Casdin Capital

Auron Therapeutics is a biotechnology company focused on cancer therapy development that seeks to change the paradigm from tumor cell killing to differentiation therapy, aiming to transform malignant cells into normal functioning cells by reactivating endogenous cellular programs. The company uses platforms that integrate large-omic datasets and miniaturized high-throughput flow cytometry to rapidly identify and validate multiple targets and drugs in primary human patient samples. This approach is designed to advance therapeutic candidates for cancer across areas of unmet medical need and to support the development of transformative medicines. Auron Therapeutics was founded in 2018 and is based in Wellesley, Massachusetts.

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.

IDRx is a clinical-stage biopharmaceutical company focused on developing precision therapies for oncology, specifically targeting gastrointestinal stromal tumors. The company specializes in designing precision combination therapies that utilize engineered drug combinations to address key tumor escape mechanisms and enhance the duration of therapeutic responses. Through its innovative approach, IDRx aims to transform cancer treatment by enabling healthcare professionals to create targeted cancer medicines tailored to individual patient needs.

BridgeBio Oncology Therapeutics is a biopharmaceutical company specializing in the development of precision oncology therapeutics. It focuses on targeting RAS-dependent cancers, leveraging a profound understanding of RAS signaling biology to create drugs that selectively inhibit RAS-driven PI3K activation and a wide spectrum of KRAS mutants. The company aims to provide patients with tailored treatments that address both active and inactive cancer-driving states.

Avidity Biosciences is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

Octave is a clinical platform developer focused on transforming the care landscape for multiple sclerosis and other neurodegenerative diseases. The company creates a platform that generates, analyzes, and integrates data to improve patient outcomes and reduce overall healthcare costs. By optimizing medication usage and healthcare services, Octave aims to enhance the efficiency of the pharmaceutical industry throughout the entire lifecycle of drug development, which includes discovery, clinical trials, and post-marketing phases. Their approach utilizes real-world evidence to inform decision-making, ultimately striving to set a new standard in multiple sclerosis care.

Orbital Therapeutics develops innovative RNA-based medicines to enhance global health. Its platform integrates RNA technology, delivery techniques, data analytics, and automation to create a broad portfolio of treatments for various diseases.

iVexSol specializes in developing and manufacturing lentiviral vectors (LVVs), which serve as critical gene delivery vehicles for cell and gene therapies. Founded in 2018, the company is headquartered in Worcester, Massachusetts. iVexSol's innovative technology addresses the shortage of LVVs by producing them in greater quantities and at a lower cost than traditional methods. This enables shorter clinical development timelines, increased access to life-saving treatments for patients worldwide.

Asimov designs and develops biologics and gene therapies, focusing on mammalian cell engineering and genetic circuit design. The company combines synthetic biology, computer-aided design, and machine learning to accelerate the design and manufacture of biologics, viral vectors, and cell and gene therapies. Its platform integrates biophysical simulations with data-driven models of biology to engineer genetic circuits and optimize production processes. Founded in 2017 and based in Cambridge, Massachusetts, Asimov supports healthcare firms in advancing therapeutic pipelines through software and engineering tooling that bridges biology and computational design.

IDRx is a clinical-stage biopharmaceutical company focused on developing precision therapies for oncology, specifically targeting gastrointestinal stromal tumors. The company specializes in designing precision combination therapies that utilize engineered drug combinations to address key tumor escape mechanisms and enhance the duration of therapeutic responses. Through its innovative approach, IDRx aims to transform cancer treatment by enabling healthcare professionals to create targeted cancer medicines tailored to individual patient needs.

Auron Therapeutics is a biotechnology company focused on cancer therapy development that seeks to change the paradigm from tumor cell killing to differentiation therapy, aiming to transform malignant cells into normal functioning cells by reactivating endogenous cellular programs. The company uses platforms that integrate large-omic datasets and miniaturized high-throughput flow cytometry to rapidly identify and validate multiple targets and drugs in primary human patient samples. This approach is designed to advance therapeutic candidates for cancer across areas of unmet medical need and to support the development of transformative medicines. Auron Therapeutics was founded in 2018 and is based in Wellesley, Massachusetts.

Founded in Seattle in 2008, Arzeda specializes in protein design technology. It creates and manufactures proteins and enzymes for pharmaceuticals, industrial chemicals, polymers, and other advanced applications.

