SpliceBio Raises $57 Million to Advance Protein Splicing Platform and Expand Gene Therapy Pipeline

Spanish biotech SpliceBio completed an oversubscribed $57 million (€50 million) series A financing to advance its protein splicing platform that enables efficient delivery of large genes with adeno-associated vectors.

Photo: Miquel Vila-Perelló, co-founder and CEO of SpliceBio

UCB Ventures and existing shareholder Ysios Capital co-led the financing with participation by new investors New Enterprise Associates, Gilde Healthcare, Novartis Venture Fund, and existing shareholder Asabys Partners. Ysios Capital and Asabys Partners provided seed funding for the company in 2020.

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. Inteins are auto-processing domains found in organisms from all domains of life. These proteins carry out a process known as protein splicing, which is a multi-step biochemical reaction comprised of both the cleavage and formation of peptide bonds. 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 for currently untreatable genetic diseases, 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 United States and European Union. 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.

“We are very pleased to attract this outstanding syndicate of institutional and corporate investors which validates our approach to developing next generation gene therapies,” said Miquel Vila-Perelló, co-founder and CEO of SpliceBio.

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.

Author: Rare Daily Staff