OrbiMed

Garuda Therapeutics is a biotechnology company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening blood, bone marrow, immune, and metabolic diseases. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants. Utilizing advanced technologies, including zebrafish, mice, and human-induced pluripotent stem cells, Garuda Therapeutics conducts research to clone and analyze novel genes and mechanisms critical for blood formation. This innovative approach is designed to provide patients with consistent, durable, and HLA-compatible transgene-free blood stem cell therapies, offering rapid and broad access to life-saving treatments.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of 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 treat aging and age-related diseases. The company utilizes a proprietary systems biology and artificial intelligence platform to identify significant drug targets that influence aging, leveraging extensive datasets to uncover the molecular drivers of age-related pathologies. Among its key offerings is BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor that activates HIF-1 target genes involved in various biological processes, including tissue regeneration and vascular remodeling. Another notable product is BGE-175, an orally administered inhibitor targeting the prostaglandin D2 DP1 signaling pathway, which is linked to an increased risk of mortality and infections, including those associated with immune aging. Founded in 2015 and headquartered in Richmond, California, BioAge Labs is dedicated to harnessing the biology of human aging to create innovative therapies for metabolic and age-related diseases.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

Rampart Bioscience is a biotechnology company focused on developing gene medicines that aim to deliver long-lasting treatments for various diseases. The company has created proprietary development and delivery platforms that generate optimized therapeutics in a revocable, non-viral format. By integrating expertise from multiple scientific disciplines, including gene delivery, protein sciences, and clinical translation, Rampart Bioscience works to assist patients with genetically driven diseases. Their innovative approach seeks to enhance the effectiveness and safety of gene therapies, ultimately improving patient outcomes.

MinervaX is a biotechnology company focused on developing maternal vaccines to prevent infections caused by Group B streptococcus (GBS), which can lead to serious adverse pregnancy outcomes and life-threatening conditions for newborns. The company leverages a specific fusion protein that has shown promise in eliciting protective immunity against various clinically relevant GBS strains. By concentrating on this innovative vaccine approach, MinervaX aims to provide medical professionals with effective tools to safeguard newborns from bacterial infections, ultimately enhancing maternal and infant health outcomes.

ReCode Therapeutics, Inc. 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 utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments for cancer patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of targeted therapies that address unmet medical needs in oncology. Their drug development process involves replicating naturally occurring mutations in laboratory settings, allowing for the testing of these mutations' effects on signaling pathways and their responsiveness to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharmaceutical companies expand the subgroups of cancer patients who may benefit from existing 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. Established in 2019, the company specializes in genetic medicine by utilizing a proprietary platform that employs endogenous enzymes, specifically adenosine deaminase acting on RNA (ADAR), to precisely target and correct single point mutations in RNA transcripts. This innovative approach aims to restore the production of functional proteins, offering potential treatments for a variety of diseases, including those affecting genetic, cardiometabolic, complement-mediated, and central nervous system areas. ADARx Pharmaceuticals is dedicated to developing life-saving therapeutics that address genetic mutations and provide solutions for conditions that are currently considered incurable.

Garuda Therapeutics is a biotechnology company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening blood, bone marrow, immune, and metabolic diseases. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants. Utilizing advanced technologies, including zebrafish, mice, and human-induced pluripotent stem cells, Garuda Therapeutics conducts research to clone and analyze novel genes and mechanisms critical for blood formation. This innovative approach is designed to provide patients with consistent, durable, and HLA-compatible transgene-free blood stem cell therapies, offering rapid and broad access to life-saving treatments.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Established in 2019, the company specializes in genetic medicine by utilizing a proprietary platform that employs endogenous enzymes, specifically adenosine deaminase acting on RNA (ADAR), to precisely target and correct single point mutations in RNA transcripts. This innovative approach aims to restore the production of functional proteins, offering potential treatments for a variety of diseases, including those affecting genetic, cardiometabolic, complement-mediated, and central nervous system areas. ADARx Pharmaceuticals is dedicated to developing life-saving therapeutics that address genetic mutations and provide solutions for conditions that are currently considered incurable.

iECURE is a biotechnology company dedicated to developing innovative gene editing therapies aimed at treating rare and life-threatening liver disorders, particularly in children. Its approach employs mutation-agnostic in vivo gene insertion techniques, which allow for targeted interventions in diseases characterized by metabolic "loss of function" disorders. By focusing on conditions with high unmet medical needs, iECURE aims to offer potential cures for devastating illnesses that currently lack effective treatment options. The company's commitment to advancing gene editing technology positions it as a key player in the field of genetic medicine.

Frontera Therapeutics is a company specializing in adeno-associated virus (AAV) gene therapy, dedicated to creating a low-cost and scalable platform for the development and production of high-quality recombinant AAV therapies. The company focuses on commercializing cost-effective gene therapies aimed at treating a range of genetic disorders, including those affecting ophthalmology, liver, metabolic, and neuromuscular diseases. Frontera Therapeutics has established a robust product pipeline that encompasses multiple therapeutic areas, with various candidates at different stages of development. Through its innovative approach, the company aims to improve patient outcomes by making genetic therapies more accessible and affordable in the global market.

