OrbiMed Advisors LLC is a healthcare-focused investment firm founded in 1989, headquartered in New York City, with additional offices in various global locations. The firm manages approximately $5 billion in assets and specializes in private equity and venture capital investments across the healthcare sector, including pharmaceuticals, biotechnology, medical devices, and healthcare services. OrbiMed invests in companies at various stages, from early startups to established multinationals, and typically seeks to take majority stakes in its private equity investments. The firm employs fundamental analysis to guide its investment decisions and offers a range of financial services, including debt financing and structured capital, to both public and private healthcare companies. OrbiMed's investment strategy encompasses a broad spectrum of healthcare-related opportunities, targeting investments globally, primarily in North America, Europe, and Asia.
Garuda Therapeutics is a biotechnology company focused on creating off-the-shelf hematopoietic stem cell therapies aimed at treating various severe and life-threatening conditions. The company specializes in addressing blood, bone marrow, immune, and metabolic diseases. By leveraging expertise in innovative research methods, including the use of zebrafish, mice, and human-induced pluripotent stem cells, Garuda analyzes novel genes and mechanisms vital for blood formation. This approach enables the development of mechanism-based therapies designed to improve patient outcomes.
Delfi Diagnostics, Inc. is a biotechnology company based in Baltimore, Maryland, that focuses on the early detection of cancer through non-invasive blood tests. Founded in 2018, the company utilizes advanced technologies such as artificial intelligence and genome sequencing to identify unique patterns of DNA fragmentation in patients' blood. By leveraging machine learning, Delfi develops innovative testing systems aimed at recognizing cancer-associated cell-free DNA fragments, thereby enabling healthcare professionals to detect cancer at its most treatable stage. This approach facilitates timely and effective treatment options, ultimately aiming to improve patient outcomes and recovery speed.
MiroBio Ltd is a biotechnology company based in Oxford, United Kingdom, focused on developing antibody modulators for immune cell receptors aimed at treating autoimmune diseases. Founded in 2018, MiroBio leverages pioneering research from the University of Oxford to understand how immune cells communicate and are activated. This foundational knowledge allows the company to create innovative antibodies that stimulate specific immune cell signals, harnessing the body's natural control mechanisms to restore balance within the immune system. By activating these natural pathways, MiroBio's therapeutics aim to provide significant benefits to patients suffering from various autoimmune conditions, enabling clinicians to effectively manage overactive immune responses associated with these diseases.
Frontera Therapeutics is an AAV gene therapy company with the mission to establish a low-cost and scalable platform, to develop and produce high-quality and affordably priced rAAV therapies for the global market.
Aspen Neuroscience, Inc. is a biotechnology company based in San Diego, California, focused on developing induced pluripotent stem cell therapies for neurological conditions, particularly Parkinson's disease. Founded in 2018, the company aims to create patient-specific restorative cell therapies that can alter the progression of the disease. Its product pipeline includes ANPD001, an autologous neuron replacement therapy designed for sporadic Parkinson's disease, and ANPD002, a gene-edited version targeting familial forms of the disease. By integrating innovative genomic approaches with stem cell biology, Aspen Neuroscience seeks to advance the field of personalized medicine and improve treatment outcomes for patients suffering from Parkinson's disease.
Developer of life sciences technology intended to develop highly effective and differentiated treatments for cystic fibrosis. The company is developing first-in-class small molecules that are focused on a unique target with the potential to fully normalize CFTR function for the first time for the vast majority of people living with cystic fibrosis, enabling patients with best-in-class efficacy and reach and long-term benefit for people with cystic fibrosis.
Electra Therapeutics is a clinical-stage biotechnology company focused on developing therapies that target signal regulatory proteins (SIRP) for treating immunological diseases and cancer. The company has one program in clinical development and two additional preclinical programs. Its lead product candidate, ELA026, is a monoclonal antibody designed to target SIRP on the surface of myeloid and T cells, effectively depleting pathological immune cells. ELA026 is currently being investigated for secondary hemophagocytic lymphohistiocytosis (sHLH), a severe hyperinflammatory condition for which no approved treatment exists.
ImmPACT Bio USA Inc. is a cell therapy company aiming to develop potent and selective engineered T-cells for the treatment of solid tumors. The Company's technology enables targeting loss-of-gene features of solid tumor cells, sparing normal, healthy tissues.
Cyrus Biotechnology builds software tools to accelerate basic research in biotech, pharma, and industrial biotechnology. Cyrus Bench delivers an enterprise version of the Rosetta molecular modeling and design toolkit, with the associated array of bio-molecular computation tools.
It was founded in 2014 and headquartered in Seattle, Washington.
Acelyrin is a late-stage clinical biopharmaceutical company dedicated to delivering innovative treatment options to patients. The company specializes in identifying, acquiring, and accelerating the development and commercialization of promising drug candidates. By leveraging its expertise, Acelyrin aims to advance transformative medicines that can significantly improve patient outcomes.
ReCode Therapeutics is a development-stage biopharmaceutical company focused on the correction of the nonsense mutations that account for 1/3 of all inherited diseases, including approximately 10% of cystic fibrosis. They have combined selected tRNAs with their unique and diverse proprietary delivery platforms to create and develop first-in-class suppressor tRNA-nanoparticle therapeutics for correcting nonsense mutations. ReCode’s diverse pipeline includes lead programs for primary ciliary dyskinesia (PCD) and nonsense mutations in cystic fibrosis (CF). The company’s proprietary non-viral lipid nanoparticle platform enables the delivery of a variety of payloads, and precise organ targeting of nucleic acid and protein payloads via both systemic and local delivery. ReCode is leveraging its nanoparticle and nucleic acid technologies across multiple modalities, including mRNA-mediated protein replacement therapies, first-in-class tRNA NanoCorrectors for diseases caused by nonsense mutations, and gene editing. The company was founded in 2015 and is based in Dallas, Texas.
Scout Bio, Inc. researches and develops one-time therapies for major chronic pet health conditions using veterinary biotechnology and gene therapy. It offers therapies for anemia associated with chronic kidney disease (CKD) in cats. The company offers pet therapeutics, including small molecular drugs, protein therapies, and gene therapies. Scout Bio, Inc. was founded in 2016 and is headquartered in Philadelphia, Pennsylvania.
Ventyx Biosciences, Inc. is a biotechnology company based in Encinitas, California, focused on developing selective inhibitors of TYK2 for the treatment of autoimmune diseases. Incorporated in 2018, the company boasts a diverse portfolio of innovative clinical and pre-clinical programs aimed at high-value therapeutic targets. Through its research and development efforts, Ventyx is committed to advancing treatments that address unmet medical needs in the autoimmune disease space.
