Vertex Pharmaceuticals
Vertex Pharmaceuticals is a global biotechnology company dedicated to discovering and developing small-molecule drugs aimed at treating serious diseases. The company has established a strong presence in the cystic fibrosis treatment market with its key therapies, such as Kalydeco, Orkambi, Symdeko, and Trikafta/Kaftrio, which are widely regarded as the standard of care. Additionally, Vertex has expanded its portfolio to include Casgevy, a gene-editing therapy addressing beta thalassemia and sickle-cell disease. The company is also exploring innovative solutions for pain management through non-opioid small-molecule inhibitors and is investigating treatments for kidney diseases linked to APOL1. Vertex's strategic approach includes both independent commercialization of its products and collaborations with major pharmaceutical companies, reflecting its commitment to addressing a range of serious health conditions.
Arbor Biotechnologies operator of a bio-discovery company intended to provide human diagnostic development service. The company's platform employs a diverse set of technologies and techniques including artificial intelligence, genome sequencing, gene synthesis and high-throughput screening, enabling drug developers to accelerate the discovery of proteins for improving human health and sustainability.
Kymera Therapeutics is a biopharmaceutical company based in Watertown, Massachusetts, established in 2015. It specializes in targeted protein degradation, developing innovative small molecule therapeutics that selectively degrade proteins associated with various diseases by utilizing the body's natural protein degradation mechanisms. The company is advancing several therapeutic programs, including the IRAK4 program aimed at treating immunology-inflammation diseases such as hidradenitis suppurativa, atopic dermatitis, and rheumatoid arthritis. Additionally, it is developing the IRAKIMiD program for MYD88-mutated diffuse large B cell lymphoma and the STAT3 program for hematologic malignancies and solid tumors, as well as autoimmune diseases. Through its proprietary predictive modeling platform, Kymera Therapeutics seeks to accelerate drug discovery and address previously untreatable conditions.
Semma Therapeutics, Inc. is focused on developing innovative cell therapies for patients with Type 1 diabetes, who traditionally rely on insulin injections. Founded based on groundbreaking research by Professor Douglas Melton, the company has licensed a method to generate functional, insulin-producing beta cells in the laboratory. Semma Therapeutics aims to combine these proprietary cells with advanced devices to create a comprehensive therapy that can replace the missing beta cells without the need for immunosuppression. By striving to bring this transformative treatment to clinical application, Semma Therapeutics seeks to significantly enhance the lives of diabetes patients. Headquartered in Cambridge, Massachusetts, the company is a subsidiary of Vertex Pharmaceuticals and collaborates strategically with Defymed.
Exonics Therapeutics, Inc. develops gene editing technologies like CRISPR/Cas9 to permanently correct the majority of duchenne muscular dystrophy mutations. The company was founded in 2017 and is headquartered in Boston, Massachusetts with additional office in Dallas, Texas. As of July 16, 2019, Exonics Therapeutics, Inc. operates as a subsidiary of Vertex Pharmaceuticals Incorporated.
Genomics PLC is focused on advancing the use of genomic data to transform healthcare. The company develops algorithms and software solutions that facilitate the analysis of large genomic and phenotypic datasets, aimed at understanding human biology and improving disease diagnosis and treatment. Its offerings serve a diverse range of clients, including governments, healthcare providers, pharmaceutical companies, and research laboratories. By providing tools that enhance the accuracy of genetic analysis, Genomics PLC helps de-risk the drug development process and enables clinicians to better identify individuals at risk for specific diseases. Founded in 2014 and based in Oxford, United Kingdom, the company aims to set standards in genomic data application, ultimately benefiting organizations and patients alike through innovative healthcare solutions.
Genomics PLC is focused on advancing the use of genomic data to transform healthcare. The company develops algorithms and software solutions that facilitate the analysis of large genomic and phenotypic datasets, aimed at understanding human biology and improving disease diagnosis and treatment. Its offerings serve a diverse range of clients, including governments, healthcare providers, pharmaceutical companies, and research laboratories. By providing tools that enhance the accuracy of genetic analysis, Genomics PLC helps de-risk the drug development process and enables clinicians to better identify individuals at risk for specific diseases. Founded in 2014 and based in Oxford, United Kingdom, the company aims to set standards in genomic data application, ultimately benefiting organizations and patients alike through innovative healthcare solutions.
CRISPR Therapeutics AG is a gene editing company dedicated to developing transformative gene-based medicines for serious human diseases using its proprietary CRISPR/Cas9 technology. This revolutionary platform allows for precise modifications to genomic DNA, enabling the company to target a variety of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The lead product candidate, CTX001, is an ex vivo CRISPR gene-edited therapy aimed at treating transfusion-dependent beta thalassemia and severe sickle cell disease by engineering patients' hematopoietic stem cells to produce elevated levels of fetal hemoglobin. Additionally, CRISPR Therapeutics is advancing other therapies, including CTX110, an allogeneic CAR-T therapy for certain malignancies, and additional programs targeting conditions such as multiple myeloma, solid tumors, diabetes, and various genetic disorders. The company collaborates with strategic partners such as Bayer Healthcare, Vertex Pharmaceuticals, and ViaCyte, and is headquartered in Zug, Switzerland.
Aurora Biosciences Corporation
Acquisition in 2001
Aurora Biosciences Corporation ("Aurora" or the "Company") designs and develops proprietary drug discovery systems, services and technologies to accelerate and enhance the discovery of new medicines. Aurora is developing an integrated technology platform comprised of a portfolio of proprietary fluorescent assay technologies and an ultra-high throughput screening ("UHTS") system designed to allow assay miniaturization and to overcome many of the limitations associated with the traditional drug discovery process.
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