CGTLive’s Weekly Rewind – March 17, 2023

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.

Data from the open-label phase 3 HOPE-B trial (NCT03569891) demonstrate that the treatment of patients with hemophilia B with etranacogene dezaparvovec (Hemgenix; CSL Behring) is superior to prophylactic factor IX treatment on annualized bleeding rate, with a favorable safety profile.

Another serious adverse event (AE) and dose-limiting toxicity is the latest blow to Bellicum Pharmaceuticals, who accounced it would halt progress on its clinical trials for its 2 GoCAR-T cell therapy candidates, BPX-601 and BPX-603. The latest in what has been a few rocky years for the biotech, this news has the company considering "strategic alternatives" for its path forward.

The associate professor of clinical pediatrics at Cincinnati Children's Hospital Medical Center discussed Sangamo Therapeutics’ gene therapy, ST-920, and the phase 2 STAAR clinical trial. In addition, Hopkin discussed preliminary findings from kidney biopsies, the potential implications for patients if ST-920 is approved, and future plans for further research for the gene therapy.

An open-label, prospective, single-arm phase 2 clinical trial (NCT05590221) of relmacabtagene autoleucel injection (relma-cel; JW Therapeutics) has been initiated in China, according to an announcement. The trial is aimed at assessing the autologous anti-CD19 CAR-T cell immunotherapy in a population of 20 individuals with high-risk large B-cell lymphoma (LBCL) as the first-line treatment and first patient infusion.

GenSight Biologics’ lenadogene nolparvovec (Lumevoq; GS010), an investigational adeno-associated viral (AAV) vector-based gene therapy being evaluated for the treatment of Leber hereditary optic neuropathy (LHON) in the phase 3 REFLECT clinical trial (NCT03293524), has demonstrated sustained efficacy and safety in 3-year follow-up data, with the best outcomes in patients who received bilateral treatment.