SpliceBio
Private Company
Total funding raised: $245M
Overview
SpliceBio is a private, clinical-stage biotech leveraging its novel Protein Splicing platform to overcome the payload limitations of standard AAV vectors, enabling the delivery of large genes for gene therapy. The company's lead asset, SB-007 for Stargardt disease, is in clinical development, supported by over $200 million in venture funding from top-tier investors. With a seasoned leadership team and a clear focus on inherited retinal diseases and beyond, SpliceBio aims to create a new class of genetic medicines for patients with limited treatment options.
Technology Platform
Proprietary Protein Splicing platform using engineered inteins to enable dual-vector AAV delivery of large genes, reconstituting full-length functional proteins in target cells.
Funding History
3Opportunities
Risk Factors
Competitive Landscape
SpliceBio competes in the niche of overcoming AAV payload limits, facing approaches like dual-vector trans-splicing (e.g., GenSight Biologics), oversized capsids, and non-viral delivery from companies like Ring Therapeutics or Generation Bio. Its specific intein-mediated protein splicing approach is a differentiated technological strategy.