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Genetic Disorder Suppliers & Manufacturers
44 companies found
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 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 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 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 inVancouver, BRITISH COLUMBIA (CANADA)
InMed Pharmaceuticals is a global leader in the pharmaceutical research, development and manufacturing of rare cannabinoids and cannabinoid analogs, including clinical and preclinical programs targeting the treatment of diseases with high unmet ...
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 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 inMerelbeke, BELGIUM
Ziphius Vaccines is named after the rare dolphin Ziphius cavirostris. This dolphin is most known for undertaking the deepest and longest dives recorded. Ziphius' mission is to reduce the global impact of infectious diseases on global health care by ...
Self-amplifying RNA (saRNA) or replicon RNA has the advantage of having self-replicating features, as the saRNA molecule encodes a viral RNA replicase in addition to the sequence of the protein(s) of interest. Upon cytoplasmic delivery of the saRNA ...
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 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 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 inMunich, GERMANY
The technological progress made in biomedical research has radically changed the view to new treatment strategies. Modern genetic technologies can deliver disease-relevant targets but are insufficient to refill the empty drug discovery pipeline. We ...
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 inSultanbeyli – Istanbul, TURKEY
Anatolia Geneworks is a biotechnology company operating in accordance with ISO 9001, ISO 13485 and 98/79/EC Total Quality Systems. The main activities of Anatolia Diagnostics and Biotechnology Inc. are to develop and manufacture Real-Time Polymerase ...
Familial Mediterranean fever (FMF) is an autosomal recessive genetic disorder, which occurs commonly in Mediterranean countries causing symptoms such as recurrent acute fever, abdominal pain, joint pain etc. FMF ...
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 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 inBentley DC, AUSTRALIA
Ozgene is one of the leading companies providing genetically modified mice for researchers around the world. We offer a full spectrum of pre-clinical capabilities from the development of mouse models through to breeding, characterisation and ...
Knock-in mice have an insertion in a specific locus in order to produce genetically modified mice for customised research purposes. Reporter genes, human genes, related genes from the same organism, or point mutations can be introduced into the ...
based inSeongnam-si, SOUTH KOREA
LASEROPTEK is a global company developing and manufacturing aesthetic and medical laser devices born from market needs and in-house R&D capability. LASEROPTEK laser devices perform aesthetic and medical dermatological treatments across a wide range ...
PALLAS is the world’s first UVB laser with highly safe and efficacious wavelengths with a therapeutic beam for treating a variety of complex skin conditions such as vitiligo, psoriasis, atopic dermatitis and ...
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 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 ...