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Rare Diseases Suppliers Near Europe
88 companies found
based inCharlottesville, 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 ...
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 ...
based inCambridge, UNITED KINGDOM
We’re driven by the shared purpose of breaking new ground in the discovery of new treatments for rare diseases. Healx is a mission-driven technology company pioneering the next wave of drug discovery in order to bring novel, effective treatments to ...
Our Rare Treatment Accelerator helps academic, patient, and industry groups advance their rare disease drug redevelopment projects. By combining our team, expertise and financial resources with your ...
based inBoston, MASSACHUSETTS (USA)
SOPHiA GENETICS is the creator of a global data-sharing network – we work with customers from over 790 institutions in over 70 countries. Our network advances data-driven medicine to improve health outcomes and economics worldwide. We offer a ...
Most rare and inherited diseases have a neurological component, likely because more than 80% of human genes are expressed in the brain.1 Neurological disorders are multifactorial and heterogeneous, meaning that ...
based inShanghai, CHINA
RareStone Group is dedicated to building the first rare disease ecosystem in China to improve the lives of patients and families impacted by rare diseases, by bringing the best-in-the-world therapeutic solutions and comprehensive and sustainable ...
Chronic kidney disease (CKD) is a non-communicable disease usually caused by diabetes and hypertension. The prevalence of chronic kidney disease in China is reported to be 10.8%, and is predicted to ...
based inBangalore, INDIA
Strand Life Sciences is a genomics-based research and diagnostics company with a mission to improve global health by decoding the genetic information contained in an individual’s genome. Strand Life Sciences partnership programs enable large-scale ...
Strand has extensive experience in validating NGS-based panels under CAP- and CLIA-settings: be it germline panels, somatic tumor panels, or liquid biopsy panels, and be it small panels with tens of genes, to large panels with thousands of ...
based inMadison, WISCONSIN (USA)
InvivoSciences, Inc. (IVS) is a precision medicine company personalizing the drug development process by introducing patient-derived and gene-edited human samples, including 3D engineered microtissues. Our unique integration of cell & tissue ...
InvivoSciences (IVS) combines its human micro tissue technology and phenotypic assay platform with the systems biology approach to achieve rare disease modeling for its drug discovery. Our strip tissues are perfect ...
based inNew 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 ...
based inWayne, PENNSYLVANIA (USA)
Palvella Therapeutics was founded to serve individuals suffering from rare diseases by developing safe and efficacious therapies that will dramatically enhance their quality of life. We focus on developing therapies that specifically target the root ...
QTORIN™ rapamycin 3.9%is a novel targeted topical therapy being studied for Gorlin syndrome. Palvella is enrolling a phase 2b clinical study (CODY) for people with Gorlin ...
based inBoston, MASSACHUSETTS (USA)
People living with rare and devastating diseases are our Guiding Star. We believe it is our responsibility to listen to, understand, and change the lives of patients and those who work tirelessly to help them. Our mission is to transform the lives ...
People with rare diseases often wait years to receive a proper diagnosis and many never receive one at all. RARE ANSWERS™ is a system of innovative and sustainable tools designed to help ...
based inNEW YORK (USA)
Our company is a globally operating service provider and one of the world leaders in the development and delivery of advanced drug therapies for rare diseases. We not only provide global customers with a wide range of services for rare disease ...
based inParis, 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 ...
based inPhiladelphia, PENNSYLVANIA (USA)
We are a biotechnology company at the forefront of advanced therapies to treat a range of devastating rare diseases. Amicus Therapeutics (Nasdaq:FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and ...
based inNaarden, NETHERLANDS
Azafaros has leveraged on decades of discovery by professors Hans Aerts, Hermen Overkleeft, and Stan van Boeckel at Leiden University and the Academic Medical Center in the Netherlands. Expanding on their research and led by highly experienced ...
Azafaros B.V. is pioneering the development of innovative oral small molecule compounds addressing the pathological effects of metabolite accumulation in rare metabolic disorders. Their lead compound, nizubaglustat, leverages unique ...
based inRostock, GERMANY
Since 2006, CENTOGENE has helped physicians diagnose thousands of rare disease patients around the world. A quick and accurate diagnosis can end an odyssey of medical tests and visits to multiple medical specialists. It also helps physicians better ...
Analyze, interpret, and report genomic variants with our decentralized diagnostic solution – no matter where you are located. CentoCloud is a cloud-based Software as a Service (SaaS) platform specifically designed for the rapid bioinformatic ...
based inChatham, NEW JERSEY (USA)
Tonix is a clinical-stage biopharmaceutical company focused on developing novel therapies and vaccines to prevent and treat central nervous system disorders, immunology conditions, infectious diseases, and rare diseases. At Tonix, we are focused on ...
TNX-2900, Tonix’s proprietary potentiated intranasal oxytocin is in the pre-Investigational New Drug (IND) stage as a candidate for the treatment of Prader-Willi syndrome (PWS) and non-organic failure to thrive disease. TNX-2900 has been ...
based inBrisbane, CALIFORNIA (USA)
Sangamo is a genomic medicine company that seeks to help millions of people who suffer from severe diseases for which today’s medicine can only offer symptom management at best. Our mission is to translate ground-breaking science into medicines that ...
Behind these are research efforts thoughtfully focused on leveraging our differentiated zinc finger technology. We are advancing a robust pipeline of wholly owned and partnered programs in rare disease, neurology, ...
based inStockholm, SWEDEN
IBT is a pharmaceutical company with its registered office in Stockholm with a vision to develop drugs influencing the human infant microbiome, and thereby prevent or treat rare diseases affecting premature infants. IBT is currently developing its ...
based inheongju-si, SOUTH KOREA
Curome Biosciences is an innovative R&D company developing therapies to treat mitochondrial dysfunction-related diseases with high unmet medical needs. Curome Biosciences wants to provide solutions for treatment of diseases with high unmet medical ...
Mitochondria are an essential cellular organelle that is responsible for the generation of energy (known as the ‘powerhouse of the cell’). Mitochondrial dysfunction is related with numerous diseases. Curome Biosciences ...
based inGermantown, MARYLAND (USA)
Cell and gene therapies (CGTs) use modified cells or cellular matter to treat, prevent or cure serious health issues like cancer, genetic disorders, immunodeficiencies, rare diseases, and more. CGTs have shown potential to replace damaged cells with ...
based inKing of Prussia, PENNSYLVANIA (USA)
Behring's parent company, CSL, was formed more than 100 years ago to save lives using the latest technologies. In the century since, CSL Behring has grown into a global biotechnology leader, driven by that same promise to save and improve lives. We ...
