Ultragenyx and Arcturus Therapeutics made the announcement on June 19th in California, USA. Their existing collaboration now expands to include up to 12 rare disease targets and includes Arcturus’ nucleic acid technologies to enable mRNA, DNA, and siRNA therapeutics.
GSD3 is one of the 12 targets. Ultragenyx also have a gene therapy in development for GSD1a which has completed preclinical studies.
Ultragenyx becomes Arcturus’ largest shareholder with $30 million of upfront payments to Arcturus, including $6 million cash for collaboration agreement amendment and a $24 million equity investment at $10 per share.
A focus on GSD3
Emil D. Kakkis, MD, PhD, Chief Executive Officer and President of Ultragenyx said:
“We are pleased with the progress of our ongoing collaboration. Our most advanced mRNA program, UX053 for the treatment of Glycogen Storage Disease Type III, is expected to move into the clinic next year, and we look forward to further building upon the initial success of this partnership.”
AGSD-UK understands this to indicate that a clinical trial in GSD3 may start before the end of 2020.