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Rare Disease Suppliers & Manufacturers
94 companies found
based inPearl 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 ...
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 inVancouver, BRITISH COLUMBIA (CANADA)
Precision NanoSystems is a global leader in technologies, solutions and services for the development of non-viral delivery of genomic medicines, including mRNA vaccines and therapeutics. We work with the world’s leading drug developers to understand ...
based inAshiya, JAPAN
Since its establishment in 1975, JCR has been working on proprietary technology developments and creation of products in a manner to be 'one step ahead' of competitors at all times under its corporate philosophy, 'Contributing towards people's ...
based inLaupheim, GERMANY
We are a leading global Contract Development and Manufacturing Organization (CDMO), focused exclusively on client projects. From our headquarters in Laupheim, Germany, and our site in Milford, MA, USA, we offer end-to-end solutions including ...
based inBudapest, HUNGARY
Cytocast, an innovative TechBio startup located in Budapest, is at the forefront of data-driven medical technology.The company's primary focus is on integrating extensive multi-omics data with high-performance computing and advanced data science ...
Cytocast is at the forefront of medical technology with its innovative Cytocast Digital Twin Platform™, a pioneering solution in drug development and personalized medicine. At its core is the CYTOCAST DIGITAL TWIN Cell™, a highly ...
based inDijon, FRANCE
Oncodesign is a biopharmaceutical company that offers you powerful technologies and tools to guide you through the development of your drug, from medicinal chemistry to drug candidate. Our therapeutic areas are expanding. In addition to oncology, ...
This is the best tool you can use to evaluate new therapeutic approaches in conditions that are as close as possible to a clinical situation: ...
based inBerwyn, PENNSYLVANIA (USA)
Virpax Pharmaceuticals specializes in developing novel and proprietary drug-delivery systems across various pain and CNS disorder indications to enhance compliance and to optimize the efficacy of each product candidate. Our transforming ...
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 inIrving, TEXAS (USA)
Avero Diagnostics is dedicated to making a positive impact on the lives of patients and the physicians who treat them. We bring a patient-centered approach to every aspect of our organization, including our laboratory and research facility. It’s ...
Expectant parents want to do everything they can before the birth of their baby. The Avero Carrier Test looks at a patient’s genes to see if they may carry a disease that they could pass on to their child. Knowing a patient’s genetic ...
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 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 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 inCounty of New Castle, DELAWARE (USA)
MedGenome Inc. is a global leader in personalized medicine with unique genomic solutions in immuno-oncology, diabetes, ophthalmology, cardiology and other rare diseases. Founded in 2013, MedGenome currently has a Next Generation Sequencing (NGS) ...
based inAustin, 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 ...
based inCarlton, AUSTRALIA
Radiopharm Theranostics has the ambition to become a recognised leader in the development of radiopharmaceutical products for both diagnostic and therapeutic uses in areas of high unmet medical needs. There is unique potential of ...
DUNP19 is the invention of Professor Ulmert at UCLA. ...
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 ...
We're building a rare disease ecosystem to bring both a support network and much-needed treatments to individuals and their families affected by rare disease. We are developing an ...
based inNangang 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 ...
based inLondon, UNITED KINGDOM
The Medical Algorithms Company supports providers and payers with analytics and documentation for evidence-based medical practice. Our platform supplies over 30,000 tested medical calculators and analytic risk scores. Nearly all specialties are ...
based inJenkintown, PENNSYLVANIA (USA)
SFA Therapeutics, Inc. is a development-stage bio-pharmaceutical startup company focused on a new advancement in the treatment of chronic inflammatory disease – the use of microbiome-derived metabolites as drugs. A completely new approach to drug ...
SFA Therapeutics, Inc. is a development-stage bio-pharmaceutical startup company focused on a new advancement in the treatment of chronic inflammatory disease – the use of microbiome-derived metabolites as ...