Pfizer

Enara Bio Limited is a biotechnology company based in Oxford, United Kingdom, specializing in the development of vaccines and immunotherapies for cancer treatment and prevention. Founded in 2016 and previously known as ErVaxx Limited, the company focuses on creating targeted cancer immunotherapies by exploring novel antigens derived from endogenous retroviral DNA sequences. Enara Bio's technology platform enables the identification and assessment of tumor specificity and immunogenic potential, facilitating improved strategies for combating various cancers. Through its innovative approach, the company aims to provide effective therapeutic options for a broad patient population.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Caribou Biosciences develops cellular engineering solutions based on CRISPR technologies, offering a gene editing platform with applications in human therapeutics, disease modeling, genomics research, and agricultural biotechnology.

Vedanta Biosciences is a clinical-stage microbiome company focused on developing therapies for immune-mediated and infectious diseases using live bacteria derived from the human microbiome. The company designs orally delivered defined bacterial consortia, leveraging libraries of microbiome-derived strains, proprietary datasets from human interventional studies, and tools for selecting potent strains. Its platform encompasses consortium design and CGMP manufacturing of bacterially defined products, enabling physicians to access live bacteria drugs for autoimmune and inflammatory conditions. Headquartered in Cambridge, Massachusetts, Vedanta Biosciences aims to create a new category of oral therapies arising from defined bacterial communities grown from clonal cell banks.

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.

Storm Therapeutics Limited is a biotechnology company based in Cambridge, United Kingdom, focused on discovering and developing small molecule drugs that target RNA-modifying enzymes for cancer treatment and other immune diseases. Incorporated in 2015 and previously known as Iceni Therapeutics Limited, the company utilizes its drug discovery and RNA analytics platform to modulate RNA modifying enzymes, identifying novel targets and first-in-class drug candidates. Storm Therapeutics aims to provide innovative therapies against RNA epigenetic targets, addressing various conditions including oncology, inflammation, viral infections, and central nervous system diseases.

Nucleome Therapeutics Limited is a biotechnology company based in Oxford, United Kingdom, founded in 2019. The company focuses on decoding the "dark matter" of the human genome, which comprises over 95% of disease-linked genetic variants that have not yet been fully explored. By leveraging a unique platform that creates high-resolution three-dimensional genome structure maps and employs machine learning techniques, Nucleome aims to link these genetic variants to gene functions and map disease pathways. This innovative approach facilitates the functional validation of variants in primary cell types, enabling the discovery and development of novel therapeutics. Initially concentrating on lymphocytes and autoimmune diseases, Nucleome Therapeutics seeks to establish a robust pipeline of drug assets along with corresponding biomarkers to improve patient outcomes through genetically guided therapeutics.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Arkuda Therapeutics, Inc. is a biotechnology company founded in 2018 and based in Cambridge, Massachusetts, focused on developing innovative treatments for patients with neurological diseases. The company utilizes new insights into progranulin and lysosomal biology to create therapies that aim to correct progranulin deficiency and address lysosomal dysfunction, particularly in genetically-defined frontotemporal dementia associated with mutations in the GRN gene. By targeting these underlying biological mechanisms, Arkuda's lead compounds are designed to improve cellular health in the brain, potentially delaying the progression of neurodegenerative diseases and the onset of their symptoms.

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

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Strata Oncology, Inc. is a precision oncology company focused on enhancing cancer care by facilitating access to precision medicine clinical trials and expediting drug approval processes. Based in Ann Arbor, Michigan, the company develops a comprehensive precision oncology platform that supports routine testing and efficient use of molecular data. Strata's offerings include The Strata Trial, a genomic testing program for systematic precision oncology implementation, and StrataNGS, a targeted assay for sequencing DNA and RNA from biopsies. Additionally, StrataPOINT integrates electronic medical record histories with molecular profiling to ensure eligible patients are tested and considered for trial enrollment. The company features a portfolio of pharma-sponsored therapeutic protocols, known as Strata Partnered Trials, aligned to specific biomarkers. Strata also operates The Strata Lab, a cancer sequencing facility, and maintains the Strata Precision Oncology Network, a collaborative entity aimed at promoting precision medicine initiatives. Formerly known as Strata Oncology Research, Inc., the company rebranded in 2016 and has been incorporated since 2015.

