New Enterprise Associates

Tune Therapeutics is focused on developing innovative cell and gene therapies that utilize epigenetic programming to address serious diseases. By harnessing the epigenome, the company aims to create new pathways for treatment without making changes to the DNA sequence itself. This approach allows for the potential development of therapies for complex and widespread medical conditions, positioning Tune Therapeutics at the forefront of advancements in the field of epigenetics.

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, an off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A, a treatment for 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, an off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A, a treatment for 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, focused on developing innovative gene therapies using its proprietary intein platform technology. Founded in 2012 and originally named Proteodesign, S.L., the company aims to address the needs of patients suffering from incurable genetic diseases. The intein platform, developed from research in the Muir Lab at Princeton University, seeks to overcome two significant limitations of traditional adeno-associated virus (AAV) therapies: it enhances the capacity for larger gene delivery and broadens the range of tissues that can be targeted for treatment.

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 focused on developing innovative cell and gene therapies that utilize epigenetic programming to address serious diseases. By harnessing the epigenome, the company aims to create new pathways for treatment without making changes to the DNA sequence itself. This approach allows for the potential development of therapies for complex and widespread medical conditions, positioning Tune Therapeutics at the forefront of advancements in the field of epigenetics.

AviadoBio is focused on developing innovative gene therapies aimed at transforming the lives of individuals affected by neurodegenerative disorders, specifically frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The company leverages advanced research from King's College London and the UK Dementia Research Institute to create its therapeutic platform. By integrating a comprehensive understanding of brain biology with proprietary gene therapy techniques, AviadoBio seeks to address the challenges of effectively delivering treatments to targeted areas in the brain. This approach aims to enhance the therapeutic potential of gene therapies, offering hope for halting or potentially reversing the progression of these debilitating diseases.

Shape Therapeutics, Inc. is a biotechnology company founded in 2018 and based in Seattle, Washington. The company specializes in RNA-editing gene therapy targeting neurodegenerative disorders, oncology, metabolic conditions, and rare genetic diseases. Its proprietary RNAfix technology enables precise editing of RNA using the body's natural cellular mechanisms, which minimizes risks associated with immunogenicity, cellular toxicity, and off-target DNA editing. Shape Therapeutics employs a comprehensive approach to RNA gene therapy, integrating innovative payload, delivery, and manufacturing platform technologies. This strategy aims to facilitate the development of effective treatments and potential cures for a wide range of diseases, with the goal of enhancing patient outcomes across the healthcare landscape.

Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.

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 focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

Personal Genome Diagnostics, Inc. specializes in patient-specific cancer genome analysis using advanced digital characterization and monitoring technologies. Founded in 2010 and based in Baltimore, Maryland, the company offers a range of products and services aimed at improving cancer diagnosis and treatment. Their METDetect Assay detects MET gene amplifications in cancer patients' circulation, providing detailed reports on tumor-specific alterations. The LungSelect product identifies actionable genetic changes in plasma from non-small cell lung cancer patients, while PGDx elio plasma resolve is a diagnostic test that detects various genetic alterations in circulating cell-free DNA. Additionally, the company offers RNAcomplete, which allows for the extraction of total RNA and genomic DNA from single tissue samples, and CancerXOME, which analyzes the coding regions of numerous genes. Personal Genome Diagnostics collaborates with institutions like Mayo Clinic and has partnerships with KingMed Diagnostics to further enhance cancer genome research and diagnostics.

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, an off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A, a treatment for hepatocellular carcinoma.

Reneo Pharmaceuticals is a clinical-stage pharmaceutical company based in San Diego, California, that focuses on developing therapies for patients with rare genetic mitochondrial diseases. These conditions are linked to defects in energy metabolism, leading to issues such as muscle weakness and wasting. The company is developing REN001, an oral drug designed to modulate genes involved in mitochondrial function, aiming to enhance adenosine triphosphate (ATP) production, which is essential for cellular energy. By improving mitochondrial activity, Reneo seeks to enhance the daily functioning and quality of life for patients suffering from these disorders. Founded in 2014, Reneo Pharmaceuticals is committed to addressing the needs of individuals with orphan metabolic diseases.

Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

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 focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

Shape Therapeutics, Inc. is a biotechnology company founded in 2018 and based in Seattle, Washington. The company specializes in RNA-editing gene therapy targeting neurodegenerative disorders, oncology, metabolic conditions, and rare genetic diseases. Its proprietary RNAfix technology enables precise editing of RNA using the body's natural cellular mechanisms, which minimizes risks associated with immunogenicity, cellular toxicity, and off-target DNA editing. Shape Therapeutics employs a comprehensive approach to RNA gene therapy, integrating innovative payload, delivery, and manufacturing platform technologies. This strategy aims to facilitate the development of effective treatments and potential cures for a wide range of diseases, with the goal of enhancing patient outcomes across the healthcare landscape.

Reneo Pharmaceuticals is a clinical-stage pharmaceutical company based in San Diego, California, that focuses on developing therapies for patients with rare genetic mitochondrial diseases. These conditions are linked to defects in energy metabolism, leading to issues such as muscle weakness and wasting. The company is developing REN001, an oral drug designed to modulate genes involved in mitochondrial function, aiming to enhance adenosine triphosphate (ATP) production, which is essential for cellular energy. By improving mitochondrial activity, Reneo seeks to enhance the daily functioning and quality of life for patients suffering from these disorders. Founded in 2014, Reneo Pharmaceuticals is committed to addressing the needs of individuals with orphan metabolic diseases.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies 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, an off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A, a treatment for hepatocellular carcinoma.

Personal Genome Diagnostics, Inc. specializes in patient-specific cancer genome analysis using advanced digital characterization and monitoring technologies. Founded in 2010 and based in Baltimore, Maryland, the company offers a range of products and services aimed at improving cancer diagnosis and treatment. Their METDetect Assay detects MET gene amplifications in cancer patients' circulation, providing detailed reports on tumor-specific alterations. The LungSelect product identifies actionable genetic changes in plasma from non-small cell lung cancer patients, while PGDx elio plasma resolve is a diagnostic test that detects various genetic alterations in circulating cell-free DNA. Additionally, the company offers RNAcomplete, which allows for the extraction of total RNA and genomic DNA from single tissue samples, and CancerXOME, which analyzes the coding regions of numerous genes. Personal Genome Diagnostics collaborates with institutions like Mayo Clinic and has partnerships with KingMed Diagnostics to further enhance cancer genome research and diagnostics.

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 based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

Cydan is an orphan drug accelerator focused on developing therapies for patients with rare genetic diseases. The organization identifies and mitigates the risks associated with compounds that have therapeutic and commercial potential. Through a rigorous process, Cydan generates data to support the development of new therapies and the formation of new companies. Their experienced team leverages strong relationships with academia and patient advocacy groups, along with deep expertise in drug development and a proven track record of successful product commercialization. By guiding therapies through clinical, regulatory, and commercial development, 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 plc is a clinical-stage gene therapy company based in London, focused on developing innovative one-time treatments for rare inherited retinal diseases. The company’s lead product candidate, NSR-REP1, is currently in phase 3 clinical development for choroideremia, while NSR-RPGR is undergoing phase 2/3 trials for X-linked retinitis pigmentosa. Additionally, Nightstar is developing NSR-BEST1 and NSR-ABCA4, with the latter in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address the unmet medical needs of patients suffering from these conditions.

