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Find Drugs for Rare Diseases and Disorders in Preclinical Development
Details :
Novasep will be in charge of developing and manufacturing the two AAV vectors designed for the Sensorion OTOF-GT project and will supply Drug Product batches to support preclinical and clinical studies.
Details :
Proceeds will be used to advance Kamari's lead program KM023, a first-in-class oral TRPV3 inhibitor, for three rare genetic skin diseases, Olmsted syndrome, severe keratoderma and ichthyosis.
Details :
The collaboration aims to advance the innovative genetic medicine, which is being evaluated in preclinical clinical trial studies for the treatment of Rett Syndrome.
Details :
BA-102 is an investigational agent that aims to target and regulate nitric oxide (NO) levels in the brain and being developed for the treatment of PMS, a syndrome associated with ASD.
Details :
RVB-003 is based on ResVita Bio's continuous protein therapy platform, which utilizes genetically engineered bacteria applied topically, being developed for Netherton Syndrome.
Details :
Agreement will focus on the development of novel topical rapamycin (sirolimus) formulations as potential treatments for a number of rare and orphan diseases.
Details :
Under the terms of the agreement, Beacon will be responsible for the development and commercialization of AAV204 capsid for potential use for the treatment of retinal diseases.
Details :
The funding will be used to support the company’s lead program, AXV-101, an adeno-associated virus (AAV9)-based gene therapy targeting retinal dystrophy associated with Bardet-Biedl Syndrome.
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 collaboration aims to advanced the clinical development of AXV-101, which is being evaluated in the early-stage clinical trial studies for the treatment of Bardet-Biedl syndrome.
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