Artios Pharma Limited is a biotechnology company based in Cambridge, United Kingdom, that specializes in developing innovative cancer treatments targeting DNA damage response (DDR) pathways. Founded in 2016, the company is dedicated to creating first-in-class therapies that selectively kill cancer cells by exploiting their reliance on DDR mechanisms. Artios’ product pipeline includes a range of promising candidates, such as a therapy that targets DNA polymerase theta, a protein involved in DNA repair processes, and another program focusing on a novel DDR target. The company collaborates with prominent research organizations, including Cancer Research Technology, to enhance its development efforts and leverage expertise in DNA repair. Artios is led by a skilled team with extensive experience in drug discovery, positioning it to advance its next-generation DDR programs and contribute significantly to the field of cancer treatment.

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company based in Emeryville, California, focused on developing innovative gene therapies for unmet medical needs. The company utilizes targeted and evolved adeno-associated virus (AAV) vectors to create its product candidates, which are concentrated in the therapeutic areas of ophthalmology, cardiology, and pulmonology. Key candidates in its portfolio include 4D-125, currently in a Phase I/II trial for X-linked retinitis pigmentosa, and 4D-110, undergoing Phase I trials for choroideremia. Additionally, 4D-310 is in Phase I/II trials for Fabry disease, while investigational drugs 4D-150 and 4D-710 are being developed for wet age-related macular degeneration and cystic fibrosis lung disease, respectively. Founded in 2013, 4D Molecular Therapeutics aims to advance its gene therapies to address significant health challenges.

Metabomed is a drug discovery company focused on developing treatments for cancer by targeting the unique metabolic pathways of cancer cells. Its proprietary platform leverages computational biology, genomics, and metabolomics to identify specific metabolic alterations that support cancer growth. By creating small molecules that specifically target these reprogrammed metabolic processes, Metabomed aims to halt tumor progression while minimizing harm to surrounding healthy tissues. This innovative approach allows for quicker initiation of treatment and increases the likelihood of successful patient outcomes.

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.

Arkuda Therapeutics, Inc. is a biotechnology company founded in 2018 and based in Cambridge, Massachusetts, focused on developing innovative treatments for patients with neurological diseases. The company utilizes new insights into progranulin and lysosomal biology to create therapies that aim to correct progranulin deficiency and address lysosomal dysfunction, particularly in genetically-defined frontotemporal dementia associated with mutations in the GRN gene. By targeting these underlying biological mechanisms, Arkuda's lead compounds are designed to improve cellular health in the brain, potentially delaying the progression of neurodegenerative diseases and the onset of their symptoms.

Storm Therapeutics Limited is a biotechnology company based in Cambridge, United Kingdom, focused on discovering and developing small molecule drugs that target RNA-modifying enzymes for cancer treatment and other immune diseases. Incorporated in 2015 and previously known as Iceni Therapeutics Limited, the company utilizes its drug discovery and RNA analytics platform to modulate RNA modifying enzymes, identifying novel targets and first-in-class drug candidates. Storm Therapeutics aims to provide innovative therapies against RNA epigenetic targets, addressing various conditions including oncology, inflammation, viral infections, and central nervous system diseases.

Therachon is a biotechnology company specializing in the development of treatments for rare genetic diseases that lack available therapies. Primarily based in Basel, Switzerland, with research labs in Nice, France, the company is advancing its lead candidate, TA-46, a novel protein therapy aimed at treating achondroplasia, the most prevalent form of short-limbed dwarfism. In addition to its focus on achondroplasia, Therachon is also committed to developing therapeutics targeting rare gastrointestinal and musculoskeletal disorders, with the goal of enabling medical professionals to effectively manage conditions such as short bowel syndrome.

Arrakis Therapeutics, Inc. is a biopharmaceutical company based in Waltham, Massachusetts, that specializes in drug discovery focused on ribonucleic acid (RNA) targets. Established in 2015, the company utilizes a proprietary platform that combines advanced RNA bioinformatics, structural tools, and chemical libraries to identify new RNA targets and develop RNA-targeted small molecules (rSMs). This innovative approach aims to address various diseases, including cancer, neurological disorders, and rare genetic conditions. Arrakis also offers SHAPEware, a software tool designed to analyze and predict RNA secondary structures and potential ligand-binding sites, further enhancing its drug development capabilities. Through its comprehensive pipeline, Arrakis Therapeutics seeks to improve treatment options and outcomes for patients across multiple therapeutic areas.

