OrbiMed

Garuda Therapeutics is a biotech company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening diseases, particularly those affecting blood, bone marrow, immunity, and metabolism. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants by leveraging its innovative platform technology to generate self-renewing blood stem cells. This approach is designed to ensure rapid and broad access to consistent, durable, HLA-compatible, and transgene-free blood stem cell therapies for patients. Garuda Therapeutics employs advanced methodologies, including the use of zebrafish, mice, and human-induced pluripotent stem cells, to clone and analyze novel genes and mechanisms critical for blood formation, ultimately developing mechanism-based therapies that address unmet medical needs.

enGene is a developer of a mucosal immunotherapy platform designed to treat inflammatory bowel disease and diabetes. The company's mucosal immunotherapy platform has developed a flexible nucleotide delivery technology targeting mucosal tissues to treat numerous prevalent, chronic diseases via the induction or suppression of protein expression levels, enabling physicians and doctors to regenerate physiologic, meal-regulated insulin secretion from the gut of subjects with diabetes.

Asceneuron develops effective therapeutics for orphan tauopathies and Alzheimer’s disease. By focusing on areas of high unmet medical need, Asceneuron aspires to become a leading biotech company specialized in small molecule drug discovery for neurodegenerative diseases. Tauopathies are currently untreatable neurodegenerative diseases that rapidly progress towards debilitating conditions. The appearance of deposits of the microtubule-associated tau protein as e.g. neurofibrillary tangles in neurons of the brain is a common feature of tauopathies that is shared with Alzheimer’s disease. Neuronal tau deposits are known to be a major contributor to neurodegeneration and mutations in the tau gene are causative of the tauopathy fronto-temporal dementia and parkinsonism linked to chromosome 17 (FTDP-17). Due to increasing life expectancy, Alzheimer’s disease is viewed as one of the largest healthcare problems of this century, imposing a major economic burden on societies in the Western and developing world. Current treatment options provide limited benefits supporting the urgent need for more efficacious and better tolerated medicines that address symptomatic relief as well as disease progression.

Fate Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing programmed cellular immunotherapies for cancer and immune disorders. The company’s pipeline includes various NK- and T-cell immuno-oncology programs, such as FT516 for acute myeloid leukemia and B-cell lymphoma, FT596 for B-cell lymphoma and chronic lymphocytic leukemia, and FT538 for multiple myeloma. Additional programs include FT500, FT576, FT819, and FT-ONO1, targeting solid tumors and hematologic malignancies. Fate Therapeutics is also collaborating with Ono Pharmaceutical Co. Ltd. and Janssen Biotech, Inc. to advance its off-the-shelf iPSC-derived CAR T-cell therapies and other innovative treatments. Founded in 2007, the company leverages insights from leading scientists to create and modulate stem cells for therapeutic applications, aiming to restore health through advanced cellular therapies.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

BioAge Labs, Inc. is a biotechnology company focused on developing therapies to address aging and age-related diseases. The company utilizes a systems biology and artificial intelligence platform to identify key drug targets that influence aging and analyze extensive datasets to uncover the molecular drivers of age-related conditions. BioAge Labs is advancing several drug candidates, including BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor designed to activate genes involved in critical biological processes such as tissue regeneration and vascular remodeling. Additionally, BGE-175, an orally administered inhibitor of the prostaglandin D2 DP1 signaling pathway, aims to mitigate risks associated with immune aging and respiratory infections. Founded in 2015 and based in Richmond, California, BioAge Labs is committed to discovering and developing innovative treatments that harness the biology of human aging.

Rampart Bioscience specializes in the development of gene medicines aimed at providing long-lasting treatments for a broad array of diseases. The company has created proprietary development and delivery platforms that enable the production of optimized therapeutics in a non-viral format. By integrating expertise from various scientific disciplines, such as gene delivery, protein sciences, and clinical translation, Rampart Bioscience focuses on assisting patients with genetically driven conditions, ultimately enhancing the effectiveness and safety of therapeutic interventions.

Minervax is focused on developing a vaccine to prevent infections caused by Group B streptococcus (GBS), which can lead to serious health issues in newborns and adverse pregnancy outcomes. The company is researching a specific fusion protein that has demonstrated the ability to stimulate strong protective immunity against various clinically significant GBS strains. By utilizing this innovative approach, Minervax aims to provide medical professionals with an effective tool to protect newborns from potentially life-threatening bacterial infections associated with GBS.

ReCode Therapeutics is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company employs a unique non-viral lipid nanoparticle platform that facilitates the precise delivery of nucleic acid and protein payloads to specific organs and tissues, enhancing the effectiveness of genetic therapies. This selective organ targeting (SORT) technology allows for the administration of corrective or disease-modifying treatments beyond the liver, addressing the challenges faced by traditional gene therapy methods. By harnessing these innovative techniques, ReCode Therapeutics aims to provide new therapeutic options for patients suffering from rare and life-limiting respiratory diseases.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

Garuda Therapeutics is a biotech company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening diseases, particularly those affecting blood, bone marrow, immunity, and metabolism. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants by leveraging its innovative platform technology to generate self-renewing blood stem cells. This approach is designed to ensure rapid and broad access to consistent, durable, HLA-compatible, and transgene-free blood stem cell therapies for patients. Garuda Therapeutics employs advanced methodologies, including the use of zebrafish, mice, and human-induced pluripotent stem cells, to clone and analyze novel genes and mechanisms critical for blood formation, ultimately developing mechanism-based therapies that address unmet medical needs.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, founded in 2017. The company focuses on developing human genetics-based therapeutics aimed at addressing ineffective red blood cell production in hematologic diseases. Its innovative pipeline includes therapies such as bitopertin for treating erythropoietic porphyrias and Diamond-Blackfan anemia. Disc Medicine targets the hepcidin metabolism axis and aims to modify fundamental biological pathways related to red blood cell formation and function, including heme biosynthesis and iron homeostasis. Through its portfolio of clinical and preclinical product candidates, the company strives to transform the lives of patients suffering from severe hematologic disorders.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

iECURE is a mutation-agnostic in vivo gene editing company striving to cure devastating diseases with high unmet needs.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, founded in 2017. The company focuses on developing human genetics-based therapeutics aimed at addressing ineffective red blood cell production in hematologic diseases. Its innovative pipeline includes therapies such as bitopertin for treating erythropoietic porphyrias and Diamond-Blackfan anemia. Disc Medicine targets the hepcidin metabolism axis and aims to modify fundamental biological pathways related to red blood cell formation and function, including heme biosynthesis and iron homeostasis. Through its portfolio of clinical and preclinical product candidates, the company strives to transform the lives of patients suffering from severe hematologic disorders.