Amolyt Pharma is a clinical-stage biotechnology company focused on developing peptide-based therapies for rare endocrine and metabolic disorders. The company's leading programs include AZP-3601, a parathyroid hormone analog aimed at treating hypoparathyroidism by maintaining sustained calcium levels in the blood, and AZP-3404, which features a novel mechanism of action that may help restore fat and glucose metabolism. Through its innovative approach, Amolyt Pharma seeks to provide effective treatments for patients suffering from critical and rare metabolic conditions.
Operator of a biopharmaceutical company intended to discover and develop next-generation medicines for neurodegenerative diseases. The company is focused on developing small-molecule activators of glucocerebrosidase and advanced programs targeting the innate immune system that can accelerate the progression of several neurological diseases, enabling healthcare professionals to transform the lives of patients with treatments for various ailments.
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 utilizing a proprietary platform that leverages endogenous enzymes, specifically adenosine deaminase acting on RNA (ADAR), to precisely target and correct single point mutations in mRNA transcripts. This innovative approach aims to restore the production of functional proteins, addressing various genetic mutations that contribute to diseases. ADARx is developing a pipeline of therapeutics designed to treat a range of conditions, including genetic, cardiometabolic, complement-mediated, and central nervous system diseases, offering potential solutions for previously incurable ailments.
Disc Medicine, Inc. is a biotechnology company focused on developing therapeutics based on human genetics to address ineffective red blood cell production in hematologic diseases. The company specializes in programs targeting the hepcidin metabolism axis for the treatment of disorders related to hepcidin. Founded in 2017 and headquartered in Cambridge, Massachusetts, Disc Medicine is led by a team of experienced scientists and industry experts, supported by a panel of advisors with a proven history in discovering and developing innovative therapies. The company was established with backing from Atlas Venture, along with contributions from Novo Ventures and Access Biotechnology.
Neurogastrx, Inc. is a venture-stage biopharmaceutical company focused on developing innovative therapies for gastrointestinal disorders. Established in 2017 and located in Campbell, California, the company aims to address the unmet medical needs associated with functional and motility disorders of the gastrointestinal tract. One of its key products, NG101, is designed to enhance gastric motility while also possessing antiemetic properties. Neurogastrx's specialty therapies target the enteric nervous system, addressing issues related to gut contractions, sensation, and the brain-gut axis, ultimately striving to alleviate the burden of these disorders on patients and the healthcare system.
Vigil will use the Series A funds to progress its lead pipeline candidate, a monoclonal antibody TREM2 agonist, through Phase 1 studies, advance their small molecule TREM2 agonist to IND, as well as pursue additional assets to grow the pipeline and increase the body of data supporting microglia biology as an important therapeutic pathway.
XinThera is a drug discovery company dedicated to developing precision medicines aimed at treating cancer and immunologic diseases. By utilizing advanced computational chemistry technologies, XinThera designs small molecule therapies that target well-validated and previously challenging targets in oncology and immunology. The company's focus on these promising targets enhances the potential for effective drug therapies, ultimately benefiting patients who face difficult-to-treat conditions.
Zentera Therapeutics
Series B in 2021
Zentera Therapeutics is a biopharmaceutical company.
NeRRe Therapeutics Ltd. is a biotechnology company based in Stevenage, United Kingdom, established in 2012. The company specializes in the development of neurokinin (NK) receptor antagonists, focusing on treatments for conditions related to intense pruritus, particularly in patients undergoing targeted anti-cancer therapies. Its lead asset, orvepitant, is an oral NK-1 antagonist designed to alleviate this specific symptom. Additionally, NeRRe is advancing other candidates, including NT-814, a dual NK-1 and NK-3 antagonist, and NT-949, which is ready for Phase I trials. The company also has NT-432, a clinical candidate targeting NK-1 receptors. NeRRe Therapeutics aims to address unmet medical needs through its innovative therapeutic approaches.
Splisense
Series B in 2021
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.
Janux Therapeutics develops immunotherapies that generate immune responses to prevent tumors and not affecting a patient’s healthy tissue. Janux technology targets all three stages of the anti-tumor immune response. Janux Therapeutics, Inc. was founded in 2017 by David Campbell and is based in La Jolla, California.
Theseus Pharmaceuticals
Series B in 2021
Theseus Pharmaceuticals is a Technology based company.
Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics using CRISPR technology to address genetic disorders. Founded in 2017 and based in Berkeley, California, Scribe specializes in engineering CRISPR enzymes known as X-Editing (XE) molecules, which enhance the efficacy, specificity, and deliverability of genome editing compared to existing methods. The company's proprietary platform enables the creation of custom-engineered enzymes and delivery systems, facilitating precision targeting within the genome. Scribe Therapeutics aims to establish CRISPR-based therapies as a standard in clinical care, expanding access to transformative treatments for a wide range of conditions, including immuno-oncology and degenerative disorders. Through its commitment to overcoming the limitations of current genome editing technologies, Scribe Therapeutics is poised to make substantial contributions to human therapeutics.
Graphite Bio is a gene editing company focused on targeted DNA integration to address severe diseases. The company employs technology that enables the insertion of new genetic sequences to replace defective genes, repair damaged portions of genes, and insert therapeutic genetic material at specific locations within the genome. This precise approach leverages natural cellular DNA repair mechanisms to enhance the efficiency of gene integration, aiming to correct the underlying causes of various diseases. Founded in 2019 and based in South San Francisco, California, Graphite Bio was previously known as Integral Medicines, Inc. and rebranded in August 2020. The company is committed to advancing gene replacement therapies and potential cures for individuals affected by serious health conditions.
Yisheng Biopharma Co., Ltd. is a biopharmaceutical company focused on the research, development, manufacturing, and marketing of immuno-oncology products and vaccines. Founded in 2002 and headquartered in Beijing, the company operates in China, the United States, Cambodia, and Singapore. Yisheng Biopharma utilizes its novel PIKA immunomodulating technology to enhance immune responses against cancers and infectious diseases. Key products under development include YS-ON-001, an investigational therapy aimed at treating advanced solid tumors, as well as YS-HBV-001, a vaccine for hepatitis B, and a PIKA-based rabies vaccine designed for rapid protection against rabies infection. The company's innovative approach targets multiple immune pathways to improve treatment outcomes.
Noctrix Health, Inc. is an early-stage medical device company based in Menlo Park, California, founded in 2018. The company specializes in the development of therapeutic wearable devices aimed at treating chronic neurological and sleep disorders. By focusing on innovative therapies, Noctrix Health seeks to address the unmet needs of patients suffering from these conditions.
Visen Pharmaceuticals is a biotechnology company dedicated to the development and commercialization of innovative endocrine drugs. The company aims to address significant unmet medical needs for patients in Greater China, focusing specifically on endocrine treatment solutions. By leveraging its expertise in biotechnology, Visen Pharmaceuticals seeks to improve patient outcomes through the creation of effective therapies in the field of endocrinology.