Creyon Bio is a pre-clinical stage company focused on advancing drug development through a data-driven methodology. It specializes in engineering RNA-based medicines and their components by creating proprietary datasets that inform advanced machine learning models. This innovative approach enables the rapid identification of design rules and engineering principles necessary for the development of safe and effective oligonucleotide-based medicines. By leveraging these insights, Creyon Bio aims to improve the efficiency of the drug development process, ultimately contributing to cost savings in the healthcare sector while catering to diverse patient populations.

Ventus Therapeutics is a biopharmaceutical company focused on discovering and developing innovative small molecule medicines that target the innate immune system. The company employs a structural immunology platform that provides deep insights into molecular structures and mechanisms, enabling precise targeting of the innate immune system. Ventus combines advanced protein engineering capabilities with cutting-edge rational and structure-based drug design tools to create therapeutics aimed at treating autoimmune diseases, inflammatory conditions, and cancer. The company is actively building a diverse pipeline of drug programs that address significant targets within the innate immune system, with the goal of advancing treatments for both acute and chronic inflammatory and immune-related diseases.

Septerna is a biotechnology company focused on discovering and developing oral small-molecule medicines that target G protein-coupled receptors (GPCRs). It operates a proprietary Native Complex Platform to accelerate GPCR drug discovery and aims to deliver therapies across endocrinology, immunology and inflammation, and metabolic diseases. The company is advancing a GPCR-focused pipeline with candidates such as SEP-786 (PTH1R) and SEP-631 (MRGPRX2), along with programs targeting TSHR, GLP-1R, GIPR, and GCGR, with the goal of translating GPCR biology into orally available medicines that address unmet patient needs.

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.

ONI specializes in super-resolution microscopy, making advanced imaging technology accessible to researchers worldwide. Its flagship product, the Nanoimager, offers easy access to techniques like dSTORM and PALM, enabling users to visualize molecules within living cells for enhanced biological research.

EQRx, Inc. is a biotechnology company based in Cambridge, Massachusetts, established in 2019. The company focuses on re-engineering the drug discovery and delivery process to provide a market-based solution to the escalating costs of medications. EQRx aims to enhance the efficiency and affordability of high-quality, patent-protected medicines by collaborating with various stakeholders across the healthcare system and leveraging advancements in science and technology. The company has developed a robust pipeline with over ten programs, including clinical and preclinical projects targeting oncology and immune-inflammatory conditions, featuring compounds such as Aumolertinib, Lerociclib, Sugemalimab, and Nofazinlimab.

Chroma Medicine is a genomic medicine company founded in 2021 and based in Cambridge, Massachusetts, specializing in epigenetic editing. The company is pioneering a new class of genomic therapies aimed at revolutionizing the treatment of genetically driven diseases. By utilizing epigenetics, which is the natural mechanism for gene regulation, Chroma Medicine develops programmable epigenetic editors that target specific genes and modulate chromatin conformation. This innovative approach enables healthcare providers to achieve precise control of gene expression, offering a promising therapeutic option for conditions linked to genetic anomalies.

Exo Therapeutics, Inc. is a drug discovery and development company focused on addressing challenging pharmaceutical targets. Founded in 2018 and based in Cambridge, Massachusetts, the company utilizes its proprietary ExoSight platform to create a pipeline of drug candidates that target exosites, which are unique and distal binding pockets on enzymes. This innovative approach aims to reprogram enzyme activity, leading to precise and effective therapeutic outcomes, particularly in oncology and inflammation, while also minimizing potential side effects for patients. Exo Therapeutics is committed to unlocking breakthrough therapeutics through its team of experienced researchers and a deep understanding of complex disease mechanisms.

Amplo Biotechnology is a biotechnology company developing adeno-associated viral therapies aimed at curing defects in neuromuscular junctions, with an initial focus on congenital myasthenic syndromes. Its lead program, AAV-Dok7, originated with a research team from the Institute of Medical Science at the University of Tokyo and is designed to be delivered via intravascular injection to trigger molecular events that address paralysis, breathing, and swallowing difficulties caused by neuromuscular junction defects.

Founded in 2008, Ginkgo Bioworks designs, engineers, and licenses organisms for various applications. It discovers molecules for flavors, sweeteners, cosmetics, crops, pharmaceuticals, and generates probiotic bacteria for health benefits. The company serves markets such as cultured ingredients, carbon mitigation, probiotics, and natural product discovery.