Frontera Therapeutics is a company specializing in adeno-associated virus (AAV) gene therapy, focused on developing and commercializing cost-effective recombinant virus-based therapies. The company aims to create a scalable platform that produces high-quality, affordable rAAV therapies for a range of genetic disorders, including ophthalmic, liver, metabolic, and neuromuscular diseases. Frontera Therapeutics has a diverse product pipeline that encompasses multiple therapeutic areas, with various candidates at different stages of development. By prioritizing affordability and quality, the company seeks to enhance the lives of patients through innovative genetic therapies.

Aspen Neuroscience, Inc. is a biotechnology company focused on developing induced pluripotent stem cells (iPSCs) to create patient-specific therapies for neurological conditions, particularly Parkinson's disease. Founded in 2018 and based in San Diego, California, the company is advancing its pipeline of products, which includes ANPD001, an autologous neuron replacement therapy for sporadic Parkinson's disease, and ANPD002, a gene-edited autologous neuron replacement therapy targeting familial forms of the disease. Aspen Neuroscience employs innovative genomic approaches in conjunction with stem cell biology to address diseases with significant unmet medical needs, aiming to provide restorative cell therapies that can modify the progression of Parkinson's disease and potentially extend to other affected organs.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Cyrus Biotechnology, Inc. specializes in developing software tools for protein structure prediction and design, aiming to enhance research in biotechnology and pharmaceuticals. The company's flagship product, Rosetta, allows for the design of biologically active proteins that can address critical health issues, such as brain cancer treatment and gluten breakdown in Celiac Disease patients. Additionally, Cyrus Bench offers an enterprise version of the Rosetta toolkit, equipped with a variety of bio-molecular computation tools. The company also provides Cyrus CryoEM services, which include advanced structure refinement and model building. Founded in 2014 and headquartered in Seattle, Washington, Cyrus Biotechnology serves a diverse clientele, including pharmaceutical companies, venture-backed startups, and mid-sized biotech firms focused on therapeutic discovery and industrial biotechnology innovation.

PierianDx, Inc. is a company that specializes in developing a cloud-based genomic software solution aimed at managing, analyzing, and interpreting complex genomic tests for clinical laboratories in the United States. Founded in 2014 and headquartered in Saint Louis, Missouri, PierianDx offers the Clinical Genomicist Workstation, a comprehensive platform that oversees patient case management, including accessioning, sequencing, analysis, and reporting, while ensuring integration with electronic health records. The company caters to health systems, children’s hospitals, comprehensive cancer centers, and reference laboratories, and provides consulting and technical services. PierianDx plays a pivotal role in enhancing the utilization of genomic sequencing in healthcare, enabling physicians to improve diagnosis and treatment for cancer and other complex diseases through its accredited laboratory and shared knowledgebase.

ReCode Therapeutics is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company employs a unique non-viral lipid nanoparticle platform that facilitates the precise delivery of nucleic acid and protein payloads to specific organs and tissues, enhancing the effectiveness of genetic therapies. This selective organ targeting (SORT) technology allows for the administration of corrective or disease-modifying treatments beyond the liver, addressing the challenges faced by traditional gene therapy methods. By harnessing these innovative techniques, ReCode Therapeutics aims to provide new therapeutic options for patients suffering from rare and life-limiting respiratory diseases.

Garuda Therapeutics is a biotech company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening diseases, particularly those affecting blood, bone marrow, immunity, and metabolism. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants by leveraging its innovative platform technology to generate self-renewing blood stem cells. This approach is designed to ensure rapid and broad access to consistent, durable, HLA-compatible, and transgene-free blood stem cell therapies for patients. Garuda Therapeutics employs advanced methodologies, including the use of zebrafish, mice, and human-induced pluripotent stem cells, to clone and analyze novel genes and mechanisms critical for blood formation, ultimately developing mechanism-based therapies that address unmet medical needs.

iECURE is a mutation-agnostic in vivo gene editing company striving to cure devastating diseases with high unmet needs.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, founded in 2017. The company focuses on developing human genetics-based therapeutics aimed at addressing ineffective red blood cell production in hematologic diseases. Its innovative pipeline includes therapies such as bitopertin for treating erythropoietic porphyrias and Diamond-Blackfan anemia. Disc Medicine targets the hepcidin metabolism axis and aims to modify fundamental biological pathways related to red blood cell formation and function, including heme biosynthesis and iron homeostasis. Through its portfolio of clinical and preclinical product candidates, the company strives to transform the lives of patients suffering from severe hematologic disorders.

GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs technologies. This innovative approach is designed to restore immune tolerance and address limitations present in current regulatory T-cell therapies. By targeting the underlying causes of diseases related to immune system dysfunction, GentiBio aims to provide effective therapeutic solutions for conditions that arise from immune system overshooting or malfunctioning. The company was co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of this emerging field.