Aspen Neuroscience, Inc. is a biotechnology company based in San Diego, California, focused on developing innovative therapies for neurological conditions, particularly Parkinson's disease. Founded in 2018, the company specializes in induced pluripotent stem cells (iPSCs) to create patient-specific cell therapies. Its product pipeline includes ANPD001, an autologous neuron replacement therapy for sporadic Parkinson's disease, and ANPD002, a gene-edited version targeting familial forms of the disease. By combining genomic approaches with stem cell biology, Aspen Neuroscience aims to address significant unmet medical needs and modify the progression of Parkinson's disease through personalized and restorative treatments.

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.

ReCode Therapeutics, Inc. 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 utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Garuda Therapeutics is a biotechnology company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening blood, bone marrow, immune, and metabolic diseases. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants. Utilizing advanced technologies, including zebrafish, mice, and human-induced pluripotent stem cells, Garuda Therapeutics conducts research to clone and analyze novel genes and mechanisms critical for blood formation. This innovative approach is designed to provide patients with consistent, durable, and HLA-compatible transgene-free blood stem cell therapies, offering rapid and broad access to life-saving treatments.

iECURE is a biotechnology company dedicated to developing innovative gene editing therapies aimed at treating rare and life-threatening liver disorders, particularly in children. Its approach employs mutation-agnostic in vivo gene insertion techniques, which allow for targeted interventions in diseases characterized by metabolic "loss of function" disorders. By focusing on conditions with high unmet medical needs, iECURE aims to offer potential cures for devastating illnesses that currently lack effective treatment options. The company's commitment to advancing gene editing technology positions it as a key player in the field of genetic medicine.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Established in 2019, the company specializes in genetic medicine by utilizing a proprietary platform that employs endogenous enzymes, specifically adenosine deaminase acting on RNA (ADAR), to precisely target and correct single point mutations in RNA transcripts. This innovative approach aims to restore the production of functional proteins, offering potential treatments for a variety of diseases, including those affecting genetic, cardiometabolic, complement-mediated, and central nervous system areas. ADARx Pharmaceuticals is dedicated to developing life-saving therapeutics that address genetic mutations and provide solutions for conditions that are currently considered incurable.

GentiBio, Inc. is a biotherapeutics company that specializes in developing engineered regulatory T cells, known as 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, Washington, GentiBio employs a proprietary platform that combines autologous and allogeneic technologies. This platform enhances the ability to restore immune tolerance and addresses significant limitations found in existing regulatory T-cell therapeutics. By focusing on the underlying mechanisms of immune system disorders, GentiBio aims to provide innovative therapeutic solutions that tackle the root causes of diseases caused by dysregulated immune responses. The company is co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of advanced immunotherapy development.

Biocytogen LLC is a biotechnology company based in Worcester, Massachusetts, that specializes in developing gene-targeted animal models for life science research. Founded in 2008, the company offers a range of services, including customized and off-the-shelf mouse models, phenotype analysis, and advanced gene-editing techniques. Its products encompass various types of mice, such as knockin and knockout models, as well as humanized mice, catering to diverse research areas including immunology, neurology, cancer, and infectious diseases. Biocytogen also operates a state-of-the-art animal facility to support its integrated platform for efficient antibody drug discovery and validation. The company collaborates with leading pharmaceutical and biotechnology firms to enhance preclinical research and development processes in the biomedical 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.

Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapies utilizing engineered CRISPR technologies to address genetic disorders. Founded in 2018 and based in Berkeley, California, Scribe Therapeutics is known for its proprietary X-Editing (XE) molecules, which are advanced CRISPR enzymes designed to enhance efficacy, specificity, and deliverability compared to existing genome editing methods. The company's platform aims to overcome the limitations of current technologies by creating custom-engineered enzymes and delivery systems that facilitate precise genetic modifications. Scribe Therapeutics is committed to establishing CRISPR-based therapies as a new standard of clinical care, with the potential to significantly impact the treatment landscape for a variety of diseases, including those related to immuno-oncology, degenerative disorders, and enzyme replacement therapy.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that focuses on targeted DNA integration to address severe genetic diseases. The firm employs advanced technology to enable the precise insertion of genetic sequences, effectively correcting mutations, replacing malfunctioning genes, and introducing therapeutic genetic materials into specific regions of the genome. By harnessing high-efficiency targeted gene integration, Graphite Bio aims to develop novel therapies that could potentially cure a wide array of serious and life-threatening conditions. Formerly known as Integral Medicines, Inc., the company rebranded in August 2020 and has operated since its incorporation in 2019. Graphite Bio is committed to the pioneering of a precision gene editing approach designed to fulfill the medical challenge of accurately "finding and replacing" genes.

Century Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies derived from induced pluripotent stem cells (iPSCs) to treat hematologic and solid malignancies. Founded in 2018 and headquartered in Philadelphia, Pennsylvania, the company utilizes a specialized platform that integrates gene editing and protein engineering to create allogeneic therapies. This platform allows for the generation of master cell banks of modified immune effector cells, specifically engineered to optimize performance and reduce the risk of rejection by the host immune system. By employing advanced technologies such as CRISPR-mediated gene editing and proprietary chimeric antigen receptors, Century Therapeutics aims to address significant unmet medical needs in cancer and other diseases.

