Abeona Therapeutics, a Cleveland-based biotechnology startup, closed a $3.6m funding round.
Investors include U.S.-based Cure Sanfilippo Foundation, Sanfilippo Research Foundation (SRF), Team Sanfilippo, the Abby Grace Foundation, and the National MPS Society. Support also came from the international Sanfilippo community, with investments from Spain-based Stop Sanfilippo and Sanfilippo B Foundation, Geneva, Switzerland-based Fondation Sanfilippo and Mexico-based Red Sanfilippo Foundation, from The Children’s Medical Research Foundation, Inc. (CMRF) and Canadian led Sanfilippo Children’s Research Foundation (SCRF).
The company intends to use the funds to complete preclinical development of therapies for children with Sanfilippo syndrome (mucopolysaccharidosis type III, MPS III), a rare terminal genetic disorder that results in death in children before they reach their mid-teens.
Led by Tim Miller, Ph.D, President/CEO, Abeona focuses on developing gene therapy based treatments for patients with lysosomal storage diseases, with lead products focusing on MPS IIIA and IIIB. The company received Orphan Product Designations for MPS IIIA and IIIB in 2014 and clinical trials are anticipated to begin in early-to-mid 2015.