New Enterprise Associates

Tune Therapeutics develops cell and gene therapies guided by epigenetic programming to treat devastating diseases. The company is building an epigenetic programming platform that enables new pathways for disease treatment by tapping into the epigenome to program biology without altering the DNA sequence. This approach aims to deliver paradigm-changing therapies by modulating gene expression through epigenetic mechanisms rather than editing the genome, expanding options for diseases where conventional treatments have limited efficacy. By integrating epigenetics and cell biology, the company seeks durable, precise interventions that can reprogram cellular states to restore function while minimizing genetic change. The emphasis is on research and development to advance candidates toward clinical use to address severe and unmet medical needs.

Senti Biosciences is a biotechnology company focused on synthetic biology. It develops gene circuit platforms to engineer therapeutics, with a focus on improving precision and control in cancer treatments.

Avenzo Therapeutics focuses on developing innovative cancer treatments. It operates a clinical-stage platform dedicated to creating novel therapies for underserved cancers, with a pipeline targeting rare and genomically defined cancers.

858 Therapeutics is a biotechnology company specializing in the discovery of small molecule therapeutics for novel targets in oncology and immunology. The company focuses on innovative approaches such as synthetic lethality, innate immunity, and RNA modulation, with its lead program being a PARG inhibitor currently in early clinical development.

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

Avenzo Therapeutics focuses on developing innovative cancer treatments. It operates a clinical-stage platform dedicated to creating novel therapies for underserved cancers, with a pipeline targeting rare and genomically defined cancers.

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company specializing in the discovery and development of microRNA-targeted therapies for various diseases. Founded in 2007 and headquartered in San Diego, California, the company focuses on innovative treatments for conditions such as kidney diseases and viral infections. Its lead product candidates include RG-012, an anti-miR targeting miR-21, currently in Phase II clinical trials for Alport syndrome, and RGLS4326, an anti-miR targeting miR-17, which is undergoing Phase Ib clinical trials for autosomal dominant polycystic kidney disease. Additionally, Regulus is advancing a pipeline of preclinical drug candidates, including RGLS5579 to inhibit miR-10b, as well as programs aimed at treating Hepatitis B virus and Non-Alcoholic Steatohepatitis. The company's work leverages the recent scientific advancements in microRNA research, which play a crucial role in regulating gene expression.

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company specializing in the discovery and development of microRNA-targeted therapies for various diseases. Founded in 2007 and headquartered in San Diego, California, the company focuses on innovative treatments for conditions such as kidney diseases and viral infections. Its lead product candidates include RG-012, an anti-miR targeting miR-21, currently in Phase II clinical trials for Alport syndrome, and RGLS4326, an anti-miR targeting miR-17, which is undergoing Phase Ib clinical trials for autosomal dominant polycystic kidney disease. Additionally, Regulus is advancing a pipeline of preclinical drug candidates, including RGLS5579 to inhibit miR-10b, as well as programs aimed at treating Hepatitis B virus and Non-Alcoholic Steatohepatitis. The company's work leverages the recent scientific advancements in microRNA research, which play a crucial role in regulating gene expression.

Senti Biosciences is a biotechnology company focused on synthetic biology. It develops gene circuit platforms to engineer therapeutics, with a focus on improving precision and control in cancer treatments.

Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies to treat serious and life-threatening genetic diseases. Code Bio leverages its novel multivalent synthetic DNA delivery platform, 3DNA, which has been engineered to overcome many of the challenges inherent with viral-based gene therapies such as immunogenicity, size and delivery limitations, re-dosability, and manufacturing complexity. The company is advancing an internal pipeline focused on select rare disease programs as well as establishing partnerships to take forward programs in both rare and prevalent diseases.

