Gene 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.
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.
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.
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 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.
Following Ribo Life Science’s recent Hong Kong IPO, an investor shares views with Scrip on the prospects for China's siRNA developers and likely deal trends in 2026.
The company announced positive preliminary results from its Phase II study of the gene therapy in geographic atrophy secondary to dry age-related macular degeneration.
The base-editing company’s risto-cel may have an efficacy edge over Vertex’s Casgevy, but both will be held back by safety concerns about chemotherapy preconditioning for patients.
Tessera, which claims its gene-writing technology offers functionality beyond gene-editing, hopes Regeneron can accelerate development of its preclinical alpha-1 antitrypsin deficiency drug.
The CEOs of two early-stage biotechs explain to Scrip why investor interest in ocular gene therapies is surging and how they aim to attract big pharma partners.
US FDA approval of the intrathecal formulation of Zolgensma means many more patients with spinal muscular atrophy will be eligible for the gene therapy.
The company announced data from patients enrolled after a protocol amendment designed to bring the study in line with non-muscle invasive bladder cancer (NMIBC) standards of care.
Deal Snapshot: Eli Lilly has become increasingly interested in genetic treatments for the eye and has secured the rights to a high-profile gene therapy for a rare form of childhood blindness.
The company’s third quarter earnings fell by $67.8m year-over-year, reflecting the tumultuous situation around its DMD therapy Elevidys in June.
UniQure’s plan for a 2026 BLA filing of gene therapy AMT-130 for Huntington’s is uncertain, as the FDA apparently reversed course after indicating it would consider accelerated approval.
The company said in its third quarter earnings that it would focus on areas that better foster growth for the company as the hemophilia A treatment only brought in $3m in Q3.