Cell and Gene Therapy

After revoking Sarepta’s award in July and awarding one to Krystal last month, the FDA’s platform technology designation program appears to be back on track. These six biotechs could be on the regulator’s radar.

The CRO market in the APAC region is thriving, particularly in China, due to intense clinical trial and innovation development, with Western investors and pharma leaning in.

Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed with the gene therapy.

Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation.

The plausible mechanism pathway “could accelerate gene therapy/editing development,” analysts at William Blair said Thursday, while adding that additional clarity is needed.

In 2025, landmark obesity drug deals, China’s biotech surge, and AI’s deeper integration into pharma operations drove a year of transformation and renewed momentum for life sciences.

MeiraGTx Holdings is licensing a genetic eye disease medicine to Eli Lilly in a deal worth up to $475 million.

The FDA previously placed two clinical studies on hold, including the Phase III trial in which the liver toxicity occurred. Intellia is working with experts to create a risk management program for nex-z.

Pfizer and Novo Nordisk continue to fight for ownership of obesity startup Metsera; CDER Director George Tidmarsh leaves his position amid an ongoing probe into his “personal conduct”; FDA reverses course on approval requirements for uniQure’s Huntington’s gene therapy; Sarepta’s exon-skipping Duchenne muscular dystrophy drugs fail confirmatory study.

The potential approval of Vertex’s IgAN therapy povetacicept in 2026 comes amid launch headwinds for the company’s non-opioid pain medicine Journavx and gene therapy Casgevy.