We are focusing our initial efforts on a rare, debilitating ciliopathy known as BBS.
BBS is a multi-faceted, life-limiting disease in which patients experience early-onset blindness, learning disabilities, severe obesity, kidney failure and life-threatening comorbidities.
Building on the most comprehensive BBS program started at University College London (UCL) over 25 years ago, Axovia is working to advance gene therapy programs that have already shown early promise in treating some of the most severe aspects of BBS, such as early onset blindness and severe obesity.
Led by BBS & Gene Therapy Pioneers
Axovia was co-founded by Professor Phil Beales, former head of Genetics and Genomic Medicine at University College London (UCL), UK, who has led the most comprehensive research and care efforts for BBS patients in the world .
Dr. Victor Hernandez is a Lecturer at Brunel University and co-founder of Axovia Therapeutics. He has been leading the development of the gene therapy solutions that Axovia is using to treat BBS patients and tested their preclinical efficacy.
Axovia is a majority owned subsidiary of Jaguar Gene Therapy, which is backed by Deerfield Management Company, and is also partially funded by University College London Technology Fund.