As part of its third-quarter earnings release on Wednesday, November 8, 2023, REGENXBIO announced a corporate restructuring that includes halting enrolment of patients into their development programmes RGX-181 and RGX-381 for CLN2 Batten disease (https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-updated-strategic-plans-and-third-quarter).

The BDFA has been an active partner to REGENXBIO from the inception of the clinical study in the UK and this news comes as a shock to us. It is our understanding that the company’s decision was not made in relation to the safety or efficacy of their gene therapy candidates for CLN2.

We recognise many challenges to developing medicines for rare diseases and in this regard, the BDFA expects commercial organisations entering this space to do so after a full consideration of their capabilities, and patient and family needs which they intend to serve.

The BDFA had an urgent meeting with REGENXBIO this afternoon to express our huge disappointment at their decision and to understand what practical steps they are taking and what progress has been made to restarting the program. Our conversation with REGENXBIO representatives Vivian Fernandez, Patient Advocate, and Paulo Falabella, Vice President, Clinical Development & Operations was productive, and we will continue our communication with the company and global partner charities to try to identify the best way forward. In the meantime, we received reassurance from the company that the patients who have been dosed in clinical trials with RGX-381 and RGX-181 will continue to be followed.

The BDFA will be holding a telephone conversation with the UK CLN2 families to discuss this development and impact on the community and the confirmation about the date and time will follow. In the meantime, if you have any questions or concerns, please do not hesitate to reach out to Liz Brownnutt (BDFA interim CEO, lizbrownnutt@bdfa-uk.org.uk).