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 company focused on developing innovative targeted nucleic acid therapeutics, primarily aimed at treating oncology and rare monogenic skeletal muscle diseases. The company utilizes its proprietary gene monoclonal antibody (GMAB) platform technology, which features a novel, cell-penetrating antibody that facilitates the non-covalent binding and delivery of various nucleic acid payloads to specific cells. This non-viral delivery system stands out from traditional gene therapy methods by allowing for multiple types of nucleic acids, enabling repeat dosing, and employing established manufacturing processes. Gennao Bio's approach aims to address significant unmet medical needs and improve patient outcomes in the relevant therapeutic areas.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that specializes in targeted DNA integration to develop innovative therapies for severe diseases. The company utilizes a precision gene editing approach to precisely correct genetic mutations, replace malfunctioning genes, and insert therapeutic genetic sequences at specific locations within the genome. Its technology aims to transform treatment paradigms by enabling the targeted integration of genetic payloads, which could potentially cure a range of serious and life-threatening conditions. Graphite Bio was formerly known as Integral Medicines, Inc. before rebranding in August 2020, and it was incorporated in 2019.

Century Therapeutics is a biotechnology company focused on developing advanced cell therapies using induced pluripotent stem cells (iPSCs) to treat various cancers, including hematologic and solid tumors. Founded in 2018 and based in Philadelphia, Pennsylvania, the company utilizes a unique allogeneic iPSC-derived cell therapy platform that integrates gene editing, protein engineering, and robust manufacturing capabilities. This innovative approach allows for the generation of modified immune effector cells, such as natural killer (NK) and T cells, designed to meet significant unmet medical needs. By employing techniques like CRISPR-mediated gene editing and proprietary chimeric antigen receptors, Century Therapeutics aims to enhance cell product performance and minimize rejection by the host immune system, thereby improving the efficacy of cancer treatments.

AnchorDx, also known as Guangzhou Baseline Medical, specializes in high-throughput sequencing technologies and molecular liquid biopsies, offering non-invasive access to genetic information. The company focuses on precision medicine and aims to be a leading global provider of medical solutions by leveraging its expertise in cancer genomics, genetics, and bioinformatics. With a core team possessing over 20 years of industry experience, AnchorDx collaborates with various downstream enterprises, hospitals, and research institutions to create an integrated industrial chain that facilitates the transformation of academic research into clinical products. The company is committed to developing independent intellectual property rights and competitive clinical testing products, thereby providing accurate and comprehensive services to patients, doctors, and medical institutions.

Ikena Oncology is a biotechnology company based in Boston, Massachusetts, focused on developing innovative therapies for cancer treatment. The company specializes in biomarker-driven and precision therapies that target specific mechanisms involved in cancer growth, spread, and resistance. Its product pipeline includes several candidates such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-930, an oral small-molecule inhibitor of the TEAD transcription factor in the Hippo signaling pathway. Additionally, Ikena is exploring a kynurenine-degrading enzyme with IK-412 and other discovery-stage programs targeting various oncogenic pathways. Established in 2016, Ikena Oncology aims to address unmet medical needs through a combination of chemistry, translational science, and patient-centered drug development.

Syros Pharmaceuticals is a life sciences company that is focused on treating disease by mapping gene regulatory circuits and modulating the factors that regulate gene expression. Syros has pioneered world-leading gene control research and drug discovery capabilities with a proven ability to integrate disease biology and genomic data--a proficiency that is not well represented in pharmaceutical R&D. Central to the Syros approach is a proprietary platform of carefully integrated assay technologies, bioinformatics, and biologic insights developed by members of Syros' senior leadership. While this scientific approach has applications in many therapeutic areas, Syros has demonstrated success initially in oncology, where it may help address numerous unmet medical needs.

Decibel Therapeutics is a developer of hearing disorder therapeutics intended to protect, repair and restore hearing. The company's therapeutics have provided insights into the link between hearing dysfunction and inner ear pathology and it has capabilities to encompass animal models, drug delivery to the inner ear, imaging, inner ear PK/PD modeling and measurement, bioinformatics, genetics and target identification, enabling patients to experience hearing loss therapies with different forms of hearing disorders for their recovery.

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.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

Escape Bio is a clinical-stage biopharmaceutical company that develops small molecule drugs intended to treat neurodegenerative diseases. The company is developing therapeutics for the novel, precisely therapies for genetic neurodegenerative diseases. Escape Bio's pipeline includes candidates targeting known genetic drivers of multiple diseases, including ESB1609, a small molecule S1P5 receptor agonist for the treatment of CNS lysosomal storage disorders and ESB5070, a small molecule kinase inhibitor for Parkinson’s disease patients who have an LRRK2 G2019S variant and an Alzheimer's disease program targeting ApoE4. E-scape Bio was founded in 2015 and is headquartered in San Francisco, California, United States.

Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

Aspen Neuroscience, Inc. is a biotechnology company focused on developing induced pluripotent stem cells (iPSCs) to create patient-specific therapies for neurological conditions, particularly Parkinson's disease. Founded in 2018 and based in San Diego, California, the company is advancing its pipeline of products, which includes ANPD001, an autologous neuron replacement therapy for sporadic Parkinson's disease, and ANPD002, a gene-edited autologous neuron replacement therapy targeting familial forms of the disease. Aspen Neuroscience employs innovative genomic approaches in conjunction with stem cell biology to address diseases with significant unmet medical needs, aiming to provide restorative cell therapies that can modify the progression of Parkinson's disease and potentially extend to other affected organs.

ReCode Therapeutics is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company employs a unique non-viral lipid nanoparticle platform that facilitates the precise delivery of nucleic acid and protein payloads to specific organs and tissues, enhancing the effectiveness of genetic therapies. This selective organ targeting (SORT) technology allows for the administration of corrective or disease-modifying treatments beyond the liver, addressing the challenges faced by traditional gene therapy methods. By harnessing these innovative techniques, ReCode Therapeutics aims to provide new therapeutic options for patients suffering from rare and life-limiting respiratory diseases.

