Redmile Group

GRO Biosciences is focused on developing advanced protein therapeutics by utilizing innovative technologies in computational protein design and synthetic biology. The company has created several genomically recoded strains of bacteria that enable the incorporation of non-standard amino acids into proteins. This approach enhances the potency, stability, and targeted delivery of these therapeutics. GRO Biosciences aims to apply its technology to improve treatments for a range of conditions, including diabetes, growth disorders, and autoimmunity, thereby addressing significant unmet medical needs.

Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

Krystal Biotech is a biopharmaceutical company using gene therapy to develop best in class treatments that provide significant clinical benefit for patients suffering from rare debilitating disorders.

GRO Biosciences is focused on developing advanced protein therapeutics by utilizing innovative technologies in computational protein design and synthetic biology. The company has created several genomically recoded strains of bacteria that enable the incorporation of non-standard amino acids into proteins. This approach enhances the potency, stability, and targeted delivery of these therapeutics. GRO Biosciences aims to apply its technology to improve treatments for a range of conditions, including diabetes, growth disorders, and autoimmunity, thereby addressing significant unmet medical needs.

Mammoth Biosciences, Inc. is a biotechnology company based in South San Francisco, California, that focuses on developing innovative CRISPR solutions for a variety of applications. The company offers DETECTR™, a diagnostic platform that identifies specific nucleic acids in samples, aiding in the detection of diseases such as bacterial infections, cancer, and viral infections. Additionally, Mammoth Biosciences provides CRISPR-Cas systems for genome editing, targeting therapeutic areas including immuno-oncology, autoimmune diseases, and liver diseases. By harnessing proprietary ultracompact proteins and leveraging advanced research and development, Mammoth aims to create affordable point-of-care tests and enhance diagnostics across healthcare, agriculture, and environmental monitoring. Co-founded by CRISPR pioneer Jennifer Doudna, the company has attracted significant investment from institutional and individual backers.

Prime Medicine is a biotechnology company focused on developing genetic therapies using its proprietary Prime Editing technology. This next-generation gene editing method allows for precise "search and replace" modifications in the genome, aiming to restore normal genetic function and address the underlying causes of various diseases. By targeting a broad spectrum of conditions with significant unmet medical needs, Prime Medicine seeks to revolutionize the field of genetic medicine through effective and efficient therapeutic solutions.

Strand Therapeutics is a developer of a biotechnology platform intended to create gene therapies powered by synthetic biology.The company's offerings include immunotherapies to help cells produce target cancer-killing proteins during immunotherapy, enabling patients to improve their body's immune system to fight cancer.

Quantum-Si is focused on revolutionizing the growing field of proteomics. The company’s suite of technologies are powered by a first-of-its-kind semiconductor chip designed to enable single-molecule next-generation protein sequencing and genomics, and digitize proteomic research in order to advance drug discovery and diagnostics beyond what has been possible with DNA sequencing.

Clear Labs, Inc. is a food analytics company based in Menlo Park, California, founded in 2013. The company specializes in providing a comprehensive analytics platform that analyzes the molecular contents of food and ingredients. Its offerings include Clear Dx™ and Clear Safety™, which utilize next-generation sequencing and data analytics for food safety testing and infectious disease surveillance. Clear Labs serves food manufacturers, suppliers, and retailers by ensuring transparency throughout the supply chain and testing products for various factors such as authenticity, contamination, allergens, and nutritional content. The platform delivers actionable insights that enhance safety and operational efficiency while supporting public health initiatives. Through its integrated genomics system, Clear Labs aims to simplify complex diagnostics across clinical and applied markets, making genomic insights accessible to labs, hospitals, and businesses.

Excision BioTherapeutics Inc. is a life science company based in Philadelphia, Pennsylvania, established in 2015. The company specializes in developing gene editing therapeutics aimed at treating life-threatening diseases caused by neurotropic viruses. Utilizing CRISPR-based technology, Excision BioTherapeutics focuses on creating advanced gene-editing medicines that can eradicate or disrupt viral genes in human patients. The company is committed to advancing these therapeutics to ensure they are safe and effective, with the goal of significantly improving the lives of individuals affected by viral infections worldwide.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing precision genetic medicines using its innovative base editing technology. Founded in 2017, the company aims to provide lifelong cures for patients with serious diseases. Its therapeutic portfolio includes treatments for sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, pediatric acute myeloid leukemia, alpha-1 antitrypsin deficiency, and glycogen storage disorder 1A, as well as therapies for ocular and central nervous system disorders. Beam's base editing technology allows for targeted modifications at a single base in the genome without creating double-stranded breaks in DNA, positioning it as a pioneer in the field of genetic medicine. The company's pipeline includes several programs, such as BEAM-101, ESCAPE, BEAM-302, BEAM-301, and BEAM-201, reflecting its commitment to advancing genetic therapies.

