Around the Helix: Cell and Gene Therapy Company Updates – May 4, 2022

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

1. FDA Signs Off on Restart of Pfizer’s Hemophilia Gene Therapy Trial

After lifting the clinical hold on Pfizer’s phase 3 AFFINE trial (NCT04370054) of giroctocogene fitelparvovec (SB-525; PF-07055480) for the potential treatment of hemophilia A in March 2022, the US Food and Drug Administration (FDA) has now cleared the trial to restart, the company announced in its first-quarter earnings call.

2. FDA Fast Tracks Graphite Bio's Sickle Cell Disease Gene-Edited Cell Therapy

The FDA has granted fast track designation to Graphite Bio's GPH101, its investigational gene-edited autologous hematopoietic stem cell therapy for the treatment of sickle cell disease.

3. Taysha Granted European Orphan Drug Designation for Giant Axonal Neuropathy Gene Therapy

The European Commission has granted orphan drug designation to TSHA-120, an investigational AAV9 gene therapy currently being evaluated for the treatment of giant axonal neuropathy.

4. FDA Places Phase 1/2 Trial of Vertex’s Type 1 Diabetes Cell Therapy VX-880 on Hold

The US Food and Drug Administration (FDA) has placed a clinical hold on Vertex Pharmaceuticals’ phase 1/2 study (NCT04786262) of VX-880 for the potential treatment of patients with type 1 diabetes (T1D), the company announced in a statement also reporting positive data based on the first 3 patients dosed in the study.

5. Alaunos Therapeutics Doses First Patient in TCR-T Library Phase 1/2 trial for Solid Tumors

Alaunos Therapeutics has dosed its first patient in its TCR-T Library phase 1/2 trial targeting KRAS, TP53, and EGFR mutations across 6 solid tumor indications using their Sleeping Beauty transposon/transposase technology.

6. Biogen and Scribe Partnership Expands

Biogen is exercising its option for an additional neurological disease target in gene therapy using Scribe Therapeutics' CRISPR technology.