GenSight Biologics
Post in 2025
GenSight Biologics is a clinical-stage biotechnology company focused on developing therapies for mitochondrial and neurodegenerative diseases of the eye. It uses gene therapy-based platforms, Mitochondrial Targeting Sequence and Optogenetics, to preserve or restore vision in patients with severe degenerative retinal diseases.
Aadi Bioscience
Post in 2025
Aadi Bioscience is a clinical-stage biopharmaceutical company developing precision therapies for cancers driven by mTOR pathway alterations. It focuses on targeted mTOR inhibitors to treat genetically defined cancers, including alterations in TSC1 or TSC2 genes, where conventional inhibitors have struggled with pharmacology, delivery, safety, or tumor targeting. The company is advancing ABI-009, a clinical-phase mTOR inhibitor intended for oncology indications as well as cardiovascular and metabolic contexts. Aadi aims to bring transformative therapies to patients with ultra-rare cancers such as PEComa, and it traces its origins to an earlier name, with its operations based in Pacific Palisades, California, and a founding year of 2011.
Solid Biosciences
Post in 2025
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
Series A in 2025
Coave Therapeutics specializes in developing gene therapies for rare ocular and central nervous system (CNS) diseases. Its platform enables targeted delivery of therapeutic genes to the CNS, enhancing autophagy and lysosomal function, with a focus on improving patient outcomes for conditions like ALS.
Aadi Bioscience
Post in 2024
Aadi Bioscience is a clinical-stage biopharmaceutical company developing precision therapies for cancers driven by mTOR pathway alterations. It focuses on targeted mTOR inhibitors to treat genetically defined cancers, including alterations in TSC1 or TSC2 genes, where conventional inhibitors have struggled with pharmacology, delivery, safety, or tumor targeting. The company is advancing ABI-009, a clinical-phase mTOR inhibitor intended for oncology indications as well as cardiovascular and metabolic contexts. Aadi aims to bring transformative therapies to patients with ultra-rare cancers such as PEComa, and it traces its origins to an earlier name, with its operations based in Pacific Palisades, California, and a founding year of 2011.
PrognomiQ
Series D in 2024
PrognomiQ is a technology company focused on transforming early disease detection and treatment through multi-omics data. It generates proteomic, genomic, and metabolomic data to provide insights into complex diseases and to improve patient outcomes. The company develops advanced test products that apply multi-omics data to detect cancers and other conditions earlier, complementing traditional diagnostics and supporting monitoring of disease recurrence. By integrating proteomic data with broader health data, PrognomiQ aims to enable clinicians to diagnose more accurately and tailor interventions, ultimately advancing preventive and precision medicine.
Triveni Bio
Series B in 2024
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.
Cartesian Therapeutics
Post in 2024
Cartesian Therapeutics is a clinical-stage biopharmaceutical company developing cell and gene immunotherapies to treat cancer and autoimmune diseases. The company designs chimeric antigen receptor therapies for multiple myeloma and other cancers and uses its ImmTOR immune tolerance platform to mitigate antigen-specific immune responses. It operates a proprietary technology and manufacturing platform to deliver one or more mRNA molecules into cells to enhance their function, aiming to improve therapeutic performance.
Alterome Therapeutics
Series B in 2024
Alterome Therapeutics is a precision oncology biotechnology company focused on developing alteration-specific therapeutics to target high-value, validated oncogenic drivers. It employs structure-guided drug discovery and advanced computational chemistry to create small-molecule therapies that selectively address oncogenic alterations, with the aim of improving patient outcomes in cancer.
GenSight Biologics
Post in 2024
GenSight Biologics is a clinical-stage biotechnology company focused on developing therapies for mitochondrial and neurodegenerative diseases of the eye. It uses gene therapy-based platforms, Mitochondrial Targeting Sequence and Optogenetics, to preserve or restore vision in patients with severe degenerative retinal diseases.
PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.
Solid Biosciences
Post in 2024
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.
