CRISPR Therapeutics AG
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- CRISPR Therapeutics AG (Nasdaq: CRSP) is a biotechnology company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 gene-editing platform. Founded in 2013 and headquartered in Zug, Switzerland, the company operates globally with research and development facilities in Boston and San Francisco, California, and a state-of-the-art manufacturing site in Framingham, Massachusetts. Its lead approved therapy, exagamglogene autotemcel (exa-cel, commercialized as CASGEVY in collaboration with Vertex Pharmaceuticals), is an autologous ex vivo CRISPR/Cas9 gene-edited cell therapy approved in multiple countries for eligible patients with sickle cell disease and transfusion-dependent beta-thalassemia; the company advances a broad pipeline including wholly owned and partnered programs across hemoglobinopathies such as next-generation conditioning agents and in vivo HSC editing; immuno-oncology and autoimmune therapies like CTX112 (anti-CD19 allogeneic CAR T) and other CAR T candidates targeting CD19, CD70, and glypican-3; in vivo cardiovascular programs including CTX310 (ANGPTL3), CTX320 (Lp(a)), and CTX340 (AGT for refractory hypertension); additional in vivo candidates such as CTX450 (ALAS1 for acute hepatic porphyria), CTX460 (SERPINA1 for alpha-1 antitrypsin deficiency), and undisclosed programs in rare and common diseases; regenerative medicine offerings like CTX211 and CTX213 (allogeneic gene-edited stem cell-derived therapies for type 1 diabetes); and partnered siRNA and other initiatives including SRSD107 (Factor XI for thromboembolic conditions with Sirius Therapeutics) and programs in Duchenne muscular dystrophy, myotonic dystrophy type 1, cystic fibrosis, and type 1 diabetes licensed to Vertex. In 2025, CRISPR Therapeutics expanded its portfolio through a strategic collaboration with Sirius Therapeutics, involving a $25 million cash upfront and $70 million equity investment to co-develop and commercialize novel siRNA therapies starting with SRSD107, with options for two additional programs and shared costs/profits; the company also continues global commercialization of CASGEVY, achieving regulatory approvals in nine countries and treating over 100 patients as of mid-2025 while advancing clinical milestones for CTX112, CTX310, CTX320, and others amid strong cash reserves exceeding $1.7 billion.
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