AnchorDx is a biotechnology company specializing in next-generation sequencing techniques, particularly through the use of molecular liquid biopsies. This innovative approach allows for non-invasive access to genetic information, enabling patients to gain insights into their physiological conditions and better prepare for potential health issues. With a core team boasting over 20 years of experience in cancer genomics, genetics, and bioinformatics, AnchorDx aims to lead in precision medicine by developing comprehensive clinical applications and research platforms. The company fosters collaborations with various stakeholders, including hospitals, research institutes, and third-party testing agencies, to create a complete industrial chain that facilitates the transformation of academic research into clinical products. Through its commitment to developing independent intellectual property and competitive clinical testing solutions, AnchorDx provides accurate and comprehensive services to patients, healthcare providers, and medical institutions.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

Decibel Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing innovative therapies aimed at protecting, repairing, and restoring hearing. The company addresses various hearing disorders, including those related to pediatric cancers, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as conditions characterized by persistent tinnitus. Decibel Therapeutics employs a multifaceted approach that includes drug delivery to the inner ear, advanced imaging techniques, and pharmacokinetic and pharmacodynamic modeling. The company leverages its expertise in inner ear biology, single-cell genomics, and bioinformatics to develop effective treatments for hearing loss and related disorders. Established in 2013 and headquartered in Boston, Massachusetts, Decibel Therapeutics was originally known as Hearing, Inc. before rebranding in 2014.

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 for cancer patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of targeted therapies that address unmet medical needs in oncology. Their drug development process involves replicating naturally occurring mutations in laboratory settings, allowing for the testing of these mutations' effects on signaling pathways and their responsiveness to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharmaceutical companies expand the subgroups of cancer patients who may benefit from existing therapies.

ESCAPE Bio is a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

GentiBio, Inc. is a biotherapeutics company that specializes in developing engineered regulatory T cells, known as 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, Washington, GentiBio employs a proprietary platform that combines autologous and allogeneic technologies. This platform enhances the ability to restore immune tolerance and addresses significant limitations found in existing regulatory T-cell therapeutics. By focusing on the underlying mechanisms of immune system disorders, GentiBio aims to provide innovative therapeutic solutions that tackle the root causes of diseases caused by dysregulated immune responses. The company is co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of advanced immunotherapy development.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

Aspen Neuroscience, Inc. is a biotechnology company based in San Diego, California, focused on developing innovative therapies for neurological conditions, particularly Parkinson's disease. Founded in 2018, the company specializes in induced pluripotent stem cells (iPSCs) to create patient-specific cell therapies. Its product pipeline includes ANPD001, an autologous neuron replacement therapy for sporadic Parkinson's disease, and ANPD002, a gene-edited version targeting familial forms of the disease. By combining genomic approaches with stem cell biology, Aspen Neuroscience aims to address significant unmet medical needs and modify the progression of Parkinson's disease through personalized and restorative treatments.

ReCode Therapeutics, Inc. 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 utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Emendo Biotherapeutics is a biotechnology company focused on advancing genome editing technologies to develop genetic medicines. Established by a team of experts with backgrounds in protein engineering and DNA repair from prestigious institutions, Emendo aims to tackle the challenges associated with gene therapy. By innovating and transforming existing tools, the company seeks to address diseases and disorders that are presently deemed untreatable. Emendo's approach fuses diverse scientific disciplines with creative problem-solving, enabling the development of advanced gene editing solutions that enhance the efficacy of gene therapy and expand its potential applications. Through this commitment to pushing the boundaries of science, Emendo Biotherapeutics strives to fulfill the promise of gene editing for improved patient outcomes.

Aspen Neuroscience, Inc. is a biotechnology company based in San Diego, California, focused on developing innovative therapies for neurological conditions, particularly Parkinson's disease. Founded in 2018, the company specializes in induced pluripotent stem cells (iPSCs) to create patient-specific cell therapies. Its product pipeline includes ANPD001, an autologous neuron replacement therapy for sporadic Parkinson's disease, and ANPD002, a gene-edited version targeting familial forms of the disease. By combining genomic approaches with stem cell biology, Aspen Neuroscience aims to address significant unmet medical needs and modify the progression of Parkinson's disease through personalized and restorative treatments.

Repare Therapeutics Inc. is a precision oncology company based in Montréal, Canada, that specializes in discovering and developing targeted therapeutics through a synthetic lethality approach. Utilizing its proprietary SNIPRx platform, which integrates genome-wide analysis and CRISPR technology, Repare systematically identifies and advances cancer therapies aimed at genomic instability, particularly in DNA damage repair. The company's lead candidate, RP-3500, is an oral small molecule inhibitor designed for solid tumors with specific genomic alterations related to DNA damage repair. In addition, Repare is developing innovative programs such as a CCNE1-SL inhibitor for tumors with CCNE1 amplification and a Polymerase Theta program targeting BRCA mutations and other genomic changes. Founded in 2016, Repare Therapeutics is focused on addressing the unique vulnerabilities of tumor cells in genetically defined patient populations to enhance cancer treatment outcomes.

