ART001: Clinical Safety and Tolerability Data for ATTR Amyloidosis Gene Therapy

An investigator-initiated trial of 10 ATTR amyloidosis patients treated with the gene editing drug ART001. The dataset, authored by Yasi Jiang and last updated in April 2026, includes safety, pharmacokinetic, pharmacodynamic, and efficacy results, showing TTR protein reductions of 84% to 92% at 72 weeks. The trial was funded by Accuredit Therapeutics and registered with ChiCTR (ChiCTR2400081216).

Use Cases

• Analyze dose-response relationships based on drug doses ranging from 0.05 mg/kg to 1.0 mg/kg.
• Model long-term protein reduction efficacy based on TTR knock-down percentages sustained over 72 weeks.
• Assess clinical safety profiles based on the reported absence of infusion-related reactions and serious adverse events.

Strengths

• Includes specific efficacy results, with TTR protein reductions averaging 84% and 92% at high doses.
• Reports a clear safety outcome: no infusion-related reactions, serious adverse events, or serious adverse reactions were observed.
• Data is openly shared under a CC-BY-4.0 license.

Limitations

• Row count is unknown, which may limit suitability assessment.
• Column-level documentation is absent; field semantics must be inferred after download.
• The dataset is very small at 71.7 KB, indicating limited scope.

Provenance

Source

figshare, author Yasi Jiang.

Collection Method

Data originates from an investigator-initiated trial (IIT) involving 10 patients.

Time Range

Trial registered on 26th February 2024; data reflects outcomes up to at least 72 weeks post-treatment.

Freshness

Last updated 2026-04-15 04:36:46; freshness should be verified.

Geography

null