Scorpion Therapeutics, Inc. is a biotechnology company focused on developing next-generation precision oncology technologies aimed at improving cancer treatment. Founded in 2020 and based in Boston, Massachusetts, the company specializes in creating precision oncology drugs that target known oncogenes, address currently undruggable cancer targets, and explore new therapeutic targets. By integrating advanced technologies in target discovery, medicinal chemistry, and translational medicine, Scorpion Therapeutics seeks to expand the reach of precision medicine to a broader patient population. The company's approach is designed to unlock novel small-molecule drugs that can overcome the limitations of existing therapies, thereby enhancing treatment options for cancer patients.
Ikena Oncology is dedicated to bringing next generation oncology therapies to patients suffering from cancer. Ikena Oncology are committed to leveraging our unique understanding of the complex biologic pathways that drive the formation and spread of cancers to discover and develop novel, patient-directed therapies. Selecting patients for treatment in clinical studies with biomarkers we develop is at the core of our strategy.
RayzeBio, Inc. is a biotechnology company based in San Diego, California, founded in 2020. The company specializes in developing tumor-targeted small molecule medicines that utilize radioisotopes, particularly focusing on alpha-emitting isotopes like Actinium-225 for the treatment of solid tumors. RayzeBio aims to improve cancer treatment outcomes by creating a robust pipeline of radiopharmaceuticals targeting validated oncology drug targets. Its portfolio includes multiple drug candidates at various stages of development, ranging from discovery to late-stage clinical programs, addressing significant market opportunities in the field of cancer therapeutics.
Invivyd is a biotechnology company focused on developing advanced antibodies to neutralize SARS-CoV-2, SARS-CoV-1, and other potentially emerging coronaviruses. Founded in 2020 and headquartered in Waltham, Massachusetts, the company aims to create best-in-class therapeutic and prophylactic treatments that surpass the efficacy of existing antibody programs. Its innovative portfolio features multiple non-competing antibodies, each targeting distinct viral epitopes, which is designed to minimize the risk of viral escape. By leveraging cutting-edge antibody discovery technologies, Invivyd seeks to enhance the potency and durability of its antibody candidates, addressing a critical need in the ongoing fight against coronaviruses.
Q32 Bio is a biotechnology company developing treatments for patients with severe autoimmune and inflammatory diseases. Q32 Bio is addressing dysregulation to help patients take back control of their lives in diseases with few to no treatment options, or those diseases where current therapeutics provide inadequate relief.
Q32 Bio consists of a team of scientists and industry veterans with strong expertise in immunology. The team has a strong track record of discovering and developing new and innovative therapies.
Q32 Bio is a biotechnology company developing treatments for patients with severe autoimmune and inflammatory diseases. Q32 Bio is addressing dysregulation to help patients take back control of their lives in diseases with few to no treatment options, or those diseases where current therapeutics provide inadequate relief.
Q32 Bio consists of a team of scientists and industry veterans with strong expertise in immunology. The team has a strong track record of discovering and developing new and innovative therapies.
Zhaoke Ophthalmology Pharmaceutical
Series B in 2020
Zhaoke Ophthalmology Pharmaceutical is a Pharmaceutical platform.
E-Scape Bio, Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule therapies for neurodegenerative diseases. Established in 2015 and headquartered 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. E-Scape Bio is advancing a pipeline of therapeutics that target specific genetic drivers of these diseases, including ESB1609, a small molecule S1P5 receptor agonist for CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor designed for Parkinson’s disease patients with the LRRK2 G2019S variant. Additionally, the company is developing a program targeting ApoE4 in Alzheimer’s disease, emphasizing its commitment to precision medicine in treating genetically defined subpopulations.
Erasca, Inc. is a clinical-stage precision oncology company focused on discovering, developing, and commercializing therapies for cancers driven by the RAS/MAPK pathway. Founded in 2018 and headquartered in San Diego, California, Erasca utilizes its oncology pattern recognition algorithm (OPRA), an artificial intelligence-driven drug discovery platform, to analyze large-scale data sets and uncover novel tumor biology that can inhibit key cancer pathways. The company is advancing multiple drug candidates, including Naporafenib, an innovative pan-RAF inhibitor aimed at treating NRAS mutant melanoma and other RAS/MAPK-driven tumors, as well as ERAS-007 and ERAS-601, which are oral inhibitors targeting ERK1/2 and SHP2, respectively. Through collaborations with academic and biopharmaceutical partners, Erasca aims to expand its pipeline and provide new therapeutic options for cancer patients.
VelosBio, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-drug conjugates (ADCs) to treat hematological cancers and solid tumors. The company specializes in therapies targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), a cell-surface protein commonly found in various tumors but absent in normal tissues, making it a promising target for cancer treatment. Its lead product, VLS-101, is designed as a ROR1-directed ADC aimed at patients with both hematologic and solid tumor malignancies. Founded in 2017, VelosBio has demonstrated positive preclinical results for its ROR1-directed therapeutics, paving the way for potential use in monotherapy or in combination with other treatments across a wide range of cancers. As of late 2020, VelosBio operates as a subsidiary of Merck & Co., Inc.
MabPlex International Ltd provides contract development and manufacturing of biopharmaceuticals including, monoclonal antibodies (mAbs), recombinant proteins, Antibody Drug Conjugates (ADCs) and bispecifics. It offers customized mammalian cell medium, cell banking, monoclonal antibodiy active pharmaceutical ingredient (API) manufacturing, and biologics and ADC fill and finish manufacturing. The company was founded in 2013 and is based in Yantai, China with an additional office in Fremont, California.
AbCellera Biologics Inc. is a biotechnology company that specializes in the discovery and development of therapeutic antibodies. Founded in 2012 and headquartered in Vancouver, Canada, it has created a full-stack, AI-powered platform that analyzes natural immune systems to identify antibodies suitable for drug development. The company employs next-generation transgenic mice, which generate fully human antibodies, enhancing its ability to discover effective therapeutic candidates. AbCellera's innovative approach addresses the limitations of traditional antibody discovery by enabling faster and more precise identification of optimal clinical candidates. It has established partnerships with various pharmaceutical companies, including a significant collaboration with Eli Lilly and Company to explore multiple targets. Through its proprietary technologies and expertise, AbCellera aims to streamline the antibody discovery process and support the development of next-generation therapeutics.
Pandion Therapeutics is a clinical-stage biotechnology company focused on developing innovative therapeutics for autoimmune diseases. The company's lead product candidate, PT101, is an engineered variant of interleukin-2 currently undergoing Phase 1a clinical trials to address various autoimmune and inflammatory conditions. In addition to PT101, Pandion's pipeline includes PT627, a systemic PD-1 agonist in preclinical studies, and two bifunctional molecules, PT001 and PT002, designed to facilitate tissue-selective immunomodulation in the gastrointestinal tract. PT001 combines a PD-1 agonist with a tether module that binds to mucosal vascular addressing cell adhesion molecule (MAdCAM), while PT002 merges an IL-2 mutein effector with a MAdCAM-binding module. The company has also established a collaboration with Astellas Pharma to develop locally acting immunomodulators for pancreatic autoimmune diseases. Headquartered in Watertown, Massachusetts, Pandion Therapeutics aims to create effective and safe treatments to improve patient outcomes in the field of autoimmune disorders.
