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Rare Disease Suppliers & Manufacturers
94 companies found
based inOxford, UNITED KINGDOM
As a leading global provider of diagnostic genomic solutions, our goal is to enable researchers and clinical decision makers to reach the right decisions for each patient, every time. The quality and innovation built into our products for NGS, ...
based inDublin, IRELAND
Our mission is to help clients accelerate the development of drugs and devices that save lives and improve quality of life. We offer a full range of consulting, clinical development and commercialisation services from a global network of offices in ...
Accelerating study start-up and delivering results with custom strategies and logistics solutions. ICON helps navigate the Cell and Gene Therapy journey by overcoming challenges and mitigating risks. Almost every cell and gene therapy clinical trial ...
based inCharlestown, MASSACHUSETTS (USA)
Seven Bridges is the leading biomedical data company, specializing in software and data analytics to drive public and private healthcare research. We deliver end-to-end bioinformatic solutions including access to datasets, analytic workflows and ...
The representation of the human reference genome as a linear haploid DNA sequence poses limitations when trying to incorporate the known genetic diversity of human populations. This has led to the development of graph-based references, able to ...
based inSan Jose, CALIFORNIA (USA)
Enhancing global compliance, creating a world where quality and compliance professionals, regulators, and government agencies come together to help the world comply with the intent and the spirit of laws, policies and mandates, ensuring continuous ...
This comprehensive training package of ten courses is aimed at helping medical device companies gain a thorough understanding of the top 10 regulations that they should comply ...
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 inEmeryville, CALIFORNIA (USA)
Nutcracker Therapeutics is an early-stage company that is pioneering a revolutionary therapeutic development and manufacturing platform designed to enable the advancement of RNA-based treatments for as many diseases as possible, as fast as possible. ...
RNA is often seen as a molecule with a supporting role in human physiology– a go-between for DNA and proteins. In fact, RNA is a macromolecule with immense versatility, which can be harnessed to tackle the most complex and difficult to treat ...
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 inConshohocken, PENNSYLVANIA (USA)
BTG Specialty Pharmaceuticals provides rescue medicines typically used in emergency rooms and intensive care units to treat patients for whom there are limited treatment options. We are dedicated to delivering quality medicines that make a real ...
based inWoburn, MASSACHUSETTS (USA)
Aegle Therapeutics is a first in class, phase 1/2a stage biotechnology company isolating extracellular vesicles including exosomes (EVs) secreted by allogeneic bone marrow derived mesenchymal stem cells to treat dystrophic epidermolysis bullosa ...
Epidermolysis bullosa (“EB”) is a group of rare genetic disorders that manifests as blistering or erosion of the skin in response to little or no apparent trauma. There are many genetic and symptomatic variations of EB. EB is always ...
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 inBoston, MASSACHUSETTS (USA)
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — ...
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 inMahwah, NEW JERSEY (USA)
KORU is, first and foremost, a patient-centric organization that will go the extra mile for our customers, providers, and partners who depend on our devices. We are a world-class manufacturer of subcutaneous infusion devices that deliver life-saving ...
Chronic inflammatory demyelinating polyneuropathy, or CIDP, is a rare disorder characterized by gradually increasing sensory loss and weakness associated with loss of reflexes. This is caused by an autoimmune process in which the body’s own ...
based inSaint Kevin`s, IRELAND
At Horizon, we believe science and compassion must work together to transform lives. Our mission to deliver medicines for rare, autoimmune and severe inflammatory diseases and provide compassionate support comes from our strong and simple philosophy ...
based inBasel, SWITZERLAND
EsoCap’s vision is to improve the lives of patients affected by esophageal diseases. The esophagus is a particularly challenging environment, as swallowed drugs only remain in the area between the mouth and the stomach for a few seconds. EsoCap, a ...
EsoCap owns a unique drug delivery platform allowing the topical application of drug substances for targeted and long-lasting treatment of diseases of the upper gastrointestinal tract for the first ...
based inAustin, TEXAS (USA)
Veravas™ is transforming laboratory medicine with a first of its kind, patented antibody detection platform to address unmet clinical needs. This innovative technology uses proprietary nano-sized magnetic beads to preanalytically clean patient ...
based inBothell, WASHINGTON (USA)
AGC Biologics offers deep industry expertise and uniquely customized services for the scale-up and cGMP manufacture of protein-based therapeutics (using mammalian and microbial systems), mRNA, pDNA, viral vector and cell therapy products, from ...
based inSan 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 ...
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 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 ...