Rare Disease Suppliers & Manufacturers

Confo Therapeutics’ unparalleled technology stabilizes functional conformations of GPCRs (G protein-coupled receptors) to uncover a wide range of previously inaccessible GPCRs as drug targets. This technology platform combined with the pharmacologic ...

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, ...

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 ...

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 ...

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 ...

SOPHiA GENETICS is the creator of a global data-sharing network – we work with customers from over 790 institutions in over 70 countries.  Our network advances data-driven medicine to improve health outcomes and economics worldwide. We offer a ...

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 ...

Behring's parent company, CSL, was formed more than 100 years ago to save lives using the latest technologies. In the century since, CSL Behring has grown into a global biotechnology leader, driven by that same promise to save and improve lives. We ...

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 ...

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 ...

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 ...

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 ...

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 ...

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 ...

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, ...

JADBio stands for Just Add Data and aims to make machine learning accessible to all regardless of expertise or programming skills. JADBio is a robust AutoML platform focused on BioMed and Multi-omics. We make it easy and affordable for data ...

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 ...

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 ...

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 ...

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 ...