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Vertex Pharmaceuticals
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Vertex Presents Long-Term CLIMB Trial Program Data of Casgevy (Exagamglogene Autotemcel) to Treat Hemoglobinopathies at EHA 2025

Shots: The global P-I/II/III (CLIMB-121 & 111) trials assessed Casgevy in sev. SCD & TDT pts (12–35 yrs), with 2yr. post-infusion follow-up, after which pts entered the ongoing 15yr. CLIMB-131 trial, with SCD pts followed for >5.5yrs & TDT for >6yrs. In SCD, 43/45 pts were VOC-free for ≥12mos., with mean VOC-free duration of 35mos.…
July 4, 2025

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Orna Collaborates with Vertex to Develop Gene Therapies for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)

Shots: Orna & Vertex have entered into a 3yrs. strategic research collaboration to utilize the former’s LNP delivery solutions for supporting the development of next-gen. gene editing therapies for SCD & TDT, with Vertex funding the partnership As per the terms, Orna will get $65M upfront (incl. investment in the form of a convertible…
January 8, 2025

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