Neuron23, Inc. is a biotechnology company focused on developing therapies for genetic disorders, particularly neurological diseases such as Parkinson's and Alzheimer's. Founded in 2018 and headquartered in South San Francisco, California, with an additional office in Munich, Germany, the company utilizes advanced artificial intelligence and genetic insights to enhance drug discovery. Neuron23's innovative platform integrates the latest advancements in human genetics with AI-driven methodologies to create targeted treatments for patients facing debilitating health challenges. By focusing on genetically defined conditions, Neuron23 aims to improve therapeutic options and outcomes for individuals affected by these disorders.

Clear Labs, Inc. is a food analytics company based in Menlo Park, California, founded in 2013. The company specializes in providing a comprehensive analytics platform that analyzes the molecular contents of food and ingredients. Its offerings include Clear Dx™ and Clear Safety™, which utilize next-generation sequencing and data analytics for food safety testing and infectious disease surveillance. Clear Labs serves food manufacturers, suppliers, and retailers by ensuring transparency throughout the supply chain and testing products for various factors such as authenticity, contamination, allergens, and nutritional content. The platform delivers actionable insights that enhance safety and operational efficiency while supporting public health initiatives. Through its integrated genomics system, Clear Labs aims to simplify complex diagnostics across clinical and applied markets, making genomic insights accessible to labs, hospitals, and businesses.

ArcherDX, Inc. is a genomics company based in Boulder, Colorado, specializing in the development and commercialization of advanced molecular pathology products and services. The company focuses on next-generation sequencing technologies to enhance genetic mutation detection, offering a range of research products such as VariantPlex, FusionPlex, LiquidPlex, and Immunoverse. ArcherDX's proprietary Anchored Multiplexed PCR (AMP™) chemistry, combined with powerful bioinformatics software, allows for efficient identification of genetic mutations, including gene fusions and point mutations. The company also provides customization services for clinical and biopharmaceutical customers through its assay designer platform, enabling the development of personalized applications. Its products are primarily aimed at academic and reference laboratories, biopharmaceutical firms, and contract research organizations, supporting therapy optimization and cancer monitoring. Founded in 2013, ArcherDX operates as a subsidiary of Invitae Corporation.

Repare Therapeutics Inc. is a precision oncology company based in Montréal, Canada, specializing in the discovery and development of novel therapeutics through a synthetic lethality approach. Utilizing its proprietary SNIPRx platform, which combines genome-wide and CRISPR-enabled technology, the company systematically identifies targeted cancer therapies addressing genomic instability, particularly in DNA damage repair. Its lead candidate, RP-3500, is an oral small molecule inhibitor designed to treat solid tumors with specific DNA damage repair-related genomic alterations. Additionally, Repare is advancing a CCNE1-SL inhibitor program targeting tumors with CCNE1 amplification, along with a Polymerase Theta program focused on BRCA mutations and related genomic alterations. Founded in 2016, the company aims to improve cancer treatment by leveraging innovative drug discovery methods and addressing the unique vulnerabilities of tumor cells.

Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing precision genetic medicines using its innovative base editing technology. Founded in 2017, the company aims to provide lifelong cures for patients with serious diseases. Its therapeutic portfolio includes treatments for sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, pediatric acute myeloid leukemia, alpha-1 antitrypsin deficiency, and glycogen storage disorder 1A, as well as therapies for ocular and central nervous system disorders. Beam's base editing technology allows for targeted modifications at a single base in the genome without creating double-stranded breaks in DNA, positioning it as a pioneer in the field of genetic medicine. The company's pipeline includes several programs, such as BEAM-101, ESCAPE, BEAM-302, BEAM-301, and BEAM-201, reflecting its commitment to advancing genetic therapies.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company headquartered in Bedford, Massachusetts, focused on developing novel antisense oligonucleotide medicines to address the underlying causes of severe genetic diseases. The company employs its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) technology to enhance gene expression by targeting RNA splicing, thereby selectively restoring protein levels. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a serious and progressive genetic epilepsy. Established in 2014, Stoke Therapeutics also collaborates with Invitae Corporation to provide genetic testing services, further supporting its mission to advance gene-targeted therapies for patients with severe genetic conditions.

