SpliceBio, a biotechnology company exploiting protein splicing to develop next generation gene therapies, today announced the completion of an oversubscribed €50 million series A financing. The financing was co-led by UCB Ventures and existing shareholder Ysios Capital and joined by new investors New Enterprise Associates (NEA), Gilde Healthcare, Novartis Venture Fund, and existing shareholder Asabys Partners. The Company was seeded in 2020 by Ysios Capital and Asabys Partners.
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 after more than 20 years of pioneering intein and protein engineering research. In this novel approach, engineered inteins catalyze highly efficient protein trans-splicing to reconstitute the desired full-length therapeutic protein in vivo.
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 programs, 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.
Miquel Vila-Perelló, PhD, Co-Founder and Chief Executive Officer of SpliceBio, 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.”
Following the closing of the financing, the Board of SpliceBio chaired by Jean Philippe Combal will include: Erica Whittaker, UCB Ventures; Joël Jean-Mairet, Ysios Capital; Ed Mathers, NEA; Arthur Franken, Gilde Healthcare; Beat Steffen, Novartis Venture Fund; and Miquel Vila- Perelló, CEO.
Erica Whittaker, Vice President and Head of UCB Ventures, stated: “We are delighted to support SpliceBio in the development of its innovative platform to create treatments for patients suffering from genetic diseases not currently addressable by existing gene therapy approaches.”
Ed Mathers, General Partner at NEA, commented: “We are very pleased to back this team, building on the founders’ early-stage research at Princeton’s Muir Laboratory to develop SpliceBio into a world leading gene therapy player. We believe SpliceBio’s innovative approach to maximizing the capacity of AAV vectors has the potential to make a meaningful impact in the delivery of much needed gene therapies, and we look forward to supporting the Company through its next stages of growth.”