OrbiMed

Garuda Therapeutics is a biotechnology company focused on creating off-the-shelf hematopoietic stem cell therapies aimed at treating various severe and life-threatening conditions. The company specializes in addressing blood, bone marrow, immune, and metabolic diseases. By leveraging expertise in innovative research methods, including the use of zebrafish, mice, and human-induced pluripotent stem cells, Garuda analyzes novel genes and mechanisms vital for blood formation. This approach enables the development of mechanism-based therapies designed to improve patient outcomes.

Developer of life sciences technology intended to develop highly effective and differentiated treatments for cystic fibrosis. The company is developing first-in-class small molecules that are focused on a unique target with the potential to fully normalize CFTR function for the first time for the vast majority of people living with cystic fibrosis, enabling patients with best-in-class efficacy and reach and long-term benefit for people with cystic fibrosis.

Nucleix develops, manufactures, and markets non-invasive molecular cancer diagnostic tests. Its sensitive and specific tests are based on identification of subtle changes in methylation patterns. Nucleix offers a urine test for monitoring of bladder cancer called Bladder EpiCheck and a blood test for early detection of lung cancer called Lung EpiCheck. Adam Wasserstrom, Danny Frumkin, and Elon Ganor founded it in 2008, with its headquarters in Rehovot in Israel.

ReCode Therapeutics is a development-stage biopharmaceutical company focused on the correction of the nonsense mutations that account for 1/3 of all inherited diseases, including approximately 10% of cystic fibrosis. They have combined selected tRNAs with their unique and diverse proprietary delivery platforms to create and develop first-in-class suppressor tRNA-nanoparticle therapeutics for correcting nonsense mutations. ReCode’s diverse pipeline includes lead programs for primary ciliary dyskinesia (PCD) and nonsense mutations in cystic fibrosis (CF). The company’s proprietary non-viral lipid nanoparticle platform enables the delivery of a variety of payloads, and precise organ targeting of nucleic acid and protein payloads via both systemic and local delivery. ReCode is leveraging its nanoparticle and nucleic acid technologies across multiple modalities, including mRNA-mediated protein replacement therapies, first-in-class tRNA NanoCorrectors for diseases caused by nonsense mutations, and gene editing. The company was founded in 2015 and is based in Dallas, Texas.

Garuda Therapeutics is a biotechnology company focused on creating off-the-shelf hematopoietic stem cell therapies aimed at treating various severe and life-threatening conditions. The company specializes in addressing blood, bone marrow, immune, and metabolic diseases. By leveraging expertise in innovative research methods, including the use of zebrafish, mice, and human-induced pluripotent stem cells, Garuda analyzes novel genes and mechanisms vital for blood formation. This approach enables the development of mechanism-based therapies designed to improve patient outcomes.

iECURE is a biotechnology company focused on developing gene editing therapies aimed at treating rare and life-threatening liver disorders, particularly in children. The company employs mutation-agnostic in vivo gene insertion techniques, which allow for targeted therapeutic interventions without being limited by specific genetic mutations. Its initial programs concentrate on addressing metabolic "loss of function" disorders, which often have significant unmet medical needs. Through its innovative approach, iECURE aims to provide effective treatments for devastating diseases that currently lack viable options.

GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) to treat a range of conditions, including autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and based in Boston, Massachusetts, with additional locations in Seattle and Israel, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs. This innovative approach aims to restore immune tolerance and address the limitations found in current regulatory T-cell therapies. The company was co-founded by experts in Treg biology and synthetic immunology, emphasizing its commitment to addressing the underlying causes of diseases linked to immune system dysfunction.

Biocytogen is a biotechnology company that specializes in developing gene-targeted animal models for life science research. Founded in 2008, with headquarters in Beijing, China, and additional facilities in the United States, Biocytogen offers a range of services including customized and off-the-shelf mouse models, as well as phenotype analysis across various biological areas such as immunology, neurology, and cancer. The company utilizes advanced gene-targeting platforms to create a variety of genetically modified animals, including humanized models and knockouts, to support preclinical studies. Biocytogen also provides pharmacology services related to tumor immunity and autoimmunity, and it markets its products online. With a commitment to integrating resources from both China and the United States, Biocytogen aims to be a leading global supplier of gene-targeted animal models.

Splisense is develops antisense oligonucleotide based therapies to target genetic diseases caused by splicing mutations. The company has developed a compound to cure the lung disease in cystic fibrosis patients carrying a specific splicing mutation. It is also developing a targeted modulation approach to correct the splicing pattern of CFTR transcripts according to the specific mutation carried.

Gennao Bio is a privately held genetic medicines company developing first-in-class targeted nucleic acid therapeutics utilizing a proprietary gene monoclonal antibody (GMAB) platform technology. GMAB is an adaptive technology that uses a novel, cell-penetrating antibody to non-covalently bind to and deliver therapeutic levels of a wide variety of nucleic acid payloads to select cells. This non-viral delivery platform is differentiated from traditional gene delivery systems as it can deliver multiple types of nucleic acids, allows for repeat dosing and employs well-established manufacturing processes. Gennao Bio is developing this delivery system with an initial focus on addressing significant unmet needs in oncology and rare monogenic skeletal muscle diseases.

Nucleix develops, manufactures, and markets non-invasive molecular cancer diagnostic tests. Its sensitive and specific tests are based on identification of subtle changes in methylation patterns. Nucleix offers a urine test for monitoring of bladder cancer called Bladder EpiCheck and a blood test for early detection of lung cancer called Lung EpiCheck. Adam Wasserstrom, Danny Frumkin, and Elon Ganor founded it in 2008, with its headquarters in Rehovot in Israel.

Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics using CRISPR technology to address genetic disorders. Founded in 2017 and based in Berkeley, California, Scribe specializes in engineering CRISPR enzymes known as X-Editing (XE) molecules, which enhance the efficacy, specificity, and deliverability of genome editing compared to existing methods. The company's proprietary platform enables the creation of custom-engineered enzymes and delivery systems, facilitating precision targeting within the genome. Scribe Therapeutics aims to establish CRISPR-based therapies as a standard in clinical care, expanding access to transformative treatments for a wide range of conditions, including immuno-oncology and degenerative disorders. Through its commitment to overcoming the limitations of current genome editing technologies, Scribe Therapeutics is poised to make substantial contributions to human therapeutics.

Graphite Bio is a gene editing company focused on targeted DNA integration to address severe diseases. The company employs technology that enables the insertion of new genetic sequences to replace defective genes, repair damaged portions of genes, and insert therapeutic genetic material at specific locations within the genome. This precise approach leverages natural cellular DNA repair mechanisms to enhance the efficiency of gene integration, aiming to correct the underlying causes of various diseases. Founded in 2019 and based in South San Francisco, California, Graphite Bio was previously known as Integral Medicines, Inc. and rebranded in August 2020. The company is committed to advancing gene replacement therapies and potential cures for individuals affected by serious health conditions.

AnchorDx is a company focused on advancing precision medicine through its development of next-generation sequencing techniques, particularly utilizing molecular liquid biopsies. This approach offers non-invasive access to genetic information, enabling patients to gain insights into their physiological conditions and prepare for potential future health issues. With a core team boasting over 20 years of experience in cancer genomics, genetics, and bioinformatics, AnchorDx aims to establish itself as a leading global provider of precision medical solutions. The company collaborates with various middle and downstream enterprises, fostering a comprehensive industrial chain that spans from academic research to clinical product transformation. Additionally, AnchorDx has formed partnerships with numerous large-scale third-party inspection agencies, hospitals, and research institutes, contributing to the development of independent intellectual property rights and competitive clinical testing products that deliver accurate and comprehensive services to patients, doctors, and medical institutions.

Gemini Therapeutics, Inc. is a precision medicine company established in 2015 and based in Cambridge, Massachusetts. The company specializes in developing therapies for patients with dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini's approach focuses on addressing genetically-defined conditions by creating therapeutic candidates that target specific molecular abnormalities prevalent in patients with significant clinical needs. Their diverse pipeline features a range of innovative treatment modalities, including monoclonal antibodies, recombinant proteins, and gene therapies. The company has secured funding from prominent life science investors and collaborates with academic institutions globally to advance its mission of delivering potentially first-in-class therapeutics.

Syros Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead candidates, SY-1425, a selective retinoic acid receptor alpha agonist currently undergoing Phase II trials for specific patients with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor in Phase I trials for select advanced solid tumors. Syros employs a proprietary platform that integrates assay technologies, bioinformatics, and biological insights to map gene regulatory circuits and modulate gene expression, positioning itself as a leader in gene control research. Additionally, Syros has established collaborations, including a target discovery agreement with Incyte Corporation focused on myeloproliferative neoplasms, and a licensing agreement with TMRC Co. Ltd. for tamibarotene's development and commercialization. Founded in 2011, Syros Pharmaceuticals aims to address significant unmet medical needs in oncology and beyond.

Novellus is a biotechnology company working to develop engineered cellular medicines. The company uses its non-immunogenic mRNA molecules to reprogram and gene-edit cells safely, and with high efficiency. They engineer precision medicines for patient segments and select from its technology toolbox for in vivo, ex vivo, allogeneic and autologous delivery. Novellus platform enables the high-level expression of gene-editing and cell-reprogramming proteins for high-specificity gene repair and both autologous and allogeneic cell therapies. The company is advancing its lead program, a breakthrough therapy for a rare dermatologic disease, through pre-clinical studies. Novellus was founded in 2015 and is headquartered in Cambridge, Massachusetts, United States.

Kinnate Biopharma develops precision oncology therapeutics to treat patients with genomically-defined cancers. Its mission is to expand the reach of targeted therapeutics by developing products for underserved populations. The company utilizes its deep expertise in structure-based drug discovery, translational research, and patient-driven precision medicine. Its programs include candidates in preclinical development for cancers that are driven by specific oncogenic alterations in either the BRAF kinase gene or in the FGFR2 and FGFR3 kinase genes.

GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) to treat a range of conditions, including autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and based in Boston, Massachusetts, with additional locations in Seattle and Israel, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs. This innovative approach aims to restore immune tolerance and address the limitations found in current regulatory T-cell therapies. The company was co-founded by experts in Treg biology and synthetic immunology, emphasizing its commitment to addressing the underlying causes of diseases linked to immune system dysfunction.

ReCode Therapeutics is a biopharmaceutical company that focuses on developing precision medicines for pulmonary diseases, particularly targeting rare conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company has created a non-viral lipid nanoparticle platform that facilitates the targeted delivery of genetic therapies. This innovative technology aims to deliver corrective or disease-modifying therapies to specific organs and tissues, addressing the limitations of current gene therapy methods. By improving the precision of drug delivery, ReCode Therapeutics seeks to provide new treatment options for patients with unmet medical needs, particularly in the realm of life-limiting respiratory diseases.

