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Rare Disease Suppliers & Manufacturers
94 companies found
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 inBrisbane, CALIFORNIA (USA)
Sangamo is a genomic medicine company that seeks to help millions of people who suffer from severe diseases for which today’s medicine can only offer symptom management at best. Our mission is to translate ground-breaking science into medicines that ...
Behind these are research efforts thoughtfully focused on leveraging our differentiated zinc finger technology. We are advancing a robust pipeline of wholly owned and partnered programs in rare disease, neurology, ...
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 ...
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 inRedwood City, CALIFORNIA (USA)
At Codexis, we apply our technology platform to engineer enzymes for biosolutions that benefit human health and environmental health. From developing biocatalysts for sustainable manufacturing of pharmaceuticals and food ingredients, to engineering ...
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 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 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 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 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 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 inLund, SWEDEN
Camurus is committed to developing and commercializing innovative and long-acting medicines for the treatment of severe and chronic conditions, including opioid dependence, pain, cancer and endocrine disorders. New drug products are based on our ...
based inCambridge, UNITED KINGDOM
We’re driven by the shared purpose of breaking new ground in the discovery of new treatments for rare diseases. Healx is a mission-driven technology company pioneering the next wave of drug discovery in order to bring novel, effective treatments to ...
Our Rare Treatment Accelerator helps academic, patient, and industry groups advance their rare disease drug redevelopment projects. By combining our team, expertise and financial resources with your ...
based inMilano, ITALY
We are a clinical-stage biotechnology company pioneering the treatment of cancers with a technology platform that allows the direct delivery of immunotherapeutic payloads within the tumor microenvironment. Intrinsic resistance significantly reduces ...
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 ...
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 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 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 inJenkintown, PENNSYLVANIA (USA)
SFA Therapeutics, Inc. is a development-stage bio-pharmaceutical startup company focused on a new advancement in the treatment of chronic inflammatory disease – the use of microbiome-derived metabolites as drugs. A completely new approach to drug ...
SFA Therapeutics, Inc. is a development-stage bio-pharmaceutical startup company focused on a new advancement in the treatment of chronic inflammatory disease – the use of microbiome-derived metabolites as ...
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 ...