
Samarth Kulkarni
Delivered the world's first approved CRISPR gene-editing therapy (Casgevy) for sickle cell disease, opening the era of genetic medicine
Samarth Kulkarni serves as CEO of CRISPR Therapeutics, the Swiss-American biotechnology company that achieved one of the most important milestones in medical history: the approval of Casgevy (exagamglogene autotemcel), the world's first CRISPR-based gene-editing therapy. Approved in the UK, US, and EU for sickle cell disease and transfusion-dependent beta-thalassemia, Casgevy represents the beginning of an entirely new therapeutic modality — using gene editing to potentially cure genetic diseases in a single treatment. CRISPR Therapeutics was co-founded by Emmanuelle Charpentier, who shared the 2020 Nobel Prize in Chemistry for the discovery of CRISPR-Cas9 gene editing. Casgevy works by editing a patient's own blood stem cells to produce fetal hemoglobin, which compensates for the defective hemoglobin that causes sickle cell disease. The treatment is complex and expensive (priced at approximately $2.2 million per patient) but potentially curative. Beyond Casgevy, CRISPR Therapeutics is developing gene-edited cell therapies for cancer (CAR-T therapies using gene editing to create more potent immune cells), in vivo gene editing (editing genes directly inside the body rather than in extracted cells), and therapies for diabetes, autoimmune diseases, and cardiovascular conditions. Key stock drivers include Casgevy commercial launch uptake, patient identification and treatment center expansion, pipeline clinical trial results, competition from other gene therapy companies (Intellia, Beam, Editas), and the evolution of gene-editing technology.
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