Silverback Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, focused on developing tissue-targeted therapeutics for cancer, chronic viral infections, and other serious diseases. The company's lead product candidate, SBT6050, is currently in a Phase I/Ib clinical trial. It is a TLR8 agonist linker-payload conjugated to a HER2-directed monoclonal antibody, specifically targeting tumors such as breast, gastric, and non-small cell lung cancers. Additionally, Silverback is advancing SBT6290, a preclinical candidate that targets Nectin4, expressed in various cancers, and SBT8230, aimed at treating chronic hepatitis B virus infection. Utilizing its proprietary ImmunoTAC technology, Silverback aims to develop a new generation of therapies that can be systemically delivered yet act locally at disease sites, thereby modulating critical disease pathways that were previously considered inaccessible.
NewMed Medical Co., Ltd. is a Shanghai-based company focused on the research, development, manufacture, and marketing of interventional artificial heart valve systems. Established in 2015, the company specializes in producing artificial heart valves and transcather implantation systems designed to replace diseased mitral valves. These innovative products aim to simplify heart valve replacement surgery, thereby reducing treatment risks and facilitating quicker patient recovery while also lowering surgical costs. Through its advancements in medical technology, NewMed Medical contributes to improved outcomes in cardiac care.
Emendo Bio, Inc. is a biotechnology company focused on developing innovative genome editing technologies aimed at repairing and eliminating genetic mutations responsible for serious diseases and disorders. Founded in 2015 and headquartered in New York, the company integrates expertise in protein engineering, DNA repair, and computational analysis to advance its gene editing capabilities. Emendo Bio is dedicated to creating genetic medicines that address currently untreatable conditions by transforming existing tools and methodologies. The company operates as a subsidiary of AnGes, Inc., emphasizing a collaborative approach that combines knowledge from diverse scientific disciplines to overcome challenges in the field of gene therapy. Through its cutting-edge research, Emendo Bio aims to push the boundaries of what is possible in the realm of genetic medicine.
Kinnate Biopharma develops precision oncology therapeutics to treat patients with genomically-defined cancers. Its mission is to expand the reach of targeted therapeutics by developing products for underserved populations.
The company utilizes its deep expertise in structure-based drug discovery, translational research, and patient-driven precision medicine. Its programs include candidates in preclinical development for cancers that are driven by specific oncogenic alterations in either the BRAF kinase gene or in the FGFR2 and FGFR3 kinase genes.
Edgewise Therapeutics is a clinical-stage biopharmaceutical company based in Boulder, Colorado, specializing in the development of small molecule therapies for severe musculoskeletal diseases. Founded in 2017, the company leverages its expertise in skeletal muscle physiology to create innovative treatments for genetically defined muscle disorders, including Duchenne, Becker, and limb girdle muscular dystrophies. Edgewise Therapeutics employs a precision medicine platform to identify and target key muscle proteins and modulators, aiming to address the underlying causes of these conditions. Additionally, the company has established a state-of-the-art research facility to enhance its capabilities in understanding muscle adaptation and injury related to disease.
Repare Therapeutics is a developer of oncology drugs designed to target specific vulnerabilities of tumor cells. The company's drugs combine a proprietary high throughput gene-editing and target discovery method with high-resolution protein crystallography, computational biology and clinical informatics, enabling patients to improve cancer treatment both with single therapy and in combination with existing drugs and treatments.
Passage Bio is a genetic medicines company based in Philadelphia, Pennsylvania, specializing in the development of therapies for rare monogenic central nervous system (CNS) diseases. Founded in 2017, the company aims to create a portfolio of five AAV-delivered therapeutics, targeting conditions such as GM1 Gangliosidosis, Frontotemporal dementia, and Krabbe Disease. Passage Bio collaborates closely with the University of Pennsylvania, leveraging its Gene Therapy Program for preclinical work while managing clinical development, regulatory affairs, manufacturing, and commercialization of its product candidates. The company also engages with the Orphan Disease Center for natural history studies, key opinion leader involvement, and patient advocacy.
Adicet Bio is a biotechnology company focused on the discovery and development of allogeneic gamma delta T cell therapies for cancer and other diseases. The company is advancing its lead product candidate, ADI-001, which is a first-in-class allogeneic gamma delta T cell therapy designed to target CD20. This therapy aims to address relapsed or refractory aggressive B cell non-Hodgkin's lymphoma and potentially autoimmune diseases. Additionally, Adicet Bio is developing another preclinical candidate, ADI-270, which is an armored gamma delta CAR T cell therapy targeting renal cell carcinoma, with prospects for treating other solid tumors and hematological malignancies that express CD70.
MDClone Ltd. is a company that specializes in software designed to analyze medical records while ensuring patient privacy. Founded in 2016 and based in Beersheba, Israel, MDClone's platform generates synthetic medical records for fictitious patients. This innovative approach allows researchers to study disease behavior, medical practices, and various healthcare dynamics without exposing actual patient data. The platform creates fictitious individuals with unique characteristics, preserving essential relationships between them, thus maintaining the integrity of medical information while safeguarding privacy. By enabling health organizations to harness the value of their data assets, MDClone significantly enhances the ways medical information is gathered, analyzed, and utilized in research and service delivery.
Prelude Therapeutics is a clinical-stage biopharmaceutical company based in Wilmington, Delaware, founded in 2016. The company focuses on discovering and developing small molecule therapies designed to target critical mechanisms driving cancer growth and resistance. Its pipeline includes several candidates currently in Phase 1 clinical trials, such as PRT543, aimed at treating solid tumors and myeloid malignancies, and PRT811, which targets solid tumors like glioblastoma multiforme. Other development programs include PRT1419, a selective inhibitor of an anti-apoptotic protein, PRT2527, an inhibitor of CDK9, PRT-SCA2 for genomically selected cancers, and PRT-K4 for solid tumors. Prelude Therapeutics aims to provide innovative treatment options for patients facing refractory cancers.
Laekna Therapeutics Shanghai Co., Ltd. is a gene therapy company, which develops therapeutics for oncology and metabolic diseases. It develops pre-clinical discovery programs for oncology and liver diseases. It develops small molecule candidate LAE001 (previously known as CFG920) for prostate cancer. The company was founded in 2016 and is based in Shanghai, China.
