SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, announced the closing of a $135 million Series B financing 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.
What SpliceBio does: SpliceBio is transforming and expanding the scope of diseases that can be treated with gene therapies by addressing a fundamental limitation of AAV vectors, namely 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 be accommodated within the AAV vectors. SpliceBio’s unique Protein Splicing platform utilizes a family of proprietary, engineered proteins called inteins, developed initially 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. After entering the cell, the DNA of each transgene is transcribed into messenger RNA and translated into protein. SpliceBio’s engineered inteins are designed to assemble then the full-length protein that is needed to treat the disease.
How the funding will be used: 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 approved by the Food and Drug Administration (FDA) for clinical development in Stargardt disease. SB-007 has also received regulatory clearance for clinical development from the UK Medicines and Healthcare products Regulatory Agency (MHRA).
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.
What is Stargardt disease? Stargardt disease is an inherited retinal disorder caused by mutations in the ABCA4 gene, resulting in progressive vision loss and eventual blindness, with no approved treatments currently available. And SB-007 was 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.
New board member: In connection with the funding, Daniela Begolo, Managing Director at EQT Life Sciences, Laia Crespo, Partner at Sanofi Ventures, and Carole Nuechterlein, Head of Roche Venture Fund, will join the SpliceBio Board of Directors.
KEY QUOTES:
“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. 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.”
Miquel Vila-Perelló, Ph.D., Chief Executive Officer and Co-Founder of SpliceBio
“We are proud to support SpliceBio, a pioneer among the next-generation of genetic medicine companies. Its Protein Splicing platform is designed to offer a novel solution to deliver large genes with AAV, one of the field’s most pressing challenges, and exemplifies our commitment to backing transformational science that can meaningfully benefit patients’ lives.”
Daniela Begolo, Managing Director at EQT Life Sciences
“With compelling data for its lead program, SB-007, and a highly differentiated platform, we are excited to support SpliceBio as it tackles a fundamental challenge for genetic medicines. By enabling the delivery of large and complex genes through its novel AAV vector Protein Splicing technology, SpliceBio has the potential to make a significant impact on the field of gene therapy and to deliver best-in-class therapies to patients.”
Laia Crespo, Partner at Sanofi Ventures
“We are impressed by the team’s strong execution, the momentum behind SB-007 in Stargardt disease, and the platform’s potential to unlock a new class of genetic medicines. We are proud to support SpliceBio at this pivotal stage of growth as they advance their lead program through clinical development and explore additional high-impact indications.”
Carole Nuechterlein, Head of Roche Venture Fund
