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 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.

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 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 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. 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 based in Pittsburgh, Pennsylvania, focused on developing gene therapies for patients with rare skin diseases in the United States. Its primary candidate, beremagene geperpavec (B-VEC), is designed to treat dystrophic epidermolysis bullosa and has completed Phase I/II clinical trials. Additionally, the company is advancing KB105, which is in Phase I/II trials for autosomal recessive congenital ichthyosis, while KB301 and KB104 are in preclinical stages targeting aesthetic skin conditions and Netherton Syndrome, respectively. Krystal Biotech utilizes its proprietary STAR-D gene therapy platform to create innovative treatments that offer significant clinical benefits. Founded in 2015, the company aims to broaden its pipeline to address various dermatological conditions in the future.

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.

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 Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2017. The company focuses on genetically programming mRNA to develop innovative therapies that enhance patient outcomes. Utilizing its mRNA programming technology, Strand Therapeutics creates gene therapies powered by synthetic biology, which include immunotherapies designed to stimulate cells to produce cancer-targeting proteins. This approach improves the immune system's ability to combat cancer by enabling precise control over the timing, location, and intensity of therapeutic protein expression. The technology leverages cell-type specific expression and responds to specific molecular signals, allowing for tailored therapeutic interventions.

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.

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.

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.

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.

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.

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.

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 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, 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.

BioNTech is a Germany-based biotechnology company that specializes in developing innovative immunotherapies for cancer and other serious diseases, as well as vaccines for infectious diseases, including COVID-19. The company’s oncology pipeline includes a range of drug classes, such as mRNA-based drugs that encode antigens and neoantigens, cell therapies, bispecific antibodies, and antibody-drug conjugates. BioNTech has established partnerships with several major pharmaceutical companies, enhancing its research and development capabilities. Its COVID-19 vaccine, Comirnaty, represents its first commercialized product, demonstrating the company's commitment to addressing urgent health challenges through advanced therapeutic approaches.

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 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.

BioNTech is a Germany-based biotechnology company that specializes in developing innovative immunotherapies for cancer and other serious diseases, as well as vaccines for infectious diseases, including COVID-19. The company’s oncology pipeline includes a range of drug classes, such as mRNA-based drugs that encode antigens and neoantigens, cell therapies, bispecific antibodies, and antibody-drug conjugates. BioNTech has established partnerships with several major pharmaceutical companies, enhancing its research and development capabilities. Its COVID-19 vaccine, Comirnaty, represents its first commercialized product, demonstrating the company's commitment to addressing urgent health challenges through advanced therapeutic approaches.

Nightstar Therapeutics plc is a clinical-stage gene therapy company based in London, focused on developing innovative one-time treatments for rare inherited retinal diseases. The company’s lead product candidate, NSR-REP1, is currently in phase 3 clinical development for choroideremia, while NSR-RPGR is undergoing phase 2/3 trials for X-linked retinitis pigmentosa. Additionally, Nightstar is developing NSR-BEST1 and NSR-ABCA4, with the latter in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address the unmet medical needs of patients suffering from these conditions.

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.

Syros Pharmaceuticals, Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead product candidates, including SY-1425, a selective retinoic acid receptor alpha agonist currently in Phase II clinical trials for specific patient populations with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor under investigation in a Phase I clinical trial for advanced solid tumors. Syros is also engaged in collaborative research, including a partnership with Incyte Corporation aimed at identifying therapeutic targets for myeloproliferative neoplasms and a licensing agreement with TMRC Co. Ltd. for the development of tamibarotene. With a focus on mapping gene regulatory circuits, Syros leverages its proprietary platform and expertise to address significant unmet medical needs in oncology. Founded in 2011, the company aims to establish new standards of care for patients with hematologic malignancies.

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 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 Therapeutics Inc. is a biopharmaceutical company based in New York, focused on developing innovative therapies for patients with neurological disorders, particularly rare diseases. Founded in 2014, the company is advancing a promising pipeline of drug candidates aimed at treating conditions such as angelman syndrome and fragile X syndrome. Its lead candidate, OV101, is in Phase III clinical trials for angelman syndrome and has completed Phase II trials for fragile X syndrome. Additionally, Ovid is developing OV935, currently in Phase II trials for cyclin-dependent kinase-like 5 deficiency disorder and Dravet syndrome. The company is also researching OV329 for infantile spasms and OV881 as a microRNA gene therapy for angelman syndrome. Ovid Therapeutics has established collaborations and licensing agreements with various pharmaceutical and research organizations to enhance its development efforts. The company's commitment to addressing unmet medical needs is underpinned by strong intellectual property and a focus on safe, effective treatments for patients facing challenging neurological conditions.

Syros Pharmaceuticals, Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead product candidates, including SY-1425, a selective retinoic acid receptor alpha agonist currently in Phase II clinical trials for specific patient populations with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor under investigation in a Phase I clinical trial for advanced solid tumors. Syros is also engaged in collaborative research, including a partnership with Incyte Corporation aimed at identifying therapeutic targets for myeloproliferative neoplasms and a licensing agreement with TMRC Co. Ltd. for the development of tamibarotene. With a focus on mapping gene regulatory circuits, Syros leverages its proprietary platform and expertise to address significant unmet medical needs in oncology. Founded in 2011, the company aims to establish new standards of care for patients with hematologic malignancies.

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.

Foundation Medicine, Inc. is a biotechnology company based in Cambridge, Massachusetts, that specializes in providing molecular information products for cancer diagnosis and treatment. The company utilizes advanced genomic profiling technologies to analyze tumor specimens, enabling healthcare providers to gain insights into individual patients' cancer profiles. This information assists in optimizing treatment strategies, particularly for targeted therapies and immunotherapies. Foundation Medicine’s product offerings include FoundationOne for solid tumors, FoundationOne Heme for blood-based cancers, and various diagnostic assays to support clinical decision-making in oncology. The company also maintains a knowledgebase, FoundationCORE, to disseminate scientific advancements and strengthen collaborations within the oncology community. Foundation Medicine has established strategic partnerships with major pharmaceutical companies and research organizations to enhance precision medicine initiatives and develop companion diagnostics for specific therapies. Founded in 2009, Foundation Medicine operates as a subsidiary of Roche Holdings, Inc.

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.