CGTLive’s Weekly Rewind – April 22, 2022

Article

Review top news and interview highlights from the week ending April 15, 2022.

Welcome to CGTLive’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

1. FDA Grants GCC19CART Fast Track Designation for Colorectal Cancer

The FDA has granted a Fast Track Designation to GCC19CART as a potential treatment for adult patients with relapsed and refractory metastatic colorectal cancer.

2. Building on CRISPR and Striving for Definitive Cures: Josh Lehrer, MD

The chief executive officer of Graphite Bio discusses the phase 1/2 CEDAR trial of GPH101, an investigative Cas9-edited autologous CD34+ cell therapy for the treatment of sickle cell disease.

3. T Cell Therapy for Post-Transplant Virus Prevention Granted RMAT Designation

This is the third regenerative medicine advanced therapy designation granted to AlloVir’s posoleucel therapy.

4. Pushing for a New Era in Cardiology: Matt Killeen, PhD

The chief scientific officer at Renovacor shares updates on the company's lead candidate, REN-001, an adeno-associated viral vector serotype 9-based gene therapy for the treatment of BAG3-associated dilated cardiomyopathy.

5. ADI-001 Granted Fast Track Designation for Non-Hodgkin Lymphoma

ADI-001 has been granted a Fast Track Designation by the FDA a potential treatment for patients with relapsed or refractory B-cell non-Hodgkin lymphoma.

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Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA
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Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
Barry J. Byrne, MD, PhD, the chief medical advisor of Muscular Dystrophy Association (MDA) and a physician-scientist at the University of Florida
John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia
Chun-Yu Chen, PhD, a research scientist at Seattle Children’s Research Institute
William Chou, MD, on Targeting Progranulin With Gene Therapy for Frontotemporal Dementia
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