This page tracks new and upcoming treatment options for Batten disease. What’s in development? What’s promising? What’s available soon? Current topics include BBDF-101, gene therapy, AT-GTX-502, AT-GTX-501, AAV2CUhCLN2, BMN 190, Brineura (cerliponase alfa), AB-202, AB-201, and more. , Projects
Last updated: 4 years ago
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News
- Abeona Therapeutics and Taysha Gene Therapies Enter into Licensing and Inventory Purchase Agreements for ABO-202, a Clinical Stage, Novel, One-time Gene Therapy for CLN1 Disease Aug 17, 2020
- Neurogene Announces FDA Orphan Drug Designation for CLN7 Batten Disease Gene Therapy Aug 11, 2020
- Gene therapy to the inner retina prevents blindness in mouse model of CLN3 Batten disease Aug 10, 2020
- Exon skipping combats Batten disease Aug 4, 2020
- Researchers develop new therapeutic approach to treat CLN3 Batten disease Jul 31, 2020
- Rosalind Franklin University Researcher awarded NIH grant for CLN3 Batten disease study Jul 31, 2020
- Role of CLN6 in Batten Discovered, Shedding Light on Disease Jul 24, 2020
- Research reveals key role of a protein in rare Batten disease Feb 28, 2020
- Theranexus and Beyond Batten Disease Foundation (BBDF) announce signing of exclusive license for Batten drug candidate BBDF-101 [PDF] Dec 12, 2019
- Gene therapy successes point to better therapies Nov 26, 2019
Research
- Successful treatment of cannabinoid administration against refractory epilepsy in Batten disease: a case report Sep 30, 2020
- AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease Sep 23, 2020
- Expanding the Neuroimaging Phenotype of Neuronal Ceroid Lipofuscinoses Aug 27, 2020
- Autosomal dominant neuronal ceroid lipofuscinosis – clinical features and molecular basis Aug 12, 2020
- Comparative analysis of the gut microbiota composition in the Cln1(R151X) and Cln2(R207X) mouse models of Batten disease and in three wild-type mouse strains Aug 4, 2020
- Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease Jul 27, 2020
- Gene therapy targeting the inner retina rescues the retinal phenotype in a mouse model of CLN3 Batten disease Jul 15, 2020
- Modulation of Kv4.2/KChIP3 interaction by the ceroid lipofuscinosis neuronal 3 protein CLN3 Jul 7, 2020
- A tailored Cln3(Q352X) mouse model for testing therapeutic interventions in CLN3 Batten disease Jun 29, 2020
- A CLN6-CLN8 complex recruits lysosomal enzymes at the endoplasmic reticulum (ER) for Golgi transfer Jun 29, 2020
- Extracellular Vesicles as Drug Carriers for Enzyme Replacement Therapy to Treat CLN2 Batten Disease: Optimization of Drug Administration Routes May 20, 2020
- Experimental gene therapies for the NCLs Mar 24, 2020
- Neuropathophysiology of Lysosomal Storage Diseases: Synaptic Dysfunction as a Starting Point for Disease Progression Feb 25, 2020
- Future perspectives: What lies ahead for Neuronal Ceroid Lipofuscinosis research? Jan 8, 2020
- The contribution of multicellular model organisms to neuronal ceroid lipofuscinosis research Nov 26, 2019
- Cellular models of Batten disease Oct 23, 2019
- Changes in motor behavior, neuropathology, and gut microbiota of a Batten disease mouse model following administration of acidified drinking water Oct 18, 2019
- The CLN3 gene and protein: What we know Sep 30, 2019
- Pharmacological approaches to tackle NCLs Sep 12, 2019
- Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease Sep 6, 2019
- Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease Jul 10, 2019
- TPP1 Delivery to Lysosomes with Extracellular Vesicles and their Enhanced Brain Distribution in the Animal Model of Batten Disease Apr 18, 2019
- Modulation of CRMP2 via (S)-Lacosamide shows therapeutic promise but is ultimately ineffective in a mouse model of CLN6-Batten disease Apr 8, 2019
- Upregulation of tripeptidyl-peptidase 1 by 3-hydroxy-(2,2)-dimethyl butyrate, a brain endogenous ligand of PPARα: Implications for late-infantile Batten disease therapy Mar 28, 2019
- Current and Emerging Treatment Strategies for Neuronal Ceroid Lipofuscinoses Mar 15, 2019
- Therapeutic landscape for Batten disease: current treatments and future prospects Feb 19, 2019
- Emerging new roles of the lysosome and neuronal ceroid lipofuscinoses Jan 16, 2019
Clinical Trials
- Safety Study of a Gene Transfer Vector (AAV2CUhCLN2) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis [Completed] Jul 24, 2020
- A Retrospective, Natural History Study in Children With CLN2 [Observational] Jul 21, 2020
- Cerliponase Alfa Observational Study in the US Jul 20, 2020
- An Observational Study in Children With CLN2 Batten Disease Jul 8, 2020
- A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease May 21, 2020
- A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease May 21, 2020
- Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) Apr 30, 2020
- Gene Therapy (AT-GTX-502) for Children With CLN3 Batten Disease Mar 25, 2020
- Long-Term Follow-Up of AT-GTX-501 scAAV9 Gene Transfer in Subjects With CLN6 Batten Disease Feb 26, 2020
