New Enterprise Associates

Tune Therapeutics is a biotechnology company focused on developing innovative cell and gene therapies. It employs an epigenetic programming platform to create novel therapeutic pathways, aiming to treat severe diseases without modifying the underlying DNA sequence.

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

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.

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

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 is a biotechnology company focused on developing innovative cell and gene therapies. It employs an epigenetic programming platform to create novel therapeutic pathways, aiming to treat severe diseases without modifying the underlying DNA sequence.

AviadoBio is a biotechnology company focused on developing transformative gene therapies for neurodegenerative disorders, specifically targeting diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The company leverages innovative research from King’s College London and the UK Dementia Research Institute to create its therapeutic platform. By integrating a deep understanding of brain biology with proprietary gene therapies, AviadoBio aims to effectively address the challenges of delivering treatments to the appropriate areas within the nervous system. The ultimate goal is to enhance the therapeutic potential of gene therapy, potentially halting or even reversing the progression of these debilitating conditions, thereby improving the quality of life for affected individuals.

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.

Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for the treatment of severe diseases affecting the muscle and central nervous system (CNS). Established in 2019 and based in Waltham, Massachusetts, the company specializes in creating adeno-associated virus (AAV) vectors and utilizes a platform that integrates synthetic and systems biology with high-throughput screening techniques. This innovative approach allows Affinia Therapeutics to design novel gene therapies with enhanced properties tailored to address significant unmet medical needs. The company aims to provide healthcare professionals with advanced therapeutic options for patients suffering from debilitating conditions.

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.

Monte Rosa Therapeutics is a biotechnology company dedicated to developing innovative cancer therapeutics through the modulation of protein degradation pathways. Specializing in molecular glue degraders (MGDs), the company utilizes the body's natural mechanisms for protein destruction to selectively degrade proteins that are relevant to therapeutic outcomes. Employing its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, Monte Rosa identifies target proteins for degradation by MGDs from its extensive library of over 50,000 molecules. The company's most advanced product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for the treatment of MYC-driven tumors. Monte Rosa Therapeutics aims to deliver pioneering therapies for cancer and potentially other diseases through its innovative approaches.

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

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.

Monte Rosa Therapeutics is a biotechnology company dedicated to developing innovative cancer therapeutics through the modulation of protein degradation pathways. Specializing in molecular glue degraders (MGDs), the company utilizes the body's natural mechanisms for protein destruction to selectively degrade proteins that are relevant to therapeutic outcomes. Employing its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, Monte Rosa identifies target proteins for degradation by MGDs from its extensive library of over 50,000 molecules. The company's most advanced product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for the treatment of MYC-driven tumors. Monte Rosa Therapeutics aims to deliver pioneering therapies for cancer and potentially other diseases through its innovative approaches.

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.

Monte Rosa Therapeutics is a biotechnology company dedicated to developing innovative cancer therapeutics through the modulation of protein degradation pathways. Specializing in molecular glue degraders (MGDs), the company utilizes the body's natural mechanisms for protein destruction to selectively degrade proteins that are relevant to therapeutic outcomes. Employing its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, Monte Rosa identifies target proteins for degradation by MGDs from its extensive library of over 50,000 molecules. The company's most advanced product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for the treatment of MYC-driven tumors. Monte Rosa Therapeutics aims to deliver pioneering therapies for cancer and potentially other diseases through its innovative approaches.

Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for the treatment of severe diseases affecting the muscle and central nervous system (CNS). Established in 2019 and based in Waltham, Massachusetts, the company specializes in creating adeno-associated virus (AAV) vectors and utilizes a platform that integrates synthetic and systems biology with high-throughput screening techniques. This innovative approach allows Affinia Therapeutics to design novel gene therapies with enhanced properties tailored to address significant unmet medical needs. The company aims to provide healthcare professionals with advanced therapeutic options for patients suffering from debilitating conditions.

Akouos, Inc. is a biotechnology company dedicated to developing precision genetic medicine aimed at restoring, improving, and preserving hearing for individuals with various forms of hearing loss. Utilizing a proprietary adeno-associated viral vector library and an innovative delivery approach, Akouos focuses on gene therapies tailored to genetically-defined patient populations, including those affected by specific genetic mutations, ototoxic drug exposure, and age-related hearing loss. The company's lead product candidate, AK-OTOF, targets hearing loss associated with mutations in the OTOF gene. Founded in 2016 and based in Boston, Massachusetts, Akouos collaborates with strategic partners, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing effective treatments for sensorineural hearing loss.

