Gene therapy
The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access to patients two years and older in what one Stanford Medicine professor called a “game changing advance” for the field.
Experts suggest the FDA’s Advanced Manufacturing Technologies designation could be a lifeline for improving production processes for approved cell and gene therapies.
Mixed headlines have plagued the cell and gene therapy space of late. We believe that a renewed case of optimism is not only warranted but essential if these therapies are to reach their full potential.
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.
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