Invus Financial Advisors

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Coave Therapeutics specializes in developing gene therapies aimed at treating rare ocular and central nervous system diseases. The company focuses on innovative solutions for rare retinal disorders, utilizing a recombinant adeno-associated virus that substitutes the viral DNA with a functional copy of the PDE6B gene. This therapeutic approach involves injecting the corrected version of the protein between the photoreceptors and the retinal pigment epithelium, providing patients with effective treatment options for various eye conditions.

Noema Pharma AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company focuses on developing therapeutic products and therapies for orphan neurological disorders characterized by imbalanced neuronal networks. Its research and development efforts aim to address disabling symptoms in conditions affecting the nervous system, including Tuberous Sclerosis Complex, Trigeminal Neuralgia, and Tourette syndrome. By creating novel therapies, Noema Pharma seeks to improve the quality of life and patient outcomes for individuals suffering from these rare conditions.

City Therapeutics is a biopharmaceutical company specializing in the development of next-generation small-interfering RNAs (siRNAs) to enhance RNA interference (RNAi) therapies. The company's pipeline comprises innovative RNAi therapeutics targeting diverse disease indications. City Therapeutics aims to improve and expand RNAi technology to achieve better patient outcomes, led by a team of experienced scientists and industry professionals committed to advancing the field of RNA interference.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

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.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

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.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Ring Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing gene therapies through its innovative Anellovector platform. This platform leverages the biology of the human commensal virome to create redosable and targetable DNA therapies that address several limitations of existing gene therapies, such as restricted access to various tissues, risks associated with genomic integration, and issues with tolerability. Founded in 2017, the company aims to expand the applications of gene therapy beyond traditional gene replacement, targeting a wide range of conditions including genetic disorders, ophthalmology, oncology, and metabolic diseases. By doing so, Ring Therapeutics seeks to provide healthcare solutions that allow for a broader array of therapeutic modalities and improved patient outcomes.

Noema Pharma AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company focuses on developing therapeutic products and therapies for orphan neurological disorders characterized by imbalanced neuronal networks. Its research and development efforts aim to address disabling symptoms in conditions affecting the nervous system, including Tuberous Sclerosis Complex, Trigeminal Neuralgia, and Tourette syndrome. By creating novel therapies, Noema Pharma seeks to improve the quality of life and patient outcomes for individuals suffering from these rare conditions.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Senti Bio, established in 2016 and headquartered in South San Francisco, specializes in synthetic biotechnology for therapeutic development. The company's core focus is engineering gene circuits, a platform technology that enables precise control over cellular behavior. Senti Bio's innovative approach allows for the creation of therapies with enhanced specificity and control, particularly in oncology. Their product pipeline includes SENTI-202, an off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A, a treatment for hepatocellular carcinoma.

LifeMine Therapeutics, Inc. is a biotechnology company focused on discovering new drug modalities from eukaryotic microbes, particularly fungi. Founded in 2016 and headquartered in Cambridge, Massachusetts, with an additional office in New York, the company employs an integrated platform that combines genomics, artificial intelligence, and synthetic biology. This innovative Avatar-Rx platform utilizes high-throughput microbiology, data science, machine learning, genome engineering, and automation to explore the fungal biosphere for novel genetically encoded molecules with specific biological functions. By integrating chemoinformatic-assisted drug optimization and advanced chemical synthesis with biotransformation, LifeMine Therapeutics aims to advance a pipeline of new medicines into development.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

Ring Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing gene therapies through its innovative Anellovector platform. This platform leverages the biology of the human commensal virome to create redosable and targetable DNA therapies that address several limitations of existing gene therapies, such as restricted access to various tissues, risks associated with genomic integration, and issues with tolerability. Founded in 2017, the company aims to expand the applications of gene therapy beyond traditional gene replacement, targeting a wide range of conditions including genetic disorders, ophthalmology, oncology, and metabolic diseases. By doing so, Ring Therapeutics seeks to provide healthcare solutions that allow for a broader array of therapeutic modalities and improved patient outcomes.