GenSight Biologics
Post in 2023
GenSight Biologics is a clinical-stage biotechnology company focused on developing therapies for mitochondrial and neurodegenerative diseases of the eye. It uses gene therapy-based platforms, Mitochondrial Targeting Sequence and Optogenetics, to preserve or restore vision in patients with severe degenerative retinal diseases.
Engine Biosciences
Series A in 2023
Engine Biosciences, established in 2014 and headquartered in Singapore, specializes in drug discovery and cellular reprogramming. The company's core offering is a validated platform that combines artificial intelligence and genetic perturbation to uncover gene interactions and biological networks. This platform enables researchers and drug developers to create targeted therapies and apply precision medicine. Engine Biosciences caters to prominent American corporations and research institutions, accelerating and reducing the costs of R&D for new medical therapies.
Triveni Bio
Series A in 2023
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
Post in 2023
Taysha Gene Therapies develops adeno-associated virus-based gene therapies to treat monogenic diseases of the central nervous system. Its pipeline includes treatments for GM2 gangliosidosis, CLN1 disease, Rett syndrome, SLC6A1 haploinsufficiency disorder, and Surfeit locus 1 deficiency. The company collaborates with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments.
ADARx Pharmaceuticals
Series C in 2023
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.
GenSight Biologics
Post in 2023
GenSight Biologics is a clinical-stage biotechnology company focused on developing therapies for mitochondrial and neurodegenerative diseases of the eye. It uses gene therapy-based platforms, Mitochondrial Targeting Sequence and Optogenetics, to preserve or restore vision in patients with severe degenerative retinal diseases.
Flare Therapeutics
Series B in 2023
Flare Therapeutics is a biotechnology company that develops small-molecule medicines by targeting transcription factors. It focuses on identifying switch sites—druggable regions that regulate transcription factor activity—to address disease-causing mutations. Leveraging insights into transcription factor biology, Flare aims to translate this understanding into precision medicines, building an early pipeline that is initially focused on precision oncology with potential expansions into neurology, rare genetic disorders, immunology, and inflammation. The company combines a drug-discovery platform with a biology-led approach to discover therapies that alter the trajectory of diseases driven by transcription factor dysregulation.
Ring Therapeutics
Series C in 2023
Ring Therapeutics is a biotechnology company developing gene therapies using Anellovector, a redosable and targetable DNA therapy platform based on the biology of the human commensal virome. The platform aims to address limitations of current DNA therapies, including limited tissue access, inability to redose, potential genomic integration, and tolerability concerns. Founded in 2017 and based in Cambridge, Massachusetts, Ring Therapeutics was established by Flagship Pioneering to pursue a multi-product platform. It focuses on discovering and developing Anellovector therapeutics to unlock the full potential of gene therapy, moving beyond gene replacement to a broader set of modalities and mechanisms. The approach targets disease sites with tissue and cellular specificity, aiming to treat conditions across genetic disorders, ophthalmology, oncology, and metabolic disorders and to expand therapeutic options for previously inaccessible diseases.
Solid Biosciences
Post in 2022
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.
Pretzel Therapeutics
Series A in 2022
Pretzel Therapeutics is a biotechnology company focused on developing therapies for mitochondrial dysfunction. Founded by leading experts in mitochondrial biology, the company aims to address the underlying causes of mitochondrial dysfunction through a thorough understanding of mitochondrial mechanisms. By creating innovative treatments, Pretzel Therapeutics seeks to provide effective solutions for a range of diseases, particularly those related to aging. The company is dedicated to advancing the field of mitochondrial medicine and improving patient outcomes through targeted therapeutic approaches.
Senti Biosciences
Post in 2022
Senti Biosciences is a biotechnology company focused on synthetic biology. It develops gene circuit platforms to engineer therapeutics, with a focus on improving precision and control in cancer treatments.
