Method to Inhibit Toxic Pathways Activated in Genetic ALS/FTD

Value Proposition

• Treatment readily acts via subcutaneous injection and crosses the blood-brain barrier without extra formulation or drug delivery mechanism.
• Treatment significantly reduces pathological and behavioral phenotypes from toxic proteins coded by genetic defects in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), which are both diseases without cures.

Technology Description

ALS and FTD are both severe neurodegenerative diseases with 2-7 year survival rates. Researchers at Johns Hopkins have developed a method to inhibit pathways that contribute to the expression of toxic proteins in ALS and FTD. Current research demonstrates that the method can rescue phenotypic ALS/FTD in experimental models.

Unmet Need

Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are incurable diseases with 30,000 and 50,000-60,000 patients in the US. Current ALS treatments are expensive and can only slow disease progression. No FDA approved treatments for FTD exist. Therefore, there exists a resounding need for a treatment that can rescue symptoms of ALS/FTD patients.

Stage of Development

• Researchers have demonstrated rescue efficacy in patient-derived induced pluripotent stem cell-differentiated neurons and validated the rescue effect in mouse model.

Data Availability: Data available upon request.

Publication

WO2024229355