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Genetic Disorder Suppliers & Manufacturers
4 companies found
Technologybased inCambridge, UNITED KINGDOM
We’re driven by the shared purpose of breaking new ground in the discovery of new treatments for rare diseases. Healx is a mission-driven technology company pioneering the next wave of drug discovery in order to bring novel, effective treatments to ...
Technologybased inSouth San Francisco, CALIFORNIA (USA)
Neurona Therapeutics is driven by rigorous scientific discovery and focused on developing breakthrough treatments for neurological disorders. Neurona Therapeutics is a privately-held, clinical-stage biotherapeutics company focused on discovering and ...
Neurona’s lead product candidate, NRTX-1001, comprises human MGE-type inhibitory GABAergic interneurons derived from pluripotent stem cells. NRTX-1001’s first indication is for the treatment of drug-resistant focal epilepsy, which is a ...
Technologybased inNess Ziona, ISRAEL
Science in Action (SIA) is a boutique preclinical contract research organization (CRO) founded in 2010 by immunologist Raanan Margalit. SIA is GLP-accredited for toxicology studies in multiple areas. SIA has extensive experience in tailor-made ...
SIA's philanthropic research supports the Israeli Duchenne Parent Association. This is an eort to develop a cure for Duchenne Muscular Dystrophy (DMD). DMD is the most common, fatal genetic disorder diagnosed in ...
Technologybased 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 ...