Biotech firm SpliceBio, which is exploiting protein splicing to develop the next generation of gene therapies, has completed a £42m fundraise.
Adeno-associated viruses (AAV) are the gene therapy vector of choice for the treatment of genetic diseases. However, their small packaging capacity is a major challenge for the development of novel gene therapies.
SpliceBio’s protein splicing platform aims to address this major limitation to enable the efficient delivery of large genes using AAV vectors.
The platform is based on technology developed in the Muir Lab at Princeton University.
The proceeds from the financing, the largest Series A round for a Spanish biotech company, will enable SpliceBio to build a pipeline of protein splicing gene therapy programmes, while advancing the lead program in Stargardt disease to the clinic.
Stargardt disease is the most common form of juvenile macular dystrophy affecting more than 80,000 people in US and EU.
The disease is caused by a loss of function mutation in the ABCA4 gene, which at 6.8 kb is too large for single AAV vectors.
The company will focus its efforts on ophthalmology as well as other disease areas of significant unmet patient need. The platform has been validated in several other organs beyond the retina.
CEO Miquel Vila-Perelló said: “We are very pleased to attract this outstanding syndicate of institutional and corporate investors which validates our approach to developing next generation gene therapies. I am excited to lead an exceptional team as we continue to build our platform and advance our pipeline of gene therapy programs into the clinic.”
SpliceBio‘s proprietary platform enables efficient delivery of large genes with adeno-associated vectors (AAV), overcoming the most fundamental challenge in the quest to curing a broad range of genetic diseases.