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.
Pluristem’s phase 3 trial (NCT03451916) of allogeneic PLX-PAD cells for the treatment of muscle injury following arthroplasty for hip fracture has failed to meet its primary endpoint of improvements in Short Physical Performance Battery test at week 26 compared to placebo, although other improvements were seen.
Ultragenyx has acquired GeneTx after promising interim data, from a phase 1/2 trial (NCT04259281) on GTX-102, the companies’ joint gene therapy program for the treatment of Angelman syndrome, were released.
The phase 3 OVAL trial (NCT03398655) of ofranergene obadenovec (VB-111; VBL Therapeutics) in combination with paclitaxel has failed to meet its primary endpoint in improving progression-free survival or overall survival in patients with platinum-resistant ovarian cancer.
After submitting an amended IND, Adverum’s gene therapy is set to be evaluated in the phase 2 LUNA trial.
The FDA has granted orphan drug designation to AVROBIO’s gene therapy AVR-RD-05 for the potential treatment of mucopolysaccharidosis type II (MPSII) also known as Hunter syndrome.
Neurona Therapeutics’ phase 1/2 clinical trial (NCT05135091) for NRTX-1001, a regenerative neural cell therapy derived from human pluripotent stem cells, has dosed its first patient with Mesial Temporal Lobe Epilepsy (MTLE).
Bone Therapeutics has made adjustments to optimize their phase 2b trial (NCT04432389) of their allogeneic osteoblast cell therapy, ALLOB, for high-risk tibial fractures. The adjustments come after the company announced a reprioritization to focus on ALLOB in September 2021.
SwanBio will use the platform to identify new targets for gene therapies for neurological diseases.
The two companies will cooperate in discovery, manufacturing, clinical trials, and related research/service projects.
Avista's scAAVengr platform will be used to develop AAV capsids, which will be evaluated and potentially licensed by Roche for use in gene therapy programs.
A collaboration has been announced between Antegene and Celularity which will focus on researching potential combination therapies for the treatment of hematological and solid tumor cancers. Notably, this collaboration will be Antegene's entrance into the field of cell therapy development.
The phase 2 (NCT03374657) sub-retinally delivered therapy candidate is in development for the treament of patients with retinitis pigmentosa due to mutations in the RLBP1 gene, and functions by delivering a working copy of the RLBP gene.
The New Jersey facility was launched by McKinsey & Company in partnership with New Jersey Innovation Institute and will give companies the opportunity to see technologies in practice and engage in hands-on educational activities.
The New York City facility will enable the company to manufacture its cell therapies in-house.