EmendoBio, Inc. is focused on advancing the field of genome editing by developing innovative gene editing tools aimed at overcoming challenges in gene therapy. The company leverages expertise in protein engineering and DNA repair, combining insights from top academic institutions to create genetic medicines that address currently untreatable disorders and diseases. By transforming existing methodologies and employing creative approaches, EmendoBio seeks to resolve significant bottlenecks in gene therapy, enabling healthcare professionals to provide effective treatments where none previously existed. Through a commitment to pushing scientific boundaries, EmendoBio strives to deliver on the potential of gene editing for transformative medical solutions.

Aspen Neuroscience, Inc. is a biotechnology company focused on developing induced pluripotent stem cells (iPSCs) to create patient-specific therapies for neurological conditions, particularly Parkinson's disease. Founded in 2018 and based in San Diego, California, the company is advancing its pipeline of products, which includes ANPD001, an autologous neuron replacement therapy for sporadic Parkinson's disease, and ANPD002, a gene-edited autologous neuron replacement therapy targeting familial forms of the disease. Aspen Neuroscience employs innovative genomic approaches in conjunction with stem cell biology to address diseases with significant unmet medical needs, aiming to provide restorative cell therapies that can modify the progression of Parkinson's disease and potentially extend to other affected organs.

Repare Therapeutics Inc. is a precision oncology company based in Montréal, Canada, specializing in the discovery and development of novel therapeutics through a synthetic lethality approach. Utilizing its proprietary SNIPRx platform, which combines genome-wide and CRISPR-enabled technology, the company systematically identifies targeted cancer therapies addressing genomic instability, particularly in DNA damage repair. Its lead candidate, RP-3500, is an oral small molecule inhibitor designed to treat solid tumors with specific DNA damage repair-related genomic alterations. Additionally, Repare is advancing a CCNE1-SL inhibitor program targeting tumors with CCNE1 amplification, along with a Polymerase Theta program focused on BRCA mutations and related genomic alterations. Founded in 2016, the company aims to improve cancer treatment by leveraging innovative drug discovery methods and addressing the unique vulnerabilities of tumor cells.

Passage Bio is a fully integrated gene therapy company with a mission to develop a portfolio of five life-transforming AAV-delivered therapeutics for the treatment of rare monogenic central nervous system diseases. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP) as well as the Orphan Disease Center (ODC). Pursuant to the research collaboration, GTP conducts IND-enabling preclinical work, and Passage Bio is responsible for clinical development, regulatory, manufacturing and commercialization of all product candidates. The ODC is responsible for natural history studies, KOL engagement, and patient advocacy outreach.

Prevail Therapeutics is a biotechnology company focused on developing novel gene therapies for patients with Parkinson’s disease and other neurodegenerative diseases. Prevail was launched in 2017 by The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, and is headquartered in New York, NY.

Passage Bio is a fully integrated gene therapy company with a mission to develop a portfolio of five life-transforming AAV-delivered therapeutics for the treatment of rare monogenic central nervous system diseases. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP) as well as the Orphan Disease Center (ODC). Pursuant to the research collaboration, GTP conducts IND-enabling preclinical work, and Passage Bio is responsible for clinical development, regulatory, manufacturing and commercialization of all product candidates. The ODC is responsible for natural history studies, KOL engagement, and patient advocacy outreach.

Modis Therapeutics is bringing disease-modifying therapies to patients with rare genetic diseases.

Therachon is a biotechnology company dedicated to developing treatments for rare genetic diseases that currently lack available therapies. Based in Basel, Switzerland, with research facilities in Nice, France, Therachon focuses on creating innovative therapeutics for conditions such as achondroplasia, the most prevalent form of short-limbed dwarfism, through its lead candidate, TA-46. The company also addresses other rare gastrointestinal and musculoskeletal disorders, aiming to provide effective solutions for severe medical conditions like short bowel syndrome.

Alector, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neurodegenerative diseases, including Alzheimer's disease and frontotemporal dementia. The company’s lead products, AL001, AL101, AL002, and AL003, are currently undergoing various stages of clinical trials, with AL001 in a phase II trial for frontotemporal dementia and AL101 in phase I trials targeting multiple neurodegenerative conditions. Alector employs advanced antibody technology combined with insights from neuroimmunology and human genetics to create therapeutics that address underlying disease mechanisms. The company has established a collaboration with Adimab, which specializes in the discovery and optimization of antibody therapeutics, to enhance its research and development efforts. Alector also has several additional programs in the research and development stage, reflecting its commitment to advancing novel treatments for complex neurological disorders. Founded in 2013 and headquartered in South San Francisco, California, Alector aims to restore healthy immune function in the brain through its unique therapeutic approaches.

Prevail Therapeutics is a biotechnology company focused on developing novel gene therapies for patients with Parkinson’s disease and other neurodegenerative diseases. Prevail was launched in 2017 by The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, and is headquartered in New York, NY.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, established in 2015. The company is dedicated to developing innovative therapies for patients with dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, ensuring that their therapeutic candidates are tailored to the specific molecular abnormalities found in patients with significant clinical needs. Their extensive pipeline includes a range of modalities such as monoclonal antibodies, recombinant proteins, and gene therapies. Supported by prominent life science investors and global academic collaborations, Gemini Therapeutics aims to create potentially first-in-class therapeutics to address unmet medical needs in the field of ophthalmology.

Cyrus Biotechnology, Inc. specializes in developing software tools for protein structure prediction and design, aiming to enhance research in biotechnology and pharmaceuticals. The company's flagship product, Rosetta, allows for the design of biologically active proteins that can address critical health issues, such as brain cancer treatment and gluten breakdown in Celiac Disease patients. Additionally, Cyrus Bench offers an enterprise version of the Rosetta toolkit, equipped with a variety of bio-molecular computation tools. The company also provides Cyrus CryoEM services, which include advanced structure refinement and model building. Founded in 2014 and headquartered in Seattle, Washington, Cyrus Biotechnology serves a diverse clientele, including pharmaceutical companies, venture-backed startups, and mid-sized biotech firms focused on therapeutic discovery and industrial biotechnology innovation.