Omega Therapeutics is a development-stage biotechnology company based in Cambridge, Massachusetts, focused on developing genomic medicines aimed at curing diseases. The company utilizes its proprietary epigenomic programming platform to create a new class of programmable epigenetic medicines, referred to as Omega Epigenomic Controllers. These controllers are designed to selectively modulate genomic activity, enabling precise tuning of the human genome to treat various medical conditions. Founded in 2016 and rebranded from VL42, Inc. in 2017, Omega Therapeutics is dedicated to transforming human medicine by harnessing the innate potential of the genome for therapeutic benefits.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

Ikena Oncology, Inc. is a biotechnology company focused on discovering and developing biomarker-driven therapies for cancer treatment. It specializes in precision oncology by targeting pathways essential for cancer growth and therapeutic resistance, particularly within the Hippo and RAS signaling networks. The company's portfolio includes several preclinical and discovery-stage programs, such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-412, a kynurenine-degrading enzyme. Additionally, Ikena is developing IK-930, an oral small-molecule inhibitor of the TEAD transcription factor involved in the Hippo pathway. Ikena Oncology, originally founded as Kyn Therapeutics in 2016, rebranded in December 2019 and is headquartered in Boston, Massachusetts. The company aims to address significant medical needs in oncology through innovative drug development.

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.

Locanabio, Inc. is a biotechnology company based in San Diego, California, founded in 2016. It specializes in developing RNA-targeted gene therapies aimed at treating a range of underserved diseases, particularly in the areas of neuromuscular, neurodegenerative, and retinal conditions. The company's innovative platform focuses on modifying disease-causing RNA, allowing for the identification and correction of specific RNA sequences associated with rare genetic disorders. This approach distinguishes Locanabio's therapies from traditional DNA-targeted methods, offering a unique solution for patients with severe and challenging health issues. The company, which rebranded from Locana, Inc. in July 2020, is dedicated to advancing therapeutic candidates that address significant medical needs.

Noema Pharma AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company focuses on developing therapeutic products and therapies for orphan neurological disorders characterized by imbalanced neuronal networks. Its research and development efforts aim to address disabling symptoms in conditions affecting the nervous system, including Tuberous Sclerosis Complex, Trigeminal Neuralgia, and Tourette syndrome. By creating novel therapies, Noema Pharma seeks to improve the quality of life and patient outcomes for individuals suffering from these rare conditions.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and headquartered in San Mateo, California, the company focuses on targeting dysregulated transcription factors and oncogenic signaling pathways. Its lead candidate, entospletinib, is a selective inhibitor of spleen tyrosine kinase aimed at treating acute myeloid leukemia. Additionally, Kronos Bio is developing KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9, intended for MYC-amplified solid tumors. By leveraging advanced technologies such as high-throughput small-molecule microarrays and targeted protein degradation, Kronos Bio aims to identify effective compounds that can modulate historically undruggable cancer targets, ultimately striving to improve patient outcomes through precision medicine.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies for both rare and prevalent diseases. Founded in 2016 and originally named Torus Therapeutics, the company focuses on treating conditions primarily affecting the liver and retina, as well as diseases of skeletal muscle, the central nervous system, and oncology. Generation Bio employs a proprietary non-viral platform that includes a high-capacity DNA construct known as closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP). This platform aims to deliver genetic payloads effectively across various tissues and allows for redosable treatments tailored to individual patient needs. Through its advanced manufacturing processes, Generation Bio seeks to expand access to gene therapies and provide durable solutions for patients with both rare and common diseases.

Triplet Therapeutics, Inc. is focused on developing innovative therapies for repeat expansion disorders, which include conditions such as Huntington’s disease, myotonic dystrophy, and spinocerebellar ataxias. Founded in 2018 and based in Cambridge, Massachusetts, the company aims to address these disorders at their source by utilizing a unique approach that involves targeting the DNA Damage Response pathway through a single oligonucleotide. This biotherapeutic technology is designed to provide a comprehensive treatment solution for multiple repeat expansion disorders, enabling healthcare professionals to better manage these complex conditions.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and headquartered in San Mateo, California, the company focuses on targeting dysregulated transcription factors and oncogenic signaling pathways. Its lead candidate, entospletinib, is a selective inhibitor of spleen tyrosine kinase aimed at treating acute myeloid leukemia. Additionally, Kronos Bio is developing KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9, intended for MYC-amplified solid tumors. By leveraging advanced technologies such as high-throughput small-molecule microarrays and targeted protein degradation, Kronos Bio aims to identify effective compounds that can modulate historically undruggable cancer targets, ultimately striving to improve patient outcomes through precision medicine.

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.

Foghorn Therapeutics Inc. is a biopharmaceutical company focused on discovering and developing medicines that target genetically determined dependencies within the chromatin regulatory system. Utilizing its proprietary Gene Traffic Control platform, the company aims to understand and manipulate gene expression to develop innovative therapies for various diseases, particularly cancer. Foghorn is advancing several drug candidates, including FHD-286, an enzymatic inhibitor targeting BRG1 and BRM for acute myeloid leukemia and uveal melanoma, and FHD-609, a protein degrader aimed at treating synovial sarcoma. Additionally, the company has ongoing preclinical and discovery programs targeting non-small-cell lung cancer and other cancers associated with ARID1B mutations. Foghorn has established a collaboration with Merck Sharp & Dohme Corp. to create novel oncology therapeutics. Founded in 2015 and headquartered in Cambridge, Massachusetts, Foghorn Therapeutics is pioneering a new class of drug targets that could transform treatment approaches for patients with serious diseases.

