Around the Helix: Cell and Gene Therapy Company Updates – January 4, 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. Tessa Therapeutics Shifts Focus to Allogeneic Therapies

Tessa Therapeutics will be shifting its focus primarily to allogeneic therapies based on its CD30.CAR-modified Epstein-Barr virus-specific T-cell (EBVST) therapy platform, according to a corporate update.

2. Cellectis Shifts to In-House Manufacturing With UCART22

Cellectis has dosed the first patient with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) with its allogeneic, in-house manufactured chimeric antigen receptor (CAR) T-cell therapy UCART22. The patient has completed the 28-day observation period and no dose-limiting toxicities (DLTs) were observed as of December 14, 2022.

3. BCMA-directed CAR-T Gets IND Clearance for R/R Multiple Myeloma

IASO Biotherapeutics’ equecabtagene autoleucel (CT103A), an investigational autologous chimeric antigen receptor T-cell (CAR-T) therapy being developed for the treatment of relapsed/refractory (r/r) multiple myeloma (MM), has received clearance of its investigational new drug (IND) application from the FDA.

4. Gene Therapies Advance in Danon Disease, CLN2 Batten Disease

Progress is being made with gene therapies in development for rare diseases, with both Rocket Pharmaceuticals and REGENXBIO announcing updates for their gene therapy programs in Danon disease and late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease (the most common form of Batten disease), respectively.

5. UT Southwestern Scientists Demonstrate Preclinical Proof-of-Concept for Dilated Cardiomyopathy Gene Therapy

The researchers were able to correct all mutations associated with the disease in human cells and a mouse model with a CRISPR-Cas9 gene editing system.

6. Mayflower Bioventures and Cellectis Collaborate to Launch New Start-up Focused on Mitochondrial Diseases

The new company, Primera Therapeutics, will work with Cellectis to develop a new gene-editing platform with the intention of correcting mutations in mitochondrial DNA.

7. Japan's Ministry of Health, Labour and Welfare Approves Yescarta for Second-Line Treatment of Large B-cell Lymphoma

The decision was informed by results from the ZUMA-7 (NCT03391466) study.

8. Opus Genetics Acquires Preclinical Gene Therapies for BEST1-related Inherited Retinal Diseases and Rhodopsin-mediated Autosomal Dominant Retinitis Pigmentosa

The company now has the rights for research, development, and commercialization of the 2 investigational therapies.

9. Capsida Biotherapeutics, Prevail to Collaborate on CNS Disease Gene Therapies

Prevail will utilize Capsida's platform for adeno-associated virus capsid identification for its therapies.

10. CANbridge Pharmaceuticals Acquires Rights to Investigational Spinal Muscular Atrophy Gene Therapy

The company also announced the successful completion of a technology transfer from LogicBio Therapeutics related to investigational gene therapies for Fabry disease and Pompe disease.

11. Hadassah Medical Organization, Bar-Ilan University, Immix BioPharma Announce Collaboration to Advance anti-BCMA CAR-T

HBI0101 is currently being evaluated in a phase 1b clinical trial for patients with multiple myeloma and AL amyloidosis.

12. Cilta-Cel NDA Submission Accepted by China's NMPA

The submission is supported by results from CARTIFAN-1, a phase 2 clinical trial (NCT03758417).

13. Final Evidence Report on Hemophilia A and B Gene Therapies Published by the Institute for Clinical and Economic Review

The report covers CSL Behring's etranacogene dezaparvovec, and also includes an update on the Institute for Clinical and Economic Review (ICER)'s previous review of Biomarin's valoctocogene roxaparvovec.