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Novartis Terminates Their Rett Syndrome Gene Replacement Program

October 26, 2021

During Novartis’s quarterly investor call this morning the company informed the public that it is terminating the Rett syndrome gene replacement program, OAV201.

Novartis has shared this information with us:

"The journey to bring new therapies from the lab to patients is complex and our team works with a sense of urgency, a need for flexibility and innovation, and an ability to accept – and successfully navigate – challenges in a developing field of science.

"In this context, we have made the decision to retire development of OAV201 for Rett syndrome. Upon consideration of the program for IND submission, the totality of the pre-clinical data does not support a path forward for this development candidate to human clinical trials. This decision was not a result of safety concerns identified in the pre-clinical program. To enhance the knowledge in the field of AAV gene therapy, we plan to share the relevant data by the end of the year. We recognize this update will be especially disappointing to the Rett syndrome patient community and we remain committed to continuing to explore therapeutic options.”

IND submission refers to the FDA’s Investigational New Drug application, which is required before any new drug can proceed to clinical trial. AAV stands for adeno-associated virus, which is the delivery mechanism used to get genes into cells.

Novartis will be providing a more comprehensive community update statement at a later time.

Everyone at RSRT is disappointed that there is one less option for our community. We prepare, however, for these types of situations by driving forward multiple curative approaches in parallel.

The Taysha Gene Therapies Rett program remains on track, and there are also gene replacement programs moving forward in companies that have not yet publicly disclosed their Rett programs.

RSRT is as dedicated as ever, as passionate as ever, and as optimistic as ever about advancing us to our goal — a cure.