CGTLive®’s Weekly Rewind – September 6, 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. Abata Therapeutics' Treg Therapy ABA-101 Garners FDA Fast Track Designation

The therapy is being developed for the treatment of patients with progressive MS who have imaging evidence of ongoing inflammatory tissue injury and who are HLA-DRB1*15:01 positive.

2. Crystal Proud, MD, on Improving Newborn Screening for Spinal Muscular Atrophy

The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, spoke about the enhancements to newborn screening for the disease.

3. Grit Biotechnology’s TIL Therapy GT201 Cleared for US Trial in Advanced Solid Tumors

GT201 previously received IND clearance from China’s Center for Drug Evaluation in July 2023.

4. REGENXBIO’s MPSII Gene Therapy RGX-121 Continues to Show Efficacy in Long-Term Trial Data

Among patients who received the pivotal dose level, a median reduction of 85% in HS D2S6 levels was seen in the CSF.

5. Biogen’s Nusinersen Shows Efficacy in SMA Compared With Sham Treatment at Experimental Higher Dose

The data come from the pivotal part B cohort of the phase 2/3 DEVOTE clinical trial.