Vtesse, Inc. is a biopharmaceutical company based in Gaithersburg, Maryland, focused on developing treatments for patients with rare diseases. The company is conducting clinical studies for VTS-270, aimed at treating Niemann-Pick disease type C (NPC), and is also engaged in pre-clinical discovery and development of additional therapies for NPC and other lysosomal storage diseases. Vtesse originated as the first spin-out from Cydan Development, an orphan-drug accelerator that identifies promising therapeutic programs. The company collaborates with the National Institutes of Health (NIH) to advance its clinical studies and is supported by a consortium of experienced investors. Vtesse's management team has a strong background, having participated in the development of over 20 approved drugs and vaccines, which underscores its commitment to addressing the needs of underserved patient populations.

Nightstar Therapeutics plc is a clinical-stage gene therapy company based in London, focused on developing innovative one-time treatments for rare inherited retinal diseases. The company’s lead product candidate, NSR-REP1, is currently in phase 3 clinical development for choroideremia, while NSR-RPGR is undergoing phase 2/3 trials for X-linked retinitis pigmentosa. Additionally, Nightstar is developing NSR-BEST1 and NSR-ABCA4, with the latter in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address the unmet medical needs of patients suffering from these conditions.

Personal Genome Diagnostics, Inc. specializes in patient-specific cancer genome analysis using advanced digital characterization and monitoring technologies. Founded in 2010 and based in Baltimore, Maryland, the company offers a range of products and services aimed at improving cancer diagnosis and treatment. Their METDetect Assay detects MET gene amplifications in cancer patients' circulation, providing detailed reports on tumor-specific alterations. The LungSelect product identifies actionable genetic changes in plasma from non-small cell lung cancer patients, while PGDx elio plasma resolve is a diagnostic test that detects various genetic alterations in circulating cell-free DNA. Additionally, the company offers RNAcomplete, which allows for the extraction of total RNA and genomic DNA from single tissue samples, and CancerXOME, which analyzes the coding regions of numerous genes. Personal Genome Diagnostics collaborates with institutions like Mayo Clinic and has partnerships with KingMed Diagnostics to further enhance cancer genome research and diagnostics.

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 for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.

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 conducting clinical studies for VTS-270, aimed at treating Niemann-Pick disease type C (NPC), and is also engaged in pre-clinical discovery and development of additional therapies for NPC and other lysosomal storage diseases. Vtesse originated as the first spin-out from Cydan Development, an orphan-drug accelerator that identifies promising therapeutic programs. The company collaborates with the National Institutes of Health (NIH) to advance its clinical studies and is supported by a consortium of experienced investors. Vtesse's management team has a strong background, having participated in the development of over 20 approved drugs and vaccines, which underscores its commitment to addressing the needs of underserved patient populations.

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.

Loxo Oncology, Inc. is a biopharmaceutical company based in Stamford, Connecticut, focused on developing and commercializing targeted therapies for genetically defined cancers in the United States. Founded in 2013, the company aims to bring innovative cancer treatments to the clinic by identifying actionable "driver mutations" in tumors. Its lead product candidate, larotrectinib, is an oral selective inhibitor of tropomyosin receptor kinase (TRK) and is undergoing various clinical trials for multiple cancer types, including lung, melanoma, and breast cancer. Loxo Oncology is also advancing several preclinical programs, including candidates targeting RET fusion proteins and FGFR isoforms. The company has established collaborations with several organizations to enhance its drug discovery efforts and optimize its clinical development strategies. As a subsidiary of Eli Lilly and Company, Loxo Oncology is dedicated to creating effective therapies that address specific genetic vulnerabilities in cancer patients.

Cydan is an orphan drug accelerator focused on developing therapies for patients with rare genetic diseases. The organization identifies and mitigates the risks associated with compounds that have therapeutic and commercial potential. Through a rigorous process, Cydan generates data to support the development of new therapies and the formation of new companies. Their experienced team leverages strong relationships with academia and patient advocacy groups, along with deep expertise in drug development and a proven track record of successful product commercialization. By guiding therapies through clinical, regulatory, and commercial development, Cydan aims to improve the lives of patients and families affected by rare diseases.

