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GeneCradle

Beijing, Beijing, Chinaen.bj-genecradle.com ↗

Ownership: Private
Development stage: Phase 3

Overview

GeneCradle is a leading Chinese biotech specializing in AAV-mediated in vivo gene therapies targeting rare neuromuscular and genetic metabolic diseases. Its most advanced programs are in Phase 1/2 clinical trials, including GC301 for infantile-onset Pompe disease, which has demonstrated safety and efficacy over more than 12 months of follow-up. The company also has ongoing research into SMA intrathecal therapy and biomarker development.

Therapeutic areas: Metabolic DisordersRare DiseasesGastroenterologyNeurology
Modalities: AAV-based gene therapy (Adeno-associated virus vectors)
Industry: Biotech

Drug pipeline

8 assets

gc301

7 more assets in the full pipeline.

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