SpringWorks Therapeutics is a clinical-stage biopharmaceutical company applying precision medicine to develop medicines for severe rare diseases and cancer. It maintains a differentiated targeted oncology portfolio with lead candidates nirogacestat, a gamma secretase inhibitor, and mirdametinib, a MEK inhibitor, both in late-stage trials; the pipeline also includes BGB-3245, an oral inhibitor of activating BRAF mutations, in Phase I, and several combination programs. The company pursues collaborations with BeiGene, GlaxoSmithKline, and Allogene and has license agreements with Pfizer for nirogacestat and mirdametinib. Founded in 2017 and headquartered in Stamford, Connecticut, SpringWorks aims to advance potentially registrational studies in rare tumor types and genetically defined cancers through partnerships that expand its portfolio and patient access.

Vivet Therapeutics SAS is a biotechnology company based in Paris, France, focused on the research, development, and commercialization of gene therapy treatments for inherited liver disorders and orphan diseases. Founded in 2016, the company utilizes a liver-tropic adeno-associated virus (AAV) to deliver therapeutic genes directly to hepatocytes, aiming to correct genetic disorders at the source. Vivet is particularly engaged in developing innovative solutions for conditions such as Wilson disease, Progressive Familial Intrahepatic Cholestasis (PFIC), and Citrullinemia Type I (CTLN1). To enhance its gene therapy approaches, Vivet collaborates with the Fundación para la Investigación Médica Aplicada at the Centro de Investigación Médica Aplicada, focusing on optimizing AAV vectors for targeted liver delivery and ensuring long-term expression of therapeutic genes.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, that specializes in developing and commercializing therapeutics for patients with rare genetic disorders of hemoglobin. Founded in 2016, the company is advancing IMR-687, an oral, once-daily therapeutic designed to treat sickle cell disease and beta-thalassemia. IMR-687 is a highly selective small molecule inhibitor of PDE9, engineered to have a multimodal mechanism of action that impacts various cell types, including red blood cells and white blood cells, as well as adhesion mediators. Through its innovative approach, Imara aims to provide disease-modifying treatments for individuals affected by hemoglobinopathies.

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company based in Emeryville, California, focused on developing innovative gene therapies for unmet medical needs. The company utilizes targeted and evolved adeno-associated virus (AAV) vectors to create its product candidates, which are concentrated in the therapeutic areas of ophthalmology, cardiology, and pulmonology. Key candidates in its portfolio include 4D-125, currently in a Phase I/II trial for X-linked retinitis pigmentosa, and 4D-110, undergoing Phase I trials for choroideremia. Additionally, 4D-310 is in Phase I/II trials for Fabry disease, while investigational drugs 4D-150 and 4D-710 are being developed for wet age-related macular degeneration and cystic fibrosis lung disease, respectively. Founded in 2013, 4D Molecular Therapeutics aims to advance its gene therapies to address significant health challenges.

Artios Pharma Limited is a biotechnology company based in Cambridge, United Kingdom, that specializes in developing innovative cancer treatments targeting DNA damage response (DDR) pathways. Founded in 2016, the company is dedicated to creating first-in-class therapies that selectively kill cancer cells by exploiting their reliance on DDR mechanisms. Artios’ product pipeline includes a range of promising candidates, such as a therapy that targets DNA polymerase theta, a protein involved in DNA repair processes, and another program focusing on a novel DDR target. The company collaborates with prominent research organizations, including Cancer Research Technology, to enhance its development efforts and leverage expertise in DNA repair. Artios is led by a skilled team with extensive experience in drug discovery, positioning it to advance its next-generation DDR programs and contribute significantly to the field of cancer treatment.

Therachon is a biotechnology company specializing in the development of treatments for rare genetic diseases that lack available therapies. Primarily based in Basel, Switzerland, with research labs in Nice, France, the company is advancing its lead candidate, TA-46, a novel protein therapy aimed at treating achondroplasia, the most prevalent form of short-limbed dwarfism. In addition to its focus on achondroplasia, Therachon is also committed to developing therapeutics targeting rare gastrointestinal and musculoskeletal disorders, with the goal of enabling medical professionals to effectively manage conditions such as short bowel syndrome.

