The group’s antisense project is the only game in town for Usher syndrome, but its latest trial is not a slam-dunk.
As several big pharma companies drop Parkinson’s projects, attention turns to gene therapies and repurposing diabetes drugs.
Followers of Nordic Nanovector and Oxford Biomedica, among others, will pay close attention to an early focus of yesterday’s deal.
Industry-sponsored clinical studies against the new coronavirus have proliferated, as have their designs and efficacy measures.
Data will soon show whether Avadel’s FT218 is more convenient than Jazz’s Xyrem, while Axovant will be looking for a dose response with its Parkinson’s gene therapy.
Despite the recent flurry of gene therapy deals there is still a fairly extensive list of affordable companies out there.
Axovant is the latest company to benefit from excitement over gene therapy data in small numbers of patients.
The French company has taken a look at its R&D portfolio, and made some radical changes in oncology and diabetes.