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, 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…
Joint Statement to the Global Batten Disease Community On Thursday, February 15, 2024, important news broke regarding CLN1 clinical research that global Batten disease patient advocacy groups wish to share with the Batten community. A patient with CLN1 disease was…
Dear Batten community, As many of you know, we have been awaiting further news regarding the future of the CLN3 and CLN6 gene therapy clinical programs led by Amicus Therapeutics. Today, we wish to share an important update relating to…
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…
Elpida Therapeutics will be hosting an open virtual meeting via Zoom on Thursday,January 18, from 3-4 p.m. EST (8-9pm UK time), to provide an update on the ongoing gene therapy trial for treating CLN7 Batten disease, which has been led…
Dear CLN3 Community, Please find below some information about the ongoing research into CLN3, the Miglustat trial hopefully coming next year, and a link to the BDSRA Australia project looking to speech and language in individuals with Batten disease .…
Dear Families, Following the news two weeks ago that Regenexbio have halted enrolment into their development programmes RGX-181 and RGX-381, the BDFA would like to invite you to a call on Wednesday 29th November at 12-1pm. This is an opportunity…
Dear Families, Thank you to those of you who have filled out the CLN2 survey. We have received some responses from both parents and teachers. However, in order to make as much of an impact as possible on the outcome of…
Dear CLN2 and CLN3 families, Lottie Morison is a speech pathologist and researcher leading a project on speech and language in individuals with CLN2/CLN3. She has had an encouraging response so far but Lottie has asked if we might be able to…