SpliceBio has announced the closing of a US$135 million Series B financing round to support the clinical advancement of its lead gene therapy candidate, SB-007, for Stargardt disease.
There are currently no approved treatments for the disease, which is caused by mutations in the ABCA4 gene and causes progressive vision loss. It typically appears before the age of 20.
SB-007, based on SpliceBio’s proprietary Protein Splicing platform, is designed to deliver a full-length, functional ABCA4 protein using a dual adeno-associated viral (AAV) vector approach.
SB-007 is the first dual AAV gene therapy for Stargardt disease cleared by the United States Food and Drug Administration to enter clinical development.
SB-007 is the first dual AAV gene therapy for Stargardt disease cleared by the United States Food and Drug Administration to enter clinical development.
The therapy also received regulatory clearance from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA). The company is currently conducting two studies: the interventional phase 1/2 ASTRA study and the observational POLARIS study.
“This financing marks a pivotal milestone for SpliceBio as we advance the clinical development of SB-007 and continue to expand our pipeline across ophthalmology, neurology and beyond,” said Dr Miquel Vila-Perello, CEO and Co-Founder of SpliceBio.
