Key takeaways:
- VG801 is a dual-AAV gene therapy in development for the treatment of Stargardt disease.
- The therapy is under investigation in a phase 1/2 clinical trial.
Editor’s note: This is a developing news story. Please check back soon for updates.
The FDA granted regenerative medicine advanced therapy designation to VG801 for the treatment of Stargardt disease, according to a press release from VeonGen Therapeutics.
VG801, a dual-AAV gene therapy, is being evaluated in a phase 1/2 clinical trial. It previously received rare pediatric disease and orphan drug designations from the FDA, according to the release.
The FDA grants regenerative medicine advanced therapy (RMAT) designation to regenerative medicine therapies, including gene therapies, that are under development for serious or life-threatening conditions. The therapy must show preliminary clinical evidence that it could potentially address unmet needs.
Read more: https://tinyurl.com/52yb52hs
Source: Healio