Fusion Pharmaceuticals Inc., a clinical-stage oncology company, focuses on developing radiopharmaceuticals as precision medicines. The company has developed Targeted Alpha Therapies platform together with its proprietary Fast-Clear linker technology to enable us to connect alpha particle emitting isotopes to antibodies and other targeting molecules in order to selectively deliver the alpha particle payloads to tumors. Its lead product candidate is FPI-1434 that is in Phase 1 clinical trials as a monotherapy in patients with solid tumors expressing insulin-like growth factor 1 receptor. The company is also conducting additional preclinical studies of FPI-1434 in combination with approved checkpoint inhibitors and DNA damage response inhibitors to further assess the anti-tumor activity, and dosing schedule and pharmacodynamics of the combinations. In addition, it is progressing its earlier-stage product candidate, FPI-1966 into clinical development for the treatment of head and neck, and bladder cancers expressing fibroblast growth factor receptor. The company was founded in 2014 and is headquartered in Hamilton, Canada.
SpringWorks Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing therapies for underserved patient populations affected by rare diseases and cancer. Founded in 2017 and headquartered in Stamford, Connecticut, the company’s lead product candidate, nirogacestat, is an oral small molecule gamma secretase inhibitor currently in Phase III trials for treating desmoid tumors. Other significant candidates include mirdametinib, an oral MEK inhibitor in Phase IIb trials for neurofibromatosis type 1-associated plexiform neurofibromas, and various combinations involving nirogacestat for relapsed or refractory multiple myeloma. The company is also exploring additional therapies, such as BGB-3245, an investigational oral inhibitor targeting BRAF mutations, currently in Phase I trials. SpringWorks has established collaborations with several biotechnology and pharmaceutical firms to enhance its research and development efforts.
Prevail Therapeutics Inc. is a biotechnology company based in New York that specializes in developing gene therapies for neurodegenerative diseases. Founded in 2017 with support from The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, the company is focused on creating innovative adeno-associated virus (AAV)-based treatments. Its lead candidate, PR001, is currently undergoing Phase I/II clinical trials aimed at treating Parkinson's disease linked to GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is developing PR006 for frontotemporal dementia associated with GRN mutations and PR004 for various synucleinopathies. The company is dedicated to advancing therapies that can modify the course of these debilitating conditions.
Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing innovative therapeutics for patients with rare genetic disorders of hemoglobin, particularly sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing IMR-687, a once-daily oral therapy designed to be a potent small molecule inhibitor of PDE9. This therapeutic aims to address the significant health challenges associated with these conditions, which can lead to reduced healthy red blood cells and various complications. Imara was established following a collaboration between Cydan Development and H. Lundbeck A/S, with initial funding from notable life science investors.
BiomX Inc. is a clinical-stage company based in Ness Ziona, Israel, focused on developing bacteriophage-based therapies that target harmful bacteria linked to various diseases. The company aims to treat conditions associated with microbiome imbalances, including skin conditions, inflammatory bowel diseases, liver disorders, and colorectal cancer. BiomX's lead product candidates include BX001, which addresses skin appearance issues, BX002 for inflammatory bowel disease, BX003 for bacteria related to progressive liver disease, and BX004 targeting chronic pulmonary infections caused by Pseudomonas aeruginosa. The company collaborates with notable institutions such as The Weizmann Institute of Science, Takeda, Keio University, JSR Corporation, and Janssen Research & Development to advance its microbiome-based therapeutic products. Founded in 2015, BiomX leverages innovative research from its scientific collaborators to develop novel therapeutics aimed at improving health outcomes.
Apollomics is a developer of oncology therapeutics intend to harness the immune system and targeting specific molecular pathways.The company's therapeutics target the growth and proliferation of cancer cells and offer an oral c-Met inhibitor that restores the body's immune system to recognize and kill cancer cells, enabling clients to treat their patients in an enhanced way.
NextCure, Inc. is a clinical-stage biopharmaceutical company based in Beltsville, Maryland, dedicated to discovering and developing innovative immunomedicines for cancer and other immune-related diseases. The company’s lead product candidate, NC318, is currently undergoing Phase 2 clinical trials aimed at treating advanced or metastatic solid tumors. Additionally, NextCure is advancing NC410, an immunomedicine designed to counteract immune suppression through targeting an immune modulator known as Leukocyte-Associated Immunoglobulin-like Receptor 1. The company is also engaged in research programs evaluating novel immunomodulatory molecules, including antibodies in preclinical development that target specific immune modulators in inflamed tissues and tumor microenvironments. NextCure has established a license agreement with Yale University and collaborates with Eli Lilly and Company for research and development efforts. Founded in 2015, the company leverages proprietary platforms to identify biologically relevant targets and develop first-in-class immunotherapy products.
Terns Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on discovering and developing oral, small-molecule drugs for the treatment of cancer and liver diseases, particularly non-alcoholic steatohepatitis (NASH). Founded in 2017 and headquartered in Foster City, California, with additional offices in Shanghai, the company leverages its expertise in disease biology and medicinal chemistry to advance a pipeline of innovative therapeutic candidates. Key products include TERN-101, a non-bile acid farnesoid X receptor agonist, and TERN-201, an inhibitor of semicarbazide-sensitive amine oxidase. Terns Pharmaceuticals aims to address significant unmet medical needs both in China and globally through its efficient drug discovery model and extensive clinical development capabilities.
Arcutis Biotherapeutics is a clinical-stage biopharmaceutical company dedicated to developing and commercializing treatments for immune-mediated dermatological diseases. The company's lead product candidate, ARQ-151, is a topical cream formulation of roflumilast currently undergoing Phase III clinical trials for the treatment of plaque psoriasis and atopic dermatitis. In addition, Arcutis is advancing ARQ-154, a topical foam formulation of ARQ-151 aimed at treating seborrheic dermatitis and scalp psoriasis, as well as ARQ-252, a selective topical small molecule inhibitor of janus kinase type 1 for hand eczema and other inflammatory skin conditions. Another candidate, ARQ-255, is being developed to penetrate deeper into the skin to address inflammation in alopecia areata. Founded in 2016 and located in Westlake Village, California, Arcutis Biotherapeutics was formerly known as Arcutis, Inc. before its name change in October 2019.
Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases that currently have no available treatments. The company’s lead pipeline candidate, TA-46, is a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism. Therachon is headquartered in Basel, Switzerland, with research labs in Nice, France.
ReViral Ltd is a biotechnology company based in Stevenage, United Kingdom, that specializes in the discovery and development of antiviral drugs targeting the respiratory syncytial virus (RSV). Founded in 2011, ReViral focuses on creating novel first-in-class compounds, including a highly potent fusion inhibitor designed to treat severe RSV infections, particularly in vulnerable populations such as neonates. The company's innovative approach aims to expand the antiviral market by providing effective treatment options for patients affected by RSV, a virus known for causing significant respiratory illness. Through its research and development efforts, ReViral seeks to address unmet medical needs in the realm of viral infections.
ENYO Pharma SA is a clinical-stage biopharmaceutical company based in Lyon, France, focused on drug discovery and development for acute and chronic viral infections. Founded in 2014 by a group of scientists from the Infectiology Research Center, the company has established a unique platform to identify intracellular therapeutic targets and molecules, particularly for human pathogens. ENYO Pharma is developing treatments for significant viral infections, with ongoing programs targeting chronic hepatitis B and severe influenza. Its lead compound, EYP001, is designed to modulate FXR and reduce viral replication, while EYP002 is currently undergoing preclinical studies. The company aims to advance its pipeline into Phase II clinical trials and collaborates closely with research institutions to leverage innovative therapeutic strategies. ENYO Pharma is committed to addressing unmet medical needs in infectious and metabolic diseases, positioning itself as a leader in antiviral therapies.