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.

Akouos, Inc. is a biotechnology company dedicated to developing precision genetic medicine aimed at restoring, improving, and preserving hearing for individuals with various forms of hearing loss. Utilizing a proprietary adeno-associated viral vector library and an innovative delivery approach, Akouos focuses on gene therapies tailored to genetically-defined patient populations, including those affected by specific genetic mutations, ototoxic drug exposure, and age-related hearing loss. The company's lead product candidate, AK-OTOF, targets hearing loss associated with mutations in the OTOF gene. Founded in 2016 and based in Boston, Massachusetts, Akouos collaborates with strategic partners, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing effective treatments for sensorineural hearing loss.

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

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, Inc. is a biotechnology company dedicated to developing precision genetic medicine aimed at restoring, improving, and preserving hearing for individuals with various forms of hearing loss. Utilizing a proprietary adeno-associated viral vector library and an innovative delivery approach, Akouos focuses on gene therapies tailored to genetically-defined patient populations, including those affected by specific genetic mutations, ototoxic drug exposure, and age-related hearing loss. The company's lead product candidate, AK-OTOF, targets hearing loss associated with mutations in the OTOF gene. Founded in 2016 and based in Boston, Massachusetts, Akouos collaborates with strategic partners, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing effective treatments for sensorineural hearing loss.

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.

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.

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.

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.

23andMe, Inc. is a consumer genetics and research company headquartered in Sunnyvale, California, founded in 2006. It specializes in providing direct-to-consumer personal genome services through home-based saliva collection kits. The company offers a range of services, including Health + Ancestry, which provides insights into ancestry, traits, and health, and Ancestry + Traits, which details ancestry breakdown and various trait reports. 23andMe aims to help individuals understand their genetic makeup, enabling users to explore their ancestry, genealogy, and inherited traits. Additionally, the company markets its services to researchers and scientists, offering categorized and searchable genetic data. With a focus on consumer and research services, 23andMe is positioned within the rapidly growing biotech and healthcare sectors, contributing to the understanding of genetics and its implications for health.

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, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics aimed at treating cystic fibrosis (CF) and other genetic and degenerative disorders. The company specializes in discovering small molecule therapeutics that regulate the Proteostasis Network, which is essential for maintaining the balance of proteins in the body. Its lead product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis Therapeutics employs a theratyping approach to tailor treatments based on individual responses, regardless of specific CFTR mutations. The company has established collaboration agreements with organizations such as the Cystic Fibrosis Foundation to advance research and commercialization efforts for CF and other pulmonary diseases. Founded in 2006 and headquartered in Boston, Massachusetts, Proteostasis Therapeutics was previously known as Proteoguard, Inc. before rebranding in 2007.

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.

CRISPR Therapeutics AG is a biotechnology company based in Zug, Switzerland, dedicated to developing transformative gene-based medicines for serious human diseases using CRISPR/Cas9 technology. This revolutionary gene-editing platform allows for precise changes to genomic DNA. The company's portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Their lead product candidate is CTX001, a gene-edited therapy for transfusion-dependent beta thalassemia or severe sickle cell disease, which has been approved as Casgevy in collaboration with Vertex Pharmaceuticals. Additionally, CRISPR Therapeutics is developing CTX110, an allogeneic CAR-T therapy targeting CD19-positive malignancies, and other programs like CTX120 for multiple myeloma and CTX130 for solid tumors and hematologic malignancies. The company also engages in regenerative medicine programs for diabetes and genetic disease treatments such as glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.

CRISPR Therapeutics AG is a biotechnology company based in Zug, Switzerland, dedicated to developing transformative gene-based medicines for serious human diseases using CRISPR/Cas9 technology. This revolutionary gene-editing platform allows for precise changes to genomic DNA. The company's portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Their lead product candidate is CTX001, a gene-edited therapy for transfusion-dependent beta thalassemia or severe sickle cell disease, which has been approved as Casgevy in collaboration with Vertex Pharmaceuticals. Additionally, CRISPR Therapeutics is developing CTX110, an allogeneic CAR-T therapy targeting CD19-positive malignancies, and other programs like CTX120 for multiple myeloma and CTX130 for solid tumors and hematologic malignancies. The company also engages in regenerative medicine programs for diabetes and genetic disease treatments such as glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.

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.

