CGTLive®’s Weekly Rewind – March 8, 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.
CGTLive®'s Weekly Rewind
1. Neurogene Steps Up the Pace in Trial for Rett Syndrome Gene Therapy NGN-401
Cohort 1, which originally was set to enroll only 5 patients, has been expanded to allow for the inclusion of 8 patients in total.
2. Carlos Moraes, PhD, on Organelle, RNA Function and Trafficking in Neuromuscular Disease
The Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed the role of organelle transport in neuromuscular diseases such as CMT.
3. Taysha Cleared to Use Higher Dose in Adolescent and Adult Trial for Rett Syndrome Gene Therapy TSHA-102
Taysha Gene Therapies also announced additional updates on the TSHA-102 program for both adolescent adult patients and pediatric patients.
4. George Tachas, PhD, on Targeting Inflammation in DMD With Antisense Oligonucleotides
The lead scientist at Percheron Therapeutics discussed how ATL1102 could target inflammation as a secondary cause of muscle damage in DMD.
5. Zolgensma Demonstrates Clinical Benefit and Safety in Heavier Patients With Spinal Muscular Atrophy
The phase 3b SMART trial included 24 patients in total who each weighed from 8.5 kg to 21 kg at the time of treatment.
