Sarepta Update on 9001 Gene Therapy Study

Sarepta has shared a brief update regarding the first three cohorts of study 9001-103, and future 9001 gene therapy studies.

For any additional questions, please reach out to

Dear US Patient Advocacy Leaders,

We have received a lot of inquiries about the enrollment status of the first three cohorts of study 9001-103, an open label micro-dystrophin gene therapy study.  At this time, the clinical trial sites involved in cohorts 1-3 are no longer accepting new patients for screening. 

Many families have reached out and expressed their interest in participating in a 9001 gene therapy study.  We recognize the unmet medical need that is driving this interest, and also recognize the urgency stated by so many families.

It is important to note that planned future trials for 9001 will be larger in size and at a greater number of locations globally.  We will be sharing details of future studies as soon as we can with the community.

Thank you,

Sarepta Patient Affairs

Sarepta Therapeutics

215 First Street, Cambridge, MA 02142