Company intelligence / Profile preview
SpliceBio
Barcelona, Catalonia, Spainsplice.bio ↗
- Ownership
- Private
- Development stage
- Other
01
Overview
SpliceBio has initiated a Phase 1/2 clinical trial for its lead candidate SB‑007, a dual-AAV gene therapy targeting Stargardt disease. The company’s proprietary protein splicing platform enables delivery of large genes that exceed the packaging capacity of standard AAV vectors. This approach addresses key limitations in current gene therapy technologies and has shown preclinical validation across multiple organs beyond the retina. The company aims to expand its pipeline into other currently untreatable genetic diseases[1][2][5].
- Therapeutic areas
- Ophthalmology
- Modalities
- Gene therapy (AAV-based)Protein splicing
- Industry
- Biotech
02
Drug pipeline
4 assets3 more assets in the full pipeline.
03
Partnerships
Spark Therapeutics – collaboration on treatments for inherited retinal diseases[5].
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