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Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"University College Cork","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Agreement","leadProduct":"Sirolimus","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Quoin Pharmaceuticals \/ University College Cork","highestDevelopmentStatusID":"4","companyTruncated":"Quoin Pharmaceuticals \/ University College Cork"}]
Find Drugs for Rare Diseases and Disorders in Preclinical Development
Details :
MDL-101 is an experimental, epigenetic editing therapy, which is under investigation in the early-stage studies for the treatment of LAMA2-congenital muscular dystrophy.
Details :
The partnership aims to manufacture AAV using suspension AAV Curator Platform to manufacture clinical grade AAV for HUB-101 for the treatment of Leber Congenital Amaurosis 16.
Details :
MDL-101 is an experimental, epigenetic editing therapy, which is under investigation in the early-stage clinical trial studies for the treatment of LAMA2-congenital muscular dystrophy.
Details :
The Company intends to use the proceeds for research and clinical development of its pipeline includling, AVCN583601 (Cannabidiol), for the treatment of epidermolysis bullosa.
Details :
The collaboartion aims to develop innovative novel plasma-derived therapy formulations for the treatment of rare conditions. Nanoform will provide non-GMP nanomaterial to Takeda for in vivo studies.
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Agreement will focus on the development of novel topical rapamycin (sirolimus) formulations as potential treatments for a number of rare and orphan diseases.
Details :
Niagen (nicotinamide riboside chloride) is an oral supplement which significantly increases NAD+ levels, it is being investigated in Ataxia Telangiectasia.
Details :
MDL-101 is an experimental, epigenetic editing therapy, which is under investigation in the early-stage clinical trial studies for the treatment of LAMA2-congenital muscular dystrophy.
Details :
MIV-711 is a potent and selective inhibitor of cathepsin K, the principal protease involved in breaking down collagen in bone and cartilage. It is being evaluated for Legg-Calvé-Perthes Disease.
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