What We Do
Various therapeutics, including gene therapy, stem cell therapy, enzyme replacement therapy, anti-inflammatories, and small molecules have emerged to halt or slow the progression of Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL or CLN2). However, all available approaches are just supportive or provide some relief of symptoms, indicating an urgent need for novel therapeutic interventions.
We are developing patient-friendly, oral small-molecules using a drug repurposing strategy, because many pediatric patients are not able to wait until novel therapies are fully assessed. Drug repurposing which applies known drugs to new indications reduces development time and generally provides extensive information on safety. This strategy is ideally suited for the treatment of rare genetic disorders, especially in children, for whom access to approved medications is critical.
Drug repurposing saves time, cost,
and generally provides extensive
information on safety.
Based on preclinical studies, our small molecule therapeutics, which utilize known safe drugs, may have a prophylactic and/or disease-modifying potential for Neuronal Ceroid Lipofuscinosis (NCL), including Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL or CLN2) and Juvenile and Infantile forms.