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Updated Global Announcement Graphic


We believe it’s important to work together with our global patient advocacy partners to share important research updates in a unified, timely, and factual manner. Our network of organizations has worked together on this statement to share with our families worldwide.

Dear families,

As some of you have already heard, last week Neurogene Inc. made the difficult decision to discontinue its CLN7 Batten disease program, including the CLN7 portion of the natural history study. We are disappointed by this development and understand this news will be deeply upsetting for many. The CLN7 families affected by this development have been contacted directly by the study’s Principal Investigator, and Neurogene has also provided an open letter to the CLN7 Batten Disease Community, which can be viewed here.

We would like to acknowledge and thank the many families, patients, and researchers who contributed to the development of this program, and hope that Neurogene will continue to seek potential industry and scientific partners to continue this work into the future. Importantly, we note that Neurogene remains committed to its CLN5 gene therapy program, which continues to enroll patients for the ongoing Phase 1/2 clinical trial.

On multiple occasions over the past year, the rare disease community has received similar disappointing news of clinical programs being discontinued amid an extremely challenging, sector-wide economic downturn. We discussed some of these issues in a joint webinar last May.

As global patient advocacy group partners, we do however remain optimistic about the future of Batten disease clinical research, including for CLN7 disease where UT Southwestern’s gene therapy clinical trial is ongoing. There remains a great deal of progress in the preclinical and clinical space across numerous NCL subtypes, and we will continue to keep our community informed, as updates come to light.

We know our global Batten community is strong, united, and resilient, and we will continue to collaborate internationally to support and advocate for everyone affected by Batten disease. We will continue to cultivate relationships with our industry partners to maximize opportunities for our patients and families, and with our donors to provide funding for research. Please feel free to contact any of our organizations for any questions related to Batten disease.


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