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Genetic Disorder Suppliers & Manufacturers
40 companies found
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 inMalvern, PENNSYLVANIA (USA)
Ocugen Inc. (NASDAQ: OCGN) is a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to fight COVID-19. Our breakthrough modifier gene therapy platform ...
OCU400 (AAV-NR2E3) is a novel gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases (“IRDs”). It consists of ...
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 inNorth Brunswick, NEW JERSEY (USA)
Ascendia Pharmaceutical Solutions specializes in comprehensive drug development services, providing tailored formulation and manufacturing solutions from pre-formulation stages to commercialization. With a focus on overcoming pharmaceutical ...
In the intricate landscape of biopharmaceuticals, development timing is a critical factor that can significantly impact the success or failure of a drug. Saving time can mean the difference between being first to market for a new drug class or a ...
based inAlameda, CALIFORNIA (USA)
Scribe is focused on the engineering, delivery, and development of next-generation CRISPR molecules to rewrite and repair the underlying cause of genetic ...
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 inSan Rafael, CALIFORNIA (USA)
Established in 1997, BioMarin is a world leader in developing and commercializing first- or best-in-class therapies for rare genetic diseases. We take pride in going where the science leads us, pioneering breakthrough treatments for debilitating and ...
In alignment with our commitment to scientific innovation and areas of unmet medical need, we may provide product and funding support for independent research in rare diseases and genetic ...
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 inClayton, GEORGIA (US) (USA)
GenSol was founded in 2008 to address the growing need for canine genetic screening and the everyday application of increasingly complex biological assays using state-of-the-art technology. Accurate and affordable genetic testing for a multitude of ...
Laryngeal paralysis (LP) is a genetic disorder that results in breathing difficulties which can be made worse by physical activity and may lead to suffocation in severe cases. The condition may require surgery to ...
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 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 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. ...
based inMontreal, QUEBEC (CANADA)
As a specialty therapeutics, clinical-stage biotech company, Inversago Pharma develops new generations of peripherally-acting cannabinoid-1 receptor (CB1) inverse agonists for the treatment of diabetic kidney disease, type 1 and type 2 diabetes, ...
based inVienna, AUSTRIA
ViennaLab Diagnostics specializes in easy-to-use in vitro diagnostic assays for the detection of genetic variants associated with genetic disorders, genetic predispositions, pharmacogenetics, cancer, and the human microbiome. Our focus is on product ...
Alpha-1 Antitrypsin (AAT) deficiency is an inherited disorder, which is still an underappreciated, but treatable cause of chronic obstructive pulmonary disease (COPD). The AAT RealFast™ Assay is designed for the simultaneous detection of ...
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 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 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 inSomerset, NEW JERSEY (USA)
Even the most sophisticated, cutting-edge medical diagnostics aren’t of value unless we can get them to those who need them most. At Access Bio, we believe every life is precious, and every human on earth deserves the opportunity to live a healthy ...
Recommended by the WHO during the Malaria Policy Advisory Meeting. G6PD deficiency is an X-linked recessive genetic disorder, resulting in no or low G6PD activity. G6PD deficienct patients are vulnerable to the ...
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 ...
Genetic testing is a type of medical test that identifies changes in genes, inherited from our parents, which we then typically pass on to our children. “Mistakes” in our genes (called “pathogenic variants”) can manifest the ...
based inVancouver, BRITISH COLUMBIA (CANADA)
Acuitas is a private company based in Vancouver, British Columbia and was founded in February 2009. We are partnered with multiple pharmaceutical companies, biotechnology companies and academic institutes to advance nucleic acid therapeutics to the ...