23andMe, Inc. is a consumer genetics and research company headquartered in Sunnyvale, California, founded in 2006. It specializes in providing direct-to-consumer personal genome services through home-based saliva collection kits. The company offers a range of services, including Health + Ancestry, which provides insights into ancestry, traits, and health, and Ancestry + Traits, which details ancestry breakdown and various trait reports. 23andMe aims to help individuals understand their genetic makeup, enabling users to explore their ancestry, genealogy, and inherited traits. Additionally, the company markets its services to researchers and scientists, offering categorized and searchable genetic data. With a focus on consumer and research services, 23andMe is positioned within the rapidly growing biotech and healthcare sectors, contributing to the understanding of genetics and its implications for health.

23andMe, Inc. is a consumer genetics and research company headquartered in Sunnyvale, California, founded in 2006. It specializes in providing direct-to-consumer personal genome services through home-based saliva collection kits. The company offers a range of services, including Health + Ancestry, which provides insights into ancestry, traits, and health, and Ancestry + Traits, which details ancestry breakdown and various trait reports. 23andMe aims to help individuals understand their genetic makeup, enabling users to explore their ancestry, genealogy, and inherited traits. Additionally, the company markets its services to researchers and scientists, offering categorized and searchable genetic data. With a focus on consumer and research services, 23andMe is positioned within the rapidly growing biotech and healthcare sectors, contributing to the understanding of genetics and its implications for health.

23andMe, Inc. is a consumer genetics and research company headquartered in Sunnyvale, California, founded in 2006. It specializes in providing direct-to-consumer personal genome services through home-based saliva collection kits. The company offers a range of services, including Health + Ancestry, which provides insights into ancestry, traits, and health, and Ancestry + Traits, which details ancestry breakdown and various trait reports. 23andMe aims to help individuals understand their genetic makeup, enabling users to explore their ancestry, genealogy, and inherited traits. Additionally, the company markets its services to researchers and scientists, offering categorized and searchable genetic data. With a focus on consumer and research services, 23andMe is positioned within the rapidly growing biotech and healthcare sectors, contributing to the understanding of genetics and its implications for health.

Sunesis Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing targeted inhibitors for the treatment of hematologic and solid cancers. Its lead product candidate, vecabrutinib, is a non-covalent inhibitor of Bruton’s tyrosine kinase (BTK) currently undergoing Phase 1b/2 clinical trials for chronic lymphocytic leukemia, mantle cell lymphoma, and other B-cell malignancies. Additionally, Sunesis is advancing SNS-510, which is in preclinical studies for various tumor types, alongside partnered programs such as TAK-580, a pan-Raf inhibitor in Phase 1 trials for pediatric low-grade glioma, and vosaroxin, an anti-cancer quinolone derivative. The company has established collaborations, including an agreement with Biogen Idec MA, Inc. for small molecule BTK inhibitors and a licensing agreement with Takeda Pharmaceutical Company Limited for preclinical PDK1 inhibitors. Founded in 1998 and headquartered in South San Francisco, California, Sunesis Pharmaceuticals is committed to innovative approaches in the ongoing fight against cancer.

Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics aimed at treating cystic fibrosis (CF) and other genetic and degenerative disorders. The company specializes in discovering small molecule therapeutics that regulate the Proteostasis Network, which is essential for maintaining the balance of proteins in the body. Its lead product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis Therapeutics employs a theratyping approach to tailor treatments based on individual responses, regardless of specific CFTR mutations. The company has established collaboration agreements with organizations such as the Cystic Fibrosis Foundation to advance research and commercialization efforts for CF and other pulmonary diseases. Founded in 2006 and headquartered in Boston, Massachusetts, Proteostasis Therapeutics was previously known as Proteoguard, Inc. before rebranding in 2007.

23andMe, Inc. is a consumer genetics and research company headquartered in Sunnyvale, California, founded in 2006. It specializes in providing direct-to-consumer personal genome services through home-based saliva collection kits. The company offers a range of services, including Health + Ancestry, which provides insights into ancestry, traits, and health, and Ancestry + Traits, which details ancestry breakdown and various trait reports. 23andMe aims to help individuals understand their genetic makeup, enabling users to explore their ancestry, genealogy, and inherited traits. Additionally, the company markets its services to researchers and scientists, offering categorized and searchable genetic data. With a focus on consumer and research services, 23andMe is positioned within the rapidly growing biotech and healthcare sectors, contributing to the understanding of genetics and its implications for health.

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.