SpliceBio is a clinical-stage genetic medicines company pioneering a Protein Splicing platform that uses engineered inteins to enable delivery of large genes via dual AAV vectors. Its lead program, SB-007, is a dual AAV gene therapy designed to restore full-length ABCA4 protein expression for Stargardt disease. SB-007 received FDA IND clearance in December 2024, has Orphan Drug Designation from both the FDA and the European Commission, and obtained MHRA clearance to enter clinical development. The company is currently running the Phase 1/2 ASTRA interventional study (first patient dosed March 2025) and the POLARIS observational natural history study. Proceeds from the financing will support continued clinical development of SB-007 and accelerate SpliceBio's broader pipeline of AAV gene therapy programs in ophthalmology, neurology, and other undisclosed indications. SpliceBio leverages technology developed in the Muir Lab at Princeton University and is based in Barcelona, Spain. SpliceBio exploits Protein Splicing to develop the next generation of gene therapies and operates a proprietary platform that enables efficient delivery of large genes with adeno-associated vectors (AAV). The platform is designed to overcome AAV cargo-size limits by using intein and protein engineering approaches developed over more than 20 years in the Muir Lab at Princeton University. Led by CEO Miquel Vila-Perelló, the company intends to use its technology to build a pipeline of Protein Splicing gene therapy programs. Its lead program in Stargardt disease is being advanced toward the clinic. Financially, SpliceBio raised €50M in a Series A financing to support pipeline development and clinical advancement. The company was seeded in 2020 by Ysios Capital and Asabys Partners. Splice Bio, formerly ProteoDesign, develops intein-based technologies for gene therapy aimed at treating genetic diseases. Its platform, based on research carried out at Princeton by the company's cofounders, is designed to overcome key limitations of adeno-associated viral (AAV) vectors. The technology could enable treatments for diseases caused by mutations in large genes and broaden the range of organs that can be targeted. The company announced a new financing round to advance the development of these therapies. Proceeds will be used to develop new therapies for genetic diseases. Splice Bio also refreshed its brand to reflect its renewed focus on gene therapy. ProteoDesign is a biotechnology spin-out from Princeton University that develops personalized cancer immunotherapies. The company says it has a novel in-house protein-engineering platform discovered after more than 20 years of pioneering research. ProteoDesign announced the close of a €1.7 million funding round led by venture firm Caixa Capital Risc, with participation from a business angel. Caixa Capital Risc was already an existing investor in the company's shareholder base. The proceeds will be used to advance ProteoDesign's immunotherapy candidates through preclinical development.

Total raised

$194M

Funding rounds

4

Latest round

Series B

Latest activity

Jun 2025