Redmile Group

Clear Labs, Inc. is a food analytics company based in Menlo Park, California, founded in 2013. The company specializes in providing a comprehensive food analytics platform that utilizes next-generation sequencing and data analytics to enhance food safety and quality control. Its offerings include Clear Dx™ and Clear Safety™, which facilitate the rapid identification of pathogens and support public health initiatives. Clear Labs' platform delivers critical insights into the molecular composition of foods and ingredients, testing for authenticity, contaminants, allergens, and other safety parameters. By offering transparency across supply chains, the company serves food manufacturers, suppliers, and retailers, enabling them to improve operational efficiency and safety through detailed analytics and reporting.

Neurogene Inc., established in 2018 and headquartered in New York, specializes in developing genetic medicines for treating neurological disorders. The company focuses on utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for conditions such as Charcot-Marie Tooth disease type 4J (CMT4J), Aspartylglucosaminuria (AGU), and an undisclosed Lysosomal Storage Disease (LSD). Neurogene aims to provide effective treatment options where none currently exist, aiming to improve the quality of life for patients and families affected by these rare neurological disorders.

GRO Biosciences is a biotechnology company focused on developing advanced protein therapeutics to address various health conditions, including diabetes, growth disorders, and autoimmunity. The company employs innovative technologies in computational protein design and synthetic biology, which enable it to create genomically recoded strains of bacteria capable of incorporating non-standard amino acids into proteins. This approach enhances the properties of the proteins, resulting in increased potency, stability, and targeted delivery. Through its technology platform, GRO Biosciences aims to provide improved treatment options across multiple disease classes, contributing to advancements in medical therapeutics.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Established in 2019, the company specializes in genetic medicine by utilizing a proprietary platform that employs endogenous enzymes, specifically adenosine deaminase acting on RNA (ADAR), to precisely target and correct single point mutations in RNA transcripts. This innovative approach aims to restore the production of functional proteins, offering potential treatments for a variety of diseases, including those affecting genetic, cardiometabolic, complement-mediated, and central nervous system areas. ADARx Pharmaceuticals is dedicated to developing life-saving therapeutics that address genetic mutations and provide solutions for conditions that are currently considered incurable.

Krystal Biotech, Inc. is a biopharmaceutical company based in Pittsburgh, Pennsylvania, specializing in gene therapy for patients with rare skin diseases. Founded in 2015, the company develops innovative treatments using its proprietary STAR-D gene therapy platform, which targets dermatological conditions caused by genetic mutations. Its lead candidate, beremagene geperpavec (B-VEC), is in clinical development for dystrophic epidermolysis bullosa, having completed Phase I/II studies. Krystal Biotech is also advancing other product candidates, including KB105, which is in Phase I/II trials for autosomal recessive congenital ichthyosis, and KB301 and KB104, which are in preclinical stages for aesthetic skin conditions and Netherton Syndrome, respectively. Additionally, the company is exploring treatments for chronic skin diseases through its discovery-stage candidate KB5xx. Krystal Biotech aims to enhance the quality of life for patients suffering from debilitating skin disorders with its gene therapy solutions.

Neuron23, Inc. is a biotechnology company focused on developing and manufacturing innovative medicines for genetic disorders, particularly targeting neurological and immunological diseases. Founded in 2018 and headquartered in South San Francisco, California, with an additional office in Munich, Germany, the company utilizes advanced artificial intelligence technology and cutting-edge genetic research to enhance drug discovery. Neuron23 aims to create effective treatments for conditions such as Parkinson's disease and Alzheimer's disease, leveraging its AI-enabled platforms to identify and develop therapeutics that address the needs of patients suffering from these debilitating disorders.

GRO Biosciences is a biotechnology company focused on developing advanced protein therapeutics to address various health conditions, including diabetes, growth disorders, and autoimmunity. The company employs innovative technologies in computational protein design and synthetic biology, which enable it to create genomically recoded strains of bacteria capable of incorporating non-standard amino acids into proteins. This approach enhances the properties of the proteins, resulting in increased potency, stability, and targeted delivery. Through its technology platform, GRO Biosciences aims to provide improved treatment options across multiple disease classes, contributing to advancements in medical therapeutics.