Escape Bio is a clinical-stage biopharmaceutical company that develops small molecule drugs intended to treat neurodegenerative diseases. The company is developing therapeutics for the novel, precisely therapies for genetic neurodegenerative diseases. Escape Bio's pipeline includes candidates targeting known genetic drivers of multiple diseases, including ESB1609, a small molecule S1P5 receptor agonist for the treatment of CNS lysosomal storage disorders and ESB5070, a small molecule kinase inhibitor for Parkinson’s disease patients who have an LRRK2 G2019S variant and an Alzheimer's disease program targeting ApoE4. E-scape Bio was founded in 2015 and is headquartered in San Francisco, California, United States.

EmendoBio, Inc. is focused on advancing the field of genome editing by developing innovative gene editing tools aimed at overcoming challenges in gene therapy. The company leverages expertise in protein engineering and DNA repair, combining insights from top academic institutions to create genetic medicines that address currently untreatable disorders and diseases. By transforming existing methodologies and employing creative approaches, EmendoBio seeks to resolve significant bottlenecks in gene therapy, enabling healthcare professionals to provide effective treatments where none previously existed. Through a commitment to pushing scientific boundaries, EmendoBio strives to deliver on the potential of gene editing for transformative medical solutions.

Arrowhead Pharmaceuticals is a biotechnology company that focuses on the development of medicine to treat diseases with a genetic origin, characterized by the overproduction of one or more proteins. Its medical solutions are aimed at the genes that trigger the diseases. Its portfolio includes drugs targeting hepatitis B, Alpha-1 Antitrypsin Deficiency, thrombosis and angioedema, clear-cell and renal cell carcinoma, and cardiovascular disease.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, established in 2015. The company is dedicated to developing innovative therapies for patients with dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, ensuring that their therapeutic candidates are tailored to the specific molecular abnormalities found in patients with significant clinical needs. Their extensive pipeline includes a range of modalities such as monoclonal antibodies, recombinant proteins, and gene therapies. Supported by prominent life science investors and global academic collaborations, Gemini Therapeutics aims to create potentially first-in-class therapeutics to address unmet medical needs in the field of ophthalmology.

LogicBio Therapeutics is a genome editing company dedicated to developing innovative treatments for rare diseases, particularly those affecting pediatric patients with significant unmet medical needs. Utilizing its proprietary GeneRide technology, LogicBio aims to integrate corrective genes into patients' genomes, offering potential therapeutic benefits. The company's lead product candidate, LB-001, targets Methylmalonic Acidemia, a severe condition that manifests at birth. Additionally, LogicBio is advancing other candidates, including LB-301, which is being developed in collaboration with Takeda Pharmaceutical Company for Crigler-Najjar syndrome. The company also partners with the Children's Medical Research Institute to create new viral vectors. Founded in 2014 and based in Lexington, Massachusetts, LogicBio is focused on delivering safe and effective genetic medicine to patients suffering from previously untreatable genetic and infectious diseases.

Alector, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neurodegenerative diseases, including Alzheimer's disease and frontotemporal dementia. The company’s lead products, AL001, AL101, AL002, and AL003, are currently undergoing various stages of clinical trials, with AL001 in a phase II trial for frontotemporal dementia and AL101 in phase I trials targeting multiple neurodegenerative conditions. Alector employs advanced antibody technology combined with insights from neuroimmunology and human genetics to create therapeutics that address underlying disease mechanisms. The company has established a collaboration with Adimab, which specializes in the discovery and optimization of antibody therapeutics, to enhance its research and development efforts. Alector also has several additional programs in the research and development stage, reflecting its commitment to advancing novel treatments for complex neurological disorders. Founded in 2013 and headquartered in South San Francisco, California, Alector aims to restore healthy immune function in the brain through its unique therapeutic approaches.

Audentes Therapeutics, a clinical-stage biotechnology company based in San Francisco, is dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapies, including AT132 and AT342, which are in Phase I/II clinical studies for treating X-linked myotubular myopathy and Crigler-Najjar syndrome, respectively. Additionally, it is exploring treatments for Pompe disease and catecholaminergic polymorphic ventricular tachycardia, along with vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Audentes has established collaborative agreements for research and development with Genethon and the University of Pennsylvania. Founded in 2012, Audentes Therapeutics became a subsidiary of Astellas US Holding in 2020 and operates as Astellas Gene Therapies, focusing on leveraging its expertise in gene therapy research and commercialization.

Therachon is a biotechnology company dedicated to developing treatments for rare genetic diseases that currently lack available therapies. Based in Basel, Switzerland, with research facilities in Nice, France, Therachon focuses on creating innovative therapeutics for conditions such as achondroplasia, the most prevalent form of short-limbed dwarfism, through its lead candidate, TA-46. The company also addresses other rare gastrointestinal and musculoskeletal disorders, aiming to provide effective solutions for severe medical conditions like short bowel syndrome.

Alector, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neurodegenerative diseases, including Alzheimer's disease and frontotemporal dementia. The company’s lead products, AL001, AL101, AL002, and AL003, are currently undergoing various stages of clinical trials, with AL001 in a phase II trial for frontotemporal dementia and AL101 in phase I trials targeting multiple neurodegenerative conditions. Alector employs advanced antibody technology combined with insights from neuroimmunology and human genetics to create therapeutics that address underlying disease mechanisms. The company has established a collaboration with Adimab, which specializes in the discovery and optimization of antibody therapeutics, to enhance its research and development efforts. Alector also has several additional programs in the research and development stage, reflecting its commitment to advancing novel treatments for complex neurological disorders. Founded in 2013 and headquartered in South San Francisco, California, Alector aims to restore healthy immune function in the brain through its unique therapeutic approaches.

Intellia Therapeutics is a genome editing company headquartered in Cambridge, Massachusetts, that specializes in developing innovative therapeutics using the CRISPR/Cas9 technology. The company focuses on in vivo 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's ex vivo pipeline includes engineered cell therapies aimed at treating various oncological and autoimmune diseases. The company has entered into collaborations with notable organizations, including Novartis and Regeneron Pharmaceuticals, to advance its research and development efforts. Founded in 2014, Intellia Therapeutics is committed to addressing severe and life-threatening diseases through its comprehensive intellectual property platform and a broad range of clinical development programs.

