CGTLive’s Weekly Rewind – July 1, 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. Peter Bross, MD, on Regulating Cell Therapies: Lessons Learned

The acting chief of the oncology branch of the Center for Biologics Evaluation and Research at the FDA discussed the evolution of cell therapies in the past decade.

2. Gene Therapy for X-Linked Retinitis Pigmentosa Shows Safety, Efficacy in Phase 1/2 Study

Patients treated with MeiraGTx’s botaretigene sparoparvovec, a gene therapy in development for the treatment of X-linked retinitis pigmentosa, saw improvements in retinal function, visual function, and functional vision at 6 months post-treatment.

3. Orphan Drug Designation Granted to MB-106 in Waldenstrom Macroglobulinemia

MB-106 is currently being investigated for patients with relapsed or refractory B cell non-Hodgkin lymphoma (B-NHL) in a phase 1/2 dose-escalation study.

4. Connecting Patient Advocacy and Industry for Gene Therapy Development

Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed the panel discussion she participated in at BIO 2022.

5. Late-Onset Pompe Disease Gene Therapy Trial Placed on Clinical Hold

The hold comes after a serious AE of peripheral sensory neuropathy occurred in the phase 1/2 FORTIS study (NCT04174105) evaluating AT845, a gene-replacement therapy for the potential treatment of late-onset Pompe disease.