Vertex Pharmaceuticals

Arbor Biotechnologies, Inc., founded in 2016 and based in Cambridge, Massachusetts, operates within the biotechnology sector, focusing on bio-discovery and human diagnostic development. The company utilizes a range of advanced technologies, including artificial intelligence, genome sequencing, gene synthesis, and high-throughput screening, to facilitate the discovery of proteins aimed at enhancing human health and sustainability. Arbor's proprietary genomic tools leverage AI and machine learning to analyze genomic sequence data, thereby improving the efficiency of developing curative treatments for genetic diseases. These innovations enable drug developers to replace entire genes and precisely correct mutations, ultimately addressing previously untreatable genetic conditions.

Alpine Immune Sciences is a clinical-stage biopharmaceutical company based in Seattle, Washington, specializing in the discovery and development of protein-based immunotherapies for cancer, autoimmune, and inflammatory disorders. The company employs a proprietary platform to transform native immune system proteins into multi-targeted therapeutics. Its lead programs include ALPN-101, a dual antagonist targeting the inducible T cell costimulator (ICOS) and CD28 pathways, aimed at treating autoimmune and inflammatory diseases, and ALPN-303, a dual antagonist of the B cell activating factor (BAFF) and the proliferation-inducing ligand (APRIL), which are crucial for B cell activation and survival. Additionally, Alpine has developed ALPN-202, an antagonist targeting programmed cell death protein ligand 1 (PD-L1) and cytotoxic T-lymphocyte associated protein 4 (CTLA-4), designed to enhance cancer treatment. The company also collaborates with Kite Pharma to advance immunotherapy approaches targeting the immune synapse in cancer therapy.

Septerna is a biotechnology company focused on discovering and advancing novel small-molecule medicines that target G protein-coupled receptors (GPCRs). The company aims to improve patient outcomes by integrating innovative drug discovery technologies with GPCR drug targets. Utilizing its Native Complex platform, Septerna facilitates industrial-scale drug discovery through advanced screening technologies and structure-based drug design. This approach enables medical researchers to efficiently identify treatments for a variety of diseases.

Arbor Biotechnologies, Inc., founded in 2016 and based in Cambridge, Massachusetts, operates within the biotechnology sector, focusing on bio-discovery and human diagnostic development. The company utilizes a range of advanced technologies, including artificial intelligence, genome sequencing, gene synthesis, and high-throughput screening, to facilitate the discovery of proteins aimed at enhancing human health and sustainability. Arbor's proprietary genomic tools leverage AI and machine learning to analyze genomic sequence data, thereby improving the efficiency of developing curative treatments for genetic diseases. These innovations enable drug developers to replace entire genes and precisely correct mutations, ultimately addressing previously untreatable genetic conditions.

Kymera Therapeutics is a biopharmaceutical company based in Watertown, Massachusetts, that specializes in the discovery and development of novel small molecule therapeutics aimed at selectively degrading disease-causing proteins. The company utilizes a proprietary platform for targeted protein degradation, leveraging the body’s natural protein recycling system. Kymera is developing several programs, including the IRAK4 program for immunology-inflammation diseases such as hidradenitis suppurativa and rheumatoid arthritis, the IRAKIMiD program to address MYD88-mutated diffuse large B cell lymphoma, and the STAT3 program targeting hematologic malignancies, solid tumors, and autoimmune diseases. Founded in 2015, Kymera Therapeutics is pioneering new approaches to treat diseases that have previously been considered untreatable, focusing on previously inaccessible therapeutic targets.

Semma Therapeutics, Inc. is focused on developing innovative cell therapies for patients with Type 1 diabetes, who typically rely on insulin injections. The company was founded based on groundbreaking research by Professor Douglas Melton, which enabled the generation of functional, insulin-producing beta cells in the laboratory. This technology has been exclusively licensed to Semma Therapeutics, and the company aims to combine these proprietary cells with advanced devices to create a functional replacement for the missing beta cells without the need for immunosuppression. Headquartered in Cambridge, Massachusetts, with an additional office in Boston, Semma Therapeutics operates as a subsidiary of Vertex Pharmaceuticals and is dedicated to advancing this new therapeutic option to improve the lives of diabetes patients.

Exonics Therapeutics, Inc. is focused on developing gene editing technologies to address Duchenne muscular dystrophy (DMD), a severe genetic neuromuscular disorder. Founded in 2017 and headquartered in Boston, Massachusetts, with an additional office in Dallas, Texas, the company utilizes CRISPR/Cas9 technology in conjunction with adeno-associated virus (AAV) delivery systems. This innovative approach targets and corrects specific exon mutations that hinder the production of dystrophin, a crucial protein for muscle fiber stability and protection. By aiming for a permanent correction of these genetic mutations, Exonics Therapeutics seeks to provide patients with lasting improvements in their quality of life. The company operates as a subsidiary of Vertex Pharmaceuticals Incorporated.

Genomics plc is focused on transforming healthcare through advanced genomic analysis. The company employs sophisticated methodologies to analyze large integrated datasets of genomic and phenotypic information, aiming to enhance understanding of human biology. By leveraging genetic inference technology, Genomics plc seeks to identify new drug targets for serious diseases and assess individual disease risk. Their analytical platform enables the exploration of genomic sequence data, providing valuable insights that support pharmaceutical companies in reducing risks associated with drug development. This approach not only aims to improve healthcare outcomes but also sets a standard for how organizations and patients can benefit from genomic data.

Genomics plc is focused on transforming healthcare through advanced genomic analysis. The company employs sophisticated methodologies to analyze large integrated datasets of genomic and phenotypic information, aiming to enhance understanding of human biology. By leveraging genetic inference technology, Genomics plc seeks to identify new drug targets for serious diseases and assess individual disease risk. Their analytical platform enables the exploration of genomic sequence data, providing valuable insights that support pharmaceutical companies in reducing risks associated with drug development. This approach not only aims to improve healthcare outcomes but also sets a standard for how organizations and patients can benefit from genomic data.

Obsidian Therapeutics is a developer of a next-generation cell and gene therapies created to extend adoptive immunotherapy to every patient with cancer.The company's next-generation cell and gene therapies with pharmacologic operating systems provide exquisite control of protein activity in cells and create adoptive cell therapies with new functions that are under control of the treating physician using simple, safe, orally-active, marketed drugs, enabling patients to improve on the current generation of cell therapies.

CRISPR Therapeutics is focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. They have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from their scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. CRISPR Therapeutics’ vision is to cure serious human diseases at the molecular level using CRISPR-Cas9. The company is headquartered in Basel, Switzerland and has operations in London, UK.

ViroChem Pharma is a privately held company focused on developing and producing pharmaceutical products for viral diseases, specifically targeting Hepatitis C (HCV) and Human Immunodeficiency Virus (HIV). The company is advancing two investigational HCV polymerase inhibitors that are currently in clinical development, aiming to provide effective treatment options for patients suffering from these viral infections. With a commitment to addressing unmet medical needs in the field of virology, ViroChem Pharma plays a critical role in the ongoing fight against these significant health challenges.

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.