FDA selects Rett syndrome gene therapy for rare disease pilot drug program

The FDA has selected an investigational AAV9 gene therapy for Rett syndrome to participate in a pilot program created to provide a boost to novel drug development for rare diseases, according to a release from the manufacturer.
The Support for Clinical Trials Advancing Rare Disease Therapeutics (START) program will allow the company to engage in enhanced communications with the FDA to accelerate the pace of development for NGN-401, as well as to address product-specific development issues, including clinical study design, choice of control group and choice of patient population, Neurogene Inc.

The FDA has selected an investigational AAV9 gene therapy for Rett syndrome to participate in a pilot program created to provide a boost to novel drug development for rare diseases, according to a release from the manufacturer.
The Support for Clinical Trials Advancing Rare Disease Therapeutics (START) program will allow the company to engage in enhanced communications with the FDA to accelerate the pace of development for NGN-401, as well as to address product-specific development issues, including clinical study design, choice of control group and choice of patient population, Neurogene Inc.