Splice Bio is a biotechnology company based in Barcelona, Spain, that specializes in developing innovative gene therapies using its proprietary intein platform technology. Founded in 2012, the company focuses on addressing the needs of patients suffering from incurable genetic diseases. Splice Bio's technology, originally developed in the Muir Lab at Princeton University, enhances the capabilities of traditional adeno-associated viruses (AAVs) by allowing for larger gene cargo delivery and targeting a broader range of tissues. This advancement aims to overcome existing limitations in gene therapy, making it a promising solution for treating complex genetic disorders. The company was formerly known as Proteodesign, S.L. before rebranding to Splice Bio in September 2020.

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

Tune Therapeutics develops cell and gene therapies guided by epigenetic programming to treat devastating diseases. The company is building an epigenetic programming platform that enables new pathways for disease treatment by tapping into the epigenome to program biology without altering the DNA sequence. This approach aims to deliver paradigm-changing therapies by modulating gene expression through epigenetic mechanisms rather than editing the genome, expanding options for diseases where conventional treatments have limited efficacy. By integrating epigenetics and cell biology, the company seeks durable, precise interventions that can reprogram cellular states to restore function while minimizing genetic change. The emphasis is on research and development to advance candidates toward clinical use to address severe and unmet medical needs.

AviadoBio focuses on developing transformative gene therapies for devastating neurodegenerative disorders such as frontotemporal dementia and amyotrophic lateral sclerosis. The company's platform combines proprietary technologies with a deep understanding of the brain to deliver effective treatments.

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company specializing in the discovery and development of microRNA-targeted therapies for various diseases. Founded in 2007 and headquartered in San Diego, California, the company focuses on innovative treatments for conditions such as kidney diseases and viral infections. Its lead product candidates include RG-012, an anti-miR targeting miR-21, currently in Phase II clinical trials for Alport syndrome, and RGLS4326, an anti-miR targeting miR-17, which is undergoing Phase Ib clinical trials for autosomal dominant polycystic kidney disease. Additionally, Regulus is advancing a pipeline of preclinical drug candidates, including RGLS5579 to inhibit miR-10b, as well as programs aimed at treating Hepatitis B virus and Non-Alcoholic Steatohepatitis. The company's work leverages the recent scientific advancements in microRNA research, which play a crucial role in regulating gene expression.

Recludix Pharma develops a platform for discovering potent and selective inhibitors of complex protein targets. The platform integrates custom-generated DNA-encoded libraries, massively parallel structure-activity relationship determination, and proprietary screening tools to ensure selectivity.

858 Therapeutics is a biotechnology company specializing in the discovery of small molecule therapeutics for novel targets in oncology and immunology. The company focuses on innovative approaches such as synthetic lethality, innate immunity, and RNA modulation, with its lead program being a PARG inhibitor currently in early clinical development.

Shape Therapeutics, Inc. is a biotechnology company focused on RNA-editing gene therapy for various medical conditions, including neurodegenerative disorders, oncology, metabolic diseases, and rare genetic disorders. Founded in 2018 and headquartered in Seattle, Washington, the company utilizes its proprietary RNAfix technology to edit RNA using the body’s natural cellular machinery. This approach minimizes risks associated with immunogenicity, cellular toxicity, and unintended DNA editing. Shape Therapeutics is dedicated to advancing its innovative platforms for payload, delivery, and manufacturing to enable the development of effective treatments, aiming to address critical health challenges and improve patient outcomes.

Stablix Therapeutics is a biotechnology company specializing in Targeted Protein Stabilization (TPS). It develops heterobifunctional small molecules that recruit deubiquitinase enzymes to stabilize or enhance targeted proteins, focusing on treating rare diseases, cancer, and immunological disorders.

Affinia Therapeutics develops adeno-associated virus (AAV) gene therapies for muscular and central nervous system diseases. The company uses an AAV vector design platform built on synthetic and systems biology, high-throughput screening, and tissue- and single-cell-resolution analyses to create vectors with improved properties. It leverages sequencing, artificial intelligence, and structural modeling to enable rational design of therapies. Headquartered in Waltham, Massachusetts, Affinia Therapeutics was founded in 2019 and targets diseases with significant unmet needs in muscle and CNS.

Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies to treat serious and life-threatening genetic diseases. Code Bio leverages its novel multivalent synthetic DNA delivery platform, 3DNA, which has been engineered to overcome many of the challenges inherent with viral-based gene therapies such as immunogenicity, size and delivery limitations, re-dosability, and manufacturing complexity. The company is advancing an internal pipeline focused on select rare disease programs as well as establishing partnerships to take forward programs in both rare and prevalent diseases.

Personal Genome Diagnostics, Inc. is a biotechnology company focused on patient-specific analyses of cancer genomes. Established in 2010 and headquartered in Baltimore, Maryland, the company has pioneered technologies for non-invasive "liquid biopsy" approaches, allowing for the detection of tumor-specific alterations in circulating cell-free DNA. Its offerings include the METDetect Assay for identifying MET gene amplifications, LungSelect for detecting actionable genetic alterations in non-small cell lung cancer patients, and PGDx elio plasma resolve, which analyzes various genetic mutations from plasma samples. Additionally, Personal Genome Diagnostics provides RNAcomplete, enabling researchers to extract both RNA and genomic DNA from a single tissue sample, and CancerXOME, which captures and analyzes coding regions of genes. The company collaborates with institutions like Mayo Clinic and partners with KingMed Diagnostics to enhance its research and diagnostic capabilities, thereby empowering oncologists and researchers with crucial genomic insights to combat cancer.

Senti Biosciences is a biotechnology company focused on synthetic biology. It develops gene circuit platforms to engineer therapeutics, with a focus on improving precision and control in cancer treatments.

Reneo Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company based in San Diego, California, dedicated to developing therapies for patients with rare genetic mitochondrial diseases. These conditions are characterized by deficits in cellular metabolism and energy production, often leading to significant health challenges. The company is focused on creating treatments that improve mitochondrial function, preserve muscle integrity, and enhance the overall quality of life for affected individuals. Its lead product candidate, REN001, is an oral medication designed to modulate genes critical to the production of adenosine triphosphate (ATP), the primary energy source for cellular processes. Founded in 2014, Reneo aims to address the unmet needs of patients suffering from orphan metabolic diseases linked to mitochondrial dysfunction.

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

Affinia Therapeutics develops adeno-associated virus (AAV) gene therapies for muscular and central nervous system diseases. The company uses an AAV vector design platform built on synthetic and systems biology, high-throughput screening, and tissue- and single-cell-resolution analyses to create vectors with improved properties. It leverages sequencing, artificial intelligence, and structural modeling to enable rational design of therapies. Headquartered in Waltham, Massachusetts, Affinia Therapeutics was founded in 2019 and targets diseases with significant unmet needs in muscle and CNS.

Akouos is a biotechnology company focused on precision genetic medicine to restore and preserve hearing. Based in Boston, it develops gene therapies using a proprietary adeno-associated viral vector platform and delivery approaches to treat sensorineural hearing loss in genetically defined populations. The company's lead program, AK-OTOF, targets hearing loss caused by mutations in the OTOF gene. Akouos pursues research and development across gene-based interventions aimed at improving auditory function and long-term sensory outcomes. It has collaborations with academic and industry partners, including Massachusetts Eye and Ear and Lonza, to advance discovery and manufacturing capabilities. Founded in 2016, Akouos addresses unmet clinical needs in auditory health by seeking to restore physiologic hearing through targeted genetic therapies.

ChromaCode Inc., established in 2012 and based in Carlsbad, California, specializes in molecular diagnostics. The company develops innovative testing solutions for infectious diseases, cancer, and non-invasive prenatal testing (NIPT) using technology licensed from Caltech. ChromaCode's core products are software, reagents, and assays that enhance the multiplexing capabilities of quantitative PCR (qPCR) instruments by fivefold. Additionally, they offer molecular assays and software for various applications such as personalized medicine and pharmaceutical research. One of their notable products is the HDPCR Tick-Borne Pathogen Panel, a multiplex PCR test detecting common tick-borne pathogens in a single assay. ChromaCode's technology focuses on improving signal processing from life science instrumentation, enabling cost-effective testing of multiple targets simultaneously while integrating into existing laboratory workflows.