Vanqua Bio is a biotechnology company dedicated to discovering and developing innovative medicines for patients with neurodegenerative diseases. The company's proprietary platform employs human genetics and patient-derived neuronal cells to identify and validate novel disease pathways related to lysosomal dysfunction or aberrant immune system activation, aiming to translate these findings into clinically effective treatments.

Zentera Therapeutics is a biopharmaceutical company.

T-knife is a biotechnology company focused on developing T cell receptor therapies aimed at treating cancer. Utilizing a humanized T cell receptor (TCR) mice platform, the company is capable of carrying human TCRαβ gene loci and recombining a diverse array of human TCRs. This innovative approach facilitates the efficient generation and virtual selection of T cell receptors tailored for specific human tumor antigens, enhancing the potential for effective cancer therapies. Through its advanced technology, T-knife aims to improve treatment options for patients battling cancer.

GeneDx specializes in delivering personalized insights from genomic data to improve health outcomes. It combines extensive genetic expertise with advanced data science capabilities.

Hexagon Bio, Inc. is a biotechnology company that specializes in discovering novel small molecule therapeutics by mining genomic data from fungal genomes. Founded in 2016 and headquartered in Menlo Park, California, Hexagon Bio leverages a proprietary platform that integrates data science, synthetic biology, and automation to identify and engineer drugs. The company's innovative approach focuses on developing targeted therapies for diseases with unmet medical needs by searching for evolutionarily refined molecules and their associated protein targets. Through its unique methodology, Hexagon Bio aims to uncover new medicines encoded within the global metagenome, ultimately striving to improve patient outcomes.

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.

Exscientia is a biotechnology company that leverages artificial intelligence and machine learning to enhance the discovery and design of novel therapeutic compounds. By integrating human expertise with advanced computational techniques, Exscientia accelerates the development of small-molecule drug candidates, aiming to produce safe and effective treatments for clinical testing. The company's innovative approach seeks to revolutionize the pharmaceutical industry by improving the precision and efficiency of drug discovery, ultimately enhancing patient outcomes.

Multiply Labs develops robotic-enabled manufacturing for personalized pharmaceutical capsules, combining pharmaceutical science with robotics to produce tailor-made capsules that align with individual patient metabolic needs. The company operates a robotic manufacturing facility and has introduced 3D-printed capsules, aiming to address scaling challenges in biotech and improve the efficiency and cost of producing patient-specific medicines.

Ventus Therapeutics is a biopharmaceutical company focused on discovering and developing innovative small molecule medicines that target the innate immune system. The company employs a structural immunology platform that provides deep insights into molecular structures and mechanisms, enabling precise targeting of the innate immune system. Ventus combines advanced protein engineering capabilities with cutting-edge rational and structure-based drug design tools to create therapeutics aimed at treating autoimmune diseases, inflammatory conditions, and cancer. The company is actively building a diverse pipeline of drug programs that address significant targets within the innate immune system, with the goal of advancing treatments for both acute and chronic inflammatory and immune-related diseases.

Absci is a drug and target discovery company that leverages deep learning AI and synthetic biology to enhance the therapeutic potential of proteins. Its Integrated Drug Creation Platform streamlines the process of identifying novel drug targets, discovering optimal biotherapeutic candidates, and generating the necessary cell lines for manufacturing. By combining biologic drug discovery and cell line development into a single, efficient workflow, Absci collaborates with biotech and pharmaceutical innovators to develop next-generation protein-based drugs. These innovations include Bionic Proteins, which feature nonstandard amino acids, and other unique drug designs that may be unattainable through conventional methods. Absci's mission is to facilitate the development of improved medicines by effectively translating innovative ideas into viable drugs.

Insitro, Inc. is a data-driven company focused on drug discovery and development, particularly targeting nonalcoholic steatohepatitis (NASH). Founded in 2018 by Daphne Koller and based in South San Francisco, California, Insitro harnesses machine learning and high-throughput biology to revolutionize the process of drug discovery. The company creates extensive functional genomic data sets and integrates them with patient data through innovative machine learning techniques. This approach allows Insitro to build predictive models that enhance target selection, streamline the design of therapeutics, and optimize drug development strategies. By addressing critical challenges in pharmaceutical research and development, Insitro aims to accelerate the delivery of new and effective treatments to patients.

