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Genetic Disorder Suppliers & Manufacturers
44 companies found
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 ...
Argininosuccinic aciduria (ASA) is a rare genetic disorder characterized by a deficiency or lack of the enzyme argininosuccinate lyase (ASL). ...
based inTorrance, CALIFORNIA (USA)
Emmaus Life Sciences, Inc. is a biopharmaceutical company engaged in the discovery, development and commercialization of innovative treatments and therapies primarily for rare and orphan diseases. Our initial efforts have focused on treatments for ...
based inCambridge, MASSACHUSETTS (USA)
Many of us pursuing work in the biotechnology industry are inspired by a loved one who is living with a disease or the loss of someone in our community. Rare genetic and oncological and immunological diseases not only affect the people living with ...
The first patient was dosed with our investigational genome editing treatment for transthyretin (ATTR) amyloidosis in November 2020. Our modular approach enables us to optimize the power and versatility of the CRISPR/Cas9 technology and, ...
based inPune, INDIA
RASA Life Science Informatics, a highly-regarded organization, has gained recognition from Biotech Consortium India Limited (BCIL) and is based in Pune, India. They not only provide research and development services but also offer a comprehensive ...
How Our Gene Expression Analysis Services Can Help You ? Genes encode proteins and proteins dictate cell function. Therefore, the thousands of genes expressed in a particular cell determine what that cell can do. Moreover, each step in the flow of ...
based inEatonton, GEORGIA (US) (USA)
Since 1979 Aalto Scientific has been a leader in the manufacture of OEM/Private label quality controls and calibrators, purified human proteins, and bulk human and animal sera to the in vitro diagnostic (IVD) manufacturing community. With over 40 ...
Homocysteine Control is a stable liquid product intended to simulate human patient samples for the purpose of monitoring the precision of laboratory testing procedures for ...
based inBrignais, FRANCE
Appolon Bioteck is a French research-focused healthcare company with combined strengths in Medical genetic diagnostics, oncology, virology and inflammation. We provide diagnostic tools that enable tangible improvements in the health, quality of life ...
based 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 ...
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 ...
based inJerusalem, ISRAEL
BrainQ Technologies developing breakthrough precision neurology therapies using Brain Computer Interface technology. BrainQ’s goal is to develop therapies that target damaged neuronal networks and facilitate neurorecovery. We are developing a ...
based inToronto, ONTARIO (CANADA)
Avicanna is a Canadian biopharmaceutical company providing the global marketplace with advanced natural cannabinoid solutions through an evidence-based approach offered across several product platforms including API, medical, pharmaceutical, and ...
based inMonmouth Junction, NEW JERSEY (USA)
MedChemExpress (MCE) offers a wide range of high-quality research chemicals and biochemicals (novel life-science reagents, reference compounds and natural compounds) for scientific use. We have professionally experienced and friendly staff to meet ...
based inZirakpur, INDIA
Opdenas Lifesciences is one of the best ophthalmic and ENT based PCD Pharma Franchise Companies in India, having developed a niche in eye, ear, and nasal drops. We carry a large selection of DCGI-approved ophthalmic and ENT products. The portfolio ...
based 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 ...
based in, MASSACHUSETTS (USA)
MtoZ Biolabs is an integrate contract research organization (CRO) providing advanced proteomics, metabolomics, bioinformatics, and biopharmaceutical analysis services to researchers in biochemistry, biotechnology, and biopharmaceutical fields. The ...
based inFargo, NORTH DAKOTA (USA)
We provide high-quality plasmid DNA, proteins, enzymes, and other biologicals to help our partners achieve ground-breaking science throughout a variety of life science applications. From contract services to comprehensive solutions, we supply what ...
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 inValencia, CALIFORNIA (USA)
AVITA Medical is a publicly listed, global regenerative medicine company that provides a novel approach to skin regeneration. Our products provide innovative treatment solutions derived from the regenerative properties of a patients own skin. AVITA ...
Epidermolysis Bullosa (EB) is a group of rare and incurable skin disorders caused by mutations in genes encoding structural proteins resulting in skin fragility and blistering, leading to chronic wounds and, in some sub-types, an increased risk of ...
based inBoston, MASSACHUSETTS (USA)
SOPHiA GENETICS is the creator of a global data-sharing network – we work with customers from over 790 institutions in over 70 countries. Our network advances data-driven medicine to improve health outcomes and economics worldwide. We offer a ...
Most rare and inherited diseases have a neurological component, likely because more than 80% of human genes are expressed in the brain.1 Neurological disorders are multifactorial and heterogeneous, meaning that their ...
based 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 ...
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 ...
We provide a quick turnaround, offering actionable results to guide Rapid Precision Medicine™ and improve patient outcomes. Phenotype-Driven Results: Rapid, robust phenotype-driven results are delivered to inform medical management. ...