Passage Bio, Inc. is a genetic medicines company based in Philadelphia, Pennsylvania, dedicated to developing transformative therapies for rare monogenic central nervous system (CNS) diseases. Established 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 taking responsibility for the clinical development, regulatory processes, manufacturing, and commercialization of its product candidates. The partnership also involves research and development collaborations that support the advancement of innovative treatments for these rare disorders.

Passage Bio, Inc. is a genetic medicines company based in Philadelphia, Pennsylvania, dedicated to developing transformative therapies for rare monogenic central nervous system (CNS) diseases. Established 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 taking responsibility for the clinical development, regulatory processes, manufacturing, and commercialization of its product candidates. The partnership also involves research and development collaborations that support the advancement of innovative treatments for these rare disorders.

Modis Therapeutics, Inc. is a biopharmaceutical company focused on developing disease-modifying therapies for rare genetic diseases characterized by high unmet medical needs. Incorporated in 2016 and based in Oakland, California, the company is known for its lead therapy, MT1621, which aims to restore mitochondrial DNA replication fidelity. This therapy specifically targets thymidine kinase 2 deficiency (TK2d), an inherited mitochondrial disorder. By improving nucleotide balance and increasing mitochondrial DNA copy number, Modis Therapeutics' approach seeks to enhance cellular function and prolong the lives of affected patients. As of September 2019, Modis Therapeutics operates as a subsidiary of Zogenix, Inc.

Therachon is a biotechnology company specializing in the development of treatments for rare genetic diseases that lack available therapies. Primarily based in Basel, Switzerland, with research labs in Nice, France, the company is advancing its lead candidate, TA-46, a novel protein therapy aimed at treating achondroplasia, the most prevalent form of short-limbed dwarfism. In addition to its focus on achondroplasia, Therachon is also committed to developing therapeutics targeting rare gastrointestinal and musculoskeletal disorders, with the goal of enabling medical professionals to effectively manage conditions such as short bowel syndrome.

Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapeutics for unmet medical needs in cancer treatment. The company specializes in the field of epigenetics, aiming to address cancers associated with abnormal gene expression and drug resistance. Its lead product candidates include CPI-0610, which is undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, currently in Phase Ib/II trials for metastatic castration-resistant prostate cancer in conjunction with androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the Phase I dose escalation stage of a Phase I/II trial aimed at treating solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. before rebranding to its current name in March 2008.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

ESCAPE Bio is a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

LogicBio Therapeutics, Inc. is a genome editing company dedicated to developing innovative treatments for rare diseases, particularly for pediatric patients with significant unmet medical needs. Utilizing its proprietary GeneRide technology platform, LogicBio aims to integrate corrective genes into patients' genomes to achieve therapeutic effects. The company's lead product candidate, LB-001, targets Methylmalonic Acidemia, a serious condition that manifests at birth. Additionally, LogicBio is advancing other product candidates, including LB-101 and LB-301, the latter developed in collaboration with Takeda Pharmaceutical Company to address Crigler-Najjar syndrome. The company also partners with the Children's Medical Research Institute to create new viral vectors, enhancing the potential of its gene therapy approaches. Founded in 2014, LogicBio is headquartered in Lexington, Massachusetts.

Mitoconix Bio Ltd is a biotechnology company established in 2016 and headquartered in Jerusalem, Israel, focused on developing innovative therapies aimed at enhancing mitochondrial health to treat neurodegenerative diseases. The company employs a disease-modifying approach that targets the functions of mitochondria, which are essential for generating ATP, regulating calcium, and maintaining cellular balance. Mitochondria play a crucial role in neuron function, undergoing constant changes in structure through processes known as fission and fusion. Disruptions in these processes can lead to reduced energy production and increased oxidative stress, contributing to cellular dysfunction and death. Mitoconix's lead drug is a first-in-class inhibitor designed to counteract pathological mitochondrial fragmentation and has shown promising efficacy in preclinical models of Huntington's disease and Parkinson's disease, as well as beneficial effects in patient-derived cells associated with these conditions and Alzheimer's disease.

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

Therachon is a biotechnology company specializing in the development of treatments for rare genetic diseases that lack available therapies. Primarily based in Basel, Switzerland, with research labs in Nice, France, the company is advancing its lead candidate, TA-46, a novel protein therapy aimed at treating achondroplasia, the most prevalent form of short-limbed dwarfism. In addition to its focus on achondroplasia, Therachon is also committed to developing therapeutics targeting rare gastrointestinal and musculoskeletal disorders, with the goal of enabling medical professionals to effectively manage conditions such as short bowel syndrome.

