{
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vertex partners with orna therapeutics to advance gene therapies for blood disorders

Vertex Pharmaceuticals has partnered with Orna Therapeutics, investing $65 million to develop next-gen gene therapies for sickle cell disease and transfusion-dependent beta thalassemia. This collaboration allows Orna to expand its research capabilities and leverage its lipid nanoparticle delivery technology, aiming to create accessible treatments that bypass traditional cell therapy limitations. Vertex's recent FDA approvals for gene therapies in these areas further enhance the significance of this partnership.
Fierce Biotech
United States Dollar Vertex Pharmaceuticals Inc Merck & Co., Inc. USA Amit Munshi Joe Bolen
Healthcare, Press Release
13:00 07.01.2025

vertex partners with orna to advance in vivo gene editing therapies

Vertex Pharmaceuticals has entered a three-year partnership with Orna Therapeutics to advance in vivo gene therapies for sickle cell disease and beta thalassemia. The collaboration includes an upfront payment of $65 million, with potential milestone payments totaling up to $635 million. This initiative aims to enhance gene editing delivery methods, potentially eliminating the need for toxic preconditioning and streamlining the treatment process.
BioPharma Dive
United States Dollar Vertex Pharmaceuticals Inc CRISPR Therapeutics AG USA Amit Munshi
Technology & Innovation, Press Release
01:00 07.01.2025
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