ReCode Therapeutics is a development-stage biopharmaceutical company focused on the correction of the nonsense mutations that account for 1/3 of all inherited diseases, including approximately 10% of cystic fibrosis. They have combined selected tRNAs with their unique and diverse proprietary delivery platforms to create and develop first-in-class suppressor tRNA-nanoparticle therapeutics for correcting nonsense mutations. ReCode’s diverse pipeline includes lead programs for primary ciliary dyskinesia (PCD) and nonsense mutations in cystic fibrosis (CF). The company’s proprietary non-viral lipid nanoparticle platform enables the delivery of a variety of payloads, and precise organ targeting of nucleic acid and protein payloads via both systemic and local delivery. ReCode is leveraging its nanoparticle and nucleic acid technologies across multiple modalities, including mRNA-mediated protein replacement therapies, first-in-class tRNA NanoCorrectors for diseases caused by nonsense mutations, and gene editing. The company was founded in 2015 and is based in Dallas, Texas.

Emendo Bio, Inc. is a biotechnology company focused on developing innovative genome editing technologies aimed at repairing and eliminating genetic mutations responsible for serious diseases and disorders. Founded in 2015 and headquartered in New York, the company integrates expertise in protein engineering, DNA repair, and computational analysis to advance its gene editing capabilities. Emendo Bio is dedicated to creating genetic medicines that address currently untreatable conditions by transforming existing tools and methodologies. The company operates as a subsidiary of AnGes, Inc., emphasizing a collaborative approach that combines knowledge from diverse scientific disciplines to overcome challenges in the field of gene therapy. Through its cutting-edge research, Emendo Bio aims to push the boundaries of what is possible in the realm of genetic medicine.

Kinnate Biopharma develops precision oncology therapeutics to treat patients with genomically-defined cancers. Its mission is to expand the reach of targeted therapeutics by developing products for underserved populations. The company utilizes its deep expertise in structure-based drug discovery, translational research, and patient-driven precision medicine. Its programs include candidates in preclinical development for cancers that are driven by specific oncogenic alterations in either the BRAF kinase gene or in the FGFR2 and FGFR3 kinase genes.

Passage Bio is a genetic medicines company based in Philadelphia, Pennsylvania, specializing in the development of therapies for rare monogenic central nervous system (CNS) diseases. Founded in 2017, the company aims to create a portfolio of five AAV-delivered therapeutics, targeting conditions such as GM1 Gangliosidosis, Frontotemporal dementia, and Krabbe Disease. Passage Bio collaborates closely with the University of Pennsylvania, leveraging its Gene Therapy Program for preclinical work while managing clinical development, regulatory affairs, manufacturing, and commercialization of its product candidates. The company also engages with the Orphan Disease Center for natural history studies, key opinion leader involvement, and patient advocacy.

Laekna Therapeutics Shanghai Co., Ltd. is a gene therapy company, which develops therapeutics for oncology and metabolic diseases. It develops pre-clinical discovery programs for oncology and liver diseases. It develops small molecule candidate LAE001 (previously known as CFG920) for prostate cancer. The company was founded in 2016 and is based in Shanghai, China.

Prevail Therapeutics Inc. is a biotechnology company based in New York that specializes in developing gene therapies for neurodegenerative diseases. Founded in 2017 with support from The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, the company is focused on creating innovative adeno-associated virus (AAV)-based treatments. Its lead candidate, PR001, is currently undergoing Phase I/II clinical trials aimed at treating Parkinson's disease linked to GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is developing PR006 for frontotemporal dementia associated with GRN mutations and PR004 for various synucleinopathies. The company is dedicated to advancing therapies that can modify the course of these debilitating conditions.

Passage Bio is a genetic medicines company based in Philadelphia, Pennsylvania, specializing in the development of therapies for rare monogenic central nervous system (CNS) diseases. Founded in 2017, the company aims to create a portfolio of five AAV-delivered therapeutics, targeting conditions such as GM1 Gangliosidosis, Frontotemporal dementia, and Krabbe Disease. Passage Bio collaborates closely with the University of Pennsylvania, leveraging its Gene Therapy Program for preclinical work while managing clinical development, regulatory affairs, manufacturing, and commercialization of its product candidates. The company also engages with the Orphan Disease Center for natural history studies, key opinion leader involvement, and patient advocacy.

Modis Therapeutics, Inc. is a biopharmaceutical company focused on developing disease-modifying therapies for rare genetic diseases with significant unmet medical needs. The company, based in Oakland, California, was incorporated in 2016 and is a subsidiary of Zogenix, Inc. Modis Therapeutics is advancing its lead therapy, MT1621, which aims to restore mitochondrial DNA replication fidelity. This therapeutic candidate is specifically designed to address thymidine kinase 2 deficiency (TK2d), an inherited mitochondrial disorder. By improving nucleotide balance, increasing mitochondrial DNA copy number, and enhancing cell function, Modis Therapeutics aims to provide meaningful treatment options that can prolong the lives of affected patients.

Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases that currently have no available treatments. The company’s lead pipeline candidate, TA-46, is a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism. Therachon is headquartered in Basel, Switzerland, with research labs in Nice, France.

Laekna Therapeutics Shanghai Co., Ltd. is a gene therapy company, which develops therapeutics for oncology and metabolic diseases. It develops pre-clinical discovery programs for oncology and liver diseases. It develops small molecule candidate LAE001 (previously known as CFG920) for prostate cancer. The company was founded in 2016 and is based in Shanghai, China.

Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapeutics aimed at addressing significant unmet medical needs in cancer treatment, particularly those related to abnormal gene expression and drug resistance. The company's lead product candidates include CPI-0610, currently undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, which is in Phase Ib/II trials for metastatic castration-resistant prostate cancer alongside androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the dose escalation phase of a Phase I/II clinical trial for solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. and has established itself as a pioneer in the emerging field of epigenetics, which focuses on selective regulators of epigenetic function crucial for gene expression control.

Prevail Therapeutics Inc. is a biotechnology company based in New York that specializes in developing gene therapies for neurodegenerative diseases. Founded in 2017 with support from The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, the company is focused on creating innovative adeno-associated virus (AAV)-based treatments. Its lead candidate, PR001, is currently undergoing Phase I/II clinical trials aimed at treating Parkinson's disease linked to GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is developing PR006 for frontotemporal dementia associated with GRN mutations and PR004 for various synucleinopathies. The company is dedicated to advancing therapies that can modify the course of these debilitating conditions.

Gemini Therapeutics, Inc. is a precision medicine company established in 2015 and based in Cambridge, Massachusetts. The company specializes in developing therapies for patients with dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini's approach focuses on addressing genetically-defined conditions by creating therapeutic candidates that target specific molecular abnormalities prevalent in patients with significant clinical needs. Their diverse pipeline features a range of innovative treatment modalities, including monoclonal antibodies, recombinant proteins, and gene therapies. The company has secured funding from prominent life science investors and collaborates with academic institutions globally to advance its mission of delivering potentially first-in-class therapeutics.

LogicBio develops gene therapy vectors for previously incurable genetic and infectious diseases, including: hemophilia, HIV/AIDS and methylmalonic acidemia (MMA). Our proprietary technology based on the non-pathogenic Adeno-Associated Viral (AAV) vector offers superior efficiency and safety compared to all competitors on the market, and is uniquely suited for the treatment of children as well as adults.

Rhythm Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing peptide therapeutics for the treatment of rare genetic disorders, particularly those leading to life-threatening metabolic conditions. Its primary product candidate, setmelanotide, is a first-in-class melanocortin-4 receptor agonist currently undergoing Phase III clinical trials for obesity related to pro-opiomelanocortin and leptin receptor deficiencies, as well as Bardet-Biedl and Alström syndromes. Additionally, setmelanotide is in Phase II trials for various other genetic obesity disorders. The company is also advancing RM-853, an orally available ghrelin O-acyltransferase inhibitor, in preclinical development for Prader-Willi syndrome. Founded in 2008 and headquartered in Boston, Massachusetts, Rhythm Pharmaceuticals aims to address significant unmet medical needs in the realm of genetic metabolic disorders through innovative therapies.

Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases that currently have no available treatments. The company’s lead pipeline candidate, TA-46, is a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism. Therachon is headquartered in Basel, Switzerland, with research labs in Nice, France.

Nucleix develops, manufactures, and markets non-invasive molecular cancer diagnostic tests. Its sensitive and specific tests are based on identification of subtle changes in methylation patterns. Nucleix offers a urine test for monitoring of bladder cancer called Bladder EpiCheck and a blood test for early detection of lung cancer called Lung EpiCheck. Adam Wasserstrom, Danny Frumkin, and Elon Ganor founded it in 2008, with its headquarters in Rehovot in Israel.

Gemini Therapeutics, Inc. is a precision medicine company established in 2015 and based in Cambridge, Massachusetts. The company specializes in developing therapies for patients with dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini's approach focuses on addressing genetically-defined conditions by creating therapeutic candidates that target specific molecular abnormalities prevalent in patients with significant clinical needs. Their diverse pipeline features a range of innovative treatment modalities, including monoclonal antibodies, recombinant proteins, and gene therapies. The company has secured funding from prominent life science investors and collaborates with academic institutions globally to advance its mission of delivering potentially first-in-class therapeutics.

LogicBio develops gene therapy vectors for previously incurable genetic and infectious diseases, including: hemophilia, HIV/AIDS and methylmalonic acidemia (MMA). Our proprietary technology based on the non-pathogenic Adeno-Associated Viral (AAV) vector offers superior efficiency and safety compared to all competitors on the market, and is uniquely suited for the treatment of children as well as adults.

Audentes Therapeutics is a clinical-stage biotechnology company dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. The company is advancing several therapies, including AT132, currently in Phase I/II clinical trials for X-linked myotubular myopathy, and AT342, also in Phase I/II studies for Crigler-Najjar syndrome. Additionally, Audentes is exploring AT845 in preclinical studies for Pompe disease and AT307 for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. The company is also working on vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Collaborations include a partnership with Genethon for XLMTM treatments and an agreement with the University of Pennsylvania for Crigler-Najjar syndrome products. Founded in 2012 and headquartered in San Francisco, California, Audentes Therapeutics operates as a subsidiary of Astellas US Holding, Inc.

Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases that currently have no available treatments. The company’s lead pipeline candidate, TA-46, is a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism. Therachon is headquartered in Basel, Switzerland, with research labs in Nice, France.

