SpliceBio, a clinical-stage genetic medicines company pioneering protein splicing to address diseases caused by mutations in large genes, has closed a $135m Series B financing.
The funding will be used to advance the clinical development of SpliceBio’s lead gene therapy candidate, SB-007 for Stargardt disease, including the ongoing interventional phase 1/2 ASTRA study and the observational POLARIS study. SB-007 is the first dual adeno-associated viral (AAV) gene therapy cleared by the Food and Drug Administration (FDA) to enter clinical development for Stargardt disease. SB-007 has also received regulatory clearance for clinical development from the UK Medicines and Healthcare products Regulatory Agency (MHRA).
Stargardt disease is an inherited retinal disorder caused by mutations in the ABCA4 gene that leads to progressive vision loss and blindness, with no approved treatments available. SB-007 is designed to address the underlying genetic cause of the disease by producing a functional copy of the full-length ABCA4 protein with the potential to treat all patients, regardless of their specific ABCA4 mutation. The proceeds will also be used to accelerate SpliceBio’s pipeline of AAV gene therapy programs in ophthalmology, neurology, and other undisclosed indications that utilise the company’s proprietary Protein Splicing platform.
“This financing marks a pivotal milestone for SpliceBio as we advance the clinical development of SB-007 for Stargardt disease and continue to expand our pipeline across ophthalmology, neurology and beyond,” said Miquel Vila-Perelló, CEO and co-founder of SpliceBio.
“The support from such high-quality investors underscores the strength of our programs and our unique Protein Splicing platform and its potential to unlock gene therapies for diseases that remain untreatable today. We are building a company positioned to lead the next wave of genetic medicines.”
SpliceBio said it is redefining and expanding the scope of diseases that can be tackled with gene therapies by addressing a fundamental limitation of AAV vectors in their inability to deliver genes that exceed their limited packaging capacity of 4.7 kilobases. Many genetic disorders remain untreatable because the necessary gene is too large to fit into the AAV vectors.
SpliceBio’s protein splicing platform leverages the use of a family of proprietary, engineered proteins called inteins, originally developed at Princeton University. The company’s technology enables the splitting of the gene into two (or more) transgenes that are then delivered using dual AAV vectors. Once inside the cell, the DNA of each transgene is transcribed into messenger RNA and translated into protein. SpliceBio’s engineered inteins are designed to then assemble the full-length protein that is needed to treat the disease.
The financing was co-led by new investors EQT Life Sciences and Sanofi Ventures, with participation from Roche Venture Fund, as well as all existing investors: New Enterprise Associates, UCB Ventures, Ysios Capital, Gilde Healthcare, Novartis Venture Fund, and Asabys Partners.
Jim Cornall is editor of Deeptech Digest and publisher at Ayr Coastal Media. He is an award-winning writer, editor, photographer, broadcaster, designer and author. Contact Jim here.