Strata Oncology, Inc. is a precision oncology company focused on enhancing cancer care by facilitating access to precision medicine clinical trials and expediting drug approval processes. Based in Ann Arbor, Michigan, the company develops a comprehensive precision oncology platform that supports routine testing and efficient use of molecular data. Strata's offerings include The Strata Trial, a genomic testing program for systematic precision oncology implementation, and StrataNGS, a targeted assay for sequencing DNA and RNA from biopsies. Additionally, StrataPOINT integrates electronic medical record histories with molecular profiling to ensure eligible patients are tested and considered for trial enrollment. The company features a portfolio of pharma-sponsored therapeutic protocols, known as Strata Partnered Trials, aligned to specific biomarkers. Strata also operates The Strata Lab, a cancer sequencing facility, and maintains the Strata Precision Oncology Network, a collaborative entity aimed at promoting precision medicine initiatives. Formerly known as Strata Oncology Research, Inc., the company rebranded in 2016 and has been incorporated since 2015.

Allogene Therapeutics is a clinical-stage biotech company focused on immuno-oncology, developing genetically engineered allogeneic T-cell therapies for cancer. The company builds a pipeline of off-the-shelf donor-derived CAR-T products using gene-editing and proprietary cell-manufacturing technologies to enable broader patient eligibility and scalable production. Its programs include UCART19 for relapsed or refractory acute lymphoblastic leukemia and other allogeneic CAR-T candidates targeting lymphoma, leukemia, autoimmune diseases, and solid tumors. Revenue is primarily generated from collaborations and licensing agreements.

Cydan is an orphan drug accelerator focused on developing therapies for patients with rare genetic diseases. The company identifies and de-risks compounds that hold therapeutic and commercial potential, employing a rigorous process to generate data that supports both development pathways and the formation of new companies. With a team that boasts deep expertise in drug development and strong relationships within academia and patient advocacy, Cydan accelerates therapies through various stages, including clinical, regulatory, and commercial development. By facilitating the creation of effective treatments, Cydan aims to improve the lives of patients and families affected by rare diseases.

SpringWorks Therapeutics is a clinical-stage biopharmaceutical company applying precision medicine to develop medicines for severe rare diseases and cancer. It maintains a differentiated targeted oncology portfolio with lead candidates nirogacestat, a gamma secretase inhibitor, and mirdametinib, a MEK inhibitor, both in late-stage trials; the pipeline also includes BGB-3245, an oral inhibitor of activating BRAF mutations, in Phase I, and several combination programs. The company pursues collaborations with BeiGene, GlaxoSmithKline, and Allogene and has license agreements with Pfizer for nirogacestat and mirdametinib. Founded in 2017 and headquartered in Stamford, Connecticut, SpringWorks aims to advance potentially registrational studies in rare tumor types and genetically defined cancers through partnerships that expand its portfolio and patient access.

Arrakis Therapeutics, Inc. is a biopharmaceutical company based in Waltham, Massachusetts, that specializes in drug discovery focused on ribonucleic acid (RNA) targets. Established in 2015, the company utilizes a proprietary platform that combines advanced RNA bioinformatics, structural tools, and chemical libraries to identify new RNA targets and develop RNA-targeted small molecules (rSMs). This innovative approach aims to address various diseases, including cancer, neurological disorders, and rare genetic conditions. Arrakis also offers SHAPEware, a software tool designed to analyze and predict RNA secondary structures and potential ligand-binding sites, further enhancing its drug development capabilities. Through its comprehensive pipeline, Arrakis Therapeutics seeks to improve treatment options and outcomes for patients across multiple therapeutic areas.

Rhythm Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing peptide therapeutics for treating rare genetic disorders that cause life-threatening metabolic issues. Its lead product candidate, setmelanotide, is a potent melanocortin-4 receptor agonist designed for precision medicine to treat hyperphagia and severe obesity resulting from rare MC4R pathway diseases.

Superfluid DX is a company focused on developing advanced molecular diagnostics technology aimed at early disease detection and monitoring. By leveraging innovative research in transcriptomics, Superfluid DX analyzes tissue-specific cell-free circulating RNA found in blood samples. This non-invasive approach enables the identification of genetic information linked to neurodegenerative diseases and other conditions, providing healthcare professionals with critical insights into a patient's health status. The company's platform is designed to facilitate early diagnosis and ongoing monitoring of organ damage, ultimately improving patient outcomes by enabling timely interventions.