Shape Therapeutics, Inc. is a biotechnology company focused on RNA-editing gene therapy for various medical conditions, including neurodegenerative disorders, oncology, metabolic diseases, and rare genetic disorders. Founded in 2018 and headquartered in Seattle, Washington, the company utilizes its proprietary RNAfix technology to edit RNA using the body’s natural cellular machinery. This approach minimizes risks associated with immunogenicity, cellular toxicity, and unintended DNA editing. Shape Therapeutics is dedicated to advancing its innovative platforms for payload, delivery, and manufacturing to enable the development of effective treatments, aiming to address critical health challenges and improve patient outcomes.

Reneo Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company based in San Diego, California, dedicated to developing therapies for patients with rare genetic mitochondrial diseases. These conditions are characterized by deficits in cellular metabolism and energy production, often leading to significant health challenges. The company is focused on creating treatments that improve mitochondrial function, preserve muscle integrity, and enhance the overall quality of life for affected individuals. Its lead product candidate, REN001, is an oral medication designed to modulate genes critical to the production of adenosine triphosphate (ATP), the primary energy source for cellular processes. Founded in 2014, Reneo aims to address the unmet needs of patients suffering from orphan metabolic diseases linked to mitochondrial dysfunction.

Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.

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.

Xenon Pharmaceuticals is a biopharmaceutical company dedicated to developing novel medicines for neurological disorders. It focuses on rare genetic defects as drug targets, leveraging its proprietary discovery platform called Extreme Genetics. The company's pipeline includes XEN1101 and XEN496 for epilepsy treatment.

Akouos is a biotechnology company focused on precision genetic medicine to restore and preserve hearing. Based in Boston, it develops gene therapies using a proprietary adeno-associated viral vector platform and delivery approaches to treat sensorineural hearing loss in genetically defined populations. The company's lead program, AK-OTOF, targets hearing loss caused by mutations in the OTOF gene. Akouos pursues research and development across gene-based interventions aimed at improving auditory function and long-term sensory outcomes. It has collaborations with academic and industry partners, including Massachusetts Eye and Ear and Lonza, to advance discovery and manufacturing capabilities. Founded in 2016, Akouos addresses unmet clinical needs in auditory health by seeking to restore physiologic hearing through targeted genetic therapies.

Catalog Technologies Inc. specializes in a DNA-based platform that revolutionizes digital data storage and computation. Founded in 2016 and headquartered in Boston, Massachusetts, the company provides a method for encoding digital information into DNA, making it a viable and cost-effective solution for long-term data archiving. By leveraging advanced synthetic biology technologies, Catalog aims to transform how information is stored, allowing customers to utilize DNA molecules as a primary medium for digital records. This innovative approach positions Catalog at the forefront of next-generation data storage solutions.

Senti Biosciences is a biotechnology company focused on synthetic biology. It develops gene circuit platforms to engineer therapeutics, with a focus on improving precision and control in cancer treatments.

Personal Genome Diagnostics, Inc. is a biotechnology company focused on patient-specific analyses of cancer genomes. Established in 2010 and headquartered in Baltimore, Maryland, the company has pioneered technologies for non-invasive "liquid biopsy" approaches, allowing for the detection of tumor-specific alterations in circulating cell-free DNA. Its offerings include the METDetect Assay for identifying MET gene amplifications, LungSelect for detecting actionable genetic alterations in non-small cell lung cancer patients, and PGDx elio plasma resolve, which analyzes various genetic mutations from plasma samples. Additionally, Personal Genome Diagnostics provides RNAcomplete, enabling researchers to extract both RNA and genomic DNA from a single tissue sample, and CancerXOME, which captures and analyzes coding regions of genes. The company collaborates with institutions like Mayo Clinic and partners with KingMed Diagnostics to enhance its research and diagnostic capabilities, thereby empowering oncologists and researchers with crucial genomic insights to combat cancer.