Mammoth Biosciences, Inc. is a biotechnology company based in South San Francisco, California, founded in 2017 by CRISPR pioneer Jennifer Doudna and other key innovators. The company specializes in developing advanced CRISPR technologies, including its proprietary DETECTR platform, which enables the rapid detection of specific nucleic acids associated with various diseases. This platform facilitates diagnostic applications such as cancer screening, bacterial infections, and viral infections, allowing for real-time and simultaneous testing of multiple conditions. In addition to diagnostics, Mammoth Biosciences works on genome editing solutions for therapeutic purposes, targeting areas such as immuno-oncology, autoimmune diseases, and liver disorders. The company aims to democratize disease detection and enhance life science research through innovative, affordable, and efficient tools, while also leveraging its exclusive licensing of various Cas proteins for improved diagnostics and therapeutic applications.

Prime Medicine is a biotechnology company focused on developing genetic therapies using its innovative Prime Editing technology. This next-generation gene editing method allows for precise "search and replace" modifications in the genome, aimed at restoring normal genetic function. The company targets a wide range of diseases with significant unmet medical needs, leveraging the efficiency and versatility of its gene editing capabilities to address the underlying causes of these conditions. Through its commitment to advancing gene editing, Prime Medicine seeks to transform the landscape of genetic medicine.

Strand Therapeutics Inc. is a biotechnology company based in Boston, Massachusetts, that specializes in genetically programming mRNA to deliver innovative therapies aimed at enhancing patient outcomes. Founded in 2017, the company develops mRNA programming technology that allows for precise control over the location, timing, and intensity of therapeutic protein expression within the body. By employing mRNA-encoded logic circuits, Strand Therapeutics can achieve cell-type specific expression by detecting unique miRNA signatures and modulating protein dosage in response to externally administered small molecules. The company focuses on creating gene therapies powered by synthetic biology, including immunotherapies designed to enable cells to produce target cancer-killing proteins, thereby strengthening the body's immune response against 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 food analytics platform that utilizes next-generation sequencing and data analytics to enhance food safety and quality control. Its offerings include Clear Dx™ and Clear Safety™, which facilitate the rapid identification of pathogens and support public health initiatives. Clear Labs' platform delivers critical insights into the molecular composition of foods and ingredients, testing for authenticity, contaminants, allergens, and other safety parameters. By offering transparency across supply chains, the company serves food manufacturers, suppliers, and retailers, enabling them to improve operational efficiency and safety through detailed analytics and reporting.

Excision BioTherapeutics Inc. is a life science company based in Philadelphia, Pennsylvania, established in 2015. The company is dedicated to the development and commercialization of 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's mission is to advance these therapeutics into safe and effective treatments, significantly improving the lives of individuals affected by viral infections globally.

Verve Therapeutics is a genetic medicines company based in Cambridge, Massachusetts, focused on innovative therapies for cardiovascular disease. Founded in 2018, the company aims to transform the management of cardiovascular conditions from chronic treatments to single-course gene editing medicines. Verve Therapeutics employs advanced gene-editing technology and human genetic analysis to develop targeted therapies that reduce the risk of coronary artery diseases. Its initial programs focus on the PCSK9 and ANGPTL3 genes, which are crucial for lowering harmful blood lipids like low-density lipoprotein cholesterol. The company collaborates with strategic partners, including Beam Therapeutics for delivery technologies and Verily for gene editing delivery vehicles, enhancing its capabilities in the biotechnology and healthcare sectors.

Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, specializing in the development of precision genetic medicines utilizing its innovative base editing technology. Founded in 2017, the company aims to provide lifelong cures for patients suffering from serious diseases by targeting specific bases in the genome without inducing double-stranded breaks in DNA. Beam Therapeutics is actively developing therapies for a range of conditions, including sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, pediatric acute myeloid leukemia, alpha-1 antitrypsin deficiency, glycogen storage disorder type 1A, as well as ocular and central nervous system disorders. The company’s diverse pipeline includes several programs such as BEAM-101, BEAM-201, BEAM-301, BEAM-302, and ESCAPE, reflecting its commitment to advancing gene correction, gene modification, gene activation, gene silencing, and multiplex editing technologies.

