CGTLive’s Weekly Rewind – February 25, 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. Novel Chemo/NK Cell Therapy Combination Shows Efficacy in Pancreatic Cancer

The novel combination of aldoxorubicin, N-803 IL-15 superagonist, and PDL1- natural killer (NK) cell therapy after chemo-radiation has shown efficacy as third-line or greater treatment in patients with metastatic or locally advanced pancreatic cancer.

2. Adrenomyeloneuropathy Gene Therapy Granted Fast Track Review

The FDA has granted fast track designation to SwanBio Therapeutics’ SBT-101, an investigational adeno-associated virus gene therapy candidate for the treatment of adrenomyeloneuropathy (AMN) after clearing their investigational new drug application earlier this month.

3. Targeting Unmet Needs in Rare Dermatologic Diseases With Gene Therapy

Matthew Gantz, president and chief executive officer, Castle Creek Biosciences, discussed FCX-013 for the potential treatment of scleroderma. He also discussed unmet needs in recessive dystrophic epidermolysis bullosa (RDEB) and the importance of collaborating with patient advocacy groups.

4. Second Phenylketonuria Gene Therapy Study Put on Clinical Hold

The FDA has placed a clinical hold on Homology Medicines’ pheNIX trial (NCT03952156), which is investigating the gene therapy HMI-102 for the potential treatment of phenylketonuria (PKU) following elevated liver function test scores.

5. CAR T-Cell Therapy Demonstrates Feasibility in Resistant Prostate Cancer

Mustang Bio’s prostate stem cell antigen (PSCA)-targeted chimeric antigen receptor (CAR) T-cell therapy MB-105 has demonstrated feasibility in metastatic castration-resistant prostate cancer mCRPC according to results presented at the 2022 American Society of Clinical Oncology Genitourinary (ASCO GU) Cancers Symposium.