Rare Disease Suppliers & Manufacturers

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 ...

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 ...

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 ...

At Zynerba Pharmaceuticals, our team is dedicated to developing next-generation transdermally-delivered cannabinoid therapeutics for patients affected by rare and near-rare neuropsychiatric conditions. Often, these diseases have few, if any, ...

Seven Bridges is the leading biomedical data company, specializing in software and data analytics to drive public and private healthcare research. We deliver end-to-end bioinformatic solutions including access to datasets, analytic workflows and ...

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 ...

We are a global healthcare company, founded in 1923 and headquartered just outside Copenhagen, Denmark. Our purpose is to drive change to defeat diabetes and other serious chronic diseases such as obesity, and rare blood and rare endocrine diseases. ...

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 ...

Chimeron Bio is building a portfolio of self-amplifying RNA therapeutics and vaccines to bring more effective gene therapies and personalized medicines to patients. We are developing a compelling portfolio of innovative and personalized saRNA-based ...

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 ...

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 ...

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 ...

MedChemExpress (MCE) offers a wide range of high-quality research chemicals and biochemicals (novel life-science reagents, reference compounds and natural compounds) for scientific use. We have professionally experienced and friendly staff to meet ...

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 ...

The drug discovery and development process is long and expensive with more failures than successes. At Certara, we anticipate and address your critical drug discovery and development risks and decisions using biosimulation, technology and services. ...

SCIREQ strives to make a strong impact on global pulmonary health by empowering scientists to better understand and eradicate lung diseases. The global burden of respiratory diseases is primarily driven by asthma, chronic obstructive pulmonary ...

SeqOne Genomics is a startup based in Montpellier in the South of France, founded in 2017. The company develops state-of-the-art genomics analysis tools for clinical applications in the fields of cancer and rare diseases. Its flagship product, ...

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 ...

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 ...

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 ...