Neurogene Inc., established in 2018 and headquartered in New York, specializes in developing genetic medicines for treating neurological disorders. The company focuses on utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for conditions such as Charcot-Marie Tooth disease type 4J (CMT4J), Aspartylglucosaminuria (AGU), and an undisclosed Lysosomal Storage Disease (LSD). Neurogene aims to provide effective treatment options where none currently exist, aiming to improve the quality of life for patients and families affected by these rare neurological disorders.

Neuron23, Inc. is a biotechnology company focused on developing and manufacturing innovative medicines for genetic disorders, particularly targeting neurological and immunological diseases. Founded in 2018 and headquartered in South San Francisco, California, with an additional office in Munich, Germany, the company utilizes advanced artificial intelligence technology and cutting-edge genetic research to enhance drug discovery. Neuron23 aims to create effective treatments for conditions such as Parkinson's disease and Alzheimer's disease, leveraging its AI-enabled platforms to identify and develop therapeutics that address the needs of patients suffering from these debilitating disorders.

Mammoth Biosciences, Inc. is a biotechnology company based in South San Francisco, California, founded in 2017 by CRISPR pioneer Jennifer Doudna and other key innovators. The company specializes in developing advanced CRISPR technologies, including its proprietary DETECTR platform, which enables the rapid detection of specific nucleic acids associated with various diseases. This platform facilitates diagnostic applications such as cancer screening, bacterial infections, and viral infections, allowing for real-time and simultaneous testing of multiple conditions. In addition to diagnostics, Mammoth Biosciences works on genome editing solutions for therapeutic purposes, targeting areas such as immuno-oncology, autoimmune diseases, and liver disorders. The company aims to democratize disease detection and enhance life science research through innovative, affordable, and efficient tools, while also leveraging its exclusive licensing of various Cas proteins for improved diagnostics and therapeutic applications.

Clear Labs, Inc. is a food analytics company based in Menlo Park, California, founded in 2013. The company specializes in providing a comprehensive food analytics platform that utilizes next-generation sequencing and data analytics to enhance food safety and quality control. Its offerings include Clear Dx™ and Clear Safety™, which facilitate the rapid identification of pathogens and support public health initiatives. Clear Labs' platform delivers critical insights into the molecular composition of foods and ingredients, testing for authenticity, contaminants, allergens, and other safety parameters. By offering transparency across supply chains, the company serves food manufacturers, suppliers, and retailers, enabling them to improve operational efficiency and safety through detailed analytics and reporting.

ArcherDX, Inc. is a genomics company headquartered in Boulder, Colorado, specializing in the development and commercialization of advanced genetic analysis products. Established in 2013, the company focuses on next-generation sequencing technologies to enhance genetic mutation detection, particularly in oncology. ArcherDX offers a range of research-use-only products, including DNA-based VariantPlex, RNA-based FusionPlex, and ctDNA-based LiquidPlex assays. These products enable laboratories to conduct genomic analyses for therapy optimization and cancer monitoring. Additionally, ArcherDX provides customized assay design services for clinical and biopharmaceutical clients, allowing them to target specific biomarkers and develop new applications. The company aims to deliver actionable genomic information for the treatment of solid tumors, blood cancers, and sarcomas, catering to academic laboratories, biopharmaceutical firms, and contract research organizations. ArcherDX also seeks regulatory approval for various companion diagnostic assays to further support precision medicine initiatives.

