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sickle cell disease

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Genetix Biotherapeutics’ partnership with the National Football League Alumni Association highlights efforts to expand sickle cell disease awareness and improve understanding of gene therapy treatment options, including LYFGENIA. Representative image.
Pharma & Biotech

Genetix Biotherapeutics targets sickle cell treatment awareness with NFL Alumni partnership

Genetix Biotherapeutics and NFL Alumni are targeting the awareness and access gap that still limits sickle cell gene therapy adoption.

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Laboratory research into sickle cell disease therapies highlights Agios Pharmaceuticals’ decision to halt tebapivat development after mixed Phase 2 results, shifting investor focus to mitapivat. Representative image.
Pharma & Biotech

Agios Pharmaceuticals stops tebapivat sickle cell development after mixed Phase 2 results

Agios Pharmaceuticals, Inc. has decided not to advance tebapivat in sickle cell disease after a randomized Phase 2 trial involving 59 participants failed to establish the differentiated clinical profile required for further investment. The Nasdaq-listed rare-disease drug developer said the once-daily pyruvate kinase activator improved haemoglobin and markers of haemolysis, but the response pattern did

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Representative image: Vertex Pharmaceuticals’ expanded FDA approval for CASGEVY opens CRISPR gene therapy to children aged 2 and older with sickle cell disease and transfusion-dependent beta thalassemia.
Pharma & Biotech

Vertex expands CASGEVY to young children with sickle cell disease and beta thalassemia

Explore how the FDA’s CASGEVY age expansion could reshape early gene therapy for sickle cell disease and beta thalassemia. Read more.

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Representative image of sickle cell disease research and treatment. This article explores how NeoImmuneTech’s U.S. licensing deal for Endari could reshape the commercial landscape for sickle cell therapies in North America.
Pharma & Biotech

NeoImmuneTech takes over Endari’s U.S. rights from Emmaus: What this signals for the future of sickle cell treatment

NeoImmuneTech takes over U.S. rights for Endari from Emmaus Life Sciences. Find out what this deal reveals about the future of sickle cell treatment.

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