Edimer Pharmaceuticals focuses on developing EDI200 as a treatment for X-linked Hypohydrotic Ectodermal Dysplasia (XLHED), a rare genetic disorder characterized by symptoms such as insufficient sweat glands, impaired temperature regulation, respiratory difficulties, and abnormal hair and tooth development. The company aims to enhance the health and quality of life for individuals affected by this orphan disease. Edimer is supported by a team of experienced biotechnology professionals and a network of esteemed clinical and scientific advisors, all committed to advancing drug development for XLHED. Through its innovative approach, Edimer seeks to provide significant and lasting improvements in the management of this condition for future generations.

Cydan is an orphan drug accelerator focused on developing therapies for patients with rare genetic diseases. The organization identifies and mitigates the risks associated with compounds that have therapeutic and commercial potential. Through a rigorous process, Cydan generates data to support the development of new therapies and the formation of new companies. Their experienced team leverages strong relationships with academia and patient advocacy groups, along with deep expertise in drug development and a proven track record of successful product commercialization. By guiding therapies through clinical, regulatory, and commercial development, 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.

Prosensa, also known as Ribpharm Inc, is a biotech company specializing in the discovery, development, and commercialization of RNA-based therapeutics. The company is particularly focused on addressing genetic disorders, with an emphasis on neuromuscular conditions, as well as anti-infectives and oncology. Prosensa aims to leverage its strong intellectual property position to capitalize on emerging market opportunities in the healthcare sector. To optimize its operations, the company adopts a focused business model by outsourcing most activities to specialized organizations. Through its innovative approach, Prosensa seeks to bring novel and commercially viable therapeutic products to market.

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.

Zyngenia, Inc. is a privately held biotherapeutics company established in 2008 that specializes in developing next-generation antibody-derived drugs. The company utilizes proprietary technology to create unique molecular entities, known as Zybody™, which combine the therapeutic activities of multiple biologic treatments into a single protein. This innovative approach allows Zyngenia to target complex diseases, particularly cancer and autoimmune disorders, by simultaneously addressing multiple biological pathways. By doing so, Zyngenia aims to facilitate quicker patient treatment initiation and improve overall clinical outcomes.

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 treating various cancers, both hematologic and solid. Its lead candidate, vecabrutinib, is a non-covalent Bruton’s tyrosine kinase (BTK) inhibitor currently undergoing Phase 1b/2 clinical trials for chronic lymphocytic leukemia, mantle cell lymphoma, and other B-cell malignancies. The company is also advancing SNS-510, which is in preclinical studies for solid tumors and hematologic malignancies. Additionally, Sunesis is involved in partnered programs, including TAK-580, a pan-Raf inhibitor in Phase 1 trials for pediatric low-grade glioma, and vosaroxin, an anti-cancer agent that affects DNA and inhibits topoisomerase II. Collaborative efforts with Biogen Idec and Takeda Pharmaceutical focus on developing small molecule BTK inhibitors and preclinical PDK1 inhibitors, respectively. Founded in 1998, Sunesis Pharmaceuticals is headquartered in South San Francisco, California.

Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.

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 clinical-stage biotechnology company based in the United States and Hungary, specializing in the development of immunotherapeutic biologics aimed at treating chronic viral infections, cancer, and allergies. The company’s Immune Therapies are designed to enhance specific immune responses, offering potential modifications or control over currently incurable diseases. Their proprietary technology includes a plasmid DNA-based antigen, a nanomedicine formulation, and a topical administration device that efficiently targets antigens to dendritic cells. Notably, Genetic Immunity’s lead product candidate, DermaVir, is a therapeutic HIV vaccine that aims to boost immune responses to eliminate HIV-infected cells, showing promise in clinical trials with an excellent safety profile. In addition to DermaVir, the company has a diverse pipeline, including candidates like ChlamyDerm for Chlamydia and DermAll for allergy treatment, both of which have demonstrated efficacy in preclinical studies. Genetic Immunity is a wholly owned subsidiary of Power of the Dream Ventures, Inc.

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.