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.

Dimension Therapeutics is a gene therapy company focused on developing novel treatments for rare diseases. Their team comprises biotech industry veterans and thought leaders in gene therapy and rare diseases. The company is focused on advancing its platform of gene therapy programs in rare diseases through clinical development, starting with lead programs in hemophilia, and building out a world-class product engine for adeno-associated virus (AAV) therapeutics. In conjunction with its launch, Dimension has entered into an exclusive license and collaboration with REGENX Biosciences. REGENX holds exclusive rights to a portfolio of over 100 patents and patent applications pertaining to its NAV vector technology and related applications. Through its license and collaboration with REGENX, Dimension has acquired preferred access to NAV vector technology and rights within REGENX product programs in multiple rare disease indications. Dimension has completed a Series A financing led by Fidelity Biosciences.

Natera, Inc. is a diagnostic company that specializes in preconception and prenatal genetic testing services. Its primary offerings include the Panorama test, which is a non-invasive prenatal test that screens for chromosomal abnormalities in a fetus through a blood sample from the mother, and the Vistara test, designed to identify single-gene disorders. Additionally, Natera provides Horizon carrier screening to determine genetic disease carrier status and Spectrum, which analyzes chromosomal anomalies during in vitro fertilization. The company also offers Anora, a test for analyzing fetal chromosomes to investigate miscarriage causes, and non-invasive paternity testing. Its Signatera product utilizes circulating tumor DNA technology to monitor cancer recurrence. Natera markets its tests through a direct sales force and a network of approximately 100 laboratory and distribution partners both domestically and internationally. The company, founded in 2003 and headquartered in San Carlos, California, maintains partnerships with BGI Genomics and Foundation Medicine for the development of genetic testing assays.

GENEWIZ, Inc. is a global contract research organization specializing in genomics and DNA sequencing services tailored for pharmaceutical, biotechnology, academic, agricultural, and government sectors. Founded in 1999 and headquartered in South Plainfield, New Jersey, GENEWIZ offers a comprehensive range of services including Sanger DNA Sequencing, gene synthesis, molecular biology applications, next-generation sequencing, and custom bioinformatics. The company is also equipped to provide regulatory-compliant laboratory services for FDA applications, such as RNA/DNA identity studies and single nucleotide polymorphism testing. With a focus on delivering high-quality data and robust technical support, GENEWIZ aims to enhance productivity in scientific research and facilitate advancements in translational medicine and healthcare. The organization operates multiple locations across the United States and internationally in countries including China, the United Kingdom, France, and Japan. As of November 2018, GENEWIZ functions as a subsidiary of Brooks Automation, Inc.

Invitae is a genetic information company focused on integrating genetic insights into everyday medical practice to enhance healthcare quality for a large population. The company specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates multiple genetic tests into a single platform. Invitae's offerings include assessments for genes linked to hereditary cancer, neurological disorders, cardiovascular conditions, pediatric issues, metabolic disorders, and other hereditary ailments. By providing high-quality testing with quicker turnaround times and competitive pricing, Invitae aims to make genetic testing more accessible and effective for patients and healthcare providers alike.

Dimension Therapeutics is a gene therapy company focused on developing novel treatments for rare diseases. Their team comprises biotech industry veterans and thought leaders in gene therapy and rare diseases. The company is focused on advancing its platform of gene therapy programs in rare diseases through clinical development, starting with lead programs in hemophilia, and building out a world-class product engine for adeno-associated virus (AAV) therapeutics. In conjunction with its launch, Dimension has entered into an exclusive license and collaboration with REGENX Biosciences. REGENX holds exclusive rights to a portfolio of over 100 patents and patent applications pertaining to its NAV vector technology and related applications. Through its license and collaboration with REGENX, Dimension has acquired preferred access to NAV vector technology and rights within REGENX product programs in multiple rare disease indications. Dimension has completed a Series A financing led by Fidelity Biosciences.

Loxo Oncology, Inc. is a biopharmaceutical company focused on developing targeted therapies for patients with genetically defined cancers in the United States. Founded in 2013 and headquartered in Stamford, Connecticut, the company is dedicated to rapidly advancing cancer treatments that demonstrate significant clinical potential. Its lead product candidate, larotrectinib, is an oral selective inhibitor of tropomyosin receptor kinase (TRK) currently undergoing various clinical trials for multiple tumor types, including lung, head and neck, melanoma, colorectal, sarcoma, and breast cancers. In addition to larotrectinib, Loxo Oncology is advancing preclinical programs, including LOXO-195, a candidate addressing resistance mechanisms, as well as inhibitors targeting RET fusion proteins and FGFR isoforms. The company collaborates with several partners, including Array BioPharma and Illumina, to enhance its drug discovery and development processes. Loxo Oncology aims to leverage insights from diagnostic advancements to identify actionable targets and develop innovative treatments tailored to patients with specific genetic vulnerabilities.

Igenica, Inc. is a biotherapeutic company based in Burlingame, California, dedicated to the discovery and development of antibodies and antibody-drug conjugates (ADCs) for cancer treatment. Founded in 2008, Igenica specializes in immunotherapies and functional antibodies designed to target tumors effectively. The company's notable products include IGN523, a humanized monoclonal antibody aimed at treating various hematologic and solid tumors; IGN786, an ADC; and IGN381, which targets immune checkpoints that complement existing therapies in the tumor microenvironment. Igenica has established a strategic oncology research agreement with MedImmune and is supported by a group of prominent life science investors, including The Column Group and OrbiMed. The company operates as a subsidiary of Pierre Fabre Medicament SA, with a leadership team that has significant experience in antibody drug discovery and commercialization.