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

Akouos is a biotechnology company focused on precision genetic medicine to restore and preserve hearing. Based in Boston, it develops gene therapies using a proprietary adeno-associated viral vector platform and delivery approaches to treat sensorineural hearing loss in genetically defined populations. The company's lead program, AK-OTOF, targets hearing loss caused by mutations in the OTOF gene. Akouos pursues research and development across gene-based interventions aimed at improving auditory function and long-term sensory outcomes. It has collaborations with academic and industry partners, including Massachusetts Eye and Ear and Lonza, to advance discovery and manufacturing capabilities. Founded in 2016, Akouos addresses unmet clinical needs in auditory health by seeking to restore physiologic hearing through targeted genetic therapies.

Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.

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.

Catalog Technologies Inc. specializes in a DNA-based platform that revolutionizes digital data storage and computation. Founded in 2016 and headquartered in Boston, Massachusetts, the company provides a method for encoding digital information into DNA, making it a viable and cost-effective solution for long-term data archiving. By leveraging advanced synthetic biology technologies, Catalog aims to transform how information is stored, allowing customers to utilize DNA molecules as a primary medium for digital records. This innovative approach positions Catalog at the forefront of next-generation data storage solutions.

Nightstar Therapeutics is a clinical-stage gene therapy company based in London, United Kingdom, specializing in the development of innovative one-time treatments for rare inherited retinal diseases. The company is advancing several product candidates, including its lead candidate NSR-REP1, which is in phase 3 clinical development for choroideremia. Additionally, Nightstar is working on NSR-RPGR, currently in phase 2/3 trials for X-linked retinitis pigmentosa, as well as NSR-BEST1. The company is also developing NSR-ABCA4, which is in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address significant unmet medical needs in the field of retinal disorders.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

ChromaCode Inc., established in 2012 and based in Carlsbad, California, specializes in molecular diagnostics. The company develops innovative testing solutions for infectious diseases, cancer, and non-invasive prenatal testing (NIPT) using technology licensed from Caltech. ChromaCode's core products are software, reagents, and assays that enhance the multiplexing capabilities of quantitative PCR (qPCR) instruments by fivefold. Additionally, they offer molecular assays and software for various applications such as personalized medicine and pharmaceutical research. One of their notable products is the HDPCR Tick-Borne Pathogen Panel, a multiplex PCR test detecting common tick-borne pathogens in a single assay. ChromaCode's technology focuses on improving signal processing from life science instrumentation, enabling cost-effective testing of multiple targets simultaneously while integrating into existing laboratory workflows.

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.

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company specializing in the discovery and development of microRNA-targeted therapies for various diseases. Founded in 2007 and headquartered in San Diego, California, the company focuses on innovative treatments for conditions such as kidney diseases and viral infections. Its lead product candidates include RG-012, an anti-miR targeting miR-21, currently in Phase II clinical trials for Alport syndrome, and RGLS4326, an anti-miR targeting miR-17, which is undergoing Phase Ib clinical trials for autosomal dominant polycystic kidney disease. Additionally, Regulus is advancing a pipeline of preclinical drug candidates, including RGLS5579 to inhibit miR-10b, as well as programs aimed at treating Hepatitis B virus and Non-Alcoholic Steatohepatitis. The company's work leverages the recent scientific advancements in microRNA research, which play a crucial role in regulating gene expression.

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.

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.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

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.

Nightstar Therapeutics is a clinical-stage gene therapy company based in London, United Kingdom, specializing in the development of innovative one-time treatments for rare inherited retinal diseases. The company is advancing several product candidates, including its lead candidate NSR-REP1, which is in phase 3 clinical development for choroideremia. Additionally, Nightstar is working on NSR-RPGR, currently in phase 2/3 trials for X-linked retinitis pigmentosa, as well as NSR-BEST1. The company is also developing NSR-ABCA4, which is in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address significant unmet medical needs in the field of retinal disorders.

