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Rare Disease Suppliers & Manufacturers
94 companies found
based inBudapest, HUNGARY
Cytocast, an innovative TechBio startup located in Budapest, is at the forefront of data-driven medical technology.The company's primary focus is on integrating extensive multi-omics data with high-performance computing and advanced data science ...
Cytocast is at the forefront of medical technology with its innovative Cytocast Digital Twin Platform™, a pioneering solution in drug development and personalized medicine. At its core is the CYTOCAST DIGITAL TWIN Cell™, a highly ...
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. ...
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 ...
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, ...
based inNaarden, NETHERLANDS
Azafaros has leveraged on decades of discovery by professors Hans Aerts, Hermen Overkleeft, and Stan van Boeckel at Leiden University and the Academic Medical Center in the Netherlands. Expanding on their research and led by highly experienced ...
Azafaros B.V. is pioneering the development of innovative oral small molecule compounds addressing the pathological effects of metabolite accumulation in rare metabolic disorders. Their lead compound, nizubaglustat, leverages unique ...
based inDublin, IRELAND
Our mission is to help clients accelerate the development of drugs and devices that save lives and improve quality of life. We offer a full range of consulting, clinical development and commercialisation services from a global network of offices in ...
Accelerating study start-up and delivering results with custom strategies and logistics solutions. ICON helps navigate the Cell and Gene Therapy journey by overcoming challenges and mitigating risks. Almost every cell and gene therapy clinical trial ...
based inSan Francisco, CAMBODIA
Verana Health is a digital health company that delivers quality drug lifecycle, medical practice, and medical society insights from an exclusive real-world data network of more than 20,000 healthcare providers. Our specialty medical society partners ...
From protocol development and clinical trial design to site selection and identifying potential participants*, we help speed patient enrollment to help you meet your recruitment goals. Find out how Verana Health can support your next ...
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 ...
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 inWhite Plains, NEW YORK (USA)
Oligomerix is a biotechnology company focused on developing disease-modifying and treatment therapeutics for neurodegenerative diseases characterized by aberrant tau protein ranging from rare tauopathies such as progressive supranuclear palsy ...
There exist numerous rare diseases that tau dysfunction is associated with known as tauopathies. While Alzheimer’s is the largest tauopathy, other tauopathies are rare ...
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 ...
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, ...
based inToronto, ONTARIO (CANADA)
Deep Genomics recruits from among the top 1% of recent graduates and seasoned experts at the intersection of genomics, drug development and AI. Programming RNA Therapies Any Gene, Any Genetic Condition. Revolutions in AI, RNA biology and automation ...
based inWoburn, MASSACHUSETTS (USA)
Aegle Therapeutics is a first in class, phase 1/2a stage biotechnology company isolating extracellular vesicles including exosomes (EVs) secreted by allogeneic bone marrow derived mesenchymal stem cells to treat dystrophic epidermolysis bullosa ...
Epidermolysis bullosa (“EB”) is a group of rare genetic disorders that manifests as blistering or erosion of the skin in response to little or no apparent trauma. There are many genetic and symptomatic variations of EB. EB is always ...
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 inPlanegg, GERMANY
MorphoSys is a commercial-stage biopharmaceutical company dedicated to the discovery, development, and delivery of innovative cancer medicines. At MorphoSys, our ambition is to redefine how cancer is treated. As a commercial-stage biopharmaceutical ...
Expanded Access to our Investigational Medicinal Products Policy. At MorphoSys, our mission is to make innovative biopharmaceuticals to improve the lives of patients suffering from serious diseases. Before these and all other investigational ...
based inStockholm, SWEDEN
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 ...
IBP-9414 for the prevention of necrotizing enterocolitis (NEC) contains the active substance Lactobacillus reuteri, which is a co-evolved human bacterial strain naturally present in breast milk. Lactobacillus reuteri is a live bacteria known to be ...
based inDallas, TEXAS (USA)
Lupagen is a privately held early stage biotech company developing first-in-class bedside enabled cell & gene therapies (CAR-T cell, gene editing and immunotherapy products) based on their patent-pending bedside point-of-care C and GT production and ...
Lupagen avoids the cost & complexity of cell therapy and the safety & clinical challenges of viral-based gene therapy by a simple patient-connected gene delivery procedure done at the bedside. Our Side CAR-T® delivery system, 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 ...
based inPrinceton, NEW JERSEY (USA)
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. ...
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 inSan Carlos, CALIFORNIA (USA)
Developing live biotherapeutics to prevent and treat diseases of high unmet medical need. We leverage our deep knowledge of the human microbiome and its regulatory and developmental functions to develop live biotherapeutic products (LBPs) and ...