Monte Rosa Therapeutics is a biotechnology company focused on cancer therapeutics that target protein degradation pathways. It specializes in developing molecular glue degraders, a class of small molecules that direct disease-relevant proteins to degradation by the cell's natural machinery. The company uses its QuEEN platform, incorporating artificial intelligence and proprietary experimental tools, to identify target proteins for degradation by molecular glue degraders. It maintains a diverse library of more than 50,000 molecules and advancing its pipeline, including MRT-2359, a candidate that targets the translation termination factor GSPT1 for potential use in MYC-driven tumors.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

Auron Therapeutics is a biotechnology company focused on cancer therapy development that seeks to change the paradigm from tumor cell killing to differentiation therapy, aiming to transform malignant cells into normal functioning cells by reactivating endogenous cellular programs. The company uses platforms that integrate large-omic datasets and miniaturized high-throughput flow cytometry to rapidly identify and validate multiple targets and drugs in primary human patient samples. This approach is designed to advance therapeutic candidates for cancer across areas of unmet medical need and to support the development of transformative medicines. Auron Therapeutics was founded in 2018 and is based in Wellesley, Massachusetts.

EQRx, Inc. is a biotechnology company based in Cambridge, Massachusetts, established in 2019. The company focuses on re-engineering the drug discovery and delivery process to provide a market-based solution to the escalating costs of medications. EQRx aims to enhance the efficiency and affordability of high-quality, patent-protected medicines by collaborating with various stakeholders across the healthcare system and leveraging advancements in science and technology. The company has developed a robust pipeline with over ten programs, including clinical and preclinical projects targeting oncology and immune-inflammatory conditions, featuring compounds such as Aumolertinib, Lerociclib, Sugemalimab, and Nofazinlimab.

Octave is a clinical platform developer focused on transforming the care landscape for multiple sclerosis and other neurodegenerative diseases. The company creates a platform that generates, analyzes, and integrates data to improve patient outcomes and reduce overall healthcare costs. By optimizing medication usage and healthcare services, Octave aims to enhance the efficiency of the pharmaceutical industry throughout the entire lifecycle of drug development, which includes discovery, clinical trials, and post-marketing phases. Their approach utilizes real-world evidence to inform decision-making, ultimately striving to set a new standard in multiple sclerosis care.

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.

PanThera CryoSolutions designs and manufactures cryopreservation solutions and systems to preserve cells, tissues, and small molecules used in clinical research. The company develops patented ice recrystallization inhibitors that minimize ice crystal formation during freezing, enabling less costly storage and transportation methods and reducing reliance on liquid nitrogen in certain cell therapies. Its products aim to maintain cellular function after extended frozen storage, reduce toxicity, enhance stability, and improve post-thaw performance, helping researchers and clinicians preserve biological material more efficiently. Through scientific expertise, PanThera CryoSolutions supports reliable, scalable cryopreservation that underpins medical research, drug development, and therapeutic applications by enabling safer and more economical cryogenic storage.

CANbridge Pharma is a biopharmaceutical company focused on the development and commercialization of healthcare products in China and North Asia, particularly for oncology and rare diseases. Established in 2012 and headquartered in Beijing, with additional offices in Shanghai, Cambridge, Taipei, and Hong Kong, the company partners with Western biopharmaceutical firms to bring clinical-stage pharmaceutical, medical device, and diagnostic products to the region. CANbridge specializes in therapies for serious medical conditions that are either unavailable or inadequately addressed in North Asia. Its key product, CAN008, is a glycosylated CD95-Fc fusion protein aimed at treating glioblastoma multiforme, while the company also has a diverse pipeline of biologics and small molecules targeting conditions such as Hunter Syndrome, hemophilia A, and various rare metabolic disorders. Through licensing and exclusive commercialization agreements, CANbridge seeks to enhance healthcare solutions for underserved patient populations in the region.

iVexSol specializes in developing and manufacturing lentiviral vectors (LVVs), which serve as critical gene delivery vehicles for cell and gene therapies. Founded in 2018, the company is headquartered in Worcester, Massachusetts. iVexSol's innovative technology addresses the shortage of LVVs by producing them in greater quantities and at a lower cost than traditional methods. This enables shorter clinical development timelines, increased access to life-saving treatments for patients worldwide.

