StrideBio Appoints Deborah D. Ascheim, M.D. as Chief Medical Officer

Research Triangle Park, N.C., April 27, 2021 – StrideBio, Inc., a leading developer of novel adeno-associated viral (AAV) based gene therapies, today announced the appointment of Deborah D. Ascheim, M.D., to the newly created position of Chief Medical Officer. In this role, she will lead all clinical initiatives including the strategy, design and execution of clinical trials, as StrideBio, both independently and in collaboration with key partners, advances a pipeline of therapeutic programs that utilize internally discovered, novel engineered AAV capsids that can improve potency, evade neutralizing antibodies and enhance specific tropism to tissues critical for efficacy.

Dr. Ascheim brings to StrideBio a 30-year track record of success as a physician, clinical investigator and director of national and international clinical trials, as well as extensive expertise in developing novel, disruptive therapeutic innovations including gene therapies that target rare and complex diseases. Prior to joining StrideBio, she was President of d2a Ltd., providing independent strategic consulting to biotech and device companies, as well as academics. From 2015 to 2019, she was Chief Medical Officer of Capricor, Inc., a biotechnology company evaluating cardiosphere-derived cells for Duchenne Muscular Dystrophy. Before Capricor, Dr. Ascheim was Professor of Health Policy and Medicine (Cardiology) at the Icahn School of Medicine at Mount Sinai in New York, where she was also the director of the International Center for Health Outcomes and Innovation Research’s Clinical Trial Unit. Previously, Dr. Ascheim was on faculty in the Department of Medicine and division of Cardiology at the College of Physicians & Surgeons at Columbia University. Since 2008, she has served on the board of Physicians for Human Rights, a not-for-profit that uses medicine and science to document and advocate against mass atrocities and severe human rights violations around the world. Dr. Ascheim received her M.D. from New York University School of Medicine and a B.A. from Wellesley College.

“Dr. Ascheim is an experienced clinician with an impressive track record in the effective and efficient execution of gene therapy investigations, and her wealth of knowledge will be invaluable as we progress StrideBio to the next stage,” said Sapan Shah, Ph.D., Chief Executive Officer, StrideBio, Inc. “We are pleased to welcome Dr. Ascheim to our leadership team as we continue to harness the potential of our proprietary platform and advance next generation gene therapies to patients in need.”

“I am thrilled to join StrideBio at this important time of growth and development for the company,” said Dr. Ascheim. “The extraordinary team in place has the scientific vision, rigor, and innovative approach necessary to advance the field of gene therapy. I look forward to taking on the leadership of the clinical development of StrideBio’s therapeutic programs, working to bring them to and through the clinic for the benefit of patients suffering from severe diseases.”

About StrideBio, Inc.

Founded in 2015 based on the groundbreaking research of Mavis Agbandje-McKenna, Ph.D., and Aravind Asokan, Ph.D., StrideBio, Inc., is a fully integrated gene therapy company focused on creating best-in-class genetic medicines with life-changing or curative potential for children and adults. Our proprietary structure-inspired adeno-associated viral (AAV) vector engineering platform (STRIVE™) creates unique and differentiated capsids that overcome current limitations of first-generation gene therapies. Key targeted improvements include reduced seroprevalence, improved tropism for specific cell types, liver de-targeting and increased gene transfer efficiency, with the potential for improved safety and reduced doses in the clinic. StrideBio is advancing a robust pipeline of gene therapy candidates enabled by these novel engineered capsids, initially focused on genetically defined CNS and cardiovascular disorders. Combined with our genetic construct design expertise and in-house manufacturing capability at a 1000L scale, we are well positioned to advance novel best-in-class AAV gene therapies. StrideBio is based in a state-of-the-art 40,000-square-foot facility in Research Triangle Park, N.C., which houses our offices, research labs and in-house AAV manufacturing facilities. For more information, please visit www.stridebio.com or follow us on LinkedIn.

Contacts

StrideBio:
Robert Hughes
984-213-7301
[email protected]

For Media:
Carolyn Noyes or Susan Sharpe
781-235-3060
MacDougall
[email protected]

StrideBio Announces a Multi-technology License and Master SRA with Duke University to Advance Next-generation Gene Therapies

StrideBio receives exclusive license to novel cross-species evolved AAV vectors and an enzymatic approach for neutralizing antibody evasion

Master Sponsored Research Agreement will support collaborative research and development of multiple novel gene therapies with ability to expand

First therapeutic program under the collaboration is a gene therapy targeting Alternating Hemiplegia of Childhood, a devastating pediatric neurological disorder with no effective treatment

Research Triangle Park, N.C., April 14, 2021 – StrideBio, Inc., a leading developer of novel adeno-associated viral (AAV)-based gene therapies, today announced the signing of a multi-technology collaboration with Duke University that will enable novel next-generation gene therapies against a broad range of disorders. StrideBio is advancing multiple products incorporating these technologies with an initial program targeting a novel treatment for the pediatric neurological disorder Alternating Hemiplegia of Childhood (AHC).

