CGTLive’s Weekly Rewind – June 24, 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. Krystal Seeks Approval for Dystrophic Epidermolysis Bullosa Gene Therapy

Krystal Biotech has submitted a biologics license application for B-VEC (beremagene geperpavec), its gene therapy for the potential treatment of dystrophic epidermolysis bullosa.

2. Steven W. Pipe, MD, on Challenges in Gene Therapy Trials for Hemophilia

The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed common challenges with gene therapy clinical trials.

3. Gene-Mediated Immunotherapy for Ovarian Cancer Clears Safety Hurdle, Will Continue Dosing

Celsion’s GEN-1, an IL-12 DNA plasmid vector, was determined to have a satisfactory safety profile and acceptable risk/benefit by an independent Data Safety Monitoring Board (DSMB) that has recommended dosing to continue.

4. Thomas Martin, MD, on Exciting Cell Therapy Options in Multiple Myeloma

The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed cilta-cel and upcoming cell therapies.

5. Children Treated With Zolgensma for Presymptomatic SMA Achieve Age-Appropriate Motor Milestones

All children in the phase 3 SPR1NT study achieved the primary end point of independent sitting.