CGTLive®’s Weekly Rewind – April 5, 2024

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. First US Patient With Hemophilia B Receives Gene Therapy

Val-rox was approved as Roctavian in December 2023.

2. Shalini Shenoy, MD, MBBS, on Comparing Treatment Options for Sickle Cell Disease

The director of the Stem Cell Transplant & Cellular Therapy Program at St. Louis Children’s Hospital discussed when the choice should be made to transition from symptom management to curative therapies.

3. Verve Moves on to Second Gen Cardiovascular CRISPR Therapy After Adverse Events

The sixth participant dosed experienced grade 3 treatment-induced ALT increases and thrombocytopenia.

4. Mitchell Horwitz, MD, on Cost-Effectiveness of Omidubicel for Allo-HCT

The professor of medicine at Duke Cancer Institute discussed uptake and research with the cord blood therapy since its approval.

5. Kyverna’s CAR T Seems Feasible in Multiple Sclerosis

KYV-101 is also being evaluated in lupus nephritis, myasthenia gravis, and systemic sclerosis.