Rare Diseases Manufacturers, Suppliers & Companies (Clinical Medicine)
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Manufacturerbased in San Rafael, CALIFORNIA (USA)
Established in 1997, BioMarin is a world leader in developing and commercializing first- or best-in-class therapies for rare genetic diseases. We take pride in going where the science leads us, pioneering breakthrough treatments for debilitating and ...
Independent Research Services
In alignment with our commitment to scientific innovation and areas of unmet medical need, we may provide product and funding support for independent research in rare diseases and genetic ...
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Manufacturerbased in San Francisco, CALIFORNIA (USA)
Invitae’s mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Like wearing sunscreen or getting your blood pressure checked at the doctor, genetic testing is another important ...
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Software vendorbased in Manassas, VIRGINIA (USA)
The Jeeva eClinical Cloud was developed by researchers with empathy who listen and learn to help clinical researchers, hospital sites, academic medical centers, CROs, and biopharmaceutical sponsors accelerate patient enrollment by 3x faster. Jeeva ...
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Service providerbased in Paris, FRANCE
Ipsen is a global biopharmaceutical group dedicated to improving lives and health outcomes through innovative medicines in Oncology, Neuroscience and Rare Disease. We also have a well-established and successful Consumer Healthcare business. We are ...
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Manufacturerbased in Charlottesville, VIRGINIA (USA)
HemoShear Therapeutics is a clinical stage company developing novel treatments for patients with rare diseases. We are implementing a new vision for how advanced biological and computational models can accelerate the discovery of innovative ...
HemoShear - Model HST5040 - Investigational Oral Small Molecule Therapy for MMA and PA
HST5040 is an investigational small molecule therapy being developed by HemoShear to reduce the levels of toxins associated with methylmalonic acidemia (MMA) and propionic acidemia (PA), rare genetic disorders caused by the de?ciency of certain ...
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Manufacturerbased in Cambridge, MASSACHUSETTS (USA)
At AVROBIO, we aim to halt or reverse disease with a single dose of gene therapy. ONE COMPANY with a vision to bring personalized gene therapy to the world. Our goal is simple and powerful: We aim to enable individuals with genetic disease to live ...
AVROBIO - Model AVR-RD-06 - Gene Therapy for Gaucher Disease Type 3
AVROBIO has a preclinical research program for an investigational gene therapy for Gaucher disease type ...
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Manufacturerbased in Toronto, ONTARIO (CANADA)
Deep Genomics recruits from among the top 1% of recent graduates and seasoned experts at the intersection of genomics, drug development and AI. Programming RNA Therapies Any Gene, Any Genetic Condition. Revolutions in AI, RNA biology and automation ...
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Manufacturerbased in Ghent/Zwijnaarde, BELGIUM
Confo Therapeutics’ unparalleled technology stabilizes functional conformations of GPCRs (G protein-coupled receptors) to uncover a wide range of previously inaccessible GPCRs as drug targets. This technology platform combined with the pharmacologic ...
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Manufacturerbased in Carlsbad, CALIFORNIA (USA)
AVACEN Medical is dedicated to the innovation and design of safe, easy to use, noninvasive drug-free alternatives for the management of pain associated with numerous chronic and acute conditions including the temporary relief of minor muscle and ...
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Manufacturerbased in Milano, ITALY
We are a clinical-stage biotechnology company pioneering the treatment of cancers with a technology platform that allows the direct delivery of immunotherapeutic payloads within the tumor microenvironment. Intrinsic resistance significantly reduces ...
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Manufacturerbased in Austin, TEXAS (USA)
Lumos Pharma is a clinical-stage biopharmaceutical company whose mission is to develop new therapies for people with rare diseases, prioritizing its focus where the medical need is high, and the pathophysiology is clear. We are committed to this ...
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Manufacturerbased in New York, NEW YORK (USA)
Protara is committed to identifying and advancing transformative therapies for people with cancer and rare diseases. The Protara team prioritizes creativity, diverse perspectives and tenacity to expedite our goal of bringing life-changing therapies ...
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Manufacturerbased in Milpitas, CALIFORNIA (USA)
ASC Therapeutics is a fully incorporated biopharmaceutical company focusing on the development of curative therapeutic products that are enabled by our proprietary gene editing platform, TARGATT and other gene editing technologies developed after 12 ...
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Manufacturerbased in Melville, NEW YORK (USA)
Population Bio, Inc. is a privately held precision medicine company that is empowering pharmaceutical companies to develop targeted therapies and companion diagnostics faster and more cost effectively. We are accelerating the delivery of precision ...
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Manufacturerbased in Holytown, UNITED KINGDOM
Lamellar Biomedical is an innovative biotechnology company developing our proprietary LAMELLASOME™ based therapies to transform the treatment of complex and rare lung disorders, either as biologically active products in their own right or by ...
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Manufacturerbased in Loos, FRANCE
GENFIT is a late-stage biopharmaceutical company dedicated to improving the lives of patients with metabolic and chronic liver diseases. Our translational disease-driven research approach is based on strong expertise, featuring in-house knowledge of ...
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Manufacturerbased in Pearl River, NEW YORK (USA)
Acorda Therapeutics is a publicly traded (NASDAQ: ACOR) biotechnology company whose mission is to develop therapies that restore neurological function and improve the lives of people with neurological disorders. We started our company in 1995 as a ...
Model GGF2 - Cimaglermin Alfa
GGF2 is a member of the neuregulin growth factor family, and has been shown to promote recovery after neurological injury, as well as to enhance heart function in animal models of heart failure. GGF2 showed positive effects on cardiac ...
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Technologybased in Nangang Distric, TAIWAN
We are a clinical-stage specialty pharmaceutical company dedicated to the development and commercialization of best-in-class novel nanomedicines that combine our proprietary lipid-assembled drug delivery platform with approved active pharmaceutical ...
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Nonprofit organization (NPO)based in San Diego, CALIFORNIA (USA)
Rady Children’s Institute for Genomic Medicine is a non-profit research organization embedded within Rady Children’s Hospital-San Diego. We pioneered a medical revolution to end the diagnostic odyssey for neonatal and pediatric rare disease. Now ...
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Software vendorbased in San Mateo, CALIFORNIA (USA)
NuMedii has been pioneering the use of Big Data, Artificial Intelligence (AI) and systems biology since 2010 to accelerate the discovery of precision therapies to address high unmet medical needs. NuMedii has active R&D collaborations with several ...
NuMedii - Artificial Intelligence (AI) Technology
NuMedii has been pioneering the use of Big Data, Artificial Intelligence (AI) and systems biology since 2010 to accelerate the discovery of precision therapies to address high unmet medical needs. ...
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