KSQ Therapeutics, Inc., founded in 2015 and based in Cambridge, Massachusetts, specializes in precision functional genomics to discover innovative drug therapies. The company employs a novel approach to research and development that emphasizes de-risking prior to the creation of therapeutic candidates. Utilizing its proprietary CRISPRomics™ drug discovery engine, KSQ Therapeutics has mapped the functions of human genes across various diseases, enhancing its understanding of disease biology. This comprehensive insight enables the identification and validation of high-confidence, patient-specific drug targets. As a result, the company is focused on developing medicines that have significant potential to improve patient outcomes and has established a pipeline of preclinical discovery programs.

Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies for both rare and prevalent diseases. Founded in 2016 and originally named Torus Therapeutics, the company focuses on treating conditions primarily affecting the liver and retina, as well as diseases of skeletal muscle, the central nervous system, and oncology. Generation Bio employs a proprietary non-viral platform that includes a high-capacity DNA construct known as closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP). This platform aims to deliver genetic payloads effectively across various tissues and allows for redosable treatments tailored to individual patient needs. Through its advanced manufacturing processes, Generation Bio seeks to expand access to gene therapies and provide durable solutions for patients with both rare and common diseases.

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.

Moderna, Inc. is a biotechnology company established in 2010 and headquartered in Cambridge, Massachusetts. The company specializes in developing therapeutics and vaccines using messenger RNA (mRNA) technology, which provides instructions for cells to produce proteins essential for various biological functions. Moderna has a diverse pipeline, with numerous programs targeting infectious diseases, immuno-oncology, rare diseases, and cardiovascular conditions. As of September 2024, it has 40 mRNA development candidates in clinical trials across multiple therapeutic areas. The company's mRNA technology gained significant recognition with the authorization of its COVID-19 vaccine in December 2020. Moderna maintains strategic alliances with various organizations, including major pharmaceutical companies and research institutions, to enhance its research and development efforts.

PrimeraDx, Inc. specializes in multiplexed infectious disease assays, focusing on advanced diagnostic technologies. The company offers Scalable Target Amplification Routine technology, enabling the simultaneous quantitative measurement of multiple target nucleic acids. Its product lineup includes ViraQuant, a multiplexed assay for the quantitative measurement of various viruses, as well as FungiQuant Yeast and FungiQuant Mold, which are fungal panels used in mycology testing. Additionally, PrimeraDx provides an instrument designed for the simultaneous detection and quantification of diverse target types, such as mRNA, miRNA, SNPs, and DNA. These products are utilized in various applications, including miRNA analysis, DNA methylation, quantitative genotyping, gene expression, and viral load measurement. Founded in 2004, PrimeraDx is headquartered in Mansfield, Massachusetts.

PrimeraDx, Inc. specializes in multiplexed infectious disease assays, focusing on advanced diagnostic technologies. The company offers Scalable Target Amplification Routine technology, enabling the simultaneous quantitative measurement of multiple target nucleic acids. Its product lineup includes ViraQuant, a multiplexed assay for the quantitative measurement of various viruses, as well as FungiQuant Yeast and FungiQuant Mold, which are fungal panels used in mycology testing. Additionally, PrimeraDx provides an instrument designed for the simultaneous detection and quantification of diverse target types, such as mRNA, miRNA, SNPs, and DNA. These products are utilized in various applications, including miRNA analysis, DNA methylation, quantitative genotyping, gene expression, and viral load measurement. Founded in 2004, PrimeraDx is headquartered in Mansfield, Massachusetts.

Standard BioTools, previously known as Fluidigm, is driven by a bold vision – unleashing tools to accelerate breakthroughs in human health. Standard BioTools has an established portfolio of essential, standardized next-generation technologies that help biomedical researchers develop medicines faster and better. As a leading solutions provider, the Company provides reliable and repeatable insights in health and disease using its proprietary mass cytometry and microfluidics technologies that help transform scientific discoveries into better patient outcomes. Standard BioTools works with leading academic, government, pharmaceutical, biotechnology, plant and animal research, and clinical laboratories worldwide, focusing on the most pressing needs in translational and clinical research, including oncology, immunology, and immunotherapy.