Emendo Biotherapeutics is a biotechnology company focused on advancing genome editing technologies to develop genetic medicines. Established by a team of experts with backgrounds in protein engineering and DNA repair from prestigious institutions, Emendo aims to tackle the challenges associated with gene therapy. By innovating and transforming existing tools, the company seeks to address diseases and disorders that are presently deemed untreatable. Emendo's approach fuses diverse scientific disciplines with creative problem-solving, enabling the development of advanced gene editing solutions that enhance the efficacy of gene therapy and expand its potential applications. Through this commitment to pushing the boundaries of science, Emendo Biotherapeutics strives to fulfill the promise of gene editing for improved patient outcomes.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments for cancer patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of targeted therapies that address unmet medical needs in oncology. Their drug development process involves replicating naturally occurring mutations in laboratory settings, allowing for the testing of these mutations' effects on signaling pathways and their responsiveness to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharmaceutical companies expand the subgroups of cancer patients who may benefit from existing therapies.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

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

LogicBio Therapeutics, Inc. is a genome editing company dedicated to developing innovative treatments for rare diseases, particularly for pediatric patients with significant unmet medical needs. Utilizing its proprietary GeneRide technology platform, LogicBio aims to integrate corrective genes into patients' genomes to achieve therapeutic effects. The company's lead product candidate, LB-001, targets Methylmalonic Acidemia, a serious condition that manifests at birth. Additionally, LogicBio is advancing other product candidates, including LB-101 and LB-301, the latter developed in collaboration with Takeda Pharmaceutical Company to address Crigler-Najjar syndrome. The company also partners with the Children's Medical Research Institute to create new viral vectors, enhancing the potential of its gene therapy approaches. Founded in 2014, LogicBio is headquartered in Lexington, Massachusetts.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

Therachon is a biotechnology company specializing in the development of treatments for rare genetic diseases that lack available therapies. Primarily based in Basel, Switzerland, with research labs in Nice, France, the company is advancing its lead candidate, TA-46, a novel protein therapy aimed at treating achondroplasia, the most prevalent form of short-limbed dwarfism. In addition to its focus on achondroplasia, Therachon is also committed to developing therapeutics targeting rare gastrointestinal and musculoskeletal disorders, with the goal of enabling medical professionals to effectively manage conditions such as short bowel syndrome.

Intellia Therapeutics, Inc. is a genome editing company headquartered in Cambridge, Massachusetts, specializing in the development of therapeutics using the CRISPR/Cas9 technology. This innovative platform allows for precise alterations of genomic DNA, enabling the company to pursue treatments for a range of severe and life-threatening diseases. Intellia's pipeline includes both in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as ex vivo programs focused on engineered cell therapies for oncological and autoimmune disorders. The company is also exploring treatments for conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Intellia has established collaborations with several organizations, enhancing its research and development capabilities. Founded in 2014, Intellia holds a significant intellectual property portfolio related to the therapeutic applications of CRISPR/Cas9, aiming to address high unmet medical needs through its advanced gene-editing approaches.

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, Inc. is a gene therapy company based in Cambridge, Massachusetts, specializing in the development of innovative treatments for rare and metabolic diseases associated with the liver. Founded in 2013, the company has a robust pipeline of gene therapy programs, including DTX101 and DTX201 for hemophilia B and A, respectively. Additionally, its portfolio encompasses DTX301 for ornithine transcarbamylase deficiency, DTX401 for glycogen storage disease type Ia, DTX501 for phenylketonuria, DTX701 for Wilson disease, and DTX601 for citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, gaining access to a significant portfolio of patents related to adeno-associated virus (AAV) therapeutics. This collaboration supports the company's aim to advance its gene therapy platform and enhance its product development capabilities in the treatment of rare diseases. Dimension Therapeutics operates as a subsidiary of Ultragenyx Pharmaceutical Inc. since November 2017.

Natera, Inc. is a diagnostic and research company specializing in genetic testing services, particularly in the areas of preconception and prenatal diagnostics. Founded in 2003 and headquartered in San Carlos, California, Natera offers a range of products including the Panorama non-invasive prenatal test, which screens for chromosomal abnormalities in fetuses, and Vistara, a test for single-gene mutations. Additionally, the company provides Horizon carrier screening to assess carrier status for various genetic diseases, and Spectrum, which analyzes genetic conditions during in vitro fertilization. Natera's Anora product analyzes fetal chromosomes to understand miscarriage causes, while its non-invasive paternity tests determine parentage through fetal DNA analysis. The company's Signatera technology focuses on circulating tumor DNA to monitor cancer recurrence. Natera distributes its products through a direct sales force and a network of approximately 100 laboratory and distribution partners, both domestically and internationally. The company collaborates with BGI Genomics to develop genetic testing assays and has partnered with Foundation Medicine for personalized cancer monitoring assays.

GENEWIZ, Inc. is a global contract research organization specializing in genomics and DNA sequencing services. Founded in 1999 and headquartered in South Plainfield, New Jersey, the company serves a diverse clientele, including pharmaceutical, biotechnology, academic, agricultural, clinical, and government institutions. GENEWIZ offers a comprehensive range of services, including Sanger DNA sequencing, gene synthesis, molecular biology techniques, genomic mutation analysis, and bioinformatics, all designed to enhance research productivity and efficiency. The company is also equipped to provide regulatory-compliant laboratory services for FDA applications, including RNA/DNA identity studies and SNP testing. With a focus on delivering high-quality data and exceptional technical support, GENEWIZ is committed to accelerating scientific discovery and advancing translational medicine. The company operates multiple locations across the United States and internationally in China, the United Kingdom, France, Germany, and Japan, solidifying its position as a preferred partner in the life sciences sector. As of November 2018, GENEWIZ operates as a subsidiary of Brooks Automation, Inc.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

