# Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy

> **NCT05858983** · PHASE1,PHASE2 · RECRUITING · sponsor: **Frontera Therapeutics** · enrollment: 9 (estimated)

## Conditions studied

- Biallelic RPE65 Mutation-associated Retinal Dystrophy

## Interventions

- **GENETIC:** FT-001 Low Dose
- **GENETIC:** FT-001 Mid Dose
- **GENETIC:** FT-001 High Dose

## Key facts

- **NCT ID:** NCT05858983
- **Lead sponsor:** Frontera Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2022-11-30
- **Primary completion:** 2025-11-30
- **Final completion:** 2029-11-30
- **Target enrollment:** 9 (ESTIMATED)
- **Last updated:** 2023-05-15


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT05858983

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT05858983, "Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT05858983. Licensed CC0.

---

*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*