Company intelligence / Profile preview
Myrtelle
Wakefield, Massachusetts, United Statesmyrtellegtx.com ↗
- Ownership
- Private
- Employees
- ~50
- Development stage
- Other
01
Overview
Myrtelle’s lead candidate, rAAV-Olig001-ASPA, has shown favorable safety and tolerability in a Phase 1/2 trial with eight patients dosed. Early results indicate significant improvements in functional scales and brain white matter content. The company has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA as well as orphan designation from EMA. Initial efficacy data are encouraging for further development of this first-in-class approach targeting oligodendrocytes in children with Canavan disease.
- Therapeutic areas
- Rare DiseasesNeurology
- Modalities
- Gene therapy (recombinant adeno-associated virus vectors / rAAV)
- Industry
- Biotech
02
Drug pipeline
3 assets2 more assets in the full pipeline.
03
Partnerships
rAAVen Therapeutics – partnership to develop novel AAV vectors for CNS/myelin-based disordersRescue Hearing Inc. – exclusive worldwide licensing agreement to develop a novel gene therapy program targeting DFNB8 genetic hearing loss
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