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, Inc. is a gene therapy company based in Cambridge, Massachusetts, specializing in the development of innovative treatments for rare and metabolic diseases associated with the liver. Founded in 2013, the company has a robust pipeline of gene therapy programs, including DTX101 and DTX201 for hemophilia B and A, respectively. Additionally, its portfolio encompasses DTX301 for ornithine transcarbamylase deficiency, DTX401 for glycogen storage disease type Ia, DTX501 for phenylketonuria, DTX701 for Wilson disease, and DTX601 for citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, gaining access to a significant portfolio of patents related to adeno-associated virus (AAV) therapeutics. This collaboration supports the company's aim to advance its gene therapy platform and enhance its product development capabilities in the treatment of rare diseases. Dimension Therapeutics operates as a subsidiary of Ultragenyx Pharmaceutical Inc. since November 2017.

Igenica, Inc. is a biotherapeutic company based in Burlingame, California, that specializes in the discovery and development of antibodies and antibody-drug conjugates (ADCs) for cancer treatment. Founded in 2008, the company focuses on creating innovative immunotherapies and functional antibodies targeting tumors. Its product pipeline includes IGN523, a humanized monoclonal antibody for various hematologic and solid tumors; IGN786, an antibody-drug conjugate; and IGN381, an immune checkpoint target that enhances the tumor microenvironment. Igenica operates with a strategic oncology research agreement with MedImmune and is supported by a leadership team experienced in antibody drug discovery and clinical development. As of 2017, Igenica operates as a subsidiary of Pierre Fabre Medicament SA, positioning it to leverage additional resources and expertise in the biotherapeutics field.

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, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

Natera, Inc. is a diagnostic and research company specializing in genetic testing services, particularly in the areas of preconception and prenatal diagnostics. Founded in 2003 and headquartered in San Carlos, California, Natera offers a range of products including the Panorama non-invasive prenatal test, which screens for chromosomal abnormalities in fetuses, and Vistara, a test for single-gene mutations. Additionally, the company provides Horizon carrier screening to assess carrier status for various genetic diseases, and Spectrum, which analyzes genetic conditions during in vitro fertilization. Natera's Anora product analyzes fetal chromosomes to understand miscarriage causes, while its non-invasive paternity tests determine parentage through fetal DNA analysis. The company's Signatera technology focuses on circulating tumor DNA to monitor cancer recurrence. Natera distributes its products through a direct sales force and a network of approximately 100 laboratory and distribution partners, both domestically and internationally. The company collaborates with BGI Genomics to develop genetic testing assays and has partnered with Foundation Medicine for personalized cancer monitoring assays.

Igenica, Inc. is a biotherapeutic company based in Burlingame, California, that specializes in the discovery and development of antibodies and antibody-drug conjugates (ADCs) for cancer treatment. Founded in 2008, the company focuses on creating innovative immunotherapies and functional antibodies targeting tumors. Its product pipeline includes IGN523, a humanized monoclonal antibody for various hematologic and solid tumors; IGN786, an antibody-drug conjugate; and IGN381, an immune checkpoint target that enhances the tumor microenvironment. Igenica operates with a strategic oncology research agreement with MedImmune and is supported by a leadership team experienced in antibody drug discovery and clinical development. As of 2017, Igenica operates as a subsidiary of Pierre Fabre Medicament SA, positioning it to leverage additional resources and expertise in the biotherapeutics field.

Good Start Genetics, Inc. is a molecular diagnostics company based in Cambridge, Massachusetts, founded in 2007. It specializes in genetic screening for inherited diseases, aiming to transform reproductive medicine by providing actionable information to physicians and patients. The company offers a range of services, including EmbryVu, a preimplantation genetic screening test designed to enhance in vitro fertilization outcomes by identifying suitable embryos for implantation. Additionally, Good Start Genetics provides a comprehensive array of genetic carrier screening tests through its GoodStart Select program, utilizing advanced next-generation DNA sequencing technology along with other established genetic screening methods. This focus on high carrier detection rates and commitment to customer care and genetic counseling positions the company as a leader in the field of pre-pregnancy genetic testing and diagnostics. 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. is a molecular diagnostics company based in Cambridge, Massachusetts, founded in 2007. It specializes in genetic screening for inherited diseases, aiming to transform reproductive medicine by providing actionable information to physicians and patients. The company offers a range of services, including EmbryVu, a preimplantation genetic screening test designed to enhance in vitro fertilization outcomes by identifying suitable embryos for implantation. Additionally, Good Start Genetics provides a comprehensive array of genetic carrier screening tests through its GoodStart Select program, utilizing advanced next-generation DNA sequencing technology along with other established genetic screening methods. This focus on high carrier detection rates and commitment to customer care and genetic counseling positions the company as a leader in the field of pre-pregnancy genetic testing and diagnostics. As of August 2017, Good Start Genetics operates as a subsidiary of Invitae Corporation.

Complete Genomics is a developer of advanced DNA sequencing technology focused on human genome sequencing and analysis. The company offers a comprehensive genomics analysis platform that integrates its proprietary sequencing technology with informatics and data management software. This combination allows Complete Genomics to deliver high-quality, affordable DNA sequencing services that facilitate large-scale research into the genetic mechanisms underlying drug responses and complex diseases. By providing ready-to-use genomic data, Complete Genomics aims to enhance the capabilities of researchers in the field of genomics and contribute to advancements in personalized medicine.