LifeMine Therapeutics
Series C in 2022
LifeMine Therapeutics, Inc. is a biotechnology company focused on the genomic discovery of new drug modalities derived from eukaryotic microbes, specifically fungi. Founded in 2016 and headquartered in Cambridge, Massachusetts, with an additional office in New York, it employs an integrated drug discovery platform that combines genomics, artificial intelligence, and synthetic biology. LifeMine’s Avatar-Rx platform utilizes high-throughput microbiology, data science, genome engineering, and automation technologies to identify novel gene-encoded molecules (GEMs) with specific biological functions. The company emphasizes chemoinformatic-assisted drug optimization and advanced chemical synthesis, enabling the progression of new product candidates into development. Founded by experts in the field, LifeMine Therapeutics aims to innovate medicine through its unique approach to leveraging the fungal biosphere.
PrognomiQ
Venture Round in 2022
PrognomiQ is a technology company focused on transforming early disease detection and treatment through multi-omics data. It generates proteomic, genomic, and metabolomic data to provide insights into complex diseases and to improve patient outcomes. The company develops advanced test products that apply multi-omics data to detect cancers and other conditions earlier, complementing traditional diagnostics and supporting monitoring of disease recurrence. By integrating proteomic data with broader health data, PrognomiQ aims to enable clinicians to diagnose more accurately and tailor interventions, ultimately advancing preventive and precision medicine.
Korro Bio
Series B in 2022
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
Series B in 2021
Ring Therapeutics is a biotechnology company developing gene therapies using Anellovector, a redosable and targetable DNA therapy platform based on the biology of the human commensal virome. The platform aims to address limitations of current DNA therapies, including limited tissue access, inability to redose, potential genomic integration, and tolerability concerns. Founded in 2017 and based in Cambridge, Massachusetts, Ring Therapeutics was established by Flagship Pioneering to pursue a multi-product platform. It focuses on discovering and developing Anellovector therapeutics to unlock the full potential of gene therapy, moving beyond gene replacement to a broader set of modalities and mechanisms. The approach targets disease sites with tissue and cellular specificity, aiming to treat conditions across genetic disorders, ophthalmology, oncology, and metabolic disorders and to expand therapeutic options for previously inaccessible diseases.
Engine Biosciences
Series A in 2021
Engine Biosciences, established in 2014 and headquartered in Singapore, specializes in drug discovery and cellular reprogramming. The company's core offering is a validated platform that combines artificial intelligence and genetic perturbation to uncover gene interactions and biological networks. This platform enables researchers and drug developers to create targeted therapies and apply precision medicine. Engine Biosciences caters to prominent American corporations and research institutions, accelerating and reducing the costs of R&D for new medical therapies.
Flare Therapeutics
Series A in 2021
Flare Therapeutics is a biotechnology company that develops small-molecule medicines by targeting transcription factors. It focuses on identifying switch sites—druggable regions that regulate transcription factor activity—to address disease-causing mutations. Leveraging insights into transcription factor biology, Flare aims to translate this understanding into precision medicines, building an early pipeline that is initially focused on precision oncology with potential expansions into neurology, rare genetic disorders, immunology, and inflammation. The company combines a drug-discovery platform with a biology-led approach to discover therapies that alter the trajectory of diseases driven by transcription factor dysregulation.
Omega Therapeutics
Series C in 2021
Omega Therapeutics, Inc. is a development-stage biotechnology company based in Cambridge, Massachusetts, founded in 2016. The company specializes in developing genomic medicines aimed at curing diseases through precision tuning of the human genome. It utilizes its proprietary epigenomic programming platform to engineer a new class of programmable epigenetic medicines known as Omega Epigenomic Controllers. These controllers are designed to selectively regulate genomic activity, allowing for the downregulation or upregulation of genes to achieve therapeutic effects. Omega Therapeutics seeks to transform human medicine by harnessing the natural capacity of the genome to treat and cure various diseases.