IGNITE Immunotherapy is focused on advancing cancer treatment through innovative immunotherapy solutions. The company specializes in the development of optimized intravenous oncolytic virus vaccines, which are designed to enhance the effectiveness of existing immune checkpoint inhibitors. By igniting a targeted immune response against cancer, IGNITE aims to improve patient outcomes and ultimately cure cancer through combination immunotherapy. Their expertise encompasses oncolytic virus design, gene therapy vector discovery, and the development of experimental cancer therapeutics, positioning them at the forefront of biotechnological advancements in the fight against cancer.

Bamboo Therapeutics is a biotechnology company focused on advancing gene therapies for the treatment of monogenic disorders, particularly those affecting the central nervous system and neuromuscular conditions. Founded to translate the pioneering work of Dr. Richard Jude Samulski, a leading figure in gene therapy, the company aims to move innovative treatments from the laboratory into human clinical trials, ultimately seeking drug approval. Dr. Samulski, recognized for his groundbreaking use of adeno-associated virus (AAV) as a vehicle for gene replacement, has significantly contributed to the field through the development of over twenty patents related to AAV therapeutic applications. Bamboo Therapeutics leverages his expertise in vector optimization and AAV re-engineering to enhance the safety and efficacy of its gene therapies.

Vtesse, Inc. is a biopharmaceutical company based in Gaithersburg, Maryland, focused on developing treatments for patients with rare diseases. The company is primarily engaged in the clinical study of VTS-270 for the treatment of Niemann-Pick Disease Type C (NPC) and is also involved in the pre-clinical discovery and development of additional novel drugs for NPC and other lysosomal storage diseases. Vtesse operates as a subsidiary of Sucampo Pharmaceuticals, Inc. and is recognized as the first spin-out from Cydan Development, Inc., an orphan-drug accelerator. The company collaborates with the National Institutes of Health (NIH) to advance its clinical programs and is supported by a seasoned management team with extensive experience in drug development. Vtesse's initiatives aim not only to provide innovative therapies but also to offer resources and support for patients and families affected by NPC and related conditions.

Storm Therapeutics Limited is a biotechnology company based in Cambridge, United Kingdom, focused on discovering and developing small molecule drugs that target RNA-modifying enzymes for cancer treatment and other immune diseases. Incorporated in 2015 and previously known as Iceni Therapeutics Limited, the company utilizes its drug discovery and RNA analytics platform to modulate RNA modifying enzymes, identifying novel targets and first-in-class drug candidates. Storm Therapeutics aims to provide innovative therapies against RNA epigenetic targets, addressing various conditions including oncology, inflammation, viral infections, and central nervous system diseases.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, that specializes in developing and commercializing therapeutics for patients with rare genetic disorders of hemoglobin. Founded in 2016, the company is advancing IMR-687, an oral, once-daily therapeutic designed to treat sickle cell disease and beta-thalassemia. IMR-687 is a highly selective small molecule inhibitor of PDE9, engineered to have a multimodal mechanism of action that impacts various cell types, including red blood cells and white blood cells, as well as adhesion mediators. Through its innovative approach, Imara aims to provide disease-modifying treatments for individuals affected by hemoglobinopathies.

Metabomed is a drug discovery company focused on developing treatments for cancer by targeting the unique metabolic pathways of cancer cells. Its proprietary platform leverages computational biology, genomics, and metabolomics to identify specific metabolic alterations that support cancer growth. By creating small molecules that specifically target these reprogrammed metabolic processes, Metabomed aims to halt tumor progression while minimizing harm to surrounding healthy tissues. This innovative approach allows for quicker initiation of treatment and increases the likelihood of successful patient outcomes.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, that specializes in developing and commercializing therapeutics for patients with rare genetic disorders of hemoglobin. Founded in 2016, the company is advancing IMR-687, an oral, once-daily therapeutic designed to treat sickle cell disease and beta-thalassemia. IMR-687 is a highly selective small molecule inhibitor of PDE9, engineered to have a multimodal mechanism of action that impacts various cell types, including red blood cells and white blood cells, as well as adhesion mediators. Through its innovative approach, Imara aims to provide disease-modifying treatments for individuals affected by hemoglobinopathies.

