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Rare Disease Suppliers & Manufacturers
94 companies found
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 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 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 inMilpitas, CALIFORNIA (USA)
ASC Therapeutics is a fully incorporated biopharmaceutical company focusing on the development of curative therapeutic products that are enabled by our proprietary gene editing platform, TARGATT and other gene editing technologies developed after 12 ...
based inGangnam-gu, SOUTH KOREA
MitoImmune Therapeutics Inc. is a drug discovery and development biotech company that harnesses the power of mitochondria and its proprietary technology targeting mitochondria as the source for developing new potential breakthrough therapeutics. Our ...
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, 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 inStockholm, SWEDEN
Gesynta Pharma is a Swedish pharmaceutical company that develops unique drug candidates with the aim to reduce harmful inflammation and increase blood flow in the body’s microvasculature, thereby providing safe and efficacious treatments for several ...
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 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 inTorrance, CALIFORNIA (USA)
Emmaus Life Sciences, Inc. is a biopharmaceutical company engaged in the discovery, development and commercialization of innovative treatments and therapies primarily for rare and orphan diseases. Our initial efforts have focused on treatments for ...
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 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 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 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 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 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 inSan Francisco, CAMBODIA
Verana Health is a digital health company that delivers quality drug lifecycle, medical practice, and medical society insights from an exclusive real-world data network of more than 20,000 healthcare providers. Our specialty medical society partners ...
From protocol development and clinical trial design to site selection and identifying potential participants*, we help speed patient enrollment to help you meet your recruitment goals. Find out how Verana Health can support your next ...
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 inPlanegg, GERMANY
TQ Therapeutics GmbH is pioneering a novel approach to cell and gene therapy with its TQx platform, which transforms the production of cellular therapies into a rapid, automated process that occurs at the patient's side. This method leverages ...
TQx is developing a modular target cell-specific modification platform including relevant technology components for the generation of gene modified cell therapy ...