SmartZyme Biopharma is a life science company based in Ness Ziona, Israel, that specializes in the development of innovative protein design and engineering solutions. Founded in 2013, the company focuses on enhancing glucose monitoring technologies for individuals with diabetes. It offers a next-generation enzyme specifically designed for improved bio-electrochemical glucose catalysis, which is intended for use in glucose test strips that facilitate self-monitoring of blood glucose levels. Additionally, SmartZyme has developed Go Patch Go, a personalized patient care system aimed at assisting diabetic patients in maintaining balanced glucose levels. The company has also formed a strategic agreement with Hemoshear Therapeutics to further its research and development initiatives. By advancing glucose-sensing capabilities, SmartZyme aims to improve the safety and effectiveness of continuous glucose monitoring devices, eliminating the need for signal mediators and reducing the occurrence of hypoglycemic errors.
Crinetics Pharmaceuticals discovers and develops novel therapeutics targeting peptide hormone receptors for the treatment of endocrine-related diseases and cancers. Their programs are derived from internal discovery efforts and use objective hormonal biomarker endpoints for preclinical and clinical studies to reduce both the risk and cost to achieve meaningful clinical data. Crinetics is seeking motivated partners to help advance Their programs to become commercially successful products.
Pionyr Immunotherapeutics Inc. is a biotechnology company based in San Francisco, California, focused on developing innovative cancer immunotherapies. Established in 2015, the company aims to enhance the body’s antitumor immunity by targeting the tumor microenvironment. Pionyr employs advanced technologies for novel target discovery and antibody generation, which underpin its next-generation immuno-oncology therapeutics. Central to its strategy is the Myeloid Tuning™ approach, designed to specifically modify the cellular composition within the tumor microenvironment, thereby improving the immune system's ability to combat tumors.
ChemomAb Ltd. is a clinical-stage biopharmaceutical company based in Tel Aviv, Israel, founded in 2011. The company focuses on developing proprietary monoclonal antibodies aimed at treating autoimmune, inflammatory, and fibrotic diseases, including rare conditions. Its leading therapeutic candidate, CM-101, has undergone extensive pre-clinical testing in animal models that simulate human disorders, demonstrating promising safety and efficacy along with a novel mechanism of action. Through its innovative approach, ChemomAb aims to address unmet medical needs in immune-mediated and fibrotic disorders.
resTORbio is a clinical-stage biopharmaceutical company focused on the development and commercialization of novel therapeutics for the treatment of aging-related diseases.The Company develops medicines and therapies to treat aging-related diseases and conditions such as cancer, heart disease, neurodegenerative diseases, and diabetes mellitus. resTORbio serves clients in the State of Massachusetts.
RDD Pharma, Ltd. is a pharmaceutical company that focuses on developing and commercializing therapeutics for anorectal diseases and gastrointestinal disorders. Established in 2008 and based in Tel-Aviv, Israel, with an office in New York, the company specializes in creating targeted pharmacological treatments for conditions such as chronic anal fissures, fecal incontinence, and pruritus ani. RDD Pharma employs specialized formulation and delivery techniques to address the unique anatomical and physiological challenges associated with these conditions. By developing effective drugs, the company aims to provide healthcare professionals with options that enable patients to achieve painless and rapid relief from their ailments. As of April 2020, RDD Pharma operates as a subsidiary of Innovate Biopharmaceuticals, Inc.
Azura Ophthalmics is a clinical-stage company focused on developing innovative treatments for Meibomian Gland Dysfunction (MGD), a leading cause of dry eye disease (DED). By addressing the underlying causes of MGD, Azura aims to improve the health and quality of life for millions affected by this and other ocular surface diseases, where effective treatment options are often limited. The company is advancing a unique portfolio of compounds in conjunction with a novel drug delivery platform, enabling healthcare professionals to better manage these conditions. Based in Tel Aviv-Yafo, Israel, Azura also has operational presence in Australia and the United States, supported by a management team with a strong track record in the development and commercialization of novel ocular therapies.
MobileODT Ltd specializes in developing optical diagnostic devices and software focused on early cancer detection, particularly cervical cancer. The company's flagship product, the EVA COLPO, is a portable, internet-connected colposcope that is FDA-cleared and designed for ease of use. MobileODT also offers EVA SANE, a secure platform for managing patient information, and EVA WELL, a digital visualization tool for gynecologic exams. Their offerings include a mobile application for remote image capture and patient tracking, along with a cloud-based portal for collaboration and reporting. In addition, the company provides tools for documenting violence and abuse and plans to expand its product line to cover other medical areas such as ENT and dermatology. Established in 2012 and headquartered in Tel Aviv, Israel, with an office in New York, MobileODT has gained recognition in various markets, particularly the United States, where it is becoming a standard for visual diagnostics in healthcare settings.
LogicBio develops gene therapy vectors for previously incurable genetic and infectious diseases, including: hemophilia, HIV/AIDS and methylmalonic acidemia (MMA). Our proprietary technology based on the non-pathogenic Adeno-Associated Viral (AAV) vector offers superior efficiency and safety compared to all competitors on the market, and is uniquely suited for the treatment of children as well as adults.
InventisBio Inc. is a biotechnology company focused on discovering and developing innovative therapeutics for cancer and metabolic diseases. Founded in 2013 and based in Pudong, China, the company is advancing a pipeline of small molecule drug candidates targeting lung cancer, breast cancer, and gout. Notably, it is developing D-0316, a third-generation EGFR-T790M tyrosine kinase inhibitor, and is exploring combinations of its drugs with other immuno-oncology therapies, such as PD-1 antibodies, for various cancer indications. Co-founded by Dr. Yaolin Wang and a team of scientists with prior experience at major pharmaceutical companies like Merck and Schering-Plough, InventisBio aims to leverage their expertise in oncology and metabolic diseases to bring effective treatments to market.
PMV Pharma is developing first-in-class p53 and p53 pathway modulators for the treatment of cancer. Bringing together leaders in the field to utilize over three decades of p53 biology, PMV Pharma combines unique biological understanding with pharmaceutical development focus.