Personal Genome Diagnostics, Inc. is a biotechnology company focused on patient-specific analyses of cancer genomes. Established in 2010 and headquartered in Baltimore, Maryland, the company has pioneered technologies for non-invasive "liquid biopsy" approaches, allowing for the detection of tumor-specific alterations in circulating cell-free DNA. Its offerings include the METDetect Assay for identifying MET gene amplifications, LungSelect for detecting actionable genetic alterations in non-small cell lung cancer patients, and PGDx elio plasma resolve, which analyzes various genetic mutations from plasma samples. Additionally, Personal Genome Diagnostics provides RNAcomplete, enabling researchers to extract both RNA and genomic DNA from a single tissue sample, and CancerXOME, which captures and analyzes coding regions of genes. The company collaborates with institutions like Mayo Clinic and partners with KingMed Diagnostics to enhance its research and diagnostic capabilities, thereby empowering oncologists and researchers with crucial genomic insights to combat cancer.

Founded in 2006, 23andMe is a biotechnology company specializing in direct-to-consumer genetic testing. It offers DNA analysis services through home-based saliva collection kits, providing insights into ancestry, traits, and health risks.

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.

Proteostasis Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule therapies that regulate the body's protein homeostasis network to treat cystic fibrosis and other genetic or degenerative diseases. It develops Proteostasis Regulators intended to restore proteostasis balance and address conditions such as cystic fibrosis, emphysema, diabetes, and neurodegenerative disorders. In CF, its lead candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. The company collaborates with the Cystic Fibrosis Foundation to research, develop, and commercialize CF-related products, and has licensing relationships with Genentech. It was founded in 2006, formerly known as Proteoguard, and was renamed Proteostasis Therapeutics in 2007, based in Boston. It was acquired by Yumanity Therapeutics in 2020 in a reverse merger.

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.

ARCA biopharma, Inc. is a Colorado-based biopharmaceutical company developing genetically-targeted therapies for cardiovascular diseases. ARCA’s principal focus is to develop personalized therapies for the treatment of cardiovascular disease through the use of genetics. The Company’s business focus combines expertise in cardiovascular pathophysiology, molecular genetics and clinical development.

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.

CRISPR Therapeutics specializes in developing transformative gene-based medicines using its proprietary CRISPR/Cas9 gene-editing platform. This technology enables precise, directed changes to genomic DNA for treating serious human diseases. The company focuses on various disease areas including hemoglobinopathies, oncology, regenerative medicine, and rare diseases.

CRISPR Therapeutics specializes in developing transformative gene-based medicines using its proprietary CRISPR/Cas9 gene-editing platform. This technology enables precise, directed changes to genomic DNA for treating serious human diseases. The company focuses on various disease areas including hemoglobinopathies, oncology, regenerative medicine, and rare diseases.

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.

Interleukin Genetics, Inc. is a company specializing in personalized health through the development of genetic tests aimed at enhancing individual wellness. The organization focuses on two primary areas: the provision of genetic test products for personal health improvement and research and development efforts that create tests linked to partners’ products for medical and dental markets. Their genetic testing technology provides clear insights into wellness, offering personalized guidance on diet and exercise choices, which empowers individuals to take proactive steps in managing their health. Through partnerships and direct sales channels, Interleukin Genetics aims to contribute to the growing personalized health market by enabling individuals to make informed decisions about their health and wellness.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

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.

Edimer Pharmaceuticals is a biotechnology company focused on developing innovative treatments for X-linked Hypohydrotic Ectodermal Dysplasia (XLHED), a rare genetic disorder characterized by symptoms such as the absence of sweat glands, impaired temperature regulation, respiratory issues, and malformations of hair and teeth. The company's lead product candidate, EDI200, aims to provide significant and lasting improvements in health and quality of life for individuals affected by this condition. Edimer is guided by a team of experienced professionals in the biotechnology sector, supported by a network of distinguished clinical and scientific advisors, all committed to addressing the needs of families impacted by XLHED.

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.

