CGTLive’s Weekly Rewind – February 2, 2024

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Review top news and interview highlights from the week ending February 2, 2024.

CGTLive Weekly Rewind

CGTLive Weekly Rewind

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 Finalizes Sets of Guidelines for Both CAR-T Products and Genome Editing Products

According to Federal Register notices, the guidance documents are updated versions of drafts originally published in March 2022.

2. Barry J Byrne, MD, PhD, on the Potential of Directed Evolution to Improve AAV Gene Therapy

The chief medical advisor of the Muscular Dystrophy Association discussed how directed evolution may help develop AAV capsids better suited for treating neuromuscular diseases.

3. FDA Approves Kite’s Axi-Cel Manufacturing Change to Reduce Turnaround Time

Kyverna has also announced data on its proprietary CAR-T manufacturing process, Ingenui-T.

4. Alfonso Sabater, MD, PhD, on Further Research With Gene Therapy in Eye Care

The associate professor of clinical ophthalmology at University of Miami discussed the first topical application of gene therapy to the eye.

5. Sarepta Therapeutics' Phase 3 Study for Limb-Girdle Muscular Dystrophy Gene Therapy SRP-9003 Begins Screening Activities

The multinational, open-label EMERGENE study will seek to enroll 15 patients with LGMD2E/R4 in total.

Recent Videos
Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA
Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen
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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