Company intelligence / Profile preview

Design House

Ownership
Private
Development stage
Commercial
01

Overview

Advancing novel small molecule therapeutics targeting the underlying causes of serious genetic diseases using their proprietary GeneTAC® platform. Initial focus on monogenic repeat expansion disorders with urgent unmet needs. Clinical results are not yet available or published.

Therapeutic areas
OphthalmologyNeurology
Modalities
Small moleculeGene-targeted chimera therapy
Industry
Biotech

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