Decibel Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing innovative therapies aimed at protecting, repairing, and restoring hearing. The company addresses various hearing disorders, including those related to pediatric cancers, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as conditions characterized by persistent tinnitus. Decibel Therapeutics employs a multifaceted approach that includes drug delivery to the inner ear, advanced imaging techniques, and pharmacokinetic and pharmacodynamic modeling. The company leverages its expertise in inner ear biology, single-cell genomics, and bioinformatics to develop effective treatments for hearing loss and related disorders. Established in 2013 and headquartered in Boston, Massachusetts, Decibel Therapeutics was originally known as Hearing, Inc. before rebranding in 2014.

Absci is a drug and target discovery company that leverages deep learning AI and synthetic biology to enhance the therapeutic potential of proteins. Its Integrated Drug Creation Platform streamlines the process of identifying novel drug targets, discovering optimal biotherapeutic candidates, and generating the necessary cell lines for manufacturing. By combining biologic drug discovery and cell line development into a single, efficient workflow, Absci collaborates with biotech and pharmaceutical innovators to develop next-generation protein-based drugs. These innovations include Bionic Proteins, which feature nonstandard amino acids, and other unique drug designs that may be unattainable through conventional methods. Absci's mission is to facilitate the development of improved medicines by effectively translating innovative ideas into viable drugs.

Cedilla Therapeutics, Inc. is a biotechnology company focused on developing targeted small-molecule therapeutics for cancer and diseases linked to protein dysregulation. The company employs innovative strategies to destabilize oncoprotein targets, utilizing methods such as direct ligand-induced degradation, disruption of stabilizing protein-protein interactions, and the identification of upstream regulators that affect protein stability. Cedilla's comprehensive approach also includes large-scale proteomic analyses to map protein susceptibility, enhancing the potential for effective therapies. By concentrating on the transitions between stable and susceptible protein states, Cedilla aims to create novel treatments that broaden the applicability of small molecule therapeutics beyond oncology. Founded in 2017 and headquartered in Cambridge, Massachusetts, the company was formerly known as Protein Stability Newco, Inc. before rebranding in 2018.

A2 Biotherapeutics, Inc. is a biotechnology company dedicated to developing innovative cell therapies for tumor cancer treatment. Based in Agoura Hills, California, the company utilizes its proprietary Tmod™ platform to create T cell therapies that accurately differentiate between solid tumors and normal cells. This platform allows for the identification and engineering of binders derived from antibodies or T cell receptors, optimizing precision in targeting cancer cells. A2 Biotherapeutics specifically addresses various cancer types, including head, neck, pancreatic, colorectal, and lung cancers, as well as cancer testis antigens. By engineering T cells to recognize genetic material loss in tumors, the company aims to selectively kill tumor cells while sparing healthy tissue. Founded in 2017, A2 Biotherapeutics continues to advance the field of cancer therapy through its innovative approaches.

Monte Rosa Therapeutics is a biotechnology company focused on cancer therapeutics that target protein degradation pathways. It specializes in developing molecular glue degraders, a class of small molecules that direct disease-relevant proteins to degradation by the cell's natural machinery. The company uses its QuEEN platform, incorporating artificial intelligence and proprietary experimental tools, to identify target proteins for degradation by molecular glue degraders. It maintains a diverse library of more than 50,000 molecules and advancing its pipeline, including MRT-2359, a candidate that targets the translation termination factor GSPT1 for potential use in MYC-driven tumors.

ONI specializes in super-resolution microscopy, making advanced imaging technology accessible to researchers worldwide. Its flagship product, the Nanoimager, offers easy access to techniques like dSTORM and PALM, enabling users to visualize molecules within living cells for enhanced biological research.

Tango Therapeutics is a biotechnology company focused on developing cancer therapies based on the genetic principle of synthetic lethality. It aims to counteract tumor suppressor gene loss, reverse immune evasion in cancer cells, and identify effective drug combinations.

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.

Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing novel therapies for patients with genetically defined diseases that have significant unmet medical needs. The company's lead product candidate, losmapimod, is a small molecule currently undergoing Phase 2b clinical trials for the treatment of facioscapulohumeral muscular dystrophy, a rare condition characterized by progressive muscle wasting and disability. Another key candidate, FTX-6058, aims to upregulate fetal hemoglobin to treat sickle cell disease and beta-thalassemia. Fulcrum is also actively researching drug targets for other genetic disorders, including Duchenne muscular dystrophy and Friedreich ataxia, as well as neurological and pulmonary diseases. The company has established collaborations with Acceleron Pharma to explore biological targets in pulmonary disease and with MyoKardia to develop therapies for genetic cardiomyopathies. Founded in 2015, Fulcrum Therapeutics continues to focus on improving patient outcomes in rare disease contexts.

