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Sio Gene Therapies

New York City, NY, USA

Ownership: Private
Employees: ~12
Development stage: Dissolved

Overview

Sio reported positive interim safety and biomarker data from its ongoing Phase 1/2 study of AXO-AAV-GM1 in GM1 gangliosidosis, showing dose-dependent improvements and normalization of key biomarkers. No serious adverse events attributable to the therapy were observed. The company also advanced manufacturing capabilities for its AXO-Lenti-PD program in Parkinson's but did not reach pivotal or commercial stages before winding down operations.

Therapeutic areas: Rare DiseasesNeurology
Modalities: AAV-based gene therapy (Adeno-associated virus vectors)Lentiviral vector gene therapy
Industry: Biotech

Drug pipeline

9 assets

AXO-AAV-FTD

Preclinical

8 more assets in the full pipeline.

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Partnerships

Oxford BioMedica (manufacturing collaboration for AXO-Lenti-PD)UMass Chan Medical School (licensed rights to GM1 & GM2 programs)

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