Gene Therapy

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.

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.

The privately owned biotech said it would pause its Phase I/II study of CAP-002 in STXBP1-related disorders after the first patient in the study died.