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 playersemerging 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.
Ocugen has added the phase 3 cell therapy platform NeoCart to its pipeline following its Regenerative Medicine Advanced Therapy (RMAT) recognition for the repair of full-thickness lesions of the knee cartilage in adults.
uniQure's biologics license application (BLA) for etranacogene dezaparvovec (EtranaDez) has been accepted by the FDA for priority review for the treatment of hemophilia B. A final analysis of the pivotal HOPE-B study was presented at ASGCT 2022.
The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) has granted a positive opinion to PTC Therapeutics’ gene therapy eladocagene exuparvovec (PTC-AADC; Upstaza) for treating aromatic L-amino acid decarboxylase (AADC) deficiency based on findings from studies conducted in Taiwan.
University of Pennsylvania Medicine and Children’s Hospital of Philadelphia are teaming up with Costa Rica’s Caja Costarricense de Seguro Social to facilitate research with chimeric antigen receptor (CAR) T-cell therapies in the country.
Bayer has decided to end their exclusive worldwide licensing agreement with Atara Biotherapeutics for next-generation mesothelin-directed CAR T-cell therapies, which includes cell therapy programs indicated for malignant pleural mesothelioma and non-small-cell lung cancer.
ASC Therapeutics has joined Joins the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium US Consortium of Gene Therapy Partners, which includes the NIH and FDA. The consortium aims to accelerate development of therapies for rare diseases by tackling challenges in research, production, and regulations.
Kelonia Therapeutics, a new start-up, has designed off-the-shelf CAR T-cell therapies that are safer than traditional CAR T therapies but still personalized to patients, created with detargeted and redirected lentivirus-like particles.
The company has raised $112.8 million to advance the development of their phase 3 gene therapy program for recessive dystrophic epidermolysis bullosa (RDEB) as well as to submit an investigational new drug application for their in vivo gene therapy program for hereditary tyrosinemia type 1.
Myrtelle has entered into a Worldwide Exclusive License Agreement with Rescue Hearing to develop a novel gene therapy for DFNB8 genetic hearing loss.
The company has acquired rights to Abeona Therapeutics' adenovirus-associated vector (AAV) gene therapy ABO-102 for the potential treatment of Sanfilippo Syndrome Type A, which is currently being evaluated in the phase 1/2 Transpher A trial (NCT02716246).
Its new platform, titled UpTempo Virtuoso, which includes optimized standard protocols for cell culture, transfection, and downstream purification, streamlines AAV manufacturing with the goal of reducing the timeline from development to clinic and to enable rapid first-in-human clinical evaluation.
The lawsuit by Roche, on behalf of their subsidiary Spark Therapeutics, was filed in response to bluebird’s “Be the Spark” campaign. The lawsuit has been dismissed and the campaign remains live.