The Life Sciences team advised Ultragenyx Pharmaceutical Inc. on its partnership with GeneTx Biotherapeutics LLC to develop GeneTx’s GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome, a serious, debilitating, rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide. Under the terms of the partnership, Ultragenyx will make an initial upfront payment of $20 million to GeneTx in exchange for the exclusive option to acquire GeneTx for additional consideration, including milestone and royalty payments.
Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for serious rare and ultra-rare diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease. GeneTx is a startup biotechnology company singularly focused on developing and commercializing a safe and effective antisense therapeutic for the treatment of Angelman Syndrome.