Redmile Group

Clear Labs is a biotechnology company specializing in next-generation sequencing and data analytics for infectious disease surveillance and food safety testing. Its platforms, Clear Dx™ and Clear Safety™, enable rapid identification of pathogens, supporting public health initiatives and enhancing safety across various sectors.

Neurogene is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases. It leverages its proprietary transgene regulation platform, EXACT, to create gene therapies with the aim of addressing high-need conditions. The company’s pipeline includes NGN-401 for Rett syndrome and NGN-101 for CLN5 Batten disease. Founded in 2018, Neurogene is headquartered in New York and concentrates on advancing therapies that can improve outcomes for patients with limited treatment options.

GRO Biosciences employs computational protein design and synthetic biology to enhance protein therapeutics. It has developed multiple genomically recoded bacterial strains capable of incorporating non-standard amino acids into proteins, resulting in improved potency, stability, and targeted delivery. The company focuses on developing advanced treatments for diabetes, growth disorders, and autoimmunity.

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 specializes in the development of RNA editing therapeutics. Founded in 2019, the company focuses on discovering and developing innovative treatments that utilize endogenous enzymes to precisely target and correct single point mutations in mRNA transcripts, restoring the production of functional proteins.

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.

Founded in 2018, Neuron23 is a biotechnology company based in South San Francisco, California, with an additional office in Munich, Germany. It specializes in developing and manufacturing medicines for genetic disorders using artificial intelligence technology and genetics.

GRO Biosciences employs computational protein design and synthetic biology to enhance protein therapeutics. It has developed multiple genomically recoded bacterial strains capable of incorporating non-standard amino acids into proteins, resulting in improved potency, stability, and targeted delivery. The company focuses on developing advanced treatments for diabetes, growth disorders, and autoimmunity.

Mammoth Biosciences harnesses diverse CRISPR systems for various applications. It offers DETECTR™ for nucleic acid detection in diseases like cancer and infections, and CRISPR-Cas systems for therapeutic genome editing in immuno-oncology, autoimmune diseases, and liver disorders. Its solutions span healthcare, agriculture, environmental monitoring, biodefense, and other sectors.

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 is a biotechnology company specializing in next-generation sequencing and data analytics for infectious disease surveillance and food safety testing. Its platforms, Clear Dx™ and Clear Safety™, enable rapid identification of pathogens, supporting public health initiatives and enhancing safety across various sectors.

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 biotechnology company developing genetic medicines for cardiovascular disease, aiming to transform treatment from chronic management to single-course gene editing therapies. It leverages human genetics analysis and gene-editing technology, with expertise in cardiovascular medicine, human genetics, gene editing, delivery technologies, drug development, and commercialization.

Beam Therapeutics is a biotechnology company developing precision genetic medicines using its proprietary base editing technology. Its focus includes therapies for hematological and genetic diseases, with ongoing programs targeting sickle cell disease, alpha-1 antitrypsin deficiency, and other serious conditions.

Neurogene is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases. It leverages its proprietary transgene regulation platform, EXACT, to create gene therapies with the aim of addressing high-need conditions. The company’s pipeline includes NGN-401 for Rett syndrome and NGN-101 for CLN5 Batten disease. Founded in 2018, Neurogene is headquartered in New York and concentrates on advancing therapies that can improve outcomes for patients with limited treatment options.

Founded in 2018, Neuron23 is a biotechnology company based in South San Francisco, California, with an additional office in Munich, Germany. It specializes in developing and manufacturing medicines for genetic disorders using artificial intelligence technology and genetics.

Mammoth Biosciences harnesses diverse CRISPR systems for various applications. It offers DETECTR™ for nucleic acid detection in diseases like cancer and infections, and CRISPR-Cas systems for therapeutic genome editing in immuno-oncology, autoimmune diseases, and liver disorders. Its solutions span healthcare, agriculture, environmental monitoring, biodefense, and other sectors.

Clear Labs is a biotechnology company specializing in next-generation sequencing and data analytics for infectious disease surveillance and food safety testing. Its platforms, Clear Dx™ and Clear Safety™, enable rapid identification of pathogens, supporting public health initiatives and enhancing safety across various sectors.

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 is a precision oncology company that discovers and develops novel therapeutics using its synthetic lethality approach. It employs its proprietary SNIPRx platform to systematically identify and target genomic instability, particularly DNA damage repair pathways in cancer cells. The company's lead product candidate, RP-3500, is an oral small molecule inhibitor for treating solid tumors with specific DNA damage repair-related alterations.

Beam Therapeutics is a biotechnology company developing precision genetic medicines using its proprietary base editing technology. Its focus includes therapies for hematological and genetic diseases, with ongoing programs targeting sickle cell disease, alpha-1 antitrypsin deficiency, and other serious conditions.

Neurogene is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases. It leverages its proprietary transgene regulation platform, EXACT, to create gene therapies with the aim of addressing high-need conditions. The company’s pipeline includes NGN-401 for Rett syndrome and NGN-101 for CLN5 Batten disease. Founded in 2018, Neurogene is headquartered in New York and concentrates on advancing therapies that can improve outcomes for patients with limited treatment options.

Stoke Therapeutics is a biopharmaceutical company developing novel antisense oligonucleotide medicines. These target RNA splicing to increase gene expression, aiming to treat severe genetic diseases. Its lead product candidate, STK-001, focuses on Dravet syndrome, a progressive genetic epilepsy.

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 is a clinical-stage biotechnology company focused on RNA medicines, designing, optimizing, and producing stereopure oligonucleotides through its PRISM platform. The company aims to address genetically defined diseases by reducing harmful protein expression or correcting dysfunctional proteins, employing modalities such as RNA editing, antisense silencing, and RNA interference. Its programs span neurological and neuromuscular conditions, reflecting an emphasis on central nervous system targets. Wave collaborates with Pfizer and Takeda on research, development, and commercialization of stereopure oligonucleotide therapeutics and antisense therapies. Founded in 2012 and based in Singapore, the company seeks to unlock the broader potential of RNA medicines to improve patient outcomes.

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.