Insitro, Inc. is a data-driven company focused on drug discovery and development, particularly targeting nonalcoholic steatohepatitis (NASH). Founded in 2018 by Daphne Koller and based in South San Francisco, California, Insitro harnesses machine learning and high-throughput biology to revolutionize the process of drug discovery. The company creates extensive functional genomic data sets and integrates them with patient data through innovative machine learning techniques. This approach allows Insitro to build predictive models that enhance target selection, streamline the design of therapeutics, and optimize drug development strategies. By addressing critical challenges in pharmaceutical research and development, Insitro aims to accelerate the delivery of new and effective treatments to patients.

Founded in 2019 and based in Cambridge, Massachusetts, MoMa Therapeutics discovers precision medicines by targeting molecular machines underlying human diseases. The company's platform enables drug development from previously intractable enzyme classes.

Cerevance develops novel therapeutics for neurological disorders such as Alzheimer's disease, Parkinson's disease, and other chronic neurodegenerative conditions. It uses a proprietary platform to profile specific brain cell types in human tissue samples, enabling the identification of promising targets for new treatments.

Tango Therapeutics is a biotechnology company focused on developing cancer therapies based on the genetic principle of synthetic lethality. It aims to counteract tumor suppressor gene loss, reverse immune evasion in cancer cells, and identify effective drug combinations.

Kallyope Inc. is a biotechnology company based in New York City that focuses on understanding the gut-brain axis to develop innovative therapeutics and consumer products aimed at enhancing human health. Established in 2014, Kallyope employs advanced technologies, including sequencing, computational biology, neural imaging, and human genetics, to explore gut-brain biology. The company aims to address significant unmet medical needs, such as diabetes, obesity, gut disorders, inflammatory diseases, migraines, and allergies, by creating effective and user-friendly oral agents. Kallyope's team consists of experienced scientists from leading institutions, working alongside a skilled team with a strong track record in drug discovery and development. Through its research, Kallyope seeks to deliver transformative solutions that improve overall health outcomes.

Amunix Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to discovering and developing innovative protein and peptide therapeutics aimed at treating cancer. The company employs several proprietary platforms, including XTEN for extending drug half-life, XPAT for analyzing protease activity in the tumor microenvironment, and ProTIA, which focuses on immuno-oncology therapeutics. Amunix's product pipeline includes prodrugs and transformative T-cell engagers designed to activate the immune system against solid tumors. The company collaborates with various academic, biotechnology, and pharmaceutical sectors, maintaining strategic partnerships with organizations such as Janssen and Biogen-Idec. Founded in 2006 and headquartered in Mountain View, California, Amunix strives to deliver breakthroughs that can harness the immune system to combat cancer more effectively.

EQRx, Inc. is a biotechnology company based in Cambridge, Massachusetts, established in 2019. The company focuses on re-engineering the drug discovery and delivery process to provide a market-based solution to the escalating costs of medications. EQRx aims to enhance the efficiency and affordability of high-quality, patent-protected medicines by collaborating with various stakeholders across the healthcare system and leveraging advancements in science and technology. The company has developed a robust pipeline with over ten programs, including clinical and preclinical projects targeting oncology and immune-inflammatory conditions, featuring compounds such as Aumolertinib, Lerociclib, Sugemalimab, and Nofazinlimab.

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 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.

Umoja Biopharma is a biotechnology company specializing in innovative immunotherapy aimed at transforming cancer treatment. The company focuses on reprogramming T cells to effectively target cancer within patients' bodies, enhancing the immune response to combat both solid tumors and hematological cancers, which often show poor responses to conventional therapies. Umoja Biopharma's proprietary CAR T-cell gene therapy platform is designed to deliver scalable therapeutic regimens, allowing for tailored treatment options that can be administered to any patient, regardless of tumor type or stage. This approach aims to improve patient outcomes and quality of life by providing a safe and controllable method to attack cancer directly.

iVexSol specializes in developing and manufacturing lentiviral vectors (LVVs), which serve as critical gene delivery vehicles for cell and gene therapies. Founded in 2018, the company is headquartered in Worcester, Massachusetts. iVexSol's innovative technology addresses the shortage of LVVs by producing them in greater quantities and at a lower cost than traditional methods. This enables shorter clinical development timelines, increased access to life-saving treatments for patients worldwide.

