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Rare Disease Suppliers & Manufacturers

89 companies found

Andelyn Biosciences

Andelyn Biosciences

based inColumbus, OHIO (USA)
Andelyn Biosciences is a biopharmaceutical CDMO Pioneering Solutions That Turn Hope into Reality™. With 20+ years of experience manufacturing viral vectors, Andelyn’s scientific expertise for development and characterization has led to GMP material ...
Clinical Manufacturing Services

Clinical Manufacturing Services

Andelyn Biosciences provides clients with a broad range of viral vector manufacturing platforms and solutions, including AAV and Lentiviral adherent and suspension systems along with plasmid manufacturing. We support our clients throughout every ...
CONTACT SUPPLIER

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

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

Gesynta Pharma AB

Gesynta Pharma AB

based inStockholm, SWEDEN
Gesynta Pharma is a Swedish pharmaceutical company that develops unique drug candidates with the aim to reduce harmful inflammation and increase blood flow in the body’s microvasculature, thereby providing safe and efficacious treatments for several ...
CONTACT SUPPLIER

CENTOGENE N.V.

CENTOGENE N.V.

based inRostock, GERMANY
Since 2006, CENTOGENE has helped physicians diagnose thousands of rare disease patients around the world. A quick and accurate diagnosis can end an odyssey of medical tests and visits to multiple medical specialists. It also helps physicians better ...
CentoCloud - Version SaaS - Cloud-Based Software

CentoCloud - Version SaaS - Cloud-Based Software

Analyze, interpret, and report genomic variants with our decentralized diagnostic solution – no matter where you are located. CentoCloud is a cloud-based Software as a Service (SaaS) platform specifically designed for the rapid bioinformatic ...
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

Strand Life Sciences Pvt. Ltd.

Strand Life Sciences Pvt. Ltd.

based inBangalore, INDIA
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 ...
SmartLab Services

SmartLab Services

Strand has extensive experience in validating NGS-based panels under CAP- and CLIA-settings: be it germline panels, somatic tumor panels, or liquid biopsy panels, and be it small panels with tens of genes, to large panels with thousands of ...
CONTACT SUPPLIER

Azafaros

Azafaros

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 ...
Oral Treatment for Lysosomal Storage Disorders

Oral Treatment for Lysosomal Storage Disorders

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 ...
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

Verana Health

Verana Health

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 ...
Clinical Development Services

Clinical Development Services

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 ...
CONTACT SUPPLIER

Certara

Certara

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. ...
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

Ipsen Group

Ipsen Group

based inParis, FRANCE
Ipsen is a global biopharmaceutical group dedicated to improving lives and health outcomes through innovative medicines in Oncology, Neuroscience and Rare Disease. We also have a well-established and successful Consumer Healthcare business. We are ...
CONTACT SUPPLIER

Palvella Therapeutics.

Palvella Therapeutics.

based inWayne, PENNSYLVANIA (USA)
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 ...
Palvella - Model QTORIN - Gorlin Syndrome

Palvella - Model QTORIN - Gorlin Syndrome

QTORIN™ rapamycin 3.9%is a novel targeted topical therapy being studied for Gorlin syndrome. Palvella is enrolling a phase 2b clinical study (CODY) for people with Gorlin ...
CONTACT SUPPLIER

Cell Guidance Systems Ltd.

Cell Guidance Systems Ltd.

based inCambridge, UNITED KINGDOM
Cell Guidance Systems' mission is to develop products and services that enhance the human condition and protect our shared environment. We develop technologies that enable the delivery of bioactive molecules, controlling cell fate for medical, ...
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

JADBio

JADBio

based inLos Angeles, CALIFORNIA (USA)
JADBio stands for Just Add Data and aims to make machine learning accessible to all regardless of expertise or programming skills. JADBio is a robust AutoML platform focused on BioMed and Multi-omics. We make it easy and affordable for data ...
Solutionn for Bio-Molecular Data

Solutionn for Bio-Molecular Data

AutoML brings democratization to the data analysis process by allowing everyone to utilize Machine Learning. Fully automate your data analysis, end-to-end, with AI and our purpose-built algorithms starting from preprocessing to interpreting the ...
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

Acorda Therapeutics, Inc.

Acorda Therapeutics, Inc.

based inPearl River, NEW YORK (USA)
Acorda Therapeutics is a publicly traded (NASDAQ: ACOR) biotechnology company whose mission is to develop therapies that restore neurological function and improve the lives of people with neurological disorders. We started our company in 1995 as a ...
CONTACT SUPPLIER

Curome BioSciences

Curome BioSciences

based inheongju-si, SOUTH KOREA
Curome Biosciences is an innovative R&D company developing therapies to treat mitochondrial dysfunction-related diseases with high unmet medical needs. Curome Biosciences wants to provide solutions for treatment of diseases with high unmet medical ...
Researching Services

Researching Services

Mitochondria are an essential cellular organelle that is responsible for the generation of energy (known as the ‘powerhouse of the cell’). Mitochondrial dysfunction is related with numerous diseases. Curome Biosciences ...
CONTACT SUPPLIER
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