Evox Therapeutics
Series C in 2021
Evox Therapeutics Limited is a biotechnology company based in Oxford, United Kingdom, that specializes in developing exosome-based therapeutics for the treatment of severe diseases with limited treatment options. Founded in 2016, the company focuses on harnessing and engineering extracellular vesicles, known as exosomes, to facilitate targeted delivery of nucleic acids and proteins. Evox's innovative platform allows for the modification of exosomes using advanced molecular engineering techniques, enabling effective drug delivery to specific organs, including the brain and central nervous system. By leveraging these natural delivery capabilities, Evox aims to overcome the limitations associated with conventional protein, antibody, and nucleic acid therapies, thereby creating novel therapeutic solutions that could significantly impact human health. The company is supported by a robust intellectual property portfolio and is positioned as a leader in this emerging therapeutic space.
Scorpion Therapeutics
Series B in 2021
Founded in 2020 and headquartered in Boston, Massachusetts, Scorpion Therapeutics is a clinical-stage biotechnology company specializing in precision oncology. It develops targeted small-molecule drugs to treat cancer by focusing on three areas: therapies against known oncogenes, agents for currently undruggable targets, and drugs for new targets.
Lexeo Therapeutics
Series A in 2021
Lexeo Therapeutics is a fully integrated biotechnology company focused on developing adeno-associated virus (AAV)-mediated gene therapies. Its lead programs target both rare and non-rare monogenic diseases, with a preclinical pipeline that spans monogenic, hereditary, and acquired conditions across populations with unmet medical need. The company aims to advance clinical programs toward commercialization while maintaining an ongoing research collaboration with Weill Cornell Medicine’s Department of Genetic Medicine to inform and accelerate its preclinical and clinical work. By leveraging AAV-based approaches and collaborations, Lexeo Therapeutics seeks to transform treatment options for patients with genetic and complex diseases.
Locanabio
Series B in 2020
Locanabio is a biotechnology company developing RNA-targeted gene therapies for underserved diseases, with a focus on neuromuscular, neurodegenerative, and retinal conditions. It employs an RNA-targeting platform to modify disease-causing RNA and correct aberrant transcripts, offering therapies distinct from DNA-based approaches. The company identifies target RNA sequences within transcripts and programs therapies to advance candidates for rare genetic neuromuscular, neurodegenerative, and retinal diseases. Located in San Diego, Locanabio aims to translate RNA-directed interventions into treatments for patients with severe conditions.
Achilles Therapeutics
Series C in 2020
Achilles Therapeutics Ltd. is a biopharmaceutical company based in London, United Kingdom, specializing in the development of patient-specific immunotherapies designed to combat cancer. Founded in 2016, the company focuses on personalized vaccines and T cell therapies that utilize the body's immune system to target and eliminate cancer cells. Achilles Therapeutics aims to identify and target truncal tumor neoantigens and other markers present on cancer cells, allowing for precise attacks on tumors while sparing healthy tissue. The company is currently conducting clinical trials, including the CHIRON trial for non-small-cell lung cancer and the THETIS trial for recurrent or metastatic melanoma, to evaluate the efficacy and safety of its innovative therapies.
Ambrx
Private Equity Round in 2020
Ambrx, Inc. is a clinical-stage biopharmaceutical company based in La Jolla, California, specializing in the development of engineered precision biologics through an expanded genetic code technology platform. This innovative approach enables the incorporation of synthetic amino acids into proteins, facilitating the creation of next-generation therapeutics. Ambrx focuses on various product candidates, including antibody drug conjugates (ADCs), bispecific antibodies, targeted immuno-oncology treatments, and long-acting therapeutic peptides aimed at addressing metabolic and cardiovascular diseases. The company has a robust development pipeline featuring multiple clinical and preclinical programs, with its lead candidate ARX788 among them. Ambrx also collaborates with other organizations, such as MabSpace Biosciences and BeiGene, to enhance its research and development efforts in oncology and other therapeutic areas. Founded in 2003, Ambrx is committed to advancing the efficacy and safety of new biologic therapies for patients.
GenSight Biologics
Post in 2020
GenSight Biologics is a clinical-stage biotechnology company focused on developing therapies for mitochondrial and neurodegenerative diseases of the eye. It uses gene therapy-based platforms, Mitochondrial Targeting Sequence and Optogenetics, to preserve or restore vision in patients with severe degenerative retinal diseases.