Codexis is an industrial biotechnology company headquartered in Redwood City, California, founded in 2002. It develops and sells protein catalysts and biocatalytic solutions for pharmaceutical and chemical manufacturing. Using its proprietary CodeEvolver directed‑evolution platform, Codexis engineers enzymes that enable scale‑up and optimization of processes from research to production. Products include protein catalysts, biocatalyst panels, kits, and services such as screening and engineering. The platform also supports discovery of biotherapeutic drug candidates, molecular‑biology enzymes, and in‑vitro diagnostics, and it is developing an ECO Synthesis platform for RNAi therapeutics manufacturing. More than 50 pharmaceutical firms use Codexis solutions.

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.

Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing novel therapies for patients with genetically defined diseases that have significant unmet medical needs. The company's lead product candidate, losmapimod, is a small molecule currently undergoing Phase 2b clinical trials for the treatment of facioscapulohumeral muscular dystrophy, a rare condition characterized by progressive muscle wasting and disability. Another key candidate, FTX-6058, aims to upregulate fetal hemoglobin to treat sickle cell disease and beta-thalassemia. Fulcrum is also actively researching drug targets for other genetic disorders, including Duchenne muscular dystrophy and Friedreich ataxia, as well as neurological and pulmonary diseases. The company has established collaborations with Acceleron Pharma to explore biological targets in pulmonary disease and with MyoKardia to develop therapies for genetic cardiomyopathies. Founded in 2015, Fulcrum Therapeutics continues to focus on improving patient outcomes in rare disease contexts.

ONI specializes in super-resolution microscopy, making advanced imaging technology accessible to researchers worldwide. Its flagship product, the Nanoimager, offers easy access to techniques like dSTORM and PALM, enabling users to visualize molecules within living cells for enhanced biological research.

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 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.

Neon Therapeutics is a clinical-stage biopharmaceutical company specializing in neoantigen-targeted therapies aimed at revolutionizing cancer treatment. By harnessing the immune system's ability to recognize and attack neoantigens, the company seeks to develop innovative therapies that improve patient outcomes. Its product pipeline includes NEO-PV-01, NEO-PTC-01, and NEO-SV-01, which are designed to enhance the immune response against tumors. Through its focused research and development efforts, Neon Therapeutics is committed to advancing the field of immuno-oncology and providing new options for cancer patients.

Codiak BioSciences is a clinical-stage biopharmaceutical company dedicated to developing exosome-based therapeutics, a novel class of medicines with the potential to address various diseases with significant unmet medical needs. The company employs its proprietary engEx Platform, which allows for the engineering and manufacturing of exosomes, leveraging their natural role as intercellular messengers. Codiak's pipeline includes product candidates such as exoSTING and exoIL-12, targeting diverse therapeutic areas including oncology, neuro-oncology, neurology, neuromuscular diseases, and infectious diseases. Additionally, the company collaborates with the Ragon Institute to explore the potential of its exoVACC platform for vaccines against SARS-CoV-2 and HIV. Founded in 2015 and headquartered in Cambridge, Massachusetts, Codiak aims to transform therapeutic approaches by harnessing the unique properties of exosomes.

Founded in Seattle in 2008, Arzeda specializes in protein design technology. It creates and manufactures proteins and enzymes for pharmaceuticals, industrial chemicals, polymers, and other advanced applications.

Peloton Therapeutics, Inc. is a clinical stage biopharmaceutical company based in Dallas, Texas, dedicated to developing innovative medicines for patients with cancer and other serious conditions. The company focuses on targeting HIF-2a, a transcription factor previously considered difficult to address with small molecules. Its lead drug candidate, PT2977, is an oral HIF-2a inhibitor currently undergoing Phase 2 clinical trials for advanced or metastatic clear cell renal cell carcinoma, and it is being evaluated in various settings, including in patients with von Hippel-Lindau disease-associated renal cell carcinoma and in combination with cabozantinib. Additionally, Peloton is advancing PT2567, another oral HIF-2a inhibitor in preclinical development for non-oncology indications such as pulmonary arterial hypertension. Founded in 2010 and previously known as Damascus Pharmaceutics, Peloton Therapeutics aims to leverage its scientific expertise and collaborations to become a leading biotech company in the region.

