Gene Therapies
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.
The company reported that a patient went to the emergency room after experiencing grade 4 transaminase elevations, which unlike a similar event in May caused symptoms.
The drugmaker has made numerous moves in the field this year, with the latest deal bringing in ixo-vec, a Phase III candidate for wet AMD.
While some big pharma companies have exited cell and gene therapy, Novartis, Astellas, Gilead’s Kite and AstraZeneca’s Alexion are diving back in, executives said at ARM’s Cell and Gene Meeting on the Mesa.
While there may be the beginnings of a turnaround in biopharma capital markets, gains in the cell and gene therapy space may lag the broader sector recovery, noted speakers at ARM’s Cell and Gene Meeting on the Mesa.
It remains to be seen whether the Trump administration’s most favored nation drug pricing policy will trickle down from big pharma to small biotech, but executives said at ARM’s Cell and Gene Meeting on the Mesa that they are preparing strategies now to manage the future.
Speakers at ARM’s Cell and Gene Meeting on the Mesa highlighted challenges of CAR-T’s evolution from niche to mainstream therapy given health care providers’ limited capacity to deliver the complex care associated with the products.