Vertex Pharmaceuticals Inc. will work with a Watertown biotech on new gene-editing therapies for people with sickle cell disease and transfusion-dependent beta thalassemia.

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Vertex turns to Watertown biotech for gene-editing medicines pact

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— Collaboration to leverage Orna’s differentiated lipid nanoparticle (LNP) delivery solutions for patients with SCD and TDT — — Orna to receive an upfront payment and is eligible to receive potential pre-clinical, clinical, and commercialization milestone payments and royalties — WATERTOWN, MA, USA I January 7, 2025 I Orna Therapeutics (through its wholly owned subsidiary ReNAgade Therapeutics Inc.) announced a three-year strategic research coll…

pipelinereview

Orna Therapeutics Establishes Strategic Collaboration with Vertex Pharmaceuticals to Develop Next Generation Approaches for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) - Pipelinereview

Vertex Pharmaceuticals signed a three-year deal with Orna Therapeutics to use the RNA biotech’s delivery particles to develop its next iteration of gene editing treatments for sickle cell disease and beta thalassemia.
The partnership announcement ...

Endpoints News

Vertex signs in vivo gene editing delivery pact for sickle cell and thalassemia as it looks beyond Casgevy

The deal is set to help Vertex’s development of next-generation gene editing therapies for sickle cell disease and beta-thalassemia.

European Medical Journal

Vertex strikes deal for Orna Therapeutics' LNP technology

Orna will receive a $65m upfront payment and is eligible to receive potential milestone payments and royalties.

pharmaceutical-technology.com

Vertex and Orna sign partnership to improve efficiencies of gene therapies  - Pharmaceutical Technology

Greetings, Dr. Roach I have thalassemia mild and am Greek. My iron content is very low, and my red blood cells are generally much smaller than normal. Each time I visit a new doctor and they do a blood test, they always stress that I need to take iron supplements since my iron levels are low. I’m not sure if this is sensible advise because, as I understand it, thalassemia isn’t a problem with iron absorption from the diet, but rather a little re…

legalizationprofiles.org

Dear Doctor: Are iron supplements recommended for patients with thalassemias? - Legalization Profiles

The three-year partnership is another example of Vertex’s interest in improving on Casgevy, its CRISPR medicine for sickle cell and beta thalassemia.

Vertex turns to Watertown biotech for gene-editing medicines pact

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