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.

CytomX Therapeutics is a clinical-stage oncology-focused biopharmaceutical company that develops proteolytically-activated antibodies based on its Probody technology platform. Probody therapeutics are designed to be activated by the tumor microenvironment, enabling targeted cancer immunotherapies with reduced activity in healthy tissues. The company advances antibody and antibody-drug conjugate programs such as CX-072 (PD-L1), CX-2009 (CD166-targeted PDC), BMS-986249 (CTLA-4 Probody), CX-2029 (CD71-targeted PDC), and BMS-986288 (CTLA-4 Probody) in clinical research, and maintains collaborations with AbbVie, Amgen, Bristol-Myers Squibb, ImmunoGen, Pfizer, and Astellas to develop Probody therapeutics. Founded in 2008 and headquartered in South San Francisco, the company aims to improve the precision and safety of antibody-based therapies for cancer and inflammatory diseases.

MyoKardia, Inc. is a clinical-stage biopharmaceutical company based in Brisbane, California, specializing in the discovery, development, and commercialization of targeted therapies for rare cardiovascular diseases. Founded in 2012, the company focuses on heritable cardiomyopathies and genetically-driven heart failure stemming from biomechanical defects in cardiac muscle contraction. Its lead product candidate, Mavacamten, is an orally administered allosteric modulator of cardiac myosin currently undergoing Phase III clinical trials for hypertrophic cardiomyopathy (HCM). Additionally, MyoKardia is developing Danicamtiv, which has completed Phase I and IIa trials for dilated cardiomyopathy, alongside several preclinical programs targeting related cardiovascular conditions. As of November 2020, MyoKardia operates as a subsidiary of Bristol-Myers Squibb Company.

MyoKardia, Inc. is a clinical-stage biopharmaceutical company based in Brisbane, California, specializing in the discovery, development, and commercialization of targeted therapies for rare cardiovascular diseases. Founded in 2012, the company focuses on heritable cardiomyopathies and genetically-driven heart failure stemming from biomechanical defects in cardiac muscle contraction. Its lead product candidate, Mavacamten, is an orally administered allosteric modulator of cardiac myosin currently undergoing Phase III clinical trials for hypertrophic cardiomyopathy (HCM). Additionally, MyoKardia is developing Danicamtiv, which has completed Phase I and IIa trials for dilated cardiomyopathy, alongside several preclinical programs targeting related cardiovascular conditions. As of November 2020, MyoKardia operates as a subsidiary of Bristol-Myers Squibb Company.

Omniox is a biopharmaceutical company focused on developing innovative treatments for diseases associated with hypoxia, a condition characterized by inadequate oxygen levels in tissues. This lack of oxygen is linked to various serious health issues, including certain cancers and cardiovascular diseases. The company utilizes its proprietary H-NOX platform technology, which is designed to deliver oxygen and nitric oxide specifically to hypoxic tissues. This targeted approach aims to restore normal tissue function and improve patient outcomes by addressing the underlying causes of hypoxia-related diseases. Through its advancements in biologics, Omniox seeks to provide meaningful therapeutic benefits and enable healthcare professionals to develop effective oxygen-based therapies that enhance patient health and recovery.

Blueprint Medicines is a Cambridge, Massachusetts-based biopharmaceutical company that develops targeted, small-molecule therapies to treat genomically defined cancers, rare diseases, and related conditions. The company focuses on precision medicines that address molecular drivers and resistance mechanisms, advancing a pipeline that includes avapritinib for systemic mastocytosis and certain gastrointestinal stromal tumors, BLU-263, a KIT inhibitor for indolent systemic mastocytosis and other mast cell disorders, fisogatinib for advanced hepatocellular carcinoma, and pralsetinib, a RET inhibitor for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. It is also pursuing BLU-782, an ALK2 inhibitor in early clinical trials for fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with partner companies such as Clementia Pharmaceuticals, CStone Pharmaceuticals, Genentech, and Hoffmann-La Roche. Founded in 2008, the company operates in the United States and Europe and combines its Insights-to-Validation platform with a proprietary chemical library to discover and develop new therapeutic compounds and combination therapies in oncology, hematology, and related areas.

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.