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.
MeiraGTx’s botaretigene sparoparvovec (AAV-RPGR), a gene therapy in development for the treatment of X-linked retinitis pigmentosa (XLRP) with disease-causing variants in the RPGR gene, has shown to be generally safe and well-tolerated and demonstrated improvements in several vision-related efficacy end points, according to data from the phase 1/2 clinical trial MGT009 (NCT03252847).
ADVM-022, an investigational adeno-associated virus (AAV) vector gene therapy by Adverum Biotechnologies, improved anatomic outcomes in patients with wet age-related macular degeneration (AMD) treated in the phase 1 OPTIC trial (NCT03748784). It was granted Priority Medicines (PRIME) designation by the EMA for the treatment of wet AMD on June 24, 2022.
BridgeBio Pharma’s BBP-812, an investigational gene therapy for the treatment of Canavan disease, has a promising pharmacodynamic and safety profile, according to early data from the first 2 patients treated in the phase 1/2 clinical trial CANaspire (NCT04998396).
The FDA has lifted a clinical hold on Vertex Pharmaceuticals’ phase 1/2 clinical trial (NCT04786262) of VX880, a pancreatic islet cell replacement therapy for the treatment of type 1 diabetes with impaired hypoglycemic awareness and severe hypoglycemia.
Coave Thereputics has announced the formation of a new scientific advisory board which will include international scientific and clinical experts on gene therapy and neurology. The scientific advisory board will provide guidance for Coave's pipeline of next-generation gene therapy candidates targeting Central Nervous System diseases.
The companies have signed a patent license agreement for Pfizer to use Touchlight's rapid, scalable, enzymatic doggybone DNA (dbDNA) technology in the clinical and commercial manufacture of mRNA-based vaccines, therapeutics, and gene therapies.
In the $146 million deal Mitsubishi Tanabe will use EditForce's proprietary pentatricopeptide repeat (PPR) protein platform technology to research and develop potential treatments for an undisclosed Central Nervous System disease.
The phase I clinical stage therapeutic antibody, TNB-486, is a CD19/CD3 T-cell engager and will be evaluated in multiple forms of lymphoma.
The investigators found that a small subset of CAR T-cells out of the total amount prepared for therapy produced most of the enduring anti-cancer activity in children with acute lymphoblastic leukemia.
It will be the first modular end-to-end manufacturing platform for allogeneic cell therapies. The collaboration combines Cytiva’s experience in supplying end-to-end manufacturing solutions with Bayer’s experience with complex therapeutics.
The company will now operate as Evotech (Modena) Srl.
Turnstone will have priority access to Moffitt's research, manufacturing, and clinical capabilities for this purpose.