Refine by
Genetic Disease Technology
8 companies found
Technologybased inBasel, SWITZERLAND
Aurealis Therapeutics, a Swiss-Finnish private biopharmaceutical company, is now entering the clinical trial phase with its patented three-in-one combination product AUP-16 for chronic non-healing wounds and other regenerative diseases. The ...
Technologybased inParis, FRANCE
Biogemma is a European plant biotechnology company founded in 1997 by seed companies and French field crop producers. Its original mission was to create new genetic variability via GM traits to be exploited by its shareholding companies. With the ...
Technologybased inWayne, PENNSYLVANIA (USA)
Palvella Therapeutics was founded to serve individuals suffering from rare diseases by developing safe and efficacious therapies that will dramatically enhance their quality of life. We focus on developing therapies that specifically target the root ...
QTORIN™ rapamycin 3.9%is a novel targeted topical therapy being studied for Gorlin syndrome. Palvella is enrolling a phase 2b clinical study (CODY) for people with Gorlin ...
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 ...
Aegle’s founder Dr. Evangelos Badiavas and his team have spent decades researching clinical applications of mesenchymal stem cells (“MSCs”) in severe dermatological disorders, including conducting clinical trials in burns and ...
Technologybased 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) — ...
Cystic fibrosis (CF) is a rare, chronic and life-shortening genetic disease. It’s a progressive, multi-system disease that affects the lungs, liver, gastrointestinal tract, pancreas, sinuses, ...
Technologybased inAustin, TEXAS (USA)
Natera is a global leader in cell-free DNA (cfDNA) testing, dedicated to oncology, women’s health, and organ health. We aim to make personalized genetic testing and diagnostics part of the standard of care to protect health and inform earlier, more ...
Horizon carrier screening helps couples determine the risk of passing on serious genetic conditions to their child. It can be performed either preconception (ideally) or during ...
Technologybased inChesterfield, MISSOURI (USA)
Partek bioinformatics software delivers powerful statistical and visualization tools in an easy-to-use interface. Researchers of all skill levels are empowered to explore genomic data quicker and easier than ever before. We turn data into discovery. ...
Technologybased inBoston, MASSACHUSETTS (USA)
Neoclease is at the forefront of advancing gene editing technologies with a mission to transform healthcare by curing genetic diseases rather than just treating them. Utilizing a bespoke approach, the company develops tailored nucleases designed to ...
These enzymes are meticulously crafted using proprietary algorithms powered by state-of-the-art GPT neural networks, which are capable of redesigning nucleases for specific genetic targets. This approach allows Neoclease to tailor ...