Talaris Therapeutics
Series B in 2020
Talaris Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies aimed at eliminating chronic immunosuppression for organ transplant recipients. The company's leading product, FCR001, is designed to enable living donor kidney transplant recipients to discontinue all chronic immunosuppression therapy within twelve months post-transplant. In addition to its applications in organ transplantation, Talaris’s technology facilitates the safe use of hematopoietic stem cells from unmatched and unrelated donors for bone marrow transplants, reducing the associated toxicity and risks of traditional methods. Founded in 1988 and originally named Regenerex, Inc., Talaris Therapeutics rebranded in March 2019. The company is headquartered in Louisville, Kentucky, with an additional office in Wellesley, Massachusetts.
PrognomiQ
Venture Round in 2020
PrognomiQ is a technology company focused on transforming early disease detection and treatment through multi-omics data. It generates proteomic, genomic, and metabolomic data to provide insights into complex diseases and to improve patient outcomes. The company develops advanced test products that apply multi-omics data to detect cancers and other conditions earlier, complementing traditional diagnostics and supporting monitoring of disease recurrence. By integrating proteomic data with broader health data, PrognomiQ aims to enable clinicians to diagnose more accurately and tailor interventions, ultimately advancing preventive and precision medicine.
Kronos Bio
Private Equity Round in 2020
Kronos Bio, Inc. is a clinical-stage biopharmaceutical company based in San Mateo, California, focused on the discovery and development of innovative cancer therapeutics. Established in 2017, the company aims to address historically challenging cancer targets through precision medicine. Its primary product candidates include entospletinib, a selective inhibitor of spleen tyrosine kinase intended for acute myeloid leukemia patients, and KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9 for treating MYC-amplified solid tumors. Kronos Bio employs advanced technologies, such as high-throughput small-molecule microarrays and targeted protein degradation, to identify potent and selective compounds that target dysregulated transcription factors and other key drivers of oncogenic signaling.
Taysha Gene Therapies
Series B in 2020
Taysha Gene Therapies develops adeno-associated virus-based gene therapies to treat monogenic diseases of the central nervous system. Its pipeline includes treatments for GM2 gangliosidosis, CLN1 disease, Rett syndrome, SLC6A1 haploinsufficiency disorder, and Surfeit locus 1 deficiency. The company collaborates with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments.
Thrive Earlier Detection
Series B in 2020
Thrive Earlier Detection is a healthcare company dedicated to saving lives through early detection of cancer. It develops CancerSEEK, a liquid biopsy test designed to identify various cancers in their earliest stages via blood analysis.
Generation Bio
Series C in 2020
Generation Bio is a genetic medicines company developing durable, redosable treatments for patients with rare and prevalent diseases. Its non-viral platform combines closed-ended DNA, cell-targeted lipid nanoparticles, and scalable manufacturing processes to deliver large and multiple genes across various tissues.
Triplet Therapeutics
Series A in 2019
Triplet Therapeutics, Inc. is a biotechnology company focused on developing innovative therapies for triplet repeat expansion disorders, such as Huntington's disease, myotonic dystrophy, and spinocerebellar ataxias. Founded in 2018 and based in Cambridge, Massachusetts, the company aims to address these conditions at their source by utilizing a unique approach that involves a single oligonucleotide targeting the DNA Damage Response pathway. This strategy is designed to provide effective treatments for multiple repeat expansion disorders, enabling healthcare professionals to offer advanced biotherapeutic options to their patients. Through its research and development efforts, Triplet Therapeutics is committed to transforming the landscape of treatment for these challenging genetic disorders.
