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Rare Disease Suppliers Serving Micronesia
65 companies found
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 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 inSan 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 ...
We support programs and activities that foster excellence in patient care and provide valuable scientific, medical, and educational information to the medical and scientific communities. We also support clinicians and the independent research they ...
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 inColumbus, OHIO (USA)
Andelyn Biosciences is a biopharmaceutical CDMO Pioneering Solutions That Turn Hope into Reality™. With 20+ years of experience manufacturing viral vectors, Andelyn’s scientific expertise for development and characterization has led to GMP material ...
Andelyn Biosciences provides clients with a broad range of viral vector manufacturing platforms and solutions, including AAV and Lentiviral adherent and suspension systems along with plasmid manufacturing. We support our clients throughout every ...
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 inCambridge, UNITED KINGDOM
Cell Guidance Systems' mission is to develop products and services that enhance the human condition and protect our shared environment. We develop technologies that enable the delivery of bioactive molecules, controlling cell fate for medical, ...
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 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 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 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 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 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 inOxford, UNITED KINGDOM
Evox Therapeutics is a privately held, Oxford-based biotechnology company focused on improving the natural delivery capabilities of exosomes, and developing an entirely new class of therapeutics. Backed by leading venture capital groups and ...
Niemann-Pick C disease (NPC) is a lysosomal storage disorder which affects the brain and multiple other ...
based inHuningue, FRANCE
FIRALIS is a biotechnology company located in the very heart of BioValley, on the true triple border of FR-DE-CH, only a few minutes walking distance to Basel, the pharmaceutical capital of Europe. Firalis creates novel values via biomarker ...
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 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 inRivoli (TO), TURKEY
Since 1978, manufacturer of CRONO pumps, portable infusion systems for the treatment of several diseases. All this has led to the creation of a series of drug-delivery pumps dedicated to the treatment of various pathologies. Canè microinfusers ...
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 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 ...