Beyond Batten Disease Foundation (BBDF) is leading the unbiased identification and development of a diversified research portfolio with the highest potential to treat juvenile Batten disease. By investing in milestone-driven academic and industry research, advised and continually evaluated by leaders in every sector and phase of the therapeutic pipeline, we are accelerating the bench to bedside timeline toward a cure for juvenile Batten disease.
Creating effective treatments for juvenile Batten disease is our driving force. To achieve this goal, we invest in: 1) rigorous research to uncover the normal function(s) of the CLN3 gene and its protein of the same name, 2) identification of (drug) targets or cellular pathways whose repair would restore the health of brain cells affected by Batten and 3) examination and development of compounds to prevent or repair neurodegeneration.
Recent evidence supports the conclusion that altered Batten disease gene function may be implicated in Parkinson’s disease, Frontotemporal lobar degeneration, Gaucher, progressive myoclonic epilepsy and amyolateral sclerosis (ALS), that neurological diseases may share common drug targets, and that amelioration of secondary effects of neurological disease such as chronic inflammation, may inhibit disease progression. 10
Potential Industry partners are encouraged to contact BBDF’s Principal Scientist to discuss potential projects that help achieve our common goal to effectively treat neurodegenerative disease.
Consider approaching us when you have or are seeking:
- information on the state of the science and ongoing projects – “Who’s who in JNCL Research”
- research tools such as preclinical models, antibodies, cell-lines, and drug discovery assays
- pilot funding to reposition existing or to test emerging medicines in JNCL cell and animal models
- proposal ideas that add to our collective knowledge of CLN3, its protein and the pathophysiology of juvenile Batten disease
- partnerships structured around any phase of the therapeutic pipeline, top drug targets, potential compounds in neurodegenerative disease
To see a list of our research partners, click here.