Intellia Therapeutics is a genome editing company based in Cambridge, Massachusetts, focused on developing curative gene-editing treatments using the CRISPR/Cas9 technology. The company aims to address severe and life-threatening genetic diseases through both in vivo and ex vivo therapeutic approaches. Its pipeline includes programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Additionally, Intellia is developing engineered cell therapies for oncological and autoimmune diseases. The company has established collaborations with various entities, including leading pharmaceutical firms and research institutions, to enhance its research and development efforts. Founded in 2014, Intellia Therapeutics holds a comprehensive intellectual property platform for the therapeutic application of CRISPR technology, positioning it at the forefront of gene editing innovation.

Dimension Therapeutics, Inc. is a gene therapy company dedicated to developing innovative treatments for rare and metabolic diseases linked to liver function. Founded in 2013 and based in Cambridge, Massachusetts, the company has established a robust pipeline of gene therapy candidates. Its lead products include DTX101 for hemophilia B and DTX201 for hemophilia A, alongside other programs targeting conditions such as ornithine transcarbamylase deficiency, glycogen storage disease type Ia, phenylketonuria, Wilson disease, and citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, leveraging its extensive patent portfolio related to NAV vector technology. This partnership enhances Dimension's ability to advance its gene therapy initiatives, particularly in the realm of adeno-associated virus therapeutics. In 2017, Dimension Therapeutics became a subsidiary of Ultragenyx Pharmaceutical Inc.

Natera is a diagnostic and research company specializing in genetic testing services, primarily focused on preconception and prenatal testing. Its flagship product, Panorama, is a non-invasive prenatal test that screens for chromosomal abnormalities in fetuses through a blood draw from the mother, including assessments for twin pregnancies. Other offerings include Vistara, which tests for single-gene disorders, and Horizon carrier screening to identify carrier status for various genetic diseases. Natera also provides the Anora test for analyzing fetal chromosomes in cases of miscarriage and non-invasive paternity testing. Additionally, the company has developed Signatera, a technology that detects circulating tumor DNA to monitor cancer recurrence. Natera utilizes a direct sales force and a network of approximately 100 laboratory and distribution partners for product distribution. The company was founded in 2003, has undergone several name changes, and is headquartered in San Carlos, California. Its collaborations with organizations like BGI Genomics and Foundation Medicine further enhance its capabilities in genetic testing and personalized medicine.

Aeglea BioTherapeutics is a clinical-stage biotechnology company focused on developing human enzyme therapeutics for rare genetic and cancer diseases. Its lead product candidate, pegzilarginase, is a recombinant human Arginase 1 enzyme currently undergoing Phase III trials to assess its safety and efficacy in treating Arginase 1 deficiency. The company's preclinical pipeline includes several innovative candidates: ACN00177 for homocystinuria, AEB5100 which degrades plasma cystine and cysteine, AEB2109 targeting the degradation of methionine, and AEB3103. Aeglea BioTherapeutics aims to exploit the amino acid dependencies of tumors by creating novel, human-derived enzymes that selectively target these vulnerabilities, differentiating the metabolic needs of tumor cells from those of normal tissues. Founded in 2013 and headquartered in Austin, Texas, the company was formerly known as Aeglea BioTherapeutics Holdings, LLC before changing its name in 2015.

GENEWIZ is a leading global genomics service company. A full-service provider, GENEWIZ provides DNA sequencing, gene synthesis, molecular biology, next generation sequencing, bioinformatics, and GLP regulatory services. From the company’s founding, GENEWIZ has maintained its unwavering commitment to providing customers with the best value. Excelling in both science and customer support, GENEWIZ is dedicated to accelerating scientific discovery, as well as to developing and delivering innovative solutions for translational medicine and healthcare. Easily accessible around the world, GENEWIZ is the preferred partner at leading academic, pharmaceutical, biotechnology, agricultural, government, and clinical institutions. At GENEWIZ, it is their mission to contribute to the advancement of life sciences and technologies. Together with their clients, they can make a difference in the pursuit of scientific discoveries, better healthcare, a greener environment, and abundant food supplies. Their goal is to be the best in the world and the best for the world. Headquartered in South Plainfield, NJ, GENEWIZ is a privately-held global enterprise with locations in Boston, MA; Washington, DC Metro; Research Triangle Park, NC; San Diego, CA; Los Angeles, CA; San Francisco, CA; and Seattle, WA. International locations include Beijing, China; Suzhou, China; London, United Kingdom; Langen, Germany; and Tokyo, Japan.

Audentes Therapeutics is a clinical-stage biotechnology company dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. The company is advancing several therapies, including AT132, currently in Phase I/II clinical trials for X-linked myotubular myopathy, and AT342, also in Phase I/II studies for Crigler-Najjar syndrome. Additionally, Audentes is exploring AT845 in preclinical studies for Pompe disease and AT307 for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. The company is also working on vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Collaborations include a partnership with Genethon for XLMTM treatments and an agreement with the University of Pennsylvania for Crigler-Najjar syndrome products. Founded in 2012 and headquartered in San Francisco, California, Audentes Therapeutics operates as a subsidiary of Astellas US Holding, Inc.

Invitae is a genetic information company focused on integrating genetic testing into routine medical practice to enhance healthcare quality worldwide. It specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates a wide array of genetic tests into one platform, ensuring faster turnaround times and lower costs compared to traditional single-gene tests. Invitae's test offerings cover various hereditary conditions, including cancers, neurological disorders, cardiovascular diseases, pediatric and metabolic disorders. The company aims to make genetic testing more affordable and accessible, develop a secure infrastructure for genome management, and foster a global community for sharing genetic information to advance scientific research and medical practices. By lowering the barriers to obtaining diagnostic genetic information, Invitae seeks to improve healthcare outcomes for millions of individuals.

