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Pharmaceuticals"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Mereo BioPharma Group","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx and Mereo BioPharma Announce Interim Phase 2 Data from Phase 2\/3 Orbit Study Demonstrating Setrusumab (UX143) Significantly Reduced Fracture Rates in Patients with Osteogenesis Imperfecta (OI)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quoin Pharmaceuticals Announces Additional Positive Clinical Data from Open-Label, Single-Arm Clinical Trial in Netherton Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Quoin Pharmaceuticals"}]
Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development
Investigational data from long-term, follow-up studies showed that olipudase alfa provided sustained improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes over time in patients with ASMD.
The STAGED-PKD study was stopped for futility following an independent analysis of the annualized rate of change in total kidney volume (TKV) in patients receiving venglustat compared to placebo.
QRX003 is a once-daily topical lotion comprised of a broad-spectrum serine protease inhibitor, formulated with the proprietary invisicare® delivery technology. It is being investigated for treating netherton syndrome.
UX143 (setrusumab) is a fully human monoclonal antibody that inhibits sclerostin, a protein that acts on a key bone-signaling pathway that inhibits the maturation and activity of bone-forming cells in Osteogenesis Imperfecta.
REN001 (mavodelpar) is a potent and selective peroxisome PPAR delta agonist currently in clinical development for two rare genetic mitochondrial diseases that typically present with myopathy and PMM and LC-FAOD.
The net proceeds will advance clinical trials for Neurophth's core products, enhancing the firm's R&D capabilities and expanding its pipeline, including NFS-01 (esonadogene imvoparvovec), a rAAV2-ND4 gene therapy being developed to treat Leber's hereditary optic neuropathy.
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