Repare Therapeutics Inc. is a precision oncology company based in Montréal, Canada, that specializes in discovering and developing targeted therapeutics through a synthetic lethality approach. Utilizing its proprietary SNIPRx platform, which integrates genome-wide analysis and CRISPR technology, Repare systematically identifies and advances cancer therapies aimed at genomic instability, particularly in DNA damage repair. The company's lead candidate, RP-3500, is an oral small molecule inhibitor designed for solid tumors with specific genomic alterations related to DNA damage repair. In addition, Repare is developing innovative programs such as a CCNE1-SL inhibitor for tumors with CCNE1 amplification and a Polymerase Theta program targeting BRCA mutations and other genomic changes. Founded in 2016, Repare Therapeutics is focused on addressing the unique vulnerabilities of tumor cells in genetically defined patient populations to enhance cancer treatment outcomes.

Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, specializing in the development of precision genetic medicines utilizing its innovative base editing technology. Founded in 2017, the company aims to provide lifelong cures for patients suffering from serious diseases by targeting specific bases in the genome without inducing double-stranded breaks in DNA. Beam Therapeutics is actively developing therapies for a range of conditions, including sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, pediatric acute myeloid leukemia, alpha-1 antitrypsin deficiency, glycogen storage disorder type 1A, as well as ocular and central nervous system disorders. The company’s diverse pipeline includes several programs such as BEAM-101, BEAM-201, BEAM-301, BEAM-302, and ESCAPE, reflecting its commitment to advancing gene correction, gene modification, gene activation, gene silencing, and multiplex editing technologies.

Neurogene Inc., established in 2018 and headquartered in New York, specializes in developing genetic medicines for treating neurological disorders. The company focuses on utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for conditions such as Charcot-Marie Tooth disease type 4J (CMT4J), Aspartylglucosaminuria (AGU), and an undisclosed Lysosomal Storage Disease (LSD). Neurogene aims to provide effective treatment options where none currently exist, aiming to improve the quality of life for patients and families affected by these rare neurological disorders.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company focused on developing novel antisense oligonucleotide medicines aimed at addressing the underlying causes of severe genetic diseases. Founded in 2014 and headquartered in Bedford, Massachusetts, the company utilizes its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach to enhance gene expression through RNA splicing. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke Therapeutics has also partnered with Invitae Corporation to offer complementary genetic testing services.

Nightstar Therapeutics is a clinical-stage gene therapy company based in London, United Kingdom, specializing in the development of innovative one-time treatments for rare inherited retinal diseases. The company is advancing several product candidates, including its lead candidate NSR-REP1, which is in phase 3 clinical development for choroideremia. Additionally, Nightstar is working on NSR-RPGR, currently in phase 2/3 trials for X-linked retinitis pigmentosa, as well as NSR-BEST1. The company is also developing NSR-ABCA4, which is in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address significant unmet medical needs in the field of retinal disorders.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

WaVe Life Sciences Ltd. is a clinical-stage genetic medicine company based in Singapore, focused on developing innovative treatments for genetically defined diseases using its proprietary PRISM platform. This platform enables the precise design, optimization, and production of stereopure oligonucleotides, which are designed to target genetic defects by either reducing harmful protein expression or converting dysfunctional proteins into functional ones. The company's primary focus is on neurology, particularly within the central and neuromuscular systems. WaVe Life Sciences collaborates with major pharmaceutical companies, including Pfizer and Takeda, to advance the research and development of its oligonucleotide therapeutics. Founded in 2012, the company aims to deliver transformative therapies to improve patient outcomes in both rare and prevalent disorders.

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.

Pacific Biosciences specializes in human, microbial, plant, and animal genomics, focusing on areas such as infectious disease research. The company designs and manufactures sequencing systems that address complex genetic challenges, utilizing its innovative single molecule real-time (SMRT) sequencing technology to detect biological processes in real time. Its product offerings include the PacBio RS II and Sequel Systems, which facilitate the monitoring and analysis of biochemical reactions at the single-molecule level, along with consumables like SMRT cells and various reagent kits. Customers range from research institutions and commercial laboratories to pharmaceutical and agricultural companies, with sales conducted through direct channels in North America and Europe, and distribution partners in Asia, the Middle East, and Latin America. Founded in 2000 and headquartered in Menlo Park, California, Pacific Biosciences continues to advance genomics research and applications.