Dimension Therapeutics, Inc. is a gene therapy company dedicated to developing innovative treatments for rare and metabolic diseases linked to liver function. Founded in 2013 and based in Cambridge, Massachusetts, the company has established a robust pipeline of gene therapy candidates. Its lead products include DTX101 for hemophilia B and DTX201 for hemophilia A, alongside other programs targeting conditions such as ornithine transcarbamylase deficiency, glycogen storage disease type Ia, phenylketonuria, Wilson disease, and citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, leveraging its extensive patent portfolio related to NAV vector technology. This partnership enhances Dimension's ability to advance its gene therapy initiatives, particularly in the realm of adeno-associated virus therapeutics. In 2017, Dimension Therapeutics became a subsidiary of Ultragenyx Pharmaceutical Inc.

Igenica, Inc. is a biotherapeutic company based in Burlingame, California, specializing in the discovery and development of antibodies and antibody-drug conjugates for cancer treatment. The company is dedicated to advancing immunotherapies and functional antibodies that target tumors, as well as creating antibody-drug conjugates. Igenica's key products include IGN523, a humanized monoclonal antibody aimed at various hematologic and solid tumors; IGN786, an antibody-drug conjugate; and IGN381, which targets immune checkpoints in the tumor microenvironment. Founded in 2008, Igenica operates with a strategic oncology research agreement with MedImmune and is backed by prominent life science investors. The leadership team at Igenica has significant experience in antibody drug discovery and has contributed to the development of multiple approved antibody therapeutic products.

Amoy Diagnostics Co., Ltd. (AmoyDx) focuses on molecular diagnostics for oncology precision medicine. With completely independent intellectual property rights of ADx-ARMS and Super-ARMS technologies, AmoyDx has a market-leading portfolio of molecular diagnostic kits including but not limited to EGFR, RAS, ALK, BRAF, PIK3CA and ROS1 testing kits, which were firstly approved by CFDA and CE certified. In European EMQN and Chinese PQCC quality control programs, AmoyDx is the NO.1 testing kit supplier for some years and always achieves the highest accuracy rate. Nowadays AmoyDx serves an extensive domestic and international oncology network reaching over 300 hospitals in China and 50 countries all over the world. Every year hundreds of thousands of cancer patients benefit from AmoyDx products.

Audentes Therapeutics is a clinical-stage biotechnology company dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. The company is advancing several therapies, including AT132, currently in Phase I/II clinical trials for X-linked myotubular myopathy, and AT342, also in Phase I/II studies for Crigler-Najjar syndrome. Additionally, Audentes is exploring AT845 in preclinical studies for Pompe disease and AT307 for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. The company is also working on vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Collaborations include a partnership with Genethon for XLMTM treatments and an agreement with the University of Pennsylvania for Crigler-Najjar syndrome products. Founded in 2012 and headquartered in San Francisco, California, Audentes Therapeutics operates as a subsidiary of Astellas US Holding, Inc.

Natera is a diagnostic and research company specializing in genetic testing services, primarily focused on preconception and prenatal testing. Its flagship product, Panorama, is a non-invasive prenatal test that screens for chromosomal abnormalities in fetuses through a blood draw from the mother, including assessments for twin pregnancies. Other offerings include Vistara, which tests for single-gene disorders, and Horizon carrier screening to identify carrier status for various genetic diseases. Natera also provides the Anora test for analyzing fetal chromosomes in cases of miscarriage and non-invasive paternity testing. Additionally, the company has developed Signatera, a technology that detects circulating tumor DNA to monitor cancer recurrence. Natera utilizes a direct sales force and a network of approximately 100 laboratory and distribution partners for product distribution. The company was founded in 2003, has undergone several name changes, and is headquartered in San Carlos, California. Its collaborations with organizations like BGI Genomics and Foundation Medicine further enhance its capabilities in genetic testing and personalized medicine.

Igenica, Inc. is a biotherapeutic company based in Burlingame, California, specializing in the discovery and development of antibodies and antibody-drug conjugates for cancer treatment. The company is dedicated to advancing immunotherapies and functional antibodies that target tumors, as well as creating antibody-drug conjugates. Igenica's key products include IGN523, a humanized monoclonal antibody aimed at various hematologic and solid tumors; IGN786, an antibody-drug conjugate; and IGN381, which targets immune checkpoints in the tumor microenvironment. Founded in 2008, Igenica operates with a strategic oncology research agreement with MedImmune and is backed by prominent life science investors. The leadership team at Igenica has significant experience in antibody drug discovery and has contributed to the development of multiple approved antibody therapeutic products.

Good Start Genetics, Inc., founded in 2007 and based in Cambridge, Massachusetts, is a molecular diagnostics company specializing in genetic screening for inherited diseases. The company aims to transform reproductive medicine by offering clinically relevant information to physicians and patients regarding genetic disorders. Its flagship product, EmbryVu, is a preimplantation genetic screening test designed to enhance in vitro fertilization by identifying suitable embryos for implantation and pregnancy. Good Start Genetics employs advanced next-generation DNA sequencing (NGS) technology, alongside other established genetic screening methods, to provide a comprehensive suite of genetic carrier screening tests through its GoodStart Select program. The company emphasizes customer care and genetic counseling, ensuring that patients receive actionable insights into known and novel genetic mutations. As of August 2017, Good Start Genetics operates as a subsidiary of Invitae Corporation.

