Advanced 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.
After recent upheaval at the US FDA, the company’s CEO is unsure that an agreed fast-track pathway will still be in place for its Duchenne gene therapy, RGX-202.
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.
Shares have surged in companies who have fallen foul of Prasad’s stricter approach, but it is not clear whether a future successor will continue or reverse his policies.
The FDA has reiterated its request for a sham surgery-controlled, Phase III trial of uniQure’s gene therapy candidate for Huntington’s disease, an idea some call unethical.
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.
Deal Snapshot: The two companies signed a deal worth potentially $1.8bn to develop macrocyclic peptides, a new therapeutic modality gaining traction among pharma companies.
Japanese venture Kidswell aiming to progress pediatric cerebral palsy cell therapy in US through new operation.
The FDA cited various issues with the CAMPSIITE trial in Hunter syndrome and offered multiple paths forward, but the biotech said that could be a challenge given the rarity of the disease.
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.
The company said the FDA placed the hold on RGX-111 and RGX-121 after a patient in its RGX-111 trials developed a brain tumor, the cause of which remains under investigation.
The US giant could pay up to $1.12bn to the German biotech after getting access to its programmable recombinase technology.
Despite the positive update, uncertainty regarding the future of Intellia's second Phase III trial for nex-z, which is till on hold, remains.
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.
The Phase II data for intismeran autogene in melanoma were mostly incremental, but the program’s continued success is a cornerstone of Moderna’s breakeven strategy.
Patrick Soon-Shiong said that lymphodepletion may end up being proven unnecessary, especially with some of the newer cell therapy approaches.