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Rare Disease Suppliers & Manufacturers
94 companies found
based inBundoora, AUSTRALIA
AdAlta is an innovative, clinical stage biotech company developing a unique range of new drug treatments. AdAlta has pioneered a technology that mimics the shape and stability of a crucial antigen-binding domain, discovered initially in sharks and ...
AdAlta is utilising the power of the i-body technology platform to develop a growing pipeline of i-bodies to treat a range of conditions, with an initial focus on treating fibrotic diseases. AdAlta identified an i-body that binds to the drug target, ...
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 inMontreal, QUEBEC (CANADA)
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 ...
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 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 ...
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 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 inOxford, UNITED KINGDOM
Evox Therapeutics is a privately held, Oxford-based biotechnology company focused on improving the natural delivery capabilities of exosomes, and developing an entirely new class of therapeutics. Backed by leading venture capital groups and ...
Niemann-Pick C disease (NPC) is a lysosomal storage disorder which affects the brain and multiple other ...
based inNew York, NEW YORK (USA)
Protara is committed to identifying and advancing transformative therapies for people with cancer and rare diseases. The Protara team prioritizes creativity, diverse perspectives and tenacity to expedite our goal of bringing life-changing therapies ...
based inIrving, TEXAS (USA)
Avero Diagnostics is dedicated to making a positive impact on the lives of patients and the physicians who treat them. We bring a patient-centered approach to every aspect of our organization, including our laboratory and research facility. It’s ...
Expectant parents want to do everything they can before the birth of their baby. The Avero Carrier Test looks at a patient’s genes to see if they may carry a disease that they could pass on to their child. Knowing a patient’s genetic ...
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 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 ...
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 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 ...
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 ...
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 ...
Our advanced patient randomization and trial supply management system. Your clinical trial command and control ...
based inMahwah, NEW JERSEY (USA)
KORU is, first and foremost, a patient-centric organization that will go the extra mile for our customers, providers, and partners who depend on our devices. We are a world-class manufacturer of subcutaneous infusion devices that deliver life-saving ...
Chronic inflammatory demyelinating polyneuropathy, or CIDP, is a rare disorder characterized by gradually increasing sensory loss and weakness associated with loss of reflexes. This is caused by an autoimmune process in which the body’s own ...
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 ...
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 ...
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 ...
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 ...
based inPhiladelphia, PENNSYLVANIA (USA)
We are a biotechnology company at the forefront of advanced therapies to treat a range of devastating rare diseases. Amicus Therapeutics (Nasdaq:FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and ...
based inVancouver, BRITISH COLUMBIA (CANADA)
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 ...