CGTLive®’s Weekly Rewind – March 8, 2024

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.