Around the Helix: Cell and Gene Therapy Company Updates – April 12, 2023

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. Freeline Goes All in on Gaucher Disease

Freeline Therapeutics is deprioritizing FLT190, its gene therapy program for Fabry disease, in favor of FLT201 for Gaucher disease. The company also announced that it would be cutting 30% of its workforce.

2. Mesothelioma KIR-CAR T-cell Therapy Gets FDA Fast Track Designation

Verismo Therapeutics’ SynKIR-110, an investigational killer immunoglobulin-like receptor - chimeric antigen receptor (KIR-CAR) T-cell therapy intended to treat mesothelin-expressing cancers including malignant pleural mesothelioma, has been granted fast track designation by the FDA.

3. Immatics’ Solid Tumor Cell Therapy Pipeline Evolves

Immatics is discontinuing a clinical stage program and delaying a preclinical solid tumor cell therapy program, according to a corporate update. These programs are IMA201, currently in a phase 1/2 trial (NCT03247309), and IMA204, which is in preclinical studies.

4. Duchenne Muscular Dystrophy Gene Therapy RGX-202 Granted FDA Fast Track Designation

REGENXBIO’s RGX-202, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Duchenne muscular dystrophy (DMD), has received fast track designation from the FDA.

5. Adaptimmune Therapeutics Expands Pipeline With Return of Rights and Materials from GSK

The agreement, which relates to the return of the PRAME and NY-ESO cell therapy programs, also includes a payment of £30 million to Adaptimmmune.

6. Ginkgo Bioworks Acquires Platform Assets from StrideBio

Among the assets included in the acquisition are AAV capsid discovery and engineering platform assets, which Gingkgo Bioworks intends to utilize in its gene therapy research and development service offerings.

7. Center for Breakthrough Medicines Picks Up Pennsylvania Gene Therapy Program's Adherent Cell Manufacturing Platform in New Technology Transfer

The Center for Breakthrough Medicines will incorporate the platform into its AAV gene therapy manufacturing services.

8. Thermo Fisher Scientific Launches New Cell Therapy Manufacturing Facility at UCSF

The new facility is strategically placed near the University of California, San Francisco (UCSF) Medical Center’s Mission Bay campus with the intention of accelerating CAR-T and other cell therapy research taking place there.

9. KromaTiD Announces Launch of New Kit for Tracking T-Cell Engineering Impacts

The dGH in-Site™ CAR-T Kit, which is intended to be used as an in-house tool for academic investigators and labs, allows for the tracking of T-cell editing outcomes at the TRAC & B2M loci.

10. Leucid Bio to Use ImaginAb's Imaging Agent in CAR-T Trial

The agreement relates to Leucid Bio's planned use of ImaginAb's 89Zr crefmirlimab berdoxam in its clinical trial evaluating LEU011, an autologous CAR-T, for the treatment of solid tumors.

11. VintaBio Announces Opening of New Cell and Gene Therapy Manufacturing Facility in Philadelphia

The facility's development was supported by $64 million in funding provided to VintaBio by a group of investors led by Decheng Capital.