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CRD-TMH-001

Development stage: Phase 1
Lead developer: Cure Rare Disease
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration: Intravenous

Overview

CRD-TMH-001 is a CRISPR-based gene therapy designed to treat a rare mutation of Duchenne muscular dystrophy (DMD) affecting the muscle promoter and exon 1 of the dystrophin gene. The therapy uses CRISPR transactivation technology to upregulate an alternate form (brain isoform) of the dystrophin protein with the goal of stabilizing or potentially reversing symptom progression of DMD. It is administered as a single intravenous infusion using an AAV9 viral vector for delivery.

Targets

DMD (Dystrophin)

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