Achilles Therapeutics
Series B in 2019
Achilles Therapeutics Ltd. is a biopharmaceutical company based in London, United Kingdom, specializing in the development of patient-specific immunotherapies designed to combat cancer. Founded in 2016, the company focuses on personalized vaccines and T cell therapies that utilize the body's immune system to target and eliminate cancer cells. Achilles Therapeutics aims to identify and target truncal tumor neoantigens and other markers present on cancer cells, allowing for precise attacks on tumors while sparing healthy tissue. The company is currently conducting clinical trials, including the CHIRON trial for non-small-cell lung cancer and the THETIS trial for recurrent or metastatic melanoma, to evaluate the efficacy and safety of its innovative therapies.
Solid Biosciences
Post in 2019
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
Series A in 2019
Kronos Bio, Inc. is a clinical-stage biopharmaceutical company based in San Mateo, California, focused on the discovery and development of innovative cancer therapeutics. Established in 2017, the company aims to address historically challenging cancer targets through precision medicine. Its primary product candidates include entospletinib, a selective inhibitor of spleen tyrosine kinase intended for acute myeloid leukemia patients, and KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9 for treating MYC-amplified solid tumors. Kronos Bio employs advanced technologies, such as high-throughput small-molecule microarrays and targeted protein degradation, to identify potent and selective compounds that target dysregulated transcription factors and other key drivers of oncogenic signaling.
Thrive Earlier Detection
Series A in 2019
Thrive Earlier Detection is a healthcare company dedicated to saving lives through early detection of cancer. It develops CancerSEEK, a liquid biopsy test designed to identify various cancers in their earliest stages via blood analysis.
Foghorn Therapeutics
Venture Round in 2019
Foghorn Therapeutics Inc. is a biopharmaceutical company focused on discovering and developing innovative medicines that target genetically determined dependencies within the chromatin regulatory system. Utilizing its proprietary Gene Traffic Control platform, Foghorn aims to manipulate gene expression to develop therapies for cancer and other serious diseases. The company is advancing multiple drug candidates, including FHD-286, a small-molecule enzymatic inhibitor designed for treating acute myeloid leukemia and uveal melanoma, and FHD-609, a protein degrader targeting BRD9 for synovial sarcoma. Additionally, Foghorn is engaged in preclinical and discovery programs that focus on selectively targeting BRM in non-small-cell lung cancer and developing modulators for ARID1B in bladder, ovarian, and endometrial cancers. Founded in 2015 and headquartered in Cambridge, Massachusetts, Foghorn Therapeutics collaborates with Merck Sharp & Dohme Corp. to explore novel oncology therapeutics.
KSQ Therapeutics
Series C in 2018
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.
PACT Pharma
Series B in 2018
PACT Pharma, Inc. is a biotechnology company focused on developing personalized cell-based therapies for cancer treatment. The company specializes in neoantigen-specific adoptive T-cell therapies aimed at solid tumors. Its innovative platform utilizes bioinformatics to analyze patients' tumor and normal DNA and RNA, identifying candidate neoantigen peptides that stimulate robust antitumor T-cell responses. By pinpointing neo-epitopes unique to each patient's cancer, PACT Pharma enables the engineering of autologous T cells that specifically target and eradicate tumors expressing these neo-antigens. Founded in 2016 and headquartered in South San Francisco, California, PACT Pharma is dedicated to advancing personalized medicine in oncology.
Generation Bio
Series B in 2018
Generation Bio is a genetic medicines company developing durable, redosable treatments for patients with rare and prevalent diseases. Its non-viral platform combines closed-ended DNA, cell-targeted lipid nanoparticles, and scalable manufacturing processes to deliver large and multiple genes across various tissues.
Standard BioTools
Series D in 2004
Standard BioTools develops, manufactures, and sells technologies that accelerate biomedical research and medicines development. The company provides standardized, next-generation tools built around proprietary mass cytometry and microfluidics platforms to generate reliable insights into health and disease. Its proteomics and genomics capabilities support a range of applications in translational and clinical research, including oncology, immunology, and immunotherapy. The products are used by academic, government, pharmaceutical, biotechnology, plant and animal research, and clinical laboratories worldwide. By delivering repeatable measurement platforms, Standard BioTools helps researchers translate discoveries into improved patient outcomes.