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Genosera

Ownership: Private
Development stage: Preclinical

Overview

Genosera is developing next-generation bicistronic AAV-based gene therapies for rare genetic muscle and metabolic diseases. All pipeline programs are currently in preclinical development. The company’s lead program targets GNE myopathy with a novel two-gene approach designed to both halt progression and reverse existing muscle damage. No clinical results have been reported yet.

Therapeutic areas: Metabolic DisordersRare DiseasesNeurology
Modalities: Gene therapyAdeno-associated virus (AAV) gene therapyBicistronic gene therapy (two-gene AAV)
Industry: Biotech

Drug pipeline

6 assets

GA-002

Preclinical

5 more assets in the full pipeline.

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Partnerships

Solve GNE LLC – sponsored research agreement to develop bicistronic AAV treatment for GNE Myopathy/HIBM

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