GeneTherapyLive’s Weekly Rewind – December 24, 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. Check it out below and have a happy holiday!

1. Eli-Cel Gene Therapy for CALD Is Granted Priority Review

bluebird bio’s biologics license application (BLA) for elivaldogene autotemcel (Skysona; eli-cel, Lenti-D®), a gene therapy for the potential treatment of cerebral adrenoleukodystrophy (CALD) in pediatric patients, has been granted priority review by the FDA. The Prescription Drug User Fee Act (PDUFA) date is set for June 17, 2022.

2. Poseida’s CAR T-Cell Therapy IND Accepted for Solid Tumors

The FDA has cleared Poseida Therapeutics’ investigational new drug application for the allogeneic chimeric antigen receptor (CAR) T-cell therapy P-MUC1C-ALLO1 targeting multiple solid tumors. A multi-center, open-label, phase 1 study will be conducted in adults with refractory locally-advanced or metastatic epithelial-derived solid tumors.

3. Addressing Unmet Needs in Hemophilia: Guy Young, MD

The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed the ATLAS-INH study of fitusiran in people with hemophilia A and B. He also discussed the goals of current clinical trials in hemophilia and unmet needs in the population.

4. Patient Dies in Phase 1b Mini-Dystrophin Gene Therapy Trial in DMD

A patient with Duchenne muscular dystrophy (DMD) participating in the non-ambulatory cohort of Pfizer’s phase 1b mini-dystrophin gene therapy trial (NCT03362502) has died, the company announced on December 20th. The open-label study is evaluating fordadistrogene movaparvovec, formerly known as PF-06939926, an investigational AAV9 gene therapy designed to treat DMD.

5. bluebird bio Sickle Cell Gene Therapy On Hold Following Adverse Event

bluebird bio's sickle cell disease gene therapy program, lovotibeglogene autotemcel (lovo-cel), has been placed on partial clinical hold following a report of persistent anemia in an adolescent patient. The HGB-206, HGB-210 and LTF-307 clinical studies will pause enrollment of patients under the age of 18.