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Encoded TherapeuticsBiotechUnited States

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Encoded Therapeutics is a biotechnology company headquartered in South San Francisco, California, dedicated to developing next-generation genetic medicines designed to address the root causes of genetic diseases.

EdiGene is a clinical-stage biotechnology company founded in 2015 that focuses on translating gene-editing technologies into therapeutic products. Headquartered in Beijing, China, the company also operates research and office facilities in locations such as Shanghai, Guangzhou, and the United States.

Excision BioTherapeutics Inc. is a San Francisco-based clinical-stage biotechnology company focused on developing advanced gene editing therapeutics to treat infectious diseases.

Beam Therapeutics Inc., founded in 2017 and based in Cambridge, Massachusetts, operates as a pioneering biopharmaceutical firm. Its core mission involves engineering precise genetic remedies to tackle a spectrum of severe human ailments, primarily within the United States. The company's developmental portfolio features several key candidates: BEAM-101 is being advanced to treat both sickle cell disease and beta thalassemia. BEAM-102 is specifically designed for addressing sickle cell disease. BEAM-201, an allogeneic chimeric antigen receptor T-cell therapy, is under investigation for individuals suffering from relapsed or refractory T-cell acute lymphoblastic leukemia. BEAM-301 is a liver-targeted candidate aimed at patients afflicted with Glycogen Storage Disease Type Ia. Beyond these flagship programs, Beam Therapeutics is also engaged in devising treatments for alpha-1 antitrypsin deficiency, various ocular conditions, and other disorders impacting the liver, muscular system, and central nervous system. To further its research and development objectives, Beam Therapeutics has forged numerous strategic alliances and partnerships. These include collaborations with Boston Children's Hospital; a research and clinical trial agreement with Magenta Therapeutics, Inc.; a licensing deal with Sana Biotechnology, Inc.; and a research partnership with the Institute of Molecular and Clinical Ophthalmology Basel. Additionally, the company maintains research collaborations with both Pfizer Inc. and Apellis Pharmaceuticals, Inc., alongside a comprehensive collaboration and license agreement with Verve Therapeutics, Inc.

Base Therapeutics is a biotechnology company that develops next-generation gene editing technologies, including its proprietary AccuBase base editing system and enhanced prime editing technology. The company focuses on creating therapeutic solutions for cancer and genetic diseases, alongside engineered immune cell therapies such as BASE-NK and CAR-T products. It serves patients and partners with pharmaceutical and agricultural enterprises by licensing its precision gene editing tools.

CRISPR Therapeutics AG (CRSP) is a biotechnology firm dedicated to pioneering gene-based medicines for severe diseases. The company achieves this through its exclusive Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) technology, which enables precise and targeted alterations to an organism's genetic code. Its extensive pipeline includes therapeutic candidates spanning multiple medical areas, such as blood disorders (hemoglobinopathies), various cancers (oncology), regenerative medicine, and rare conditions. The company's flagship investigational therapy is CTX001, an ex vivo CRISPR gene-edited treatment. This therapy aims to benefit patients with transfusion-dependent beta-thalassemia or severe sickle cell disease by modifying their own hematopoietic stem cells to markedly boost the production of fetal hemoglobin within red blood cells. CRISPR Therapeutics is also advancing several other genetically engineered allogeneic (donor-sourced) CAR-T investigational therapies: CTX110, designed to combat cluster of differentiation 19-positive malignancies; CTX120, which targets B-cell maturation antigen for multiple myeloma that has relapsed or proven resistant to previous treatments; and CTX130, focused on Cluster of Differentiation 70 for a spectrum of solid tumors and blood cancers. Furthermore, the company is developing VCTX210, an immune-evasive, gene-edited stem cell-derived product candidate for treating type 1 diabetes. It is also pursuing various in vivo gene-editing initiatives aimed at addressing disorders affecting the liver, lungs, muscles, and central nervous system. The company has forged strategic alliances with significant partners, including Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, ViaCyte, Inc., Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was established in 2013 and maintains its headquarters in Zug, Switzerland.