Private clinical stage drug company.
We create novel biological agents to treat Orphan Diseases with an initial focus on Tay-Sachs Disease and Gaucher Disease..
Tay-Sachs Disease, Gaucher Disease, other lysosomal storage diseases are orphan designated diseases.
Tremendous unmet clinical need
- No therapeutic options for Tay-Sachs Disease .
- No therapeutic options for the neurological complications Gaucher Disease Type 1 and 3.
- Recently a link has been established between Gaucher Type 1 and Parkinsonism
- The company is poised to address the neurological and non-neurological manifestations of ERAD diseases with a drug delivery system that will cross the Blood-Brain-Barrier and deliver the enzyme to all cells in the human body
Large & profitable market opportunity
- No current treatment for Tay-Sachs Disease
- Type 1 Gaucher Disease market currently exceeds $1.6 billion per year and is growing
- Ability to address neurological aspects of Gaucher disease may significantly expand this market
Experienced management team and Board of Directors.