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.
Senti Bio will be prioritizing the development of its logic gated cell therapy candidates which are based on the company’s Gene Circuit technology, in particular SENTI-202 and SENTI-401, according to a corporate update.
The phase 3 CARTITUDE-4 clinical trial (NCT04181827), which is evaluating Janssen’s and Legend Biotech's ciltacabtagene autoleucel (cilta-cel; CARVYKTI) against standards of care in the treatment of relapsed/refractory multiple myeloma, will be unblinded according to an independent data monitoring committee recommendation following the study meeting its primary end point in a pre-specified interim analysis.
Mesoblast has resubmitted a biologics license application (BLA) for remestemcel-L, its mesenchymal stromal cell therapy for treating steroid-refractory acute graft versus host disease (SR-aGVHD) in children, almost 4 years after it first initiated a rolling BLA for the therapy.
The FDA has recommended Taysha Gene Therapies to dose additional patients with TSHA-120, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat giant axonal neuropathy, in order to support a BLA submission.
Sensorion's investigational gene therapy for otoferlin-related hearing loss, OTOF-GT, could potentially be among the therapies able to be delivered with such an injection system.
Spark Therapeutics expects that Neurochase's technology will improve targeting of its AAV gene therapy candidates for delivery to neural structures via Convection Enhanced Delivery.
CART-ddBCMA is currently under evaluation in a pivotal phase 2 clinical trial.
The preclinical platform technology is intended to enable the transplant of insulin producing stem cells without the need for life-long immunosuppression.
APB-102, which is intended to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), recently received clearance of its IND application by the FDA.
The research will attempt to use optimer binders to target specific cell types for siRNA -based therapeutics.
The collaboration will seek to identify improved methods of delivering complex genetic material in the production of cell therapies.
Viking Global Investors and Casdin Capital led the new CDMO's first round of funding.
CellProthera's ProtheraCytes is currently being evaluated in a phase 1/2b clinical trial with the use of BioCardia's Helix transendocardial biotherapeutic delivery system.
ISP-001, which has previously received orphan drug and rare pediatric disease designations from the FDA, will be the first ever engineered B-cell therapy to be evaluated in a clinical trial.