Ultragenyx Pharmaceutical, a California-based biotech company focussed on research and ultra-rare diseases, ventures in the field of gene therapy as it enters a collaboration with Solid Biosciences, a life sciences company focused on the development of therapies for Duchenne Muscular Dystrophy. This strategic collaboration allows both the companies to work together for developing products that combine differentiated microdystrophin construct developed by Solid Biosciences and Ultragenyx’s HeLa producer cell line (PCL) manufacturing platform and Adeno Associate Virus 8 (AAV8) variants.
Under the terms of agreement, Solid Biosciences granted an exclusive licence to Ultragenyx Biosciences for any pharmaceutical drug that expresses Solid’s proprietary microdystrophin construct from AAV8 variants. Solid will retain complete rights to all other uses of its microdystrophins and the lead candidate, SGT-001. Ultragenyx paid an upfront of USD 40 million to Solid Biosciences and Solid will additionally also receive up to USD 255 million in the form of milestone payment and tiered royalties on worldwide sale post commercialization. Upon approval of proof-of-concept, Solid will have the right to opt for co–funding programs in return for participation in profit share or increased royalty payments.
The target investigational drug, SGT-001, is undergoing an open label, Phase I/II clinical trial to evaluate safety, tolerability and effectiveness in 16 subjects aged 4 to 17 years (IGNITE DMD). It is a systematically administered candidate that delivers the proprietary microdystrophin construct developed by Solid, encoding the functional protein surrogate to stabilize essential associate proteins. The candidate has proven effective in preclinical models. Solid Biosciences is currently competing against Pfizer’s, Astellas and Sarepta Therapeutics gene therapy for Duchenne Muscular Dystrophy as its candidate stands at an advantage most advanced in Phase III clinical trial.