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HuidaGene Therapeutics clinical trials

Source: ClinicalTrials.gov, U.S. National Library of Medicine — current trials in our China and USA registers with HuidaGene Therapeutics as lead sponsor. Synced 26 Sep 2026
5Current trials
4Recruiting now
0Drugs in these trials
2Trial registers
Maintained by Sreepriya Prasannan
MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 — Top 14 finalist
Compiled from ClinicalTrials.gov and CTIS registry records · Last verified · Editorial standards
At a glance

HuidaGene Therapeutics is the lead sponsor of 5 current clinical trials in our China and USA registers. 4 are recruiting patients now. The most common phase is Phase EARLY_PHASE1. The most-studied condition is Leber Congenital Amaurosis.

ClinicalTrials.gov trials by status

  • Recruiting480%
  • Active, closed120%

Trials by phase

  • Phase EARLY_PHASE1360%
  • Phase 1/2120%
  • Not applicable120%
01

Clinical trials sponsored by HuidaGene Therapeutics

5 on ClinicalTrials.gov
StudyPhaseStatusStartRegisters
A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)
NCT06615206 · ClinicalTrials.gov ↗ · MECP2 Duplication Syndrome
Not applicable Recruiting 2024-10 China
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
NCT05906953 · ClinicalTrials.gov ↗ · Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
Phase 1/2 Recruiting 2023-10 USA, China
An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT Disease
NCT07063251 · ClinicalTrials.gov ↗ · Stargardt Disease Type 1 (STGD1)
Phase EARLY_PHASE1 Recruiting 2025-08 China
CRISPR/cas13-medIated RNA TarGeting THerapy for the Treatment of Neovascular Age-related Macular Degeneration Investigator-initiated Trial (SIGHT-I)
NCT06031727 · ClinicalTrials.gov ↗ · Neovascular Age-related Macular Degeneration(nAMD)
Phase EARLY_PHASE1 Recruiting 2023-09 China
An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE)
NCT06594094 · ClinicalTrials.gov ↗ · Duchenne Muscular Dystrophin (DMD)
Phase EARLY_PHASE1 Active, closed 2024-11 China

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.

02

Conditions in HuidaGene Therapeutics trials

6 conditions
Leber Congenital Amaurosis1Inherited Retinal Diseases Caused by RPE65 Mutations1MECP2 Duplication Syndrome1Stargardt Disease Type 1 (STGD1)1Duchenne Muscular Dystrophin (DMD)1Neovascular Age-related Macular Degeneration(nAMD)1
03

By trial register

RegisterTrialsRecruiting
Clinical trials in China54
Clinical trials in USA11

A trial with sites in more than one country appears in each of those registers.

04

Other sponsors in Leber Congenital Amaurosis

SponsorTrials
Spark Therapeutics2
Laboratoires Thea1

Sponsors with the most current trials registered for this condition.

05

Questions about HuidaGene Therapeutics trials

How many clinical trials does HuidaGene Therapeutics sponsor?

HuidaGene Therapeutics is the lead sponsor of 5 current trials on ClinicalTrials.gov in our China and USA registers, as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.

Are HuidaGene Therapeutics trials recruiting?

Yes. 4 of its 5 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Where does this information come from?

Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.

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How this page is built

Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 26 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.