GeneTherapyLive’s Weekly Rewind – November 19, 2021

Welcome to GeneTherapyLive’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. FDA Manufacturing Data Request Delays Omidubicel BLA Submission

The FDA has requested a revised analysis of manufacturing data from Gamida Cellon omidubicel (NiCord), a cell therapy for bone marrow transplant in patients with hematologic malignancies. Following their meeting, Gamida Cell expects to submit a biologics license application (BLA) in early 2022 as opposed to late 2021.

2. Assessing Tab-Cel for EBV+ Post-Transplant Lymphoproliferative Disease

AJ Joshi, MD, chief medical officer, Atara Biotherapeutics, discussed data from the ALLELE study to be presented at ASH 2021. He discussed positive data seen in enrolled participants, including safety findings and response rates.

3. OpRegen Shows Durable Efficacy in Dry AMD Geographic Atrophy

OpRegen (Lineage Cell Therapeutics) has continued to show safety and efficacy in patients with dry age-related macular degeneration with geographic atrophy, according to recent data from a phase 1/2a clinical trial (NCT02286089).

4. A New Indication for Gene Therapy: Aging

Dan Oliver, cofounder and chief executive officer, Rejuvenate Bio, discussed the company’s ultimate goal of reversing aging. He discussed their lead programs including RJB-01, which targets FGF21 and TGFβ1 and is being investigated in both humans and dogs. 

5. CYNK-101 Demonstrates Anti-Tumor Activity in HNSCC Cells

The allogeneic natural killer cell therapy CYNK-101 has shown promising anti-tumor activity for EGFR+ lung cancer and head and neck squamous cell carcinoma in preclinical studies.