CGTLive’s Weekly Rewind – February 18, 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. Achieving Developmental Milestones With GM1 Gangliosidosis Gene Therapy: David Weinstein, MD

The senior vice president of clinical development at Passage Bio discussed new data from the IMAGINE-1 trial presented at WORLDSymposium. He discussed the encouraging data in biomarkers, safety, and development seen so far in the 2 patients dosed.

2. The Potential Impact of Gene and Cell Therapy in Cystinosis: Stephanie Cherqui, PhD

The associate professor from UC San Diego discussed the gene-edited cell therapy that her lab has developed. She discussed the need for curative therapies in cystinosis and the potential of gene and cell therapies to fulfill this need.

3. Around the Helix: Cell and Gene Therapy Company Updates - February 16, 2022

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

4. Gene Therapy Well-Tolerated in Late-Onset Pompe Disease

The gene therapy AT845 (Astellas Pharma) has been well-tolerated in adult patients with late-onset Pompe disease (LOPD), according to positive interim safety data from the phase 1/2 FORTIS study (NCT04174105) presented at the 18th Annual WORLDSymposium.

5. Memory T Cell CAR T Therapy Showcases Anti-Tumor Activity in Resistant Prostate Cancer

P-PSMA-101, Poseida Therapeutics’ autologous chimeric antigen receptor (CAR) T-cell candidate, has shown promising anti-tumor activity in metastatic castration-resistant prostate cancer (mCRPC), with significant declines in prostate-specific antigen recorded