rare disease treatment

Ex-Genzyme CEO’s new company looks to rare diseases for insight into treating Parkinson’s

Scientists suspect there’s a connection between many common neurodegenerative diseases and malfunctioning lysosomes, or processing centers in human cells that break down proteins and eliminate debris. Now a new company called Lysosomal Therapeutics is tapping into the relationship between the two to discover and develop new treatments for rare diseases and common neurodegenerative diseases, starting […]


Rare disease biotech picks up $8M, drug development partner for DNA-based therapies

Amid somewhat of a renaissance in drug development for orphan diseases, another gene therapy company announced funding today for a potential treatment for a rare neurodegenerative condition called Friedreich’s ataxia (FRDA). Agilis Biotherapeutics said it’s raised $8 million and will work with synthetic biology company Intrexon Corp. to develop gene therapies and genetically modified cell […]

Quartet of advocacy groups seed an Ohio startup’s gene therapy for rare Sanfilippo syndrome

A Cleveland biotech startup focused on a rare genetic disease provides an interesting example of the growth of alternative funding for life science companies. Abeona Therapeutics’ just-announced $750,000 seed round, which will allow the startup to complete preclinical development of gene therapies for Sanfilippo syndrome, came not from angel or venture capital investors but from […]