Patient access, antisense oligo key to Angelman deal for Ultragenyx

Ultragenyx's partnership and option deal with Angelman syndrome company GeneTX delivers two components key to success in the rare disease: a candidate that targets the root cause of the disease, and a direct link to patients.

The companies teamed up to advance GeneTX Biotherapeutics LLC's preclinical antisense oligonucleotide, GTX-102, into clinical development, and Ultragenyx Pharmaceuticals Inc. (NASDAQ:RARE) received an option to acquire GeneTX any time up to 30 days after the IND is accepted by FDA...