Founded in 2006, 23andMe is a biotechnology company specializing in direct-to-consumer genetic testing. It offers DNA analysis services through home-based saliva collection kits, providing insights into ancestry, traits, and health risks.

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.

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.

Founded in 2006, 23andMe is a biotechnology company specializing in direct-to-consumer genetic testing. It offers DNA analysis services through home-based saliva collection kits, providing insights into ancestry, traits, and health risks.

Predictive Biosciences, Inc. is a molecular diagnostics company focused on developing diagnostic products aimed at enhancing cancer management. Founded in 2006 and headquartered in Lexington, Massachusetts, the company specializes in proteomics and oncology, particularly in the area of tumor biomarkers. Predictive Biosciences offers a range of assays that detect urinary biomarkers, which are indicative of the physiological changes associated with cancer development and progression. Among its key products are the Triage Monitoring Assay, which enables physicians to postpone invasive diagnostic procedures, the Interval Monitoring Assay for cancer surveillance between follow-up appointments, and the Stratification Assay to assist physicians in managing patient care decisions.

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.

Nova IVF Fertility operates a network of fertility centers across India, providing a comprehensive range of services related to assisted reproductive technology (ART). Established in 2011 and based in Bengaluru, the company offers various treatments including endoscopy, andrology, infertility counseling and assessment, intrauterine insemination (IUI), in vitro fertilization (IVF), and embryo or sperm donation. Additionally, it provides advanced options like preimplantation genetic screening and assisted hatching. Nova IVF Fertility was created through a collaboration with IVI, a prominent leader in reproductive medicine, to enhance the quality of IVF treatments available in India. The organization aims to raise awareness about infertility as a medical condition that requires treatment, addressing the gap in public knowledge regarding fertility issues. By emphasizing standardized and ethical practices, Nova IVF Fertility seeks to meet the growing demand for infertility services in a country where many couples still remain unaware of their options.

Epizyme, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to the discovery, development, and commercialization of innovative epigenetic medicines for cancer and other serious diseases. Founded in 2007, the company has developed Tazemetostat, an approved treatment for metastatic or locally advanced epithelioid sarcoma, and is exploring its use in various combinations for other malignancies, including follicular lymphoma, diffuse large B-cell lymphoma, and platinum-resistant solid tumors. In addition to Tazemetostat, Epizyme is advancing other drug candidates, such as pinometostat for acute myeloid leukemia and acute lymphoblastic leukemia, and inhibitors targeting PRMT5 and PRMT1 for various solid tumors and blood cancers. The company collaborates with several prominent organizations in the pharmaceutical sector to enhance its research and development efforts.

Founded in 2006, 23andMe is a biotechnology company specializing in direct-to-consumer genetic testing. It offers DNA analysis services through home-based saliva collection kits, providing insights into ancestry, traits, and health risks.

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.

Predictive Biosciences, Inc. is a molecular diagnostics company focused on developing diagnostic products aimed at enhancing cancer management. Founded in 2006 and headquartered in Lexington, Massachusetts, the company specializes in proteomics and oncology, particularly in the area of tumor biomarkers. Predictive Biosciences offers a range of assays that detect urinary biomarkers, which are indicative of the physiological changes associated with cancer development and progression. Among its key products are the Triage Monitoring Assay, which enables physicians to postpone invasive diagnostic procedures, the Interval Monitoring Assay for cancer surveillance between follow-up appointments, and the Stratification Assay to assist physicians in managing patient care decisions.

Proteostasis Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule therapies that regulate the body's protein homeostasis network to treat cystic fibrosis and other genetic or degenerative diseases. It develops Proteostasis Regulators intended to restore proteostasis balance and address conditions such as cystic fibrosis, emphysema, diabetes, and neurodegenerative disorders. In CF, its lead candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. The company collaborates with the Cystic Fibrosis Foundation to research, develop, and commercialize CF-related products, and has licensing relationships with Genentech. It was founded in 2006, formerly known as Proteoguard, and was renamed Proteostasis Therapeutics in 2007, based in Boston. It was acquired by Yumanity Therapeutics in 2020 in a reverse merger.

