Vertex, Orna Therapeutics Collaborate on Gene Editing Therapies for Sickle Cell Disease, Beta Thalassemia

MT Newswires Live
07 Jan

Vertex Pharmaceuticals (VRTX) has entered into a three-year research collaboration with Orna Therapeutics to develop gene editing therapies for sickle cell disease and transfusion-dependent beta thalassemia, Orna said Tuesday.

The collaboration will use Orna's lipid nanoparticle delivery system to advance Vertex's therapeutic development, Orna said.

Under the agreement, Orna said it will receive $65 million upfront, including a convertible note investment, and could earn up to $635 million based on achieving milestones for SCD/TDT products.

Orna said it is eligible for up to $365 million in additional option fees and milestones per product for up to 10 additional products if Vertex exercises rights for new indications, and will also receive tiered royalties on future sales. Vertex is financing the research collaboration and has the option to extend its duration.

Shares of Vertex were up 1.9% in recent Tuesday trading.

Price: 409.97, Change: +7.48, Percent Change: +1.86

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