Gene Therapy
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.
Multiple Chinese biotechs are seeking to raise new funds through IPOs on the Hong Kong and Beijing stock exchanges to support international clinical trials for their first-in-class molecules.
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.
Executives leading the commercialization of cell and gene therapies are informing how the industry approaches everything from manufacturing strategy to physician education.
After two deaths tied to the gene therapy, Sarepta and the FDA agreed to new labeling for Elevidys, adding a black box warning about liver injury along with suggested liver and cardiac monitoring.
After two deaths tied to the gene therapy, Sarepta and the FDA agreed to new labeling for Elevidys, limiting treatment to ambulatory Duchenne muscular dystrophy patients.
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.
Patient advocacy organizations are transforming gene therapy development by funding research, de-risking programs and driving ultra-rare disease treatments from concept to clinic at unprecedented speed.
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.