Rhythm Metabolic is the subsidiary of Rhythm Pharmaceuticals that develops setmelanotide (RM-493), a novel peptide MC4 agonist for the treatment of obesity caused by genetic deficiencies in the MC4 pathway. It was established in 2013 and is headquartered in Boston, Massachusetts.

Translate Bio, Inc. is a clinical-stage biotechnology company focused on developing messenger RNA (mRNA) therapeutics aimed at addressing diseases linked to protein or gene dysfunction. Founded in 2011 and headquartered in Lexington, Massachusetts, the company is advancing its lead candidates, MRT5005 and MRT5201. MRT5005 is currently undergoing Phase I/II clinical trials for the treatment of cystic fibrosis, while MRT5201 is being developed for ornithine transcarbamylase deficiency. Translate Bio was previously known as RaNA Therapeutics, Inc. before rebranding in June 2017. The company specializes in innovative therapies for rare diseases, leveraging the potential of RNA-based medicine.

Mirna Therapeutics, Inc. is a discovery-stage biopharmaceutical company that specializes in developing miRNA-directed therapies for human oncology. With a strong focus on research and a deep understanding of microRNA and cancer biology, Mirna is positioned to advance the emerging field of miRNA-based therapeutics. The company boasts a robust intellectual property portfolio that supports its innovative approach to cancer treatment.

Vtesse, Inc. is a biopharmaceutical company based in Gaithersburg, Maryland, focused on developing treatments for patients with rare diseases. The company is primarily engaged in the clinical study of VTS-270 for the treatment of Niemann-Pick Disease Type C (NPC) and is also involved in the pre-clinical discovery and development of additional novel drugs for NPC and other lysosomal storage diseases. Vtesse operates as a subsidiary of Sucampo Pharmaceuticals, Inc. and is recognized as the first spin-out from Cydan Development, Inc., an orphan-drug accelerator. The company collaborates with the National Institutes of Health (NIH) to advance its clinical programs and is supported by a seasoned management team with extensive experience in drug development. Vtesse's initiatives aim not only to provide innovative therapies but also to offer resources and support for patients and families affected by NPC and related conditions.

Quartet Medicine is a private biotechnology company based in Cambridge, Massachusetts, focused on developing novel treatments for chronic pain and inflammation. Founded in 2013 by scientists at Boston Children’s Hospital and École Polytechnique Fédérale de Lausanne (EPFL) in Switzerland, the company aims to restore tetrahydrobiopterin (BH4) homeostasis in neuronal and inflammatory cells. This approach is based on insights from human genetics and preclinical target validation data, which highlight BH4's role as a critical mediator of peripheral nerve dysfunction and immune cell regulation. Quartet Medicine's efforts are directed towards creating non-opioid medications that safely address chronic pain and inflammation by targeting BH4 levels at the nexus of the peripheral nervous system.

Epic Sciences is a diagnostics company focused on cancer management through blood-based tests that identify and characterize circulating tumor cells from a minimally invasive sample. Its platform profiles single-cell phenotype and genotype, including biomarker expression levels, subcellular localization, morphologic features, and genotypic metrics, and supports next-generation sequencing, FISH, and single-cell genomics to detect and characterize CTCs. The company develops tests such as the Oncotype DX AR-V7 Nucleus Detect for metastatic castration-resistant prostate cancer and offers biopharma solutions. It collaborates with biotechnology and pharmaceutical companies and cancer centers worldwide to provide real-time biopsy material and data to guide personalized medicine. By enabling analysis of rare cells before and after treatment, Epic Sciences aims to improve cancer management. The company was founded in 2008 and is headquartered in San Diego, California.

Cydan is an orphan drug accelerator focused on developing therapies for patients with rare genetic diseases. The company identifies and de-risks compounds that hold therapeutic and commercial potential, employing a rigorous process to generate data that supports both development pathways and the formation of new companies. With a team that boasts deep expertise in drug development and strong relationships within academia and patient advocacy, Cydan accelerates therapies through various stages, including clinical, regulatory, and commercial development. By facilitating the creation of effective treatments, Cydan aims to improve the lives of patients and families affected by rare diseases.

Rhythm Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing peptide therapeutics for treating rare genetic disorders that cause life-threatening metabolic issues. Its lead product candidate, setmelanotide, is a potent melanocortin-4 receptor agonist designed for precision medicine to treat hyperphagia and severe obesity resulting from rare MC4R pathway diseases.

