Pages
Leadership Team
- Edward M. Kaye, M.D.
- Jonathan Allan, J.D.
- Dawn Kalmar
- Huw M. Nash, Ph.D.
- Shamim Ruff
- Barry S. Ticho, M.D., Ph.D., FACC
- Stephen J. Tulipano, CPA, MBA
- Joan Wood
Board of Directors
- Edward M. Kaye, M.D.
- Adrian Krainer, Ph.D.
- Seth L. Harrison, M.D.
- Jennifer Burstein
- Arthur Levin, Ph.D.
- Garry E. Menzel, Ph.D.
- Julie Anne Smith
- Arthur Tzianabos, Ph.D.
Publications
- Antisense Oligonucleotide STK-002 Increases OPA1 in Retina and Improves Mitochondrial Function in Autosomal Dominant Optic Atrophy Cells
- 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)
- 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
- 24-Month Analysis of BUTTERFLY A Prospective, Observational Study to Investigate Seizures and Comorbidities in Children and Adolescents with Dravet Syndrome
- FALCON: A Prospective Natural History Study of Patients with OPA1-Autosomal Dominant Optic Atrophy (ADOA)
- 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)
- Mitochondrial Dysfunction in Autosomal Dominant Optic Atrophy (ADOA) Assessed in FALCON, A Non-interventional, Natural History Study
- Adaptive functioning and neurodevelopment in patients with Dravet syndrome: 12-month interim analysis of the BUTTERFLY observational study
- 24-Month Analysis of BUTTERFLY: A Prospective, Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children & Adolescents with Dravet Syndrome (DS)
- MONARCH & ADMIRAL: Phase 1/2a Studies in US & UK Investigating Safety and Drug Exposure of STK-001, an Antisense Oligonucleotide (ASO), in Children & Adolescents with Dravet Syndrome (DS)
- SWALLOWTAIL & LONGWING: Open-Label Extension (OLE) Studies for Children and Adolescents with Dravet Syndrome (DS) who Previously Participated in a Study of Antisense Oligonucleotide (ASO) STK-001
- Utilization of a Pharmacokinetic (PK) Model for STK-001 in Patients with Dravet Syndrome (DS) To Support Selection of Dosing Regimens in Clinic
- MONARCH and ADMIRAL Interim Analyses: Phase 1/2a Studies Investigating Safety and Drug Exposure of STK-001, an Antisense Oligonucleotide (ASO), in Children and Adolescents with Dravet Syndrome (DS)
- SWALLOWTAIL: An Open-Label Extension (OLE) Study for Children and Adolescents with Dravet Syndrome (DS) who Previously Participated in a Study of Antisense Oligonucleotide (ASO) STK-001
- Twelve-month Analysis of BUTTERFLY: An Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children and Adolescents with Dravet Syndrome (DS)
- Utilization of a Pharmacokinetic (PK) Model for STK-001 in Patients with Dravet Syndrome (DS) To Predict Pharmacologically Active Doses in Clinic
- Quantitative EEG Analysis in Patients with Dravet Syndrome (DS) Treated in the Phase 1/2a MONARCH and ADMIRAL Studies of STK-001, an Antisense Oligonucleotide (ASO)
- Interim results of adaptive functioning and neurodevelopment in BUTTERFLY – An observational study of children and adolescents with Dravet syndrome
- Interim Analyses: An Open-Label Study to Investigate the Safety and Pharmacokinetics of Single and Multiple Ascending Doses of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome
- 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)
- Models of Autosomal Dominant Optic Atrophy (ADOA) using iPSCs and Response to Targeted Augmentation of Nuclear Gene Output (TANGO) Antisense Oligonucleotides (ASOs) Treatment
- Interim Safety, PK, and CSF Exposure Data from the Phase 1/2a MONARCH Study of STK-001, an Antisense Oligonucleotide (ASO), in Children and Adolescents with Dravet Syndrome (DS)
- SWALLOWTAIL: An Open-Label Extension (OLE) Study for Patients with Dravet Syndrome (DS) who Previously Participated in Studies of STK-001
- A Pharmacokinetic (PK) Model for STK-001, an Antisense Oligonucleotide (ASO), Based on Data from Non-human Primates (NHP) Enables Dose Selection in Patients with Dravet Syndrome (DS)
- ADMIRAL: A UK Open-Label Study to Investigate the Safety and Pharmacokinetics (PK) of Multiple Ascending Doses of Antisense Oligonucleotide (ASO) STK-001 in Children and Adolescents with Dravet Syndrome
- BUTTERFLY, An Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children and Adolescents with Dravet Syndrome
- Antisense oligonucleotide mediated increase in OPA1 improves mitochondrial function in fibroblasts derived from patients with autosomal dