Orphan Disease Manufacturers, Suppliers & Companies
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Manufacturerbased in Philadelphia, 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 ...
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Manufacturerbased in Torrance, 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 ...
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Manufacturerbased in Woodcliff Lake, NEW JERSEY (USA)
Eagle is a specialty pharmaceutical company working to advance safe and efficient injectable treatments for patients across oncology, critical care, and orphan diseases. At Eagle, we are committed to developing innovative drugs that result in ...
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Manufacturerbased in Balerna, SWITZERLAND
APR Applied Pharma Research s.a. (APR) is a Swiss Pharma company dedicated to providing patients suffering from rare diseases with innovative treatments that make a real difference in their life. Our focus as a Swiss global health care company is ...
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Manufacturerbased in London, UNITED KINGDOM
Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM) is a pharmaceutical company dedicated to developing and commercializing novel therapeutics to tackle unmet medical needs. Aptorum Group is pursuing therapeutic projects in orphan diseases, ...
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Technologybased in Rixensart, BELGIUM
CuraVac is a clinical-stage biotech company developing the medical and commercial potential of a revolutionary technique for the production of specific Therapeutic Vaccines for the treatment of autoimmune diseases. The company was founded by a ...
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Manufacturerbased in Loos, FRANCE
GENFIT is a late-stage biopharmaceutical company dedicated to improving the lives of patients with metabolic and chronic liver diseases. Our translational disease-driven research approach is based on strong expertise, featuring in-house knowledge of ...
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Manufacturerbased in Oxford, UNITED KINGDOM
At OMass Therapeutics, we are identifying new medicines against highly validated but inadequately drugged targets using novel biochemistry techniques, native mass spectrometry and custom chemistry. We distil biology to its essential elements – ...
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Manufacturerbased in Basel, 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 ...
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Professional associationbased in Bloomington, MINNESOTA (USA)
The National Wildlife Rehabilitator's Association (NWRA) is dedicated to improving and promoting the profession of wildlife rehabilitation and its contributions to preserving natural ecosystems. Wildlife rehabilitators are committed to the treatment ...
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Manufacturerbased in Bothell, 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 ...
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Technologybased in Bubendorf, SWITZERLAND
CIS Pharma is an innovator in the life science industry in the field of Oncology and Ophthalmolog. We were awarded with the European Frost & Sullivan Technology Innovation Award for our unique smart polymer technologyœ, says Christoph Schäfer, CEO ...
CIS Pharma - Polymer-based Delivery System
RNA interference (RNAi) is a mechanism used to defend against external invasion. Oligo- and poly-nucleotide based RNAi therapeutics comprise a high clinical potential for unmet medical diseases and are seen as promising approach to ...
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Technologybased in Cambridge, 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 ...
GW - Autism Spectrum Disorder Treatment
Many of the childhood-onset intractable epilepsy conditions within the expanded access program share considerable overlap with ASD and these conditions often fall within the orphan disease space. Initial clinical ...
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Manufacturerbased in Huningue, 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 ...
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Manufacturerbased in Berwyn, 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 ...
Virpax - Model NobrXiol - Intranasal Pharmaceutical-Grade Cannabidiol (CBD)
Benefits: NobrXiol may be using significantly less pharmaceutical-grade CBD than current FDA approved oral CBD dosing. NobrXiol may achieve higher efficiency via the nasal route and reduce peripheral side effects like dose dumping due to high-fat ...
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Software vendorbased in Moscow, RUSSIA
DM RWE team has been working with clinical data since 2009. We process data, analyse it and develop documents to be accepted by the Russian Ministry of Health, FDA, and EMA. Our products automate the processes of clinical trials, and users manage ...
Real World Evidence (RWD) Services
Real world evidence (RWE) and real world data (RWD) have been gaining a lot of interest with their role in global healthcare becoming more and more apparent. Real world data is the data relating to patient health status and/or the delivery of health ...
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Manufacturerbased in St-Jean, QUEBEC (CANADA)
We are an innovative pharmaceutical company determined to improve the lige of people with allergy. We have developed our biotechnology with you in mind. How could we turn on sharp insights on allergy and precision medicine into a better life for ...
Angany - 3rd Generation Plant-based Production Platform
We aim to provide safe, dependable and lasting relief from allergy. We have developed a unique therapeutic approach based on a proprietary allergy vaccine technology to address the specific challenge of allergy (eBioparticle-Potentiated ...
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Consulting firmbased in Dublin, 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 ...
Cell and Gene Therapies Service
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 ...
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Manufacturerbased in Varedo, ITALY
MERYLO’ is developing a better therapy for patients with liquid cancers. With our innovative medical device that, it will be possible to entrap the chemotherapeutical agent into the patient’s Red Blood Cells, obtaining a sustained release. The ...
Drug in a Cell
MERYLO’ is developing a platform that will revolutionize the chemotherapeutic practice, proposing an innovative medical device that will work directly on the patients’ own blood at their bedside. By connecting our system to the patient ...
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Service providerbased in Wroclaw, POLAND
Pure Biologics is a biopharmaceutical company focused on the biological drug and non-systemic therapies discovery and development. We operate in the areas of immuno-oncology, autoimmunology, and rare neurological diseases, conducting research based ...
Pure Biologics - Model PB002 AptaPheresis - Therapeutic Medical Device
Neuromyelitis optica (NMO) is an neurological autoimmune condition classified as a rare disease. Because of this classification, NMO lacks effective treatment options, since it is not of primary interest to the pharmaceutical ...
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