Igenica, Inc. is a biotherapeutic company based in Burlingame, California, that specializes in the discovery and development of antibodies and antibody-drug conjugates (ADCs) for cancer treatment. Founded in 2008, the company focuses on creating innovative immunotherapies and functional antibodies targeting tumors. Its product pipeline includes IGN523, a humanized monoclonal antibody for various hematologic and solid tumors; IGN786, an antibody-drug conjugate; and IGN381, an immune checkpoint target that enhances the tumor microenvironment. Igenica operates with a strategic oncology research agreement with MedImmune and is supported by a leadership team experienced in antibody drug discovery and clinical development. As of 2017, Igenica operates as a subsidiary of Pierre Fabre Medicament SA, positioning it to leverage additional resources and expertise in the biotherapeutics field.

Complete Genomics is a developer of advanced DNA sequencing technology focused on human genome sequencing and analysis. The company offers a comprehensive genomics analysis platform that integrates its proprietary sequencing technology with informatics and data management software. This combination allows Complete Genomics to deliver high-quality, affordable DNA sequencing services that facilitate large-scale research into the genetic mechanisms underlying drug responses and complex diseases. By providing ready-to-use genomic data, Complete Genomics aims to enhance the capabilities of researchers in the field of genomics and contribute to advancements in personalized medicine.

Enobia Pharma is a biotechnology company focused on developing innovative therapies for serious genetic bone disorders that currently lack approved treatment options. The company specializes in addressing conditions such as hypophosphatasia (HPP), a rare inherited disorder that affects bone mineralization and can lead to severe complications. By concentrating on these unmet medical needs, Enobia aims to provide effective solutions that improve patient outcomes and quality of life for individuals suffering from these challenging conditions.

MacroGenics, Inc. is a biopharmaceutical company based in Rockville, Maryland, specializing in the discovery and development of antibody-based therapeutics for cancer treatment. Founded in 2000, the company focuses on immuno-oncology and has a diverse pipeline of product candidates. Key developments include Margetuximab, a monoclonal antibody in Phase III clinical trials targeting HER2-expressing tumors, and Flotetuzumab, a DART molecule aimed at treating acute myeloid leukemia. Other investigational products include MGA012, MGD013, MGD019, and Enoblituzumab, which target various immune checkpoints and tumor-associated antigens. MacroGenics is also advancing combination therapies and antibody-drug conjugates, such as MGC018, which targets solid tumors expressing B7-H3. The company has formed strategic collaborations with notable partners, enhancing its research and development efforts in the biopharmaceutical landscape.

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 Pharma is a biotechnology company focused on developing innovative therapies for serious genetic bone disorders that currently lack approved treatment options. The company specializes in addressing conditions such as hypophosphatasia (HPP), a rare inherited disorder that affects bone mineralization and can lead to severe complications. By concentrating on these unmet medical needs, Enobia aims to provide effective solutions that improve patient outcomes and quality of life for individuals suffering from these challenging conditions.

MacroGenics, Inc. is a biopharmaceutical company based in Rockville, Maryland, specializing in the discovery and development of antibody-based therapeutics for cancer treatment. Founded in 2000, the company focuses on immuno-oncology and has a diverse pipeline of product candidates. Key developments include Margetuximab, a monoclonal antibody in Phase III clinical trials targeting HER2-expressing tumors, and Flotetuzumab, a DART molecule aimed at treating acute myeloid leukemia. Other investigational products include MGA012, MGD013, MGD019, and Enoblituzumab, which target various immune checkpoints and tumor-associated antigens. MacroGenics is also advancing combination therapies and antibody-drug conjugates, such as MGC018, which targets solid tumors expressing B7-H3. The company has formed strategic collaborations with notable partners, enhancing its research and development efforts in the biopharmaceutical landscape.

Predix Pharmaceuticals Holdings is a drug discovery and development company specializing in small-molecule drugs targeting G-protein coupled receptors (GPCRs) and ion channels. The company employs an innovative 3D Discovery and Optimization Engine, which includes its proprietary PREDICT algorithm for modeling the 3D structure of GPCR targets. Additionally, Predix utilizes advanced technologies such as HYPERION and RISS for high-throughput in silico screening of millions of compounds against these models. Its integrated medicinal and computational platform, ICELR-3D, aids in lead optimization by employing predictive algorithms for crucial drug properties. Predix's research focuses on developing treatments for various conditions, including anxiety, Alzheimer's disease, and pulmonary arterial hypertension. In a strategic move to enhance its capabilities, Predix merged with Physiome Sciences Inc., thereby expanding its expertise into ion channel drug discovery.

MacroGenics, Inc. is a biopharmaceutical company based in Rockville, Maryland, specializing in the discovery and development of antibody-based therapeutics for cancer treatment. Founded in 2000, the company focuses on immuno-oncology and has a diverse pipeline of product candidates. Key developments include Margetuximab, a monoclonal antibody in Phase III clinical trials targeting HER2-expressing tumors, and Flotetuzumab, a DART molecule aimed at treating acute myeloid leukemia. Other investigational products include MGA012, MGD013, MGD019, and Enoblituzumab, which target various immune checkpoints and tumor-associated antigens. MacroGenics is also advancing combination therapies and antibody-drug conjugates, such as MGC018, which targets solid tumors expressing B7-H3. The company has formed strategic collaborations with notable partners, enhancing its research and development efforts in the biopharmaceutical landscape.

