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Rare Disease Suppliers & Manufacturers
94 companies found
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 inGermantown, MARYLAND (USA)
Cell and gene therapies (CGTs) use modified cells or cellular matter to treat, prevent or cure serious health issues like cancer, genetic disorders, immunodeficiencies, rare diseases, and more. CGTs have shown potential to replace damaged cells with ...
based inLaupheim, GERMANY
We are a leading global Contract Development and Manufacturing Organization (CDMO), focused exclusively on client projects. From our headquarters in Laupheim, Germany, and our site in Milford, MA, USA, we offer end-to-end solutions including ...
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 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 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 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 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 inBalerna, 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 ...
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 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 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 ...
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 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 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 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 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 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 inVancouver, BRITISH COLUMBIA (CANADA)
Precision NanoSystems is a global leader in technologies, solutions and services for the development of non-viral delivery of genomic medicines, including mRNA vaccines and therapeutics. We work with the world’s leading drug developers to understand ...
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 ...