Vertex has signed a three-year strategic research collaboration with Orna Therapeutics’ subsidiary ReNAgade Therapeutics to develop next-gen gene editing therapies for patients with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
The deal allows Vertex to utilize Orna’s novel and proprietary LNP delivery solutions to develop in vivo gene editing therapies that are ideally safer and more effective. Orna’s scalable LNP delivery solutions are designed to reach parts of the body other than the liver, such as stem cells in the bone marrow.
Under the terms of the agreement, Orna will receive upfront payments of $65 million, including an investment in the form of a convertible note, and is eligible to receive up to $635 million based on certain milestones related to SCD/TDT products. Additionally, Orna is further eligible to receive up to $365 million in additional option fees and milestones per product for up to ten additional products if Vertex options rights in additional indications.
Orna acquired ReNAgade in May 2024 for an undisclosed amount. ReNAgade, which launched last year, combines novel RNA delivery platforms with a comprehensive RNA platform allowing for an all-RNA system for coding, editing, and gene insertion to develop new medicines.
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