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Rare Disease Suppliers & Manufacturers

89 companies found

Taiwan Liposome Company, Ltd. (TLC)

Taiwan Liposome Company, Ltd. (TLC)

based inNangang Distric, TAIWAN
We are a clinical-stage specialty pharmaceutical company dedicated to the development and commercialization of best-in-class novel nanomedicines that combine our proprietary lipid-assembled drug delivery platform with approved active pharmaceutical ...
CONTACT SUPPLIER

MedGenome Inc.

MedGenome Inc.

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) ...
CONTACT SUPPLIER

Orgenesis Inc.

Orgenesis Inc.

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 ...
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

MedChemExpress LLC (MCE)

MedChemExpress LLC (MCE)

based inMonmouth Junction, NEW JERSEY (USA)
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 ...
MedChemExpress - Model Maralixibat chloride - 228113-66-4

MedChemExpress - Model Maralixibat chloride - 228113-66-4

Maralixibat (SHP625) chloride is an orally active ileal bile acid transporter (IBAT) inhibitor. Maralixibat chloride can be used for the research of rare cholestatic liver diseases including Alagille syndrome ...
CONTACT SUPPLIER

Chimeron Bio

Chimeron Bio

based inPhiladelphia, PENNSYLVANIA (USA)
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 ...
CONTACT SUPPLIER

SeqOne S.A.S.

SeqOne S.A.S.

based inMontpellier, FRANCE
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, ...
Rare and Hereditary Diseases Services

Rare and Hereditary Diseases Services

We offer a comprehensive solution for hereditary and rare disease data that detects both commonly accessible variants and relevant challenging variants such as mid-sized deletions, Alu elements and larger structural ...
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

SOPHiA Genetics

SOPHiA Genetics

based inBoston, MASSACHUSETTS (USA)
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 ...
SOPHiA DDM - Version DDM - Cloud-Based Software Platform for Neurological Disorders

SOPHiA DDM - Version DDM - Cloud-Based Software Platform for Neurological Disorders

Most rare and inherited diseases have a neurological component, likely because more than 80% of human genes are expressed in the brain.1 Neurological disorders are multifactorial and heterogeneous, meaning that ...
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

Pluristem Therapeutics Inc.

Pluristem Therapeutics Inc.

based inHaifa, ISRAEL
Pluristem Therapeutics Inc. is a clinical-stage regenerative medicine company using placental cells and a unique, proprietary, three-dimensional (3D) technology platform to develop cell therapies for conditions such as inflammation, muscle injuries, ...
Bone Marrow Deficiency

Bone Marrow Deficiency

Bone marrow deficiency refers to a condition in which the hematopoietic stem cells in the bone marrow fail to produce enough, or produce abnormal, red blood cells, white blood cells and platelets. Hematopoietic cell transplantation (HCT), the ...
CONTACT SUPPLIER

ComplianceOnline

ComplianceOnline

based inSan Jose, CALIFORNIA (USA)
Enhancing global compliance, creating a world where quality and compliance professionals, regulators, and government agencies come together to help the world comply with the intent and the spirit of laws, policies and mandates, ensuring continuous ...
Top 10 Medical Device Regulations Training (10 Courses)

Top 10 Medical Device Regulations Training (10 Courses)

This comprehensive training package of ten courses is aimed at helping medical device companies gain a thorough understanding of the top 10 regulations that they should comply ...
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

Suvoda

Suvoda

based inConshohocken, PENNSYLVANIA (USA)
Suvoda is a global clinical trial technology company dedicated to supporting complex studies for life-sustaining therapies.We shape digital experiences that empower you to take command over mission-critical and time-sensitive patient interactions in ...
Model IRT - Advanced Patient Randomization and Trial Supply Management System

Model IRT - Advanced Patient Randomization and Trial Supply Management System

Our advanced patient randomization and trial supply management system. Your clinical trial command and control ...
CONTACT SUPPLIER

Horizon Therapeutics plc

Horizon Therapeutics plc

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 ...
CONTACT SUPPLIER

Jeeva Informatics Solutions Inc.

Jeeva Informatics Solutions Inc.

based inManassas, VIRGINIA (USA)
The Jeeva eClinical Cloud was developed by researchers with empathy who listen and learn to help clinical researchers, hospital sites, academic medical centers, CROs, and biopharmaceutical sponsors accelerate patient enrollment by 3x faster. Jeeva ...
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

Applied Pharma Research s.a.

Applied Pharma Research s.a.

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 ...
CONTACT SUPPLIER

Camurus AB

Camurus AB

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 ...
CONTACT SUPPLIER

CSL Behring

CSL Behring

based inKing of Prussia, PENNSYLVANIA (USA)
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 ...
CONTACT SUPPLIER
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