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Rare Disease Suppliers & Manufacturers

94 companies found

Rady Children`s Institute for Genomic Medicine

Rady Children`s Institute for Genomic Medicine

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 ...
CONTACT SUPPLIER

Seven Bridges Genomics

Seven Bridges Genomics

based inCharlestown, MASSACHUSETTS (USA)
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 ...
Seven Bridges - Version GRAF - Graph-Based Workflows and Tools for Next-Generation Sequencing Data

Seven Bridges - Version GRAF - Graph-Based Workflows and Tools for Next-Generation Sequencing Data

The representation of the human reference genome as a linear haploid DNA sequence poses limitations when trying to incorporate the known genetic diversity of human populations. This has led to the development of graph-based references, able to ...
CONTACT SUPPLIER

Lumos Pharma

Lumos Pharma

based inAustin, TEXAS (USA)
Lumos Pharma is a clinical-stage biopharmaceutical company whose mission is to develop new therapies for people with rare diseases, prioritizing its focus where the medical need is high, and the pathophysiology is clear. We are committed to this ...
CONTACT SUPPLIER

BioMarin

BioMarin

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 ...
Funding & Support

Funding & Support

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 ...
CONTACT SUPPLIER

CycloLab Cyclodextrin Research and Development Laboratory Ltd.

CycloLab Cyclodextrin Research and Development Laboratory Ltd.

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 ...
CONTACT SUPPLIER

Novo Nordisk A/S

Novo Nordisk A/S

based inBagsværd, DENMARK
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. ...
Collaborating Services

Collaborating Services

We continuously explore strategic collaborations to advance our pipeline toward commercialization and to further our work in the treatment of both rare diseases and common diseases that have a ...
CONTACT SUPPLIER

Woolsey Pharmaceuticals, Inc.

Woolsey Pharmaceuticals, Inc.

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, ...
CONTACT SUPPLIER

Tonix Pharmaceuticals Holding Corp.

Tonix Pharmaceuticals Holding Corp.

based inChatham, NEW JERSEY (USA)
Tonix is a clinical-stage biopharmaceutical company focused on developing novel therapies and vaccines to prevent and treat central nervous system disorders, immunology conditions, infectious diseases, and rare diseases. At Tonix, we are focused on ...
Tonix - Model TNX-2900 - Prader-Willi Syndrome Drug

Tonix - Model TNX-2900 - Prader-Willi Syndrome Drug

TNX-2900, Tonix’s proprietary potentiated intranasal oxytocin is in the pre-Investigational New Drug (IND) stage as a candidate for the treatment of Prader-Willi syndrome (PWS) and non-organic failure to thrive disease. TNX-2900 has been ...
CONTACT SUPPLIER

EsoCap AG

EsoCap AG

based inBasel, 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 ...
EsoCap - Drug Delivery Platform

EsoCap - Drug Delivery Platform

EsoCap owns a unique drug delivery platform allowing the topical application of drug substances for targeted and long-lasting treatment of diseases of the upper gastrointestinal tract for the first ...
CONTACT SUPPLIER

Alexion Pharmaceuticals, Inc.

Alexion Pharmaceuticals, Inc.

based inBoston, MASSACHUSETTS (USA)
People living with rare and devastating diseases are our Guiding Star. We believe it is our responsibility to listen to, understand, and change the lives of patients and those who work tirelessly to help them. Our mission is to transform the lives ...
Alexio - Model RARE ANSWERS - System of Innovative and Sustainable Diagnostic Tools

Alexio - Model RARE ANSWERS - System of Innovative and Sustainable Diagnostic Tools

People with rare diseases often wait years to receive a proper diagnosis and many never receive one at all. RARE ANSWERS™ is a system of innovative and sustainable tools designed to help ...
CONTACT SUPPLIER

MitoImmune Therapeutics Inc.

MitoImmune Therapeutics Inc.

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 ...
CONTACT SUPPLIER

BTG International Inc.

BTG International Inc.

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 ...
CONTACT SUPPLIER

Veravas, Inc.

Veravas, Inc.

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 ...
CONTACT SUPPLIER

Vertex Pharmaceuticals

Vertex Pharmaceuticals

based inBoston, MASSACHUSETTS (USA)
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — ...
CONTACT SUPPLIER

SoftGenetics, LLC.

SoftGenetics, LLC.

based inState College, PENNSYLVANIA (USA)
SoftGenetics, LLC specializes in the development of genetic analysis tools for both research and diagnostic ...
NextGENe - Next Generation Sequencing Software

NextGENe - Next Generation Sequencing Software

NextGENe software is the perfect analytical partner for the analysis of desktop sequencing data produced by Illumina® iSeq, Miniseq, MiSeq, NextSeq, HiSeq, and NovaSeq systems, Ion Torrent Ion GeneStudio S5, PGM, and Proton systems as well as ...
CONTACT SUPPLIER

Invitae Corporation

Invitae Corporation

based inSan Francisco, CALIFORNIA (USA)
Invitae’s mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Like wearing sunscreen or getting your blood pressure checked at the doctor, genetic testing is another important ...
CONTACT SUPPLIER

Sangamo Therapeutics

Sangamo Therapeutics

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 ...
Sangamo - Programs Services

Sangamo - Programs Services

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, ...
CONTACT SUPPLIER

AGC Biologics

AGC Biologics

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 ...
CONTACT SUPPLIER

Canè S.p.A.

Canè S.p.A.

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 ...
CONTACT SUPPLIER

Population Bio, Inc. a Precision Medicine Company

Population Bio, Inc. a Precision Medicine Company

based inMelville, NEW YORK (USA)
Population Bio, Inc. is a privately held precision medicine company that is empowering pharmaceutical companies to develop targeted therapies and companion diagnostics faster and more cost effectively. We are accelerating the delivery of precision ...
CONTACT SUPPLIER
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