News

Updates Delayed on Regenxbio’s CLN2 Gene Therapy Programs

Regenxbio plans to provide updates on its RGX-181 and RGX-381 gene therapy candidates for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease, in the second half of this year. The company is currently reviewing information requests from…

Maypole Project Opens Counseling and Activities for Batten Families in UK

Families of a child with Batten disease in the U.K. are welcome take part in counseling and other wellness services being offered at no cost by the Maypole Project in a three-year collaboration with the Batten Disease Family Association (BDFA). Services include one-on-one therapy sessions, as well as sessions tailored to couples,…

Neurogene Workshop Seeks Families Affected by CLN5

Editor’s note: This story was updated March 30, 2021 to clarify that Neurogene is hosting videoconferences workshops for families in any country. Neurogene, which is developing treatments for Batten disease and other rare neurological disorders, is inviting families affected by CLN5, a form of late infantile…

Actelion to Help Develop BBDF-101 for Juvenile Batten Disease

Beyond Batten Disease Foundation (BBDF) has struck a deal with Actelion Pharmaceuticals to help further the development of BBDF-101, an investigational treatment for juvenile Batten disease. Under the terms of the new agreement, Actelion (a Janssen Pharmaceutical Company of Johnson & Johnson) will provide a component of…