Company intelligence / Profile preview

Lysogene

Neuilly-sur-Seine, Francesofinnovapartners.com ↗

Ownership: Private
Development stage: Phase 3

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

Drug pipeline

5 assets

LYS-FXS01

Preclinical

4 more assets in the full pipeline.

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Partnerships

Previously partnered with Sarepta (terminated in 2021)

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