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.
Vertex Pharmaceuticals and CRISPR Therapeutics today announced that they had completed the rolling submission of their biologics license applications (BLAs) for the investigational therapy exagamglogene autotemcel (exa-cel; previously known as CTX001) for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
bluebird bio’s biologics license application (BLA) submission for lovotibeglogene autotemcel (lovo-cel) to treat sickle cell disease (SCD) will likely be delayed as the company awaits FDA feedback on newly submitted manufacturing compatibility data.
Caribou Biosciences has announced that the first patient has been dosed in its phase 1 CaMMouflage clinical trial (NCT05722418) evaluating CB-011 for the treatment of relapsed/refractory (r/r) multiple myeloma (MM) and that it has initiated the dose expansion portion of its phase ANTLER clinical trial (NCT04637763) evaluating CB-010 for the treatment of r/r B-cell non-Hodgkin lymphoma (B-NHL).
The first patient has been dosed in the phase 1/1b dose-escalation and -expansion study (NCT05694364) of PRGN-3007, Precigen’s investigational and first-in-class multigenic, autologous CAR-T cell therapy, according to a company announcement.
The partnership is focused on the use of wearable technology and invisible contactless digital health technology for disease monitoring. BlueRock may use the technology for monitoring in future clinical trials of its Parkinson disease cell therapy BRT-DA01.
Polyplus specializes in the manufacture of DNA and RNA delivery reagents and plasmid DNA.
The company cited the results of preclinical efficacy research as the key factor in its decision.
The targets include CD19 and B-cell maturation antigen. The new agreement also gives Precigen exclusive rights to its IL-12 gene therapy.
The agreement specifically regards AAV gene therapies for certain cardiac conditions targeted at genes identified by Forcefield Therapeutics.
The patents include a US patent for a “Multi-Directional Steerable Catheter” and an India patent for "Steerable Endoluminal Devices and Methods for Use”.
AGC obtained the investigational product in a technology transfer agreement from the Jikei University in Japan.
The company stated that it is now focusing its resources on Gaucher disease gene therapy FLT201.