scAVV9.CB.CLN6
/ Amicus
- LARVOL DELTA
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October 03, 2025
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
(clinicaltrials.gov)
- P=N/A | N=10 | Active, not recruiting | Sponsor: Emily de los Reyes | Trial completion date: Oct 2024 ➔ Dec 2027 | Trial primary completion date: Oct 2024 ➔ Dec 2026
Trial completion date • Trial primary completion date • CNS Disorders • Gene Therapies
February 06, 2023
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
(clinicaltrials.gov)
- P=N/A | N=10 | Active, not recruiting | Sponsor: Amicus Therapeutics | Trial completion date: Jan 2035 ➔ Oct 2024 | Trial primary completion date: Jan 2035 ➔ Oct 2024
Trial completion date • Trial primary completion date • CNS Disorders • Gene Therapies
March 06, 2021
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice.
(PubMed, Mol Ther Methods Clin Dev)
- "Lastly, scAAV9.CB.CLN6 treatment partially preserved visual acuity in Cln6 mice as measured by optokinetic response. Taken together, we report the first instance of CSF-delivered viral gene reaching and rescuing pathology in both the brain parenchyma and retinal neurons, thereby partially slowing visual deterioration."
Journal • CNS Disorders • Gene Therapies • Immunology • Ophthalmology • Pediatrics
December 29, 2020
An altered secretome is an early marker of the pathogenesis of CLN6 Batten disease.
(PubMed, J Neurochem)
- "This work suggests the secretome plays a role in CLN6 pathogenesis and highlights its potential use as an in vitro model. Proteomic changes present a list of candidate biomarkers for monitoring disease and assessing potential therapeutics in future studies."
Journal • CNS Disorders • Gene Therapies • Immunology • Inflammation • CTSL
May 31, 2019
Batten CLN6 Gene Therapy
(clinicaltrials.gov)
- P1/2; N=12; Active, not recruiting; Sponsor: Emily de los Reyes; Trial completion date: Mar 2019 ➔ Mar 2023; Trial primary completion date: Mar 2019 ➔ Mar 2022
Clinical • Trial completion date • Trial primary completion date
January 23, 2019
Batten CLN6 Gene Therapy
(clinicaltrials.gov)
- P1/2; N=12; Active, not recruiting; Sponsor: Nationwide Children's Hospital; Recruiting ➔ Active, not recruiting
Clinical • Enrollment closed
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