sufficiency of its cash and cash equivalents to fund operating expenses and capital expenditure requirements. 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. In particular, the impact of the COVID-19 pandemic on the Companys ability to progress with its research, development, manufacturing and regulatory efforts, including the Companys plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Companys 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 Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on March 16, 2020 with the SEC, the Companys Quarterly Report on Form 10-Q filed on May 11, 2020, 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:

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

Media:Stephanie SimonTen Bridge CommunicationsStephanie@tenbridgecommunications.com617-581-9333

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LogicBio Therapeutics Reports Second Quarter 2020 Financial Results and Provides Business UpdatesFDA Clears IND Application for LB-001 for the...

Injections of a natural energy molecule prompted regrowth of almost half of the cartilage lost with aging in knees, a new study in rodents shows.

The study results revolve around the long-established idea that machines within animal and human cells turn the sugars, fats, and proteins we eat into energy used by the bodys millions of cells. The molecule most used to store that energy is called adenosine triphosphate, or ATP. Along with this central role in metabolism, adenosine also helps signal other cells and serves as a building block of genetic material, and so is central to the growth of human tissue.

Previous research had shown that maintaining supplies of adenosine, known to nourish the chondrocyte cells that make cartilage, also prevented osteoarthritis in similar animal models of the disease.

In the new NYU Grossman School of Medicineled study, researchers injected adenosine into the joints of rodents whose limbs had been damaged by inflammation resulting from either traumatic injury, such as a torn ligament, or from massive weight gain placing pressure on joints. The biological damage in these cases is similar, researchers say, to that sustained in human osteoarthritis.

Published online in the journal Scientific Reports on August 10, the study rodents received 8 weekly injections of adenosine, which prompted regrowth rates of cartilage tissue between 50 percent and 35 percent as measured by standard laboratory scores.

Our latest study shows that replenishing adenosine stores by injection works well as a treatment for osteoarthritis in animal models of the disease, and with no apparent side effects, says lead study author Carmen Corciulo, PhD, a postdoctoral fellow at NYU Langone.

Dr. Corciulo says it is too soon to use this experimental model as a therapy in people. Clinical trials must await a test drug that can be safely stored for days if not weeks, and experiments in larger mammals.

Study senior investigator Bruce N. Cronstein, MD, the Dr. Paul R. Esserman Professor of Medicine at NYU Langone Health, says the teams research is important because the few existing drug therapies for osteoarthritis such as acetaminophen and COX-2 inhibitor drugs, including naproxen and ibuprofen, only numb joint pain, or like hyaluronic acid just lubricate its tissues. None stall disease progression or reverse the damage. Painkillers, such as opioids, are often prescribed, but are also highly addictive, he cautions.

People with osteoarthritis desperately need more treatment options with fewer side effects, and our research advances that effort, says Dr. Cronstein, who also serves as the director of NYU Langones Clinical and Translational Science Institute. He notes that other experimental medications are being developed elsewhere, including parathyroid hormone to stimulate bone growth, WNT inhibitor drugs to block the bone and cartilage degradation, and growth factor chemicals to promote cartilage growth.

Dr. Cronstein, Dr. Corciulo, and NYU Grossman School of Medicine have a patent application pending for the use of adenosine and other agents that help with its binding to chondrocytes, called A2A receptor agonists, for the treatment of osteoarthritis.

Among the studys other key findings was that a cell-signaling pathway, known as transforming growth factor beta (TGF-beta) and involved in many forms of tissue growth, death, and differentiation, was highly active in cartilage tissue damaged by osteoarthritis, as well as in cartilage tissue undergoing repair after being treated with adenosine. Additional testing in lab-grown chondrocytes from people with osteoarthritis showed different chemical profiles of TGF-beta signaling during breakdown than during growth, providing the first evidence that the pathway switched function in the presence of adenosine (from assisting in cartilage breakdown to encouraging its repair.)

Developing treatments to halt or slow the disease is important, Dr. Cronstein says, because well over 100 million people worldwide are estimated to have osteoarthritis, which is tied to aging, especially in women. This figure, he says, is only expected to grow as more people live longer and obesity rates climb.

Right now, the only way to stop osteoarthritis is to have affected joints surgically replaced, which not only comes with pain and risk of infection, but is also quite costly, says Dr. Cronstein. If new therapies can delay or prevent disease onset and progression, then fewer joint replacements will save people from a lot of pain and expense.

The study was funded by National Institutes of Health grants R01 AR056672 and R01 AR068593, NYU-HHC Clinical and Translational Science Institute grant UL1 TR000038, and the Arthritis Foundation.

Dr. Corciulo and Dr. Cronstein have a patent for the methods and compositions for treating osteoarthritis and promoting cartilage formation (U.S. Patent 10,441,541), which has been assigned to NYU Grossman School of Medicine. They are cofounders of Regenosine Inc., a company that is developing new treatments for osteoarthritis, and in which they hold a financial interest. Dr. Cronstein has consulted for Eli Lilly, Horizon Pharmaceuticals, Bristol Myers Squibb, and Astrazeneca. He also has grants from Arcus Biopharma. All relationships are being managed in accordance with the policies and practices of NYU Langone.

Besides Dr. Cronstein and Dr. Corciulo, other NYU Langone investigators involved in this study are Cristina Castro, MD; Thomas Coughlin, PhD; Samson Jacob, MS; David Fenyo, PhD; Daniel B. Rifkin, PhD; and Oran Kennedy, PhD.

David MarchPhone: 212-404-3528david.march@nyulangone.org

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New Approach to Treating Osteoarthritis Advances | NYU Langone News - NYU Langone Health