Cell Therapies
Cell and gene therapy is entering a new phase, shifting beyond oncology and rare diseases toward broader autoimmune and neurological indications, but progress will hinge on speed across regulation, manufacturing, financing and clinical development, the Advanced Therapies UK conference heard.
With the FDA accepting its refiled BLA and setting an Aug. 22 PDUFA date, Capricor said its cell therapy could offer skeletal and cardiac benefits to Duchenne muscular dystrophy patients.
Gilead’s takeover of Arcellx tightens its grip on anito-cel as it readies for a US FDA ruling and a high stakes entry into the crowded myeloma market.
Pioneering immunologist, Prof. Bruce Levine from UPenn, wears his passion for CAR-T cell therapy on his sleeve as he highlights future uses of the therapy, predictive markers and off-the-shelf CAR-T while speaking about a journey from Kymriah co-inventor to country music co-writer
Japan has granted the first positive approval recommendations worldwide for two allogenic cell therapies derived from induced pluripotent stem cells, Sumitomo’s raguneprocel for Parkinson’s and Cuorips’ cardiomyocyte patches for heart failure.
Japanese venture Kidswell aiming to progress pediatric cerebral palsy cell therapy in US through new operation.
Orna’s lead candidate, ORN252, which targets CD19 positive autoimmune diseases, is expected to reach the clinic in 2026.
Enthusiastic predictions from industry leaders for ongoing progress in obesity, oncology, immunology, neuroscience and more were supported by optimism for the potential of AI and other technological advances to enable breakthroughs in processes as well as pipelines.
The company said the NEAT trial in ataxia telangiectasia did not meet the primary and key secondary endpoints.
While several companies await regulatory approval decisions on their cell therapies in Japan, BioCardia proceeds with discussions on an NDA filing and Innovacel readies an IPO.
Patrick Soon-Shiong said that lymphodepletion may end up being proven unnecessary, especially with some of the newer cell therapy approaches.
Immunis thinks its secretome-based biologics could provide off-the-shelf therapies for metabolic conditions related to aging. Monotherapy or combination with GLP-1 agents is being considered.
The firm founded by Treg cell discoverer Shimon Sakaguchi is building up its US team and refining R&D strategy and manufacturing processes to create an example and global path for other Japanese ventures.
With registrational Phase II data in Stiff Person Syndrome, Kyverna intends to file a BLA for mivocabtagene autoleucel in 2026. Longer term, it hopes to get the CAR-T approved for myasthenia gravis.
Next-gen CAR-T therapies showcased at the American Society of Hematology's annual meeting promise improved efficacy and safety, with Novartis, Gilead, and Kelonia advancing pivotal trials and exploring in vivo approaches.
The company presented data on four patients as a late-breaking abstract at the American Society of Hematology meeting.
The company presented positive efficacy and safety data from its registrational Phase II iMMagine-1 trial at the 2025 ASH meeting.
With Gilead reporting that both of its approved CAR-Ts saw sales declines as other products saw growth, a mixture of different competitive headwinds seems to be behind the disparities.
The biotech will present data at the upcoming ASH meeting on three patients who achieved PRs or VGPRs with minimal residual disease negativity and no cases of neurotoxicity.
Data presented at the Berlin meeting highlight the potential of the company's cell therapy anzu-cel to redefine the treatment paradigm for uveal melanoma.