Founded in 2006, 23andMe is a biotechnology company specializing in direct-to-consumer genetic testing. It offers DNA analysis services through home-based saliva collection kits, providing insights into ancestry, traits, and health risks.

Amicus Therapeutics is a biotechnology company focused on discovering, developing, and delivering therapies for rare and orphan diseases. It specializes in oral pharmacological chaperones—small molecules that bind, stabilize, and enhance activity of misfolded enzymes—to treat lysosomal storage disorders such as Fabry and Pompe disease, and it explores combination approaches with enzyme replacement therapy. The company maintains a growing pipeline targeting additional lysosomal diseases, including Gaucher, and collaborates with academic and clinical partners to advance its programs. Amicus was founded in 2002 and is headquartered in Cranbury, New Jersey.

Amicus Therapeutics is a biotechnology company focused on discovering, developing, and delivering therapies for rare and orphan diseases. It specializes in oral pharmacological chaperones—small molecules that bind, stabilize, and enhance activity of misfolded enzymes—to treat lysosomal storage disorders such as Fabry and Pompe disease, and it explores combination approaches with enzyme replacement therapy. The company maintains a growing pipeline targeting additional lysosomal diseases, including Gaucher, and collaborates with academic and clinical partners to advance its programs. Amicus was founded in 2002 and is headquartered in Cranbury, New Jersey.

Amicus Therapeutics is a biotechnology company focused on discovering, developing, and delivering therapies for rare and orphan diseases. It specializes in oral pharmacological chaperones—small molecules that bind, stabilize, and enhance activity of misfolded enzymes—to treat lysosomal storage disorders such as Fabry and Pompe disease, and it explores combination approaches with enzyme replacement therapy. The company maintains a growing pipeline targeting additional lysosomal diseases, including Gaucher, and collaborates with academic and clinical partners to advance its programs. Amicus was founded in 2002 and is headquartered in Cranbury, New Jersey.

Genetic Immunity is a US-Hungarian clinical-stage biotech company developing immunotherapeutic biologics for treating chronic viral infections, cancer, and allergies. Their proprietary platform combines plasmid DNA-based antigens, nanomedicine formulations, and topical administration devices targeting antigens to dendritic cells. Lead product DermaVir, an HIV therapeutic vaccine, has shown promising results in clinical trials, boosting HIV-specific T-cell responses and reducing infected cell counts with a favorable safety profile. The company's pipeline includes ChlamyDerm for Chlamydia treatment and DermAll for allergen-specific immunotherapy, both demonstrating positive preclinical outcomes. Genetic Immunity aims to modify disease progression, decrease drug exposure, and ultimately achieve remission in patients.

Vertex Pharmaceuticals is a global biotechnology company focused on discovering and developing small-molecule therapies for serious diseases. Its pipeline covers viral diseases, cystic fibrosis, inflammation and autoimmune disorders, cancer, and pain, with efforts to commercialize products independently and in collaboration with other pharmaceutical companies. In addition to traditional small molecules, Vertex is pursuing gene-editing therapies and non-opioid pain treatments, and it conducts research into APOL1-associated kidney diseases and cell therapies.

Life Technologies Corporation is a biotechnology tools company formed in November 2008 from the merger of Invitrogen Corporation and Applied Biosystems, Inc. The company operates under these well-known brands, providing a comprehensive range of systems, consumables, and services that facilitate scientific research and development. Invitrogen specializes in reagents and consumables applicable across various platforms, while Applied Biosystems offers integrated solutions in critical areas such as polymerase chain reaction (PCR), genetic sequencing, and mass spectrometry. Life Technologies' diverse product portfolio includes technologies for capillary electrophoresis-based sequencing, sample preparation, cell culture, RNA interference analysis, functional genomics, proteomics, and cell biology, in addition to applications in clinical diagnostics and water testing analysis. Through its offerings, Life Technologies supports researchers in accelerating their scientific endeavors.