Ethris GmbH is a biotechnology company based in Planegg, Germany, specializing in the development of innovative therapeutic solutions for diseases with unmet medical needs. The company has a focus on transcript therapeutics, particularly through its SNIM RNA platform, which offers a novel approach to treating various conditions by enabling the production of therapeutic proteins within the patient’s body. This platform presents an alternative to traditional recombinant protein and gene therapies, addressing a range of diseases, including rare and genetically predetermined conditions. Ethris also develops Magnetovax, a personalized tumor vaccine platform that utilizes magnetic fields for localized activation in tumors. The company has established strategic research collaborations with major pharmaceutical firms, enhancing its capabilities in regenerative medicine and therapeutic development. Founded in 2009, Ethris continues to advance its technologies to meet diverse medical needs.

Good Start Genetics, Inc., founded in 2007 and based in Cambridge, Massachusetts, is a molecular diagnostics company specializing in genetic screening for inherited diseases. The company aims to transform reproductive medicine by offering clinically relevant information to physicians and patients regarding genetic disorders. Its flagship product, EmbryVu, is a preimplantation genetic screening test designed to enhance in vitro fertilization by identifying suitable embryos for implantation and pregnancy. Good Start Genetics employs advanced next-generation DNA sequencing (NGS) technology, alongside other established genetic screening methods, to provide a comprehensive suite of genetic carrier screening tests through its GoodStart Select program. The company emphasizes customer care and genetic counseling, ensuring that patients receive actionable insights into known and novel genetic mutations. As of August 2017, Good Start Genetics operates as a subsidiary of Invitae Corporation.

Complete Genomics is a developer of a DNA sequencing platform that focuses on human genome sequencing and analysis. The company offers high-quality and affordable DNA sequencing services, which facilitate large-scale research into the genetic mechanisms that influence drug responses and complex diseases. By integrating proprietary sequencing technology with advanced informatics and data management software, Complete Genomics provides customers with readily usable data for genome-based research. This combination of innovative technology and a disruptive approach positions the company to significantly impact the field of DNA sequencing.

Igenica, Inc. is a biotherapeutic company based in Burlingame, California, specializing in the discovery and development of antibodies and antibody-drug conjugates for cancer treatment. The company is dedicated to advancing immunotherapies and functional antibodies that target tumors, as well as creating antibody-drug conjugates. Igenica's key products include IGN523, a humanized monoclonal antibody aimed at various hematologic and solid tumors; IGN786, an antibody-drug conjugate; and IGN381, which targets immune checkpoints in the tumor microenvironment. Founded in 2008, Igenica operates with a strategic oncology research agreement with MedImmune and is backed by prominent life science investors. The leadership team at Igenica has significant experience in antibody drug discovery and has contributed to the development of multiple approved antibody therapeutic products.

Complete Genomics is a developer of a DNA sequencing platform that focuses on human genome sequencing and analysis. The company offers high-quality and affordable DNA sequencing services, which facilitate large-scale research into the genetic mechanisms that influence drug responses and complex diseases. By integrating proprietary sequencing technology with advanced informatics and data management software, Complete Genomics provides customers with readily usable data for genome-based research. This combination of innovative technology and a disruptive approach positions the company to significantly impact the field of DNA sequencing.

Enobia Pharma focuses on developing therapies for serious genetic bone disorders that currently lack approved treatments. The company specifically targets conditions such as hypophosphatasia (HPP), a rare metabolic disorder that affects bone mineralization and can lead to severe skeletal deformities and other health complications. Through its innovative research and development efforts, Enobia aims to provide effective solutions for patients suffering from these debilitating conditions.

MacroGenics, Inc. is a biopharmaceutical company based in Rockville, Maryland, that specializes in the discovery and development of antibody-based therapeutics for cancer treatment. The company's pipeline includes several immuno-oncology candidates, such as Margetuximab, a monoclonal antibody currently in Phase III trials targeting HER2-expressing tumors, and Flotetuzumab, a DART molecule aimed at treating acute myeloid leukemia. Other investigational products include various monoclonal antibodies targeting immune checkpoints like PD-1 and B7-H3, as well as combination therapies that leverage multiple targets. MacroGenics employs advanced Fc engineering technology to enhance antibody functionality and is also exploring applications for autoimmune disorders and infectious diseases. The company has established strategic collaborations with several partners to advance its research and development efforts. Founded in 2000, MacroGenics is committed to innovating immunotherapeutics to address significant medical needs.

Enobia Pharma focuses on developing therapies for serious genetic bone disorders that currently lack approved treatments. The company specifically targets conditions such as hypophosphatasia (HPP), a rare metabolic disorder that affects bone mineralization and can lead to severe skeletal deformities and other health complications. Through its innovative research and development efforts, Enobia aims to provide effective solutions for patients suffering from these debilitating conditions.

Agensys is a biotechnology company focused on the research and development of therapeutic human monoclonal antibodies and antibody-drug conjugates specifically for cancer treatment. The company offers products aimed at addressing various tumors, including those associated with prostate, pancreatic, bladder, kidney, lung, and colon cancers. Founded in 1997 and based in Santa Monica, California, Agensys operates as a subsidiary of Astellas Pharma US, Inc. It is dedicated to providing innovative therapeutic solutions to improve patient outcomes in oncology.

