Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
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.
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.
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.
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.
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.
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.
The decision was informed by results from the ZUMA-7 (NCT03391466) study.
The company now has the rights for research, development, and commercialization of the 2 investigational therapies.
Prevail will utilize Capsida's platform for adeno-associated virus capsid identification for its therapies.
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.
HBI0101 is currently being evaluated in a phase 1b clinical trial for patients with multiple myeloma and AL amyloidosis.
The submission is supported by results from CARTIFAN-1, a phase 2 clinical trial (NCT03758417).
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.