SpliceBio wins first FDA clearance for protein splicing gene therapy

SpliceBio announced that the U.S. FDA has cleared the IND application for the Barcelona-based biotech’s lead program, SB-007, addressing the root genetic cause of Stargardt disease — marking the first ever IND clearance for a protein splicing gene therapy.

Stargardt disease, the most common form of inherited juvenile macular degeneration, is caused by mutations in the ABCA4 gene. The condition leads to the progressive loss of central vision due to damage to the region of the retina known as the macula. The disease has remained elusive to genetic medicines due to the large size of the ABCA4 gene.

SB-007 is a protein splicing dual AAV gene therapy that delivers the full-length ABCA4 gene and aims to restore expression of the native ABCA4 protein in the retina. It has been granted orphan drug designation by the FDA and the European Commission. SpliceBio plans to initiate enrolment in the phase 1/2 ASTRA study in the first half of 2025. ASTRA will evaluate the safety and efficacy of a single dose of SB-007 administered subretinally in patients with Stargardt disease.

SpiceBio made headlines in 2022 when it revealed the largest Series A round for a Spanish biotech company. The oversubscribed $52 million (€50 million) financing was co-led by UCB Ventures and existing shareholder Ysios Capital and joined by new investors New Enterprise Associates, Gilde Healthcare, Novartis Venture Fund, and Asabys Partners. The following year, biotech inked a $216 million collaboration with Roche’s Spark Therapeutics to utilize SpliceBio’s proprietary protein splicing platform to develop a gene therapy for an undisclosed inherited retinal disease.