Company intelligence / Profile preview
Lysogene
Neuilly-sur-Seine, Francelysogene.com ↗
- Ownership
- Private
- Development stage
- Other
01
Overview
Lead candidate LYS-SAF302 failed a Phase 2/3 trial for Sanfilippo syndrome in November 2022. Among the six youngest participants (children younger than 30 months), there was a statistically significant improvement in cognitive function compared to natural history two years after dosing, but no treatment effect was observed among the remaining 12 participants in the main cohort[5].
- Therapeutic areas
- Rare DiseasesNeurology
- Modalities
- AAV gene therapy
- Industry
- Biotech
02
Drug pipeline
5 assets4 more assets in the full pipeline.
03
Partnerships
Previously partnered with Sarepta (terminated in 2021)
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