Vertex partners with Orna to advance next-gen gene therapies
Vertex Pharmaceuticals (Nasdaq: VRTX) has announced a three-year research deal with Orna Therapeutics, including its wholly owned subsidiary ReNAgade Therapeutics, worth up to $4 billion.
The collab, which will see the firms developing next-generation gene editing therapies for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), comes with a $65 million upfront payment.
Orna will use its lipid nanoparticle delivery solutions to enable in vivo gene therapies targeting hematopoietic stem cells. The firm is able to bypass traditional active targeting, reducing complexity.
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