NeRRe Therapeutics Ltd. is a biotechnology company based in Stevenage, United Kingdom, established in 2012. The company specializes in the development of neurokinin (NK) receptor antagonists, focusing on treatments for conditions related to intense pruritus, particularly in patients undergoing targeted anti-cancer therapies. Its lead asset, orvepitant, is an oral NK-1 antagonist designed to alleviate this specific symptom. Additionally, NeRRe is advancing other candidates, including NT-814, a dual NK-1 and NK-3 antagonist, and NT-949, which is ready for Phase I trials. The company also has NT-432, a clinical candidate targeting NK-1 receptors. NeRRe Therapeutics aims to address unmet medical needs through its innovative therapeutic approaches.
scPharmaceticals is creating innovative and important new therapeutic options by administering drugs subcutaneously using a convenient two component delivery system suitable for patient self-administration.In February 2013 scPharmaceuticals was formed to continue the groundbreaking work of SpringLeaf Therapeutics, a Boston area company that started in 2007. scPharmaceuticals targets the untapped opportunity of treating patients with serious medical conditions with a convenient safe and comfortable subcutaneous administration of a drug. This is therapeutically equivalent to intravenous administration while avoiding the cost, burden and risks associated with intravenous administration.
Turnstone Biologics Inc. is a biotechnology company dedicated to developing viral immunotherapies aimed at enhancing cancer survival rates. Founded in 2015 and headquartered in Ottawa, Canada, with an additional office in New York, the company’s lead candidate, RIVAL-01, is based on a vaccinia virus backbone that incorporates three effective immunomodulators: Flt3 ligand, anti-CTLA-4 antibody, and IL-12 cytokine. These components are designed to synergistically stimulate immune responses and modify the tumor microenvironment to facilitate tumor eradication. Turnstone's platform leverages discoveries from prominent researchers in the field of oncolytic viral immunotherapy and has progressed RIVAL-01 into a Phase I/II clinical development study in collaboration with four Canadian academic institutions and the Fight Against Cancer Innovation Trust (FACIT).
Cleave Therapeutics, Inc. is a biopharmaceutical company based in San Francisco, California, founded in 2010. The company specializes in developing novel small-molecule drugs that target valosin-containing protein and other protein-degradation pathways critical for the survival of cancer cells. By focusing on these mechanisms, Cleave Therapeutics aims to create enzyme inhibitors that can effectively treat cancer and neurodegenerative diseases. Its research efforts are directed toward discovering innovative therapies that may improve patient outcomes in oncology.
Graybug Vision, Inc. is a clinical-stage biopharmaceutical company dedicated to developing innovative treatments for diseases affecting the retina and optic nerve. Founded in 2011 and based in Redwood City, California, the company is advancing several product candidates, including GB-102, an intravitreal injection formulation currently undergoing Phase I/IIa and IIb clinical trials for wet age-related macular degeneration and Phase IIa trials for diabetic macular edema. Additionally, Graybug is developing GB-103, an annual formulation of GB-102 for diabetic retinopathy, and GB-401, a depot formulation aimed at treating primary open-angle glaucoma. The company's proprietary micro- and nanoparticle controlled release technologies are designed to deliver medications at a sustained rate, enhancing patient compliance and improving clinical outcomes.
Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is an oral therapy currently in Phase I/IIa clinical trials for the treatment of phenylketonuria. Additionally, Synlogic is developing SYNB1891, an intratumoral synthetic biotic medicine that is undergoing Phase I clinical trials for the treatment of solid tumors and lymphoma. The company is also exploring synthetic biotics for more common conditions, including inflammatory bowel disease and secondary hyperoxaluria. Synlogic has established a collaboration with Ginkgo Bioworks to advance its development of these innovative therapies.
Zai Lab Limited is a Shanghai-based biopharmaceutical company founded in 2013, dedicated to discovering, developing, and commercializing innovative therapeutics for oncology, autoimmune, and infectious diseases. The company offers several proprietary products, including Niraparib for treating solid tumors, Optune for glioblastoma multiforme, and Ripretinib for KIT and PDGFRa-driven cancers. Additionally, Zai Lab develops other investigational therapies such as REGN1979, Margetuximab, INCMGA0012, Bemarituzumab, Omadacycline, and Durlobactam. With a focus on the Chinese market, Zai Lab has established partnerships with leading global biopharmaceutical firms to enhance its drug pipeline and improve access to transformative medicines. The company leverages its expertise in drug development and favorable regulatory conditions in China, along with in-house manufacturing capabilities, to support both clinical and commercial production. Zai Lab aims to expand its operations and build a specialized commercial team to effectively market its products in China and beyond.
ORIC Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing therapies to overcome resistance in cancer treatment. Founded in 2014 and based in South San Francisco, California, the company is advancing several product candidates, including ORIC-101, a small molecule antagonist of the glucocorticoid receptor associated with resistance to various cancer therapies in solid tumors. Another key candidate, ORIC-533, is an orally bioavailable small molecule inhibitor of CD73, which plays a significant role in resistance to chemotherapy and immunotherapy regimens. In addition to these, ORIC is working on multiple precision medicines aimed at addressing other cancer resistance mechanisms. The company's founders, Charles Sawyers and Scott Lowe, are recognized for their contributions to identifying novel cancer targets that have led to innovative treatment options.
ObsEva SA is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, dedicated to developing innovative therapeutics for women's reproductive health and pregnancy. Founded in 2012 by Ernest Loumaye and André Chollet, the company focuses on addressing serious conditions such as endometriosis, heavy menstrual bleeding, and preterm labor. Its key products include Linzagolix, an oral gonadotropin-releasing hormone receptor antagonist aimed at treating pain from endometriosis and uterine fibroids, and OBE022, a selective prostaglandin F2α receptor antagonist designed for once-daily treatment of preterm labor. Additionally, ObsEva is advancing Nolasiban, an oral oxytocin receptor antagonist intended to enhance clinical pregnancy and live birth rates in women undergoing in vitro fertilization. The company operates primarily in Switzerland and is committed to improving outcomes for women facing reproductive health challenges.
Arvinas is a pharmaceutical company that focuses on developing novel therapeutics to treat a variety of diseases including cancer, pro-inflammatory, autoimmune and rare diseases. Its proprietary approach is the targeting of disease-causing proteins for degradation thus eliminating them from the body.
The company was founded in 2013 and is headquartered in Connecticut, United States.
Corvus Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Burlingame, California, that specializes in developing innovative immuno-oncology therapies. The company's lead product candidate, Ciforadenant (CPI-444), is an oral small molecule antagonist of the A2A receptor and is currently undergoing Phase Ib/2 clinical trials targeting adenosine, an important immune checkpoint. Additionally, Corvus is advancing CPI-006, an anti-CD73 monoclonal antibody, which is in Phase I/Ib trials to inhibit adenosine production and stimulate immune responses, and is also being evaluated for its potential use in COVID-19. Other candidates in development include an antagonist of the A2B receptor and CPI-818, a small molecule inhibitor targeting interleukin-2-inducible T-cell kinase, which is also in Phase I/Ib trials. The company has established a strategic collaboration with Angel Pharmaceuticals to further develop its pipeline of targeted investigational medicines.