Alector, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neurodegenerative diseases, including Alzheimer's disease and frontotemporal dementia. The company’s lead products, AL001, AL101, AL002, and AL003, are currently undergoing various stages of clinical trials, with AL001 in a phase II trial for frontotemporal dementia and AL101 in phase I trials targeting multiple neurodegenerative conditions. Alector employs advanced antibody technology combined with insights from neuroimmunology and human genetics to create therapeutics that address underlying disease mechanisms. The company has established a collaboration with Adimab, which specializes in the discovery and optimization of antibody therapeutics, to enhance its research and development efforts. Alector also has several additional programs in the research and development stage, reflecting its commitment to advancing novel treatments for complex neurological disorders. Founded in 2013 and headquartered in South San Francisco, California, Alector aims to restore healthy immune function in the brain through its unique therapeutic approaches.

Loxo Oncology, Inc. is a biopharmaceutical company focused on developing targeted therapies for patients with genetically defined cancers in the United States. Founded in 2013 and headquartered in Stamford, Connecticut, the company is dedicated to rapidly advancing cancer treatments that demonstrate significant clinical potential. Its lead product candidate, larotrectinib, is an oral selective inhibitor of tropomyosin receptor kinase (TRK) currently undergoing various clinical trials for multiple tumor types, including lung, head and neck, melanoma, colorectal, sarcoma, and breast cancers. In addition to larotrectinib, Loxo Oncology is advancing preclinical programs, including LOXO-195, a candidate addressing resistance mechanisms, as well as inhibitors targeting RET fusion proteins and FGFR isoforms. The company collaborates with several partners, including Array BioPharma and Illumina, to enhance its drug discovery and development processes. Loxo Oncology aims to leverage insights from diagnostic advancements to identify actionable targets and develop innovative treatments tailored to patients with specific genetic vulnerabilities.

Amoy Diagnostics is a research and development based diagnostic company focusing on molecular diagnostics for oncology precision medicine. AmoyDx has a portfolio of molecular diagnostic assays. Based on multiple technology platforms including PCR, NGS, FISH and IHC, AmoyDx's core product categories include single gene testing kits, multi-gene testing panels, NGS panels, and NGS analytical systems.

Audentes Therapeutics, a clinical-stage biotechnology company based in San Francisco, is dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapies, including AT132 and AT342, which are in Phase I/II clinical studies for treating X-linked myotubular myopathy and Crigler-Najjar syndrome, respectively. Additionally, it is exploring treatments for Pompe disease and catecholaminergic polymorphic ventricular tachycardia, along with vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Audentes has established collaborative agreements for research and development with Genethon and the University of Pennsylvania. Founded in 2012, Audentes Therapeutics became a subsidiary of Astellas US Holding in 2020 and operates as Astellas Gene Therapies, focusing on leveraging its expertise in gene therapy research and commercialization.

Natera, Inc. is a diagnostic company that specializes in preconception and prenatal genetic testing services. Its primary offerings include the Panorama test, which is a non-invasive prenatal test that screens for chromosomal abnormalities in a fetus through a blood sample from the mother, and the Vistara test, designed to identify single-gene disorders. Additionally, Natera provides Horizon carrier screening to determine genetic disease carrier status and Spectrum, which analyzes chromosomal anomalies during in vitro fertilization. The company also offers Anora, a test for analyzing fetal chromosomes to investigate miscarriage causes, and non-invasive paternity testing. Its Signatera product utilizes circulating tumor DNA technology to monitor cancer recurrence. Natera markets its tests through a direct sales force and a network of approximately 100 laboratory and distribution partners both domestically and internationally. The company, founded in 2003 and headquartered in San Carlos, California, maintains partnerships with BGI Genomics and Foundation Medicine for the development of genetic testing assays.

Igenica, Inc. is a biotherapeutic company based in Burlingame, California, dedicated to the discovery and development of antibodies and antibody-drug conjugates (ADCs) for cancer treatment. Founded in 2008, Igenica specializes in immunotherapies and functional antibodies designed to target tumors effectively. The company's notable products include IGN523, a humanized monoclonal antibody aimed at treating various hematologic and solid tumors; IGN786, an ADC; and IGN381, which targets immune checkpoints that complement existing therapies in the tumor microenvironment. Igenica has established a strategic oncology research agreement with MedImmune and is supported by a group of prominent life science investors, including The Column Group and OrbiMed. The company operates as a subsidiary of Pierre Fabre Medicament SA, with a leadership team that has significant experience in antibody drug discovery and commercialization.

Crown Bioscience International, through its subsidiary, Crown Bioscience Inc., is a global drug discovery and development solutions company headquartered in Taipei, Taiwan. Established in 2006, the company provides a range of translational platforms aimed at enhancing the understanding of drug efficacy, pharmacological profiles, and patient responses in oncology, cardiovascular, and metabolic diseases. Crown Bioscience specializes in offering preclinical research products, including antibodies for in vivo and in vitro studies, bioactive recombinant proteins, recombinant cell lines, and tumor tissue microarrays. The company serves the biotech and pharmaceutical industries, helping them accelerate drug development while reducing clinical failure rates. Crown Bioscience has strategic partnerships with organizations such as the Shanghai Model Organisms Center and Hubrecht Organoid Technology to enhance its offerings in genetically engineered models and organoid-based preclinical drug development services. The company operates facilities in the United States, United Kingdom, China, and Taiwan, positioning itself as a leader in precision medicine and drug discovery.

Complete Genomics specializes in high-quality, affordable DNA sequencing and analysis, facilitating large-scale research into the genetic mechanisms of drug responses and complex diseases. The company has developed a comprehensive genomics analysis platform that integrates proprietary human sequencing technology with advanced informatics and data management software. This approach ensures that customers receive ready-to-use data for genome-based research, supporting advancements in personalized medicine and broader genetic studies. By combining innovative technology with a unique market strategy, Complete Genomics aims to transform the landscape of DNA sequencing.

