Biotechnology company AAVantgarde Bio has raised $141 million to advance its clinical programmes in inherited retinal disorders (IRD) Stargardt disease and Usher 1B syndrome. The investment follows a successful Series B financing round, led by Schroders Capital and existing investors Atlas Venture and Forbion. AAVantgarde aims to use the proceeds to complete the clinical study of AAVB-039… Read More
SpliceBio has dosed the first patient in the Phase I/II ASTRA study of SB-007, a dual adeno-associated viral (AAV) vector, for the treatment of Stargardt disease. Stargardt disease is an inherited retinal disorder caused by mutations in the ABCA4 gene. It is the most common form of juvenile macular degeneration,… Read More
Researchers have developed a gene editing therapy that could potentially treat Stargardt disease, the most common form of inherited macular degeneration. The study, published in Nature Medicine, demonstrates the therapeutic use of a precision gene editing technology called base editing. … Read More
The US Food & Drug Administration (FDA) has cleared an investigational new drug (IND) application for SpliceBio’s lead programme SB-007, a clinical-stage therapeutic addressing the root genetic cause of Stargardt disease with the potential to treat all patients across all ABCA4 mutations. The SpliceBio study uses protein splicing to address… Read More
