Dear Batten community, Over the past few years, Lexeo Therapeutics has been involved in developing an AAV-mediated gene therapy program for treating CLN2 Batten disease. However, additional funding and resources are required for further studies, and Lexeo is now looking…
Dear Families,The BDFA Team and Trustees would like to invite you to our next quarterly Town Hall Meeting at 7pm on Wednesday 13th March.The purpose of the meeting is to provide families with an update from the team and to…
Dear Families, You may be aware that there was a press release last week from Theranexus about the Batten-1 clinical trial for CLN3 (Theranexus_PR_Cash_Position_Dec_31_2023_VDEF.pdf). Beyond Batten have followed this up in an e-mail to their registered families and the BDFA…
CLN5 Program Update Neurogene has completed enrolment of Cohorts 1 and 2 in the ongoing Phase 1/2 clinical trial for CLN5 Batten disease, and interim clinical data are expected in the second half of 2024. Neurogene is currently enrolling a…
Dear all, As we approach the end of the year I would like to express our grateful thanks to all the families in our community for their tremendous support this year. It has been a challenging year for the BDFA,…
Thank you so much for supporting me and helping me to feel welcomed (and useful!) in my role. I have enjoyed a busy 6 months in post and feel I am developing a stronger understanding of who we support and…
Dear Families,As per our communication on social media yesterday the BDFA would like to invite you to attend a short call on Thursday 21st December at 12pm.The purpose of the call is to update you on our PR and…
Bertie’s Helpers provides a week’s food shop delivered to the door for families with children with Batten disease who are going through a time of crisis. Mimi and Andrew Petty set up Bertie’s Helpers in memory of their son Bertie…
Recently we shared some updates on REGENXBIO's investigational gene therapy programs for the treatment of CLN2 Batten disease. We are pleased to share this letter with the CLN2 Batten disease community, where REGENXBIO reports some initial interim data from a…
We are very pleased to share the following developments on REGENEXBIO’s first-in-human gene therapy program for the treatment of CLN2 Batten disease. REGENEXBIO announced that the first patient was dosed in the Phase 1/11 ocular trial of RGX-381 at Great…