Vertex Pharmaceuticals and Orna Therapeutics announced a three-year strategic research collaboration focused on developing next-generation gene editing therapies for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The collaboration will leverage Orna's proprietary lipid nanoparticle (LNP) delivery solutions.
Under the terms of the agreement, Vertex will provide Orna with an upfront payment of $65 million, which includes an investment in the form of a convertible note. Orna is also eligible to receive up to $635 million in potential preclinical, clinical, regulatory, and commercial milestone payments related to SCD/TDT products.
Additionally, Orna could receive up to $365 million in further option fees and milestones per product for up to ten additional products if Vertex exercises rights in other indications. Orna will also be eligible for tiered royalties on future net sales of any products resulting from this collaboration.
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