The agreements announced today provide StrideBio an exclusive license to multiple technologies that will enable best-in-class next-generation gene therapies developed at Duke University. Included are novel engineered AAV vectors which complement StrideBio’s existing STRIVETM capsid engineering platform, having been selected through a cross-species evolution that results in significantly enhanced tropism and potency versus AAV9 across a wide range of tissues such as CNS, skeletal and cardiac muscle. Data on these novel vectors were presented by Duke researcher and StrideBio co-founder, Aravind Asokan, Ph.D., at the American Society of Gene & Cell Therapy 23rd Annual Meeting in an abstract titled “Cross Species Evolution of Synthetic AAV Strains for Clinical Translation” (Gonzalez et al., ASGCT 2020, Abstract 24). In addition, StrideBio has licensed exclusive rights covering a novel use of IgG-degrading enzyme IdeZ to clear neutralizing antibodies in conjunction with AAV gene therapy administration. This innovative approach was recently published by members of the Asokan Lab in a manuscript titled “Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme” (Elmore et al., JCI Insight, 2020, 5(19): e139881). Finally, StrideBio obtained license rights to a novel gene therapy approach for the treatment of AHC recently published by Duke researcher Mohamad Mikati, M.D., in a manuscript titled “AAV Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood” (Hunanyan et al., Human Gene Therapy, February 12, 2021).

Under the Master Sponsored Research Agreement (SRA), StrideBio will fund collaborative work to advance novel gene therapies initially against AHC and other undisclosed targets. AHC is a devastating pediatric neurological disorder with mutations in a causative gene, ATP1A3, that was first identified by a team of Duke University researchers, including Dr. Mikati, in 2012. StrideBio will work closely with Dr. Mikati using a mouse model of AHC developed in his lab to select and rapidly advance a novel gene therapy candidate to the clinic, leveraging the engineered AAV vectors developed by StrideBio along with its manufacturing and translational development capabilities.

The Master SRA between StrideBio and Duke University also provides a framework for additional new programs to be brought under the collaboration. These programs will aim to utilize novel engineered AAV capsids developed by StrideBio to improve potency, evade neutralizing antibodies and enhance specific tropism to tissues of interest. One additional undisclosed program targeting the CNS vasculature has been initiated.

“We are very excited to partner with Duke University to advance these technologies that can improve and expand on the potential benefits of gene therapies for patients who desperately need them,” stated Sapan Shah, Ph.D., Chief Executive Officer, StrideBio, Inc. “We look forward to working together with a fantastic group of Duke researchers and clinicians to bring next-generation AAV-based gene therapies to patients with rare CNS diseases and beyond, starting with Alternating Hemiplegia of Childhood.”

“This License and Master Sponsored Research Agreement will ensure that these innovative technologies receive the resources and expertise necessary to develop treatments that can ultimately benefit patients. We are delighted to have StrideBio as a partner on this important effort in the gene therapy area,” commented Robin Rasor, Executive Director of the Office of Licensing and Ventures, Duke University.

Specific terms were not disclosed, but include equity, upfront and milestone-driven payments, and sponsored research commitments from StrideBio to Duke University, along with royalties on future product sales.

About StrideBio, Inc.

Founded in 2015 based on the groundbreaking research of Mavis Agbandje-McKenna, Ph.D., and Aravind Asokan, Ph.D., StrideBio, Inc., is a fully integrated gene therapy company focused on creating best-in-class genetic medicines with life-changing or curative potential for children and adults. Our proprietary structure-inspired adeno-associated viral (AAV) vector engineering platform (STRIVE™) creates unique and differentiated capsids that overcome current limitations of first-generation gene therapies. Key targeted improvements include reduced seroprevalence, improved tropism for specific cell types, liver de-targeting and increased gene transfer efficiency, with the potential for improved safety and reduced doses in the clinic. StrideBio is advancing a robust pipeline of gene therapy candidates enabled by these novel engineered capsids, initially focused on genetically defined CNS and cardiovascular disorders. Combined with our genetic construct design expertise and in-house manufacturing capability at a 1000L scale, we are well positioned to advance novel best-in-class AAV gene therapies. StrideBio is based in a state-of-the-art 40,000-square-foot facility in Research Triangle Park, N.C., which houses our offices, research labs and in-house AAV manufacturing facilities. For more information, please visit www.stridebio.com or follow us on LinkedIn.

Contacts

StrideBio:
[email protected]

For Media:
Carolyn Noyes or Susan Sharpe
781-235-3060
MacDougall
[email protected]