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Novartis OAV201 Update

Dear families and supporters,

We want to share with you the update below from Novartis Gene Therapies regarding their gene replacement program for Rett Syndrome, OAV201. Novartis reports that they continue working closely with the FDA to progress to an Investigational New Drug (IND) application, which they plan to submit by the end of this year. Pending an accepted IND, the gene therapy clinical trial could start late this year or early next year.

Like you, we’re parents of children with Rett Syndrome at RSRT and we share your profound sense of urgency to heal all our loved ones as soon as possible. While we are disappointed that the clinical trial can’t start sooner we are encouraged by Novartis’ commitment to the Rett program. We also recognize the critical importance and the time that it takes to get things exactly right for the IND and for the trial.

RSRT has aggressively championed and financially supported gene replacement strategies since 2010. We thank every Rett family and their network of relatives and friends who have been behind us so strongly. It is because of you that programs such as the Novartis one are moving forward. We won’t rest until we have a cure for Rett Syndrome.

Monica Coenraads

Dear Rett Syndrome Community,

We at Novartis Gene Therapies hope that you and your families have had a good start to 2021, and we would like to begin the year together by expressing that we deeply appreciate your collaboration and partnership and providing you with an update on our OAV201 program for Rett syndrome.

Our advocacy partners are strong allies who have deepened our understanding of this disease as we advance promising science with transformative potential for Rett. We have been fortunate to partner with and Rett Syndrome Research Trust on awareness initiatives like October Rett syndrome Awareness Month, presentations to our colleagues by Rett advocacy leaders, registry, biomarker and outcome development, and support of educational programming.

As we indicated in our last update, we have been working closely with the FDA to progress OAV201 towards an Investigational New Drug (IND) application. Today, we are pleased to let you know that we expect to submit an IND by the end of the year. As a reminder, an accepted IND is required before we can proceed to clinical trials in patients with Rett syndrome.

We recognize the significant unmet need among patients with Rett syndrome and want to again assure the Rett community that we are fully committed to pursuing a gene therapy for Rett syndrome, and that we are continuing our work with a sense of urgency and purpose.

We look forward to continued partnership with the Rett community and commit to providing you with updates on a regular basis as we have more information available to share.


The Novartis Gene Therapies OAV201 Development Team