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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 :
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.
Details :
PPL-002 is an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, being evaluated for the treatment of Danon disease.
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 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 :
The collaboration aims to support the development of Axovia’s gene therapies, including AXV101, for ciliopathies, including Bardet-Biedl Syndrome, a condition with limited options or no cure.
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