Antisense Oligonucleotide STK-002 Increases OPA1 in Retina and Improves Mitochondrial Function in Autosomal Dominant Optic Atrophy Cells September 12, 2024 Source Nucleic Acid Therapeutics
Adaptive functioning and neurodevelopment in patients with Dravet syndrome: 12-month interim analysis of the BUTTERFLY observational study January 13, 2024 Source Epilepsy & Behavior
Interim results of adaptive functioning and neurodevelopment in BUTTERFLY – An observational study of children and adolescents with Dravet syndrome November 1, 2022 Source Epilepsy & Behavior
Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome August 26, 2020 Source Science Translational Medicine
Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression July 9, 2020 Source Nature Communications
SWALLOWTAIL and LONGWING Open-label Extension Studies for Children and Adolescents with Dravet Syndrome who Previously Participated in a Study of Antisense Oligonucleotide Zorevunersen (STK-001) September 10, 2024 Source EEC 2024
MONARCH and ADMIRAL: Open-label, Phase 1/2a studies in USA and UK investigating safety, drug exposure, and clinical effect of zorevunersen(STK-001), an antisense oligonucleotide, in children and adolescents with Dravet syndrome September 10, 2024 Source EEC 2024
24-Month Analysis of BUTTERFLY A Prospective, Observational Study to Investigate Seizures and Comorbidities in Children and Adolescents with Dravet Syndrome September 10, 2024 Source EEC 2024
Utilization of a Pharmacokinetic (PK) Model for STK-001 in Patients with Dravet Syndrome (DS) To Support Selection of Dosing Regimens in Clinic December 1, 2023 Source AES 2023
FALCON: A Prospective Natural History Study of Patients with OPA1-Autosomal Dominant Optic Atrophy (ADOA) May 6, 2024 Source ARVO 2024
OSPREY: An Open-label Study to Investigate Safety, Tolerability, and Exposure of the Antisense Oligonucleotide (ASO) STK-002 in Patients with OPA1 Autosomal Dominant Optic Atrophy (ADOA) May 6, 2024 Source ARVO 2024
Mitochondrial Dysfunction in Autosomal Dominant Optic Atrophy (ADOA) Assessed in FALCON, A Non-interventional, Natural History Study May 6, 2024 Source ARVO 2024
STK-002, an Antisense Oligonucleotide (ASO) for the Treatment of Autosomal Dominant Optic Atrophy (ADOA), is Taken Up by Retinal Ganglion Cells (RGCs) and Upregulates OPA-1 Protein Expression After Intravitreal Administration to Non-human Primates (NHPs) May 16, 2022 Source ASGCT 2022
Models of Autosomal Dominant Optic Atrophy (ADOA) using iPSCs and Response to Targeted Augmentation of Nuclear Gene Output (TANGO) Antisense Oligonucleotides (ASOs) Treatment May 2, 2022 Source ARVO 2022
Antisense oligonucleotide mediated increase in OPA1 improves mitochondrial function in fibroblasts derived from patients with autosomal dominant optic atrophy (ADOA) May 4, 2021 Source ARVO 2021
Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for the treatment of autosomal dominant optic atrophy June 12, 2020 Source ARVO 2020
Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for treatment of autosomal dominant optic atrophy May 12, 2020 Source ASGCT 2020