NightstaRx Ltd. is a U.K. startup company. Gene therapy is the use of a gene to treat disease. The approach could enable treatments for a several rare conditions caused by mutations in a single gene.

Yumanity Therapeutics is transforming drug discovery for neurodegenerative diseases caused by protein misfolding. Founded in December 2014 by award-winning protein folding expert, Susan Lindquist, and renowned biotech industry leader, Tony Coles, Yumanity is working to identify and develop new, disease-modifying therapies that address several illnesses with critical unmet medical needs. The initial focus of the company is neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis (ALS). The company’s proprietary platforms have already identified one potential new target for treating Parkinson’s disease, and Yumanity is actively advancing its new chemical lead series for this condition, as well as identifying additional compounds for Alzheimer’s disease and ALS.

Audentes Therapeutics, a clinical-stage biotechnology company based in San Francisco, is dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapies, including AT132 and AT342, which are in Phase I/II clinical studies for treating X-linked myotubular myopathy and Crigler-Najjar syndrome, respectively. Additionally, it is exploring treatments for Pompe disease and catecholaminergic polymorphic ventricular tachycardia, along with vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Audentes has established collaborative agreements for research and development with Genethon and the University of Pennsylvania. Founded in 2012, Audentes Therapeutics became a subsidiary of Astellas US Holding in 2020 and operates as Astellas Gene Therapies, focusing on leveraging its expertise in gene therapy research and commercialization.

Wave Life Sciences Ltd. is a clinical-stage genetic medicine company focused on developing innovative RNA-based therapies. Utilizing its proprietary PRISM platform, the company designs, optimizes, and produces stereopure oligonucleotides aimed at addressing genetic defects. By targeting ribonucleic acid (RNA), Wave aims to either diminish the production of harmful proteins or convert dysfunctional proteins into functional ones. The company's primary focus is on neurology, particularly disorders affecting the central and neuromuscular systems. Wave Life Sciences has established research and collaboration agreements with major pharmaceutical companies, including Pfizer and Takeda, to further advance its therapeutic programs. Founded in 2012 and headquartered in Singapore, Wave Life Sciences is dedicated to delivering transformative treatments for patients with genetically defined diseases.

Ovid is a pure play neurology company focused on rare and orphan diseases of the brain. They were founded in 2014 to leverage recent advances in science and medicine to find optimal therapies. They seek to identify and develop late stage, de-risked medicines with significant potential. Their rationale for developing these medicines is based on key insights from their scientific founders and confirmed in discussions with leading experts in the field. Their products are protected by strong intellectual property. They apply the most efficient processes to bring safe and effective therapies to patients. They are based in New York City but have collaborators and are working with patients across the United States and the globe.

Syros Pharmaceuticals is a life sciences company that is focused on treating disease by mapping gene regulatory circuits and modulating the factors that regulate gene expression. Syros has pioneered world-leading gene control research and drug discovery capabilities with a proven ability to integrate disease biology and genomic data--a proficiency that is not well represented in pharmaceutical R&D. Central to the Syros approach is a proprietary platform of carefully integrated assay technologies, bioinformatics, and biologic insights developed by members of Syros' senior leadership. While this scientific approach has applications in many therapeutic areas, Syros has demonstrated success initially in oncology, where it may help address numerous unmet medical needs.

Invitae is a genetic information company focused on integrating genetic insights into everyday medical practice to enhance healthcare quality for a large population. The company specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates multiple genetic tests into a single platform. Invitae's offerings include assessments for genes linked to hereditary cancer, neurological disorders, cardiovascular conditions, pediatric issues, metabolic disorders, and other hereditary ailments. By providing high-quality testing with quicker turnaround times and competitive pricing, Invitae aims to make genetic testing more accessible and effective for patients and healthcare providers alike.

Invitae is a genetic information company focused on integrating genetic insights into everyday medical practice to enhance healthcare quality for a large population. The company specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates multiple genetic tests into a single platform. Invitae's offerings include assessments for genes linked to hereditary cancer, neurological disorders, cardiovascular conditions, pediatric issues, metabolic disorders, and other hereditary ailments. By providing high-quality testing with quicker turnaround times and competitive pricing, Invitae aims to make genetic testing more accessible and effective for patients and healthcare providers alike.

PacBio designs, develops, and manufactures sequencing systems to resolve genetically complex problems. Its single molecule real-time (SMRT) sequencing technology enables single molecule real-time detection of biological processes.