Igenica, Inc. is a biotherapeutic company based in Burlingame, California, dedicated to the discovery and development of antibodies and antibody-drug conjugates (ADCs) for cancer treatment. Founded in 2008, Igenica specializes in immunotherapies and functional antibodies designed to target tumors effectively. The company's notable products include IGN523, a humanized monoclonal antibody aimed at treating various hematologic and solid tumors; IGN786, an ADC; and IGN381, which targets immune checkpoints that complement existing therapies in the tumor microenvironment. Igenica has established a strategic oncology research agreement with MedImmune and is supported by a group of prominent life science investors, including The Column Group and OrbiMed. The company operates as a subsidiary of Pierre Fabre Medicament SA, with a leadership team that has significant experience in antibody drug discovery and commercialization.

Enobia develops therapies to treat serious genetic bone disorders for which there are no approved treatments.

As of August 20, 2008, SGX Pharmaceuticals, Inc. was acquired by Eli Lilly & Co. SGX Pharmaceuticals, Inc., a biotechnology company, focuses on the discovery, development, and commercialization of therapeutics for addressing unmet medical needs in oncology. The company's product pipeline includes drug candidates from its FAST drug discovery platform, which uses X-ray crystallography and complementary biophysical and biochemical methods, combined with medicinal and computational chemistry. Its drug development programs target the c-MET receptor tyrosine kinase (MET), an enzyme implicated in an array of cancers; and the BCR-ABL tyrosine kinase enzyme for the treatment of chronic myelogenous leukemia (CML), a bone marrow cancer. Under the MET development program, the company develops SGX523, which is in Phase I clinical development studies for solid tumor indications; and SGX126, a preclinical development product for solid tumors indications. Under the BCR-ABL Development Program, SGX Pharmaceuticals develops SGX393, an oral therapy for the second-line treatment of CML, which is in preclinical development studies. Its drug discovery technologies are also applied to a portfolio of oncology targets, including JAK2, a non-receptor tyrosine kinase involved in cytokine-induced signaling and growth regulation, survival, and differentiation of cells; RAS, a protein that regulates cell growth; and three other tyrosine kinases. The company has a license and collaboration agreement with Novartis Institutes for Biomedical Research, Inc., Cystic Fibrosis Foundation Therapeutics, Inc., National Institutes of Health, and Eli Lilly & Company. SGX Pharmaceuticals, formerly known as Structural GenomiX, Inc., was founded in 1998 and is headquartered in San Diego, California.

Enobia develops therapies to treat serious genetic bone disorders for which there are no approved treatments.

Predix Pharmaceuticals is a drug discovery and development company specializing in small-molecule drugs that target G-protein coupled receptors (GPCRs) and ion channels. The company employs an innovative 3D Discovery and Optimization Engine that utilizes a proprietary algorithm for computer modeling the 3D structures of GPCR targets. Its advanced technologies enable high-throughput in silico screening of millions of compounds against these models, facilitating lead optimization through an integrated medicinal and computational platform. Predix's drug candidates are aimed at treating various conditions, including anxiety, Alzheimer's disease, and pulmonary arterial hypertension. In August, the company expanded its capabilities in drug discovery through a merger with Physiome Sciences Inc., enhancing its expertise in ion channel research.

Predix Pharmaceuticals is a drug discovery and development company specializing in small-molecule drugs that target G-protein coupled receptors (GPCRs) and ion channels. The company employs an innovative 3D Discovery and Optimization Engine that utilizes a proprietary algorithm for computer modeling the 3D structures of GPCR targets. Its advanced technologies enable high-throughput in silico screening of millions of compounds against these models, facilitating lead optimization through an integrated medicinal and computational platform. Predix's drug candidates are aimed at treating various conditions, including anxiety, Alzheimer's disease, and pulmonary arterial hypertension. In August, the company expanded its capabilities in drug discovery through a merger with Physiome Sciences Inc., enhancing its expertise in ion channel research.

GeneFormatics is a biotechnology company specializing in structural proteomics.

Predix Pharmaceuticals is a drug discovery and development company specializing in small-molecule drugs that target G-protein coupled receptors (GPCRs) and ion channels. The company employs an innovative 3D Discovery and Optimization Engine that utilizes a proprietary algorithm for computer modeling the 3D structures of GPCR targets. Its advanced technologies enable high-throughput in silico screening of millions of compounds against these models, facilitating lead optimization through an integrated medicinal and computational platform. Predix's drug candidates are aimed at treating various conditions, including anxiety, Alzheimer's disease, and pulmonary arterial hypertension. In August, the company expanded its capabilities in drug discovery through a merger with Physiome Sciences Inc., enhancing its expertise in ion channel research.

Molecular Staging, Inc. is a life sciences tool company focused on developing technologies for the detection and measurement of proteins and nucleic acids. Its offerings encompass various applications in proteomics, genomics, pharmacogenomics, and diagnostics. Notable products include protein microarrays, which allow researchers to analyze multiple proteins simultaneously, and whole-genome amplification technology that addresses challenges posed by limited DNA sample quantities. Additionally, the company provides multiplexed diagnostics, facilitating the earlier detection of diseases and enhancing confirmatory diagnoses. Through these innovations, Molecular Staging supports advancements in research and clinical applications.

Predix Pharmaceuticals is a drug discovery and development company specializing in small-molecule drugs that target G-protein coupled receptors (GPCRs) and ion channels. The company employs an innovative 3D Discovery and Optimization Engine that utilizes a proprietary algorithm for computer modeling the 3D structures of GPCR targets. Its advanced technologies enable high-throughput in silico screening of millions of compounds against these models, facilitating lead optimization through an integrated medicinal and computational platform. Predix's drug candidates are aimed at treating various conditions, including anxiety, Alzheimer's disease, and pulmonary arterial hypertension. In August, the company expanded its capabilities in drug discovery through a merger with Physiome Sciences Inc., enhancing its expertise in ion channel research.

GeneFormatics is a biotechnology company specializing in structural proteomics.