How Ultragenyx delivered win for biotech venture philanthropy via GeneTx deal
Rare disease company invested in a spinout of a non-profit that was making science decisions, not just writing checks
Ultragenyx’s acquisition of the Foundation for Angelman Syndrome Therapeutics’ GeneTx is the capstone of a three-year partnership that has yielded the company a de-risked treatment for Angelman syndrome, financial upside for the non-profit, and the rare disease community another successful case study in venture philanthropy, an area Ultragenyx CEO Emil Kakkis has helped pioneer.
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) paid $75 million up front last month to acquire partner GeneTx Biotherapeutics LLC via an option received in a 2019 deal to advance antisense oligonucleotide GTX-102 for Angelman syndrome, a rare neurodevelopment disorder. That deal came a year after GeneTx’s debut as the first for-profit biotech launched by a non-profit patient organization, the Foundation for Angelman Syndrome Therapeutics (FAST)...