REGENXBIO halts CLN2 gene therapies

The Batten Disease community are heartbroken to learn that the biotech company REGENXBIO have decided to stop further enrollment and development of their AAV gene therapy programs for the treatment of CLN2. To date, very few children have received this therapy.

Only in August 2023, the company released a 6-month follow-up report on a 5-year-old child, showing stable neurodevelopmental measures, meaningful improvements in fine motor and expressive language skills, and an 86% reduction in seizures, with concomitant withdrawal of anti-epileptic medication.

https://www.regenxbio.com/getmedia/334aee0e-2b36-44b2-be6b-08863396787d/Poster-Fischinger-RGX181-SSIEM-08-10-23-(1)-(1).pdf?ext=.pdf

This is a devastating blow to the BD community, where there are no curative therapies.

A joint statement has been released by the Batten Disease Support and Research Association (BDSRA), to the worldwide batten community.

https://bdsrafoundation.org/joint-statement-to-our-worldwide-batten-community/