Around the Helix: Cell and Gene Therapy Company Updates – June 8, 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. Tevogen Initiates Dosing in Final Cohort of COVID Cell Therapy Trial

Tevogen Bio has initiated dosing in the fourth and final dose level OF 3 x 106 cells/kg of their phase 1 study (NCT04765449) of TVGN-489, an investigational, genetically unmodified SARS-CoV-2-specific cytotoxic CD8+ T lymphocyte therapy.

2. ICER Finds Beti-Cel Superior to Standard-of-Care for β-Thalassemia

The Institute for Clinical and Economic Review (ICER) has rated bluebird bio’s lentiviral gene therapy betibeglogeneautotemcel a B+, concluding that is superior to standard of care for patients with β-thalassemia but the magnitude of this superiority is still uncertain.

3. Immatics and Editas Collaborate to Develop γδ T-cell Therapies

Immatics is gaining non-exclusive rights to Editas Medicine’s CRISPR technology developed at Harvard and Broad Institute. Immatics plans to use the CRISPR technology in combination with their ACTallo adoptive gamma-delta T cell platform to develop therapies for cancer.

4. Allogene’s Large B-Cell Lymphoma Therapy Receives RMAT Designation

ALLO-501A is being evaluated in the phase 1/2 ALPHA2 study (NCT04416984), positive data from which were previously presented at the ASH 2021 meeting. Allogene plans to initiate a phase 2 Pivotal Trial in mid-2022.

5. Prescient Therapeutics Debuts New CAR T-Cell Platform

The platform is dubbed CellPryme-M and is designed to produce CAR T-cells that are less prone to exhaustion and are capable of improved tumor trafficking and penetrance compared to the current generation of CAR-T cells.

6. Integral and Optimeos to Develop Novel mRNA- and DNA-Based Gene Therapies

Integral Molecular has expertise in antibody discovery and mRNA immunization with the use of its MPS Antibody Discovery platform while Optimeos Life Sciences has developed technologies for nanoparticle-based drug delivery systems.

7. FDA Clears IND Application for Passage Bio’s Metachromatic Leukodystrophy Therapy

PBML04 is an adeno-associated virus-delivery gene therapy developed through partnership with the University of Pennsylvania’s Gene Therapy Program. The therapy, developed via the intra-cisterna magna, will soon be evaluated in a phase 1 trial. 

8. UCLA Discovers Receptor to Reduce Lymphodepletion Need Before Cell Therapy

A research team led by Anusha Kalbasi, MD, in collaboration with scientists from Stanford and the University of Pennsylvania, has shown that T cells engineered with a synthetic IL-9 receptor demonstrated strong anti-tumor activity without lymphodepletion in mice.

9. Gene Therapy Startup GeneVentiv Receives Loan From North Carolina Biotechnology Center

The $250K loan will allow GenVentiv to conduct pre-clinical studies for gene therapies for blood diseases and disorders. It will also go toward supporting the IND application of their lead program GENV HEM, which was previously granted Orphan Drug Designation for the treatment of hemophilia A and B.

10. Code Bio Receives Funding for Gene Therapy Studies in DMD, Type 1 Diabetes

The company recently closed a $75 million Series A financing round that will support the development of its 2 lead programs in Duchenne Muscular Dystrophy and Type 1 Diabetes, as well as progress IND-enabling studies, pipeline and manufacturing expansions.