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Rare Disease Suppliers & Manufacturers
89 companies found
based inManassas, 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 ...
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 inGrove City, OHIO (USA)
Forge is developing innovative gene therapies that aim to help patients suffering from devastating rare diseases. Forge is building capacity to directly address the growing demand for gene therapy manufacturing. Forge is currently building the ...
based inIffeldorf, GERMANY
diaserve laboratories is one of Europe’s leading suppliers of customized IVD calibrators and controls based on human sera. In constant cooperation with renowned institutes, we use the most up-to-date research findings to continuously further develop ...
Human products are frozen liquid materials based on voluntary blood donations. Materials are intended to support the industries to manufacture their own controls and calibrators. All listed products are selected and processed according to customer's ...
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 inConshohocken, PENNSYLVANIA (USA)
Suvoda is a global clinical trial technology company dedicated to supporting complex studies for life-sustaining therapies.We shape digital experiences that empower you to take command over mission-critical and time-sensitive patient interactions in ...
Our advanced patient randomization and trial supply management system. Your clinical trial command and control ...
based inSan 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 ...
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 inMilano, 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 ...
based inBudapest, HUNGARY
CycloLab is a worldwide recognized and all-around cyclodextrin research and development company, having the longest tradition and broadest circle of customers. Since 1972, a staff of internationally acknowledged experts have been working in the ...
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 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 inPlymouth Meeting, PENNSYLVANIA (USA)
Harmony Biosciences is a commercial-stage pharmaceutical company dedicated to developing and delivering innovative therapies for people living with rare neurological diseases, especially sleep/wake, neurobehavioral, and rare epilepsy disorders. The ...
based inCambridge, UNITED KINGDOM
GW is the global leader in developing cannabinoid-based medicines. GW has been researching cannabinoids since 1998 and has established a world leading position in the development of plant-derived cannabinoid therapeutics. GW has established a world ...
based inPrinceton, NEW JERSEY (USA)
The drug discovery and development process is long and expensive with more failures than successes. At Certara, we anticipate and address your critical drug discovery and development risks and decisions using biosimulation, technology and services. ...
based inPlanegg, GERMANY
MorphoSys is a commercial-stage biopharmaceutical company dedicated to the discovery, development, and delivery of innovative cancer medicines. At MorphoSys, our ambition is to redefine how cancer is treated. As a commercial-stage biopharmaceutical ...
Expanded Access to our Investigational Medicinal Products Policy. At MorphoSys, our mission is to make innovative biopharmaceuticals to improve the lives of patients suffering from serious diseases. Before these and all other investigational ...
based inBundoora, AUSTRALIA
AdAlta is an innovative, clinical stage biotech company developing a unique range of new drug treatments. AdAlta has pioneered a technology that mimics the shape and stability of a crucial antigen-binding domain, discovered initially in sharks and ...
AdAlta is utilising the power of the i-body technology platform to develop a growing pipeline of i-bodies to treat a range of conditions, with an initial focus on treating fibrotic diseases. AdAlta identified an i-body that binds to the drug target, ...
based inNew York City, NEW YORK (USA)
Woolsey Pharmaceuticals is focused on the treatment of rare, neurodegenerative diseases. Many neurodegenerative diseases – including Alzheimer’s, vascular dementia, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, Huntington’s disease, ...
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 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 ...
IBP-9414 for the prevention of necrotizing enterocolitis (NEC) contains the active substance Lactobacillus reuteri, which is a co-evolved human bacterial strain naturally present in breast milk. Lactobacillus reuteri is a live bacteria known to be ...