Intellia Therapeutics is a genome editing company based in Cambridge, Massachusetts, focused on developing curative gene-editing treatments using the CRISPR/Cas9 technology. The company aims to address severe and life-threatening genetic diseases through both in vivo and ex vivo therapeutic approaches. Its pipeline includes programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Additionally, Intellia is developing engineered cell therapies for oncological and autoimmune diseases. The company has established collaborations with various entities, including leading pharmaceutical firms and research institutions, to enhance its research and development efforts. Founded in 2014, Intellia Therapeutics holds a comprehensive intellectual property platform for the therapeutic application of CRISPR technology, positioning it at the forefront of gene editing innovation.
Principia Biopharma Inc. is a late-stage biopharmaceutical company based in South San Francisco, California, focused on developing innovative therapies for immune-mediated diseases. The company's lead candidate, rilzabrutinib, is an inhibitor currently undergoing Phase III clinical trials for pemphigus and pemphigus foliaceus, as well as Phase 1/2 and Phase 2 trials for immune thrombocytopenia and IgG4-related disease, respectively. Additionally, Principia is advancing PRN2246/SAR442168, aimed at treating multiple sclerosis and other central nervous system disorders, which is in Phase II trials. Another candidate, PRN473, is in Phase I trials for various immune-mediated diseases. Principia Biopharma collaborates with Genzyme Corporation to explore treatments for relapsing and progressive multiple sclerosis, enhancing its commitment to addressing significant unmet medical needs in immunology and oncology. Established in 2008, the company operates as a subsidiary of Sanofi.
TytoCare Ltd. is a healthcare technology company that develops handheld devices and applications for on-demand medical examinations and remote diagnoses. Founded in 2012 and based in Netanya, Israel, with an office in New York, the company offers three main telehealth products: TytoHome for consumers, TytoPro for professionals, and TytoClinic for remote care settings. These tools enable users to conduct self-examinations of various health conditions, such as ear infections, respiratory issues, and abdominal pain, by assessing the heart, lungs, skin, throat, ears, and body temperature. The platform facilitates virtual consultations by connecting patients with healthcare professionals, allowing for diagnosis and prescriptions as needed. TytoCare's technology incorporates a cloud-based data repository, analytics, and machine learning algorithms to enhance user experience and diagnostic accuracy. The company has established a strategic partnership with Novant Health to further its mission of making healthcare accessible and convenient.
Dimension Therapeutics, Inc. is a gene therapy company dedicated to developing innovative treatments for rare and metabolic diseases linked to liver function. Founded in 2013 and based in Cambridge, Massachusetts, the company has established a robust pipeline of gene therapy candidates. Its lead products include DTX101 for hemophilia B and DTX201 for hemophilia A, alongside other programs targeting conditions such as ornithine transcarbamylase deficiency, glycogen storage disease type Ia, phenylketonuria, Wilson disease, and citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, leveraging its extensive patent portfolio related to NAV vector technology. This partnership enhances Dimension's ability to advance its gene therapy initiatives, particularly in the realm of adeno-associated virus therapeutics. In 2017, Dimension Therapeutics became a subsidiary of Ultragenyx Pharmaceutical Inc.
Nabriva Therapeutics is a biopharmaceutical company focused on developing and commercializing innovative anti-infective agents to address serious bacterial infections. The company's lead product, lefamulin, is a semi-synthetic pleuromutilin antibiotic designed to treat community-acquired bacterial pneumonia and acute bacterial skin infections. It is currently undergoing clinical trials for additional applications, including pediatric infections, sexually transmitted infections, osteomyelitis, and prosthetic joint infections. Nabriva is also advancing CONTEPO, an epoxide antibiotic aimed at treating complicated urinary tract infections, and it is in clinical trials for peri-operative prophylaxis. Founded in 2005 and headquartered in Dublin, Ireland, Nabriva Therapeutics has evolved from its earlier identity as a research institute focused on antibiotic development.
True North Therapeutics, Inc. is a biotechnology company focused on developing therapies that target the complement pathway of the immune system, specifically for rare diseases with high unmet clinical needs. Founded in 2013 and based in South San Francisco, California, the company’s lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway. This approach aims to prevent inflammatory mediators and phagocytosis in conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, and other complement-mediated disorders. True North is also exploring additional treatment options for a variety of diseases in hematology, dermatology, and neurology. The company operates as a subsidiary of Bioverativ Inc.
Aeglea BioTherapeutics is a clinical-stage biotechnology company focused on developing human enzyme therapeutics for rare genetic and cancer diseases. Its lead product candidate, pegzilarginase, is a recombinant human Arginase 1 enzyme currently undergoing Phase III trials to assess its safety and efficacy in treating Arginase 1 deficiency. The company's preclinical pipeline includes several innovative candidates: ACN00177 for homocystinuria, AEB5100 which degrades plasma cystine and cysteine, AEB2109 targeting the degradation of methionine, and AEB3103. Aeglea BioTherapeutics aims to exploit the amino acid dependencies of tumors by creating novel, human-derived enzymes that selectively target these vulnerabilities, differentiating the metabolic needs of tumor cells from those of normal tissues. Founded in 2013 and headquartered in Austin, Texas, the company was formerly known as Aeglea BioTherapeutics Holdings, LLC before changing its name in 2015.
Tricida, Inc. is a pharmaceutical company based in South San Francisco, California, established in 2013. The company is dedicated to the development and commercialization of its lead product candidate, TRC101, a non-absorbed, orally-administered polymer designed to treat metabolic acidosis in patients with chronic kidney disease (CKD). Metabolic acidosis, a condition often associated with CKD, can hasten kidney deterioration and lead to serious complications such as muscle wasting and loss of bone density. Tricida has successfully completed a Phase 3, double-blind, placebo-controlled trial of TRC101, which aims to bind and remove acid from the gastrointestinal tract, thereby addressing the underlying metabolic issues and potentially slowing the progression of CKD.
Audentes Therapeutics is a clinical-stage biotechnology company dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. The company is advancing several therapies, including AT132, currently in Phase I/II clinical trials for X-linked myotubular myopathy, and AT342, also in Phase I/II studies for Crigler-Najjar syndrome. Additionally, Audentes is exploring AT845 in preclinical studies for Pompe disease and AT307 for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. The company is also working on vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Collaborations include a partnership with Genethon for XLMTM treatments and an agreement with the University of Pennsylvania for Crigler-Najjar syndrome products. Founded in 2012 and headquartered in San Francisco, California, Audentes Therapeutics operates as a subsidiary of Astellas US Holding, Inc.
GC-Rise Pharmaceutical Co., Ltd. is a solely-foreign-owned enterprise invested by INVENTAGES, a famous European investment group engaged in healthcare industry. Taking “Devoted to human healthcare and a better life” as our mission, the company makes full use of the business networks of our strategic cooperation partner in America, Europe and Japan, their affluent experiences in clinical development, clinical trials and registration in China, and their abundant strength in commercial distribution, hospital sale, marketing, so as to provide novel, safe, and highly efficient medical products and treatment ideas for Chinese patients.
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