Research & Clinical News
CLN2 Batten Disease Update from Tern Therapeutics
More positive news for our CLN2 community this week, with Tern Therapeutics announcing that its investigational gene therapy, TTX-381, for the treatment of ocular manifestations of CLN2 disease, has been selected by the FDA [...]
CLN2 Batten Disease Update from Latus Bio
Latus Bio announced today clearance by the U.S. Food and Drug Administration (FDA) of the Company’s Investigational New Drug (IND) application for LTS-101, a gene therapy candidate that’s intended to treat the CNS manifestations [...]
Kennedy Krieger Institute Awarded $8.17 Million Grant To Lead National Research Consortium; Will Work Closely With The BDSRA Foundation
PRESS RELEASE: Read the following press release below or on Kennedy Krieger Institute's website here. Kennedy Krieger Institute wrote this press release in collaboration with the BDSRA [...]
NOVEMBER ASK-AN-EXPERT EVENT POSTPONED; WILL BE RESCHEDULED
The next Ask-An-Expert session, which was scheduled for Tuesday, November 18, has been postponed and will be rescheduled. Please check our social media channels, and keep an eye on your email for the rescheduled [...]
PUBLICATION ALERT: Batten Disease Research Goals Published in The Lancet Neurology
In 2024, recognizing the urgent need to establish research priorities across the basic, translational, and clinical sciences to tackle the most pressing concerns of the Batten disease community, the Batten Disease Clinical Research Consortium [...]
BDSRA Foundation Announces Mount Sinai Health System as New Site in Clinical Centers of Excellence Program
GAHANNA, OH (September 22, 2025) – The BDSRA Foundation has officially announced Mount Sinai Health System as the newest site in the Batten Disease Clinical Center of Excellence Program. “We are delighted to welcome [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | Summer 2025
It's time for Batten disease research updates! BDSRA Foundation's Head of Research & Medical Affairs, Dr. Ineka Whiteman, breaks down Batten disease clinical program updates, research news, and resources in her monthly column, as [...]
Batten Disease Advocates Applaud B.C. Government’s Reversal on Brineura Funding
July 18, 2025—Batten Disease Support, Research, and Advocacy (BDSRA) Canada, the BDSRA Foundation, and its Batten Disease Clinical Centers of Excellence (CoEs) applaud the British Columbia Government’s decision to reinstate funding for Charleigh Pollock’s life-sustaining [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | April 2025
It's time for Batten disease research updates! BDSRA Foundation's Head of Research & Medical Affairs, Dr. Ineka Whiteman, breaks down Batten disease clinical program updates, research news, and resources in her monthly column, as [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | February & March 2025
It's time for Batten disease research updates! BDSRA Foundation's Head of Research & Medical Affairs Dr. Ineka Whiteman breaks down Batten disease clinical program updates, research news & opportunities, and resources in her monthly [...]
BREAKING: Batten Disease Global Research Initiative Announces Grant Recipients
We are proud to announce the recipients of the Batten Disease Global Research Initiative inaugural Research Grant round. Learn more about the recipients and their research by clicking the button below. Read the Full [...]
CLINICAL UPDATE: Tern Therapeutics Gives CLN2 Batten Disease Update
The following letter from Tern Therapeutics shares the latest on TTX-381 and TTX-181, gene therapies being developed for the treatment of CLN2 Batten disease. Read the PDF of this letter here. February 27, 2025 Dear [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | January 2025
It's time for the first Batten disease research updates of 2025! BDSRA Foundation's Head of Research & Medical Affairs Dr. Ineka Whiteman breaks down Batten disease clinical program updates, research news & opportunities, and [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | November 2024
It's time for research updates! BDSRA Foundation's Head of Research & Medical Affairs Dr. Ineka Whiteman breaks down Batten disease clinical program updates, research news & opportunities, and resources in her monthly column, as [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | October 2024 Illuminator Edition
It's time for research updates! BDSRA Foundation's Head of Research & Medical Affairs Dr. Ineka Whiteman breaks down Batten disease clinical program updates, research news & opportunities, and resources in her monthly column, as [...]
BDSRA Applauds Latest News From ForeBatten Foundation on Zebronkysen
We applaud the news from our friends at ForeBatten Foundation that the FDA has provided IND clearance for the clinical investigation for Zebronkysen (FBF-001) under the N-of-1 guidance for individualized Antisense Oligonucleotide (ASO) drug products. [...]
Batten Disease Research Updates with Dr. Ineka Whiteman | August 2024 Illuminator Edition
It's time for research updates! BDSRA Foundation's Head of Research & Medical Affairs Dr. Ineka Whiteman breaks down Batten disease clinical program updates, research news & opportunities, and resources in her monthly column, as [...]
Tern Therapeutics Enters into a Global Licensing Agreement with REGENXBIO Inc. for RGX-381 and RGX-181
Tern Therapeutics has announced its launch and entered into a global licensing agreement with REGENXBIO for RGX-381 and RGX-181 (now designated TTX-381 and 181, respectively) to form its initial therapeutic pipeline. TTX-381 and TTX-181 [...]