Epic Sciences is a diagnostics company focused on cancer management through blood-based tests that identify and characterize circulating tumor cells from a minimally invasive sample. Its platform profiles single-cell phenotype and genotype, including biomarker expression levels, subcellular localization, morphologic features, and genotypic metrics, and supports next-generation sequencing, FISH, and single-cell genomics to detect and characterize CTCs. The company develops tests such as the Oncotype DX AR-V7 Nucleus Detect for metastatic castration-resistant prostate cancer and offers biopharma solutions. It collaborates with biotechnology and pharmaceutical companies and cancer centers worldwide to provide real-time biopsy material and data to guide personalized medicine. By enabling analysis of rare cells before and after treatment, Epic Sciences aims to improve cancer management. The company was founded in 2008 and is headquartered in San Diego, California.

Mirna Therapeutics, Inc. is a discovery-stage biopharmaceutical company that specializes in developing miRNA-directed therapies for human oncology. With a strong focus on research and a deep understanding of microRNA and cancer biology, Mirna is positioned to advance the emerging field of miRNA-based therapeutics. The company boasts a robust intellectual property portfolio that supports its innovative approach to cancer treatment.

Clovis Oncology, Inc. is a biopharmaceutical company dedicated to acquiring, developing, and commercializing innovative cancer treatments in the United States, Europe, and other international markets. The company focuses on precision medicine, targeting specific cancer subsets to ensure that therapeutics are directed to patients who are most likely to benefit. Clovis Oncology's primary product is Rubraca, an oral small molecule inhibitor of poly ADP-ribose polymerase, approved for the treatment of recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer. Additionally, the company is developing lucitanib, an oral inhibitor targeting multiple tyrosine kinase receptors involved in cancer progression. Clovis distributes its products through specialty distributors and pharmacy providers, and it engages in various collaborations and licensing agreements with prominent pharmaceutical companies such as Pfizer, AstraZeneca, and Bristol-Myers Squibb. Founded in 2009 and headquartered in Boulder, Colorado, Clovis Oncology is committed to advancing cancer care through targeted therapeutic approaches.

FoldRx Pharmaceuticals, Inc. is engaged in the discovery and development of disease-modifying drug therapies targeting diseases caused by protein misfolding and amyloidosis. Based in Cambridge, Massachusetts, the company focuses on addressing conditions related to the accumulation of misfolded proteins, which can result from genetic mutations or defects in cellular quality control mechanisms. Its pipeline includes advanced clinical programs aimed at treating transthyretin-associated amyloidoses, specifically those affecting the nervous and cardiovascular systems. Additionally, FoldRx is exploring therapeutic options for Parkinson's disease and cystic fibrosis. Founded in 2003, the company aims to provide healthcare professionals with effective treatments for these complex conditions.

HandyLab is a medical technology company that develops and manufactures molecular diagnostic assays and automation platforms The company’s proprietary platform reduces the time, cost, and complexity of testing while improving the quality of results. Using patented real-time microfluidic PCR technology, its products are positioned to decentralize nucleic acid testing. HandyLab was founded in 2000 and is based in Ann Arbor, Michigan. It was acquired by Becton, Dickinson and Company in 2009.

Catapult Genetics Pty., Ltd. is a developer and provider of DNA testing services focused on the livestock and aquaculture industries. The company specializes in commercializing DNA tests and gene markers designed to enhance meat production and quality, thus improving efficiency for food producers, processors, and retailers. Its product offerings include GeneSTAR tests for feed efficiency, marbling, and tenderness, as well as SireTRACE for parentage identification. Additionally, Catapult Genetics provides SureTRAK for traceability of meat products, GH Exon 5 for evaluating wagyu cattle, and SCD for selecting cattle based on genotype. The company serves a diverse clientele, including stud and commercial breeders, pastoral companies, and biotechnology firms, through distribution partners in regions such as the United Kingdom, North and South America, Europe, and South Africa. Founded in 1998 and based in Albion, Australia, Catapult Genetics operates in Australia and New Zealand and was previously known as Genetic Solutions Pty Ltd.

Genizon Biosciences Inc. discovers genes and biomarkers in common diseases using genome wide association studies (GWAS), leveraging its proven and proprietary technology platform and access to patients from the Quebec Founder Population.

Antibody discovery and optimization company developing a rational approach to antibody engineering.