Predix Pharmaceuticals Holdings is a drug discovery and development company specializing in small-molecule drugs targeting G-protein coupled receptors (GPCRs) and ion channels. The company employs an innovative 3D Discovery and Optimization Engine, which includes its proprietary PREDICT algorithm for modeling the 3D structure of GPCR targets. Additionally, Predix utilizes advanced technologies such as HYPERION and RISS for high-throughput in silico screening of millions of compounds against these models. Its integrated medicinal and computational platform, ICELR-3D, aids in lead optimization by employing predictive algorithms for crucial drug properties. Predix's research focuses on developing treatments for various conditions, including anxiety, Alzheimer's disease, and pulmonary arterial hypertension. In a strategic move to enhance its capabilities, Predix merged with Physiome Sciences Inc., thereby expanding its expertise into ion channel drug discovery.

MacroGenics, Inc. is a biopharmaceutical company based in Rockville, Maryland, specializing in the discovery and development of antibody-based therapeutics for cancer treatment. Founded in 2000, the company focuses on immuno-oncology and has a diverse pipeline of product candidates. Key developments include Margetuximab, a monoclonal antibody in Phase III clinical trials targeting HER2-expressing tumors, and Flotetuzumab, a DART molecule aimed at treating acute myeloid leukemia. Other investigational products include MGA012, MGD013, MGD019, and Enoblituzumab, which target various immune checkpoints and tumor-associated antigens. MacroGenics is also advancing combination therapies and antibody-drug conjugates, such as MGC018, which targets solid tumors expressing B7-H3. The company has formed strategic collaborations with notable partners, enhancing its research and development efforts in the biopharmaceutical landscape.

MacroGenics, Inc. is a biopharmaceutical company based in Rockville, Maryland, specializing in the discovery and development of antibody-based therapeutics for cancer treatment. Founded in 2000, the company focuses on immuno-oncology and has a diverse pipeline of product candidates. Key developments include Margetuximab, a monoclonal antibody in Phase III clinical trials targeting HER2-expressing tumors, and Flotetuzumab, a DART molecule aimed at treating acute myeloid leukemia. Other investigational products include MGA012, MGD013, MGD019, and Enoblituzumab, which target various immune checkpoints and tumor-associated antigens. MacroGenics is also advancing combination therapies and antibody-drug conjugates, such as MGC018, which targets solid tumors expressing B7-H3. The company has formed strategic collaborations with notable partners, enhancing its research and development efforts in the biopharmaceutical landscape.

Predix Pharmaceuticals Holdings is a drug discovery and development company specializing in small-molecule drugs targeting G-protein coupled receptors (GPCRs) and ion channels. The company employs an innovative 3D Discovery and Optimization Engine, which includes its proprietary PREDICT algorithm for modeling the 3D structure of GPCR targets. Additionally, Predix utilizes advanced technologies such as HYPERION and RISS for high-throughput in silico screening of millions of compounds against these models. Its integrated medicinal and computational platform, ICELR-3D, aids in lead optimization by employing predictive algorithms for crucial drug properties. Predix's research focuses on developing treatments for various conditions, including anxiety, Alzheimer's disease, and pulmonary arterial hypertension. In a strategic move to enhance its capabilities, Predix merged with Physiome Sciences Inc., thereby expanding its expertise into ion channel drug discovery.

Molecular Staging Inc., a life sciences tool company, specializes in developing technologies for the detection and measurement of proteins and nucleic acids. Its offerings include protein microarrays for proteomics research, whole-genome amplification tools to overcome limited DNA sample constraints, and multiplexed diagnostics for enhanced disease detection. These products cater to applications in genomics, pharmacogenomics, and diagnostics.

Predix Pharmaceuticals Holdings is a drug discovery and development company specializing in small-molecule drugs targeting G-protein coupled receptors (GPCRs) and ion channels. The company employs an innovative 3D Discovery and Optimization Engine, which includes its proprietary PREDICT algorithm for modeling the 3D structure of GPCR targets. Additionally, Predix utilizes advanced technologies such as HYPERION and RISS for high-throughput in silico screening of millions of compounds against these models. Its integrated medicinal and computational platform, ICELR-3D, aids in lead optimization by employing predictive algorithms for crucial drug properties. Predix's research focuses on developing treatments for various conditions, including anxiety, Alzheimer's disease, and pulmonary arterial hypertension. In a strategic move to enhance its capabilities, Predix merged with Physiome Sciences Inc., thereby expanding its expertise into ion channel drug discovery.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments for cancer patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of targeted therapies that address unmet medical needs in oncology. Their drug development process involves replicating naturally occurring mutations in laboratory settings, allowing for the testing of these mutations' effects on signaling pathways and their responsiveness to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharmaceutical companies expand the subgroups of cancer patients who may benefit from existing therapies.