CoGenesys is a biopharmaceutical company focused on the development of peptide- and protein-based medicines. The company develops peptide and protein-based medicines for cancer, cardiovascular, metabolic, infectious, autoimmune, and CNS diseases.

MacroGenics, Inc. is a biopharmaceutical company based in Rockville, Maryland, that specializes in the discovery and development of antibody-based therapeutics for cancer treatment. The company's pipeline includes several immuno-oncology candidates, such as Margetuximab, a monoclonal antibody currently in Phase III trials targeting HER2-expressing tumors, and Flotetuzumab, a DART molecule aimed at treating acute myeloid leukemia. Other investigational products include various monoclonal antibodies targeting immune checkpoints like PD-1 and B7-H3, as well as combination therapies that leverage multiple targets. MacroGenics employs advanced Fc engineering technology to enhance antibody functionality and is also exploring applications for autoimmune disorders and infectious diseases. The company has established strategic collaborations with several partners to advance its research and development efforts. Founded in 2000, MacroGenics is committed to innovating immunotherapeutics to address significant medical needs.

MacroGenics, Inc. is a biopharmaceutical company based in Rockville, Maryland, that specializes in the discovery and development of antibody-based therapeutics for cancer treatment. The company's pipeline includes several immuno-oncology candidates, such as Margetuximab, a monoclonal antibody currently in Phase III trials targeting HER2-expressing tumors, and Flotetuzumab, a DART molecule aimed at treating acute myeloid leukemia. Other investigational products include various monoclonal antibodies targeting immune checkpoints like PD-1 and B7-H3, as well as combination therapies that leverage multiple targets. MacroGenics employs advanced Fc engineering technology to enhance antibody functionality and is also exploring applications for autoimmune disorders and infectious diseases. The company has established strategic collaborations with several partners to advance its research and development efforts. Founded in 2000, MacroGenics is committed to innovating immunotherapeutics to address significant medical needs.

MacroGenics, Inc. is a biopharmaceutical company based in Rockville, Maryland, that specializes in the discovery and development of antibody-based therapeutics for cancer treatment. The company's pipeline includes several immuno-oncology candidates, such as Margetuximab, a monoclonal antibody currently in Phase III trials targeting HER2-expressing tumors, and Flotetuzumab, a DART molecule aimed at treating acute myeloid leukemia. Other investigational products include various monoclonal antibodies targeting immune checkpoints like PD-1 and B7-H3, as well as combination therapies that leverage multiple targets. MacroGenics employs advanced Fc engineering technology to enhance antibody functionality and is also exploring applications for autoimmune disorders and infectious diseases. The company has established strategic collaborations with several partners to advance its research and development efforts. Founded in 2000, MacroGenics is committed to innovating immunotherapeutics to address significant medical needs.

Agensys is a biotechnology company focused on the research and development of therapeutic human monoclonal antibodies and antibody-drug conjugates specifically for cancer treatment. The company offers products aimed at addressing various tumors, including those associated with prostate, pancreatic, bladder, kidney, lung, and colon cancers. Founded in 1997 and based in Santa Monica, California, Agensys operates as a subsidiary of Astellas Pharma US, Inc. It is dedicated to providing innovative therapeutic solutions to improve patient outcomes in oncology.

GeneFormatics is a biotechnology company that focuses on structural proteomics and the development of innovative function prediction methods. The company offers tools designed for the rapid identification of biologically relevant targets from extensive sequence data generated by global genomics projects. By leveraging advanced technologies, GeneFormatics aims to enhance the understanding of protein structures and functions, facilitating significant advancements in the field of biotechnology.

MacroGenics, Inc. is a biopharmaceutical company based in Rockville, Maryland, that specializes in the discovery and development of antibody-based therapeutics for cancer treatment. The company's pipeline includes several immuno-oncology candidates, such as Margetuximab, a monoclonal antibody currently in Phase III trials targeting HER2-expressing tumors, and Flotetuzumab, a DART molecule aimed at treating acute myeloid leukemia. Other investigational products include various monoclonal antibodies targeting immune checkpoints like PD-1 and B7-H3, as well as combination therapies that leverage multiple targets. MacroGenics employs advanced Fc engineering technology to enhance antibody functionality and is also exploring applications for autoimmune disorders and infectious diseases. The company has established strategic collaborations with several partners to advance its research and development efforts. Founded in 2000, MacroGenics is committed to innovating immunotherapeutics to address significant medical needs.

NeoGenesis Pharmaceuticals, Inc. is a drug discovery company based in Cambridge, Massachusetts, founded in 1997. The company specializes in the development of small molecule drugs through its proprietary affinity-based screening technologies and an integrated chemical library. NeoGenesis employs its Automated Ligand Identification System (ALIS) to efficiently identify and optimize novel drug leads that traditional methods may overlook. This technology, combined with the company's diverse NeoMorph compound library, allows for targeted drug discovery against both established and emerging biological targets. In addition to its internal drug development efforts, NeoGenesis collaborates with partners to enhance their drug discovery processes.

Molecular Staging, Inc., a life sciences tool company, develops technologies for the detection and measurement of proteins and nucleic acids. It provides technologies for proteomics, genomics, pharmacogenomics, and diagnostics applications. The company’s products and services include protein microarrays analogous to the DNA microarray, enabling researchers to study multiple proteins; whole-genome amplification that allows DNA samples as resources, enabling researchers to return to them for new tests or confirmatory diagnoses; and multiplexed diagnostics that enable earlier detection of disease.