scAVV9.CB.CLN6 / Amicus - LARVOL DELTA

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

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

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

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

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

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