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Rare Disease Day 2017 With A Commitment To Provide New Insights Into Rare Diseases Suppliers In North America
39 companies found
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) — ...
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 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 ...
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 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 ...
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 ...
based inRedwood City, CALIFORNIA (USA)
At Codexis, we apply our technology platform to engineer enzymes for biosolutions that benefit human health and environmental health. From developing biocatalysts for sustainable manufacturing of pharmaceuticals and food ingredients, to engineering ...
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 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. ...
Drug development works best as a team endeavor. 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 genetic ...
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 ...
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 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 diseases that present accelerated regulatory pathways and significant ...
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 shorten the diagnostic journey and time to initiating ...
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 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 is using groundbreaking science and novel technology to replace today’s symptomatic treatments with tomorrow’s genomic cures. We are pursuing programs across the spectrum of genomic medicine, with gene and cell therapies in the ...
based inCharlottesville, VIRGINIA (USA)
HemoShear Therapeutics is a clinical stage company developing novel treatments for patients with rare diseases. We are implementing a new vision for how advanced biological and computational models can accelerate the discovery of innovative ...
HST5040 is an investigational small molecule therapy being developed by HemoShear to reduce the levels of toxins associated with methylmalonic acidemia (MMA) and propionic acidemia (PA), rare genetic disorders caused by the de?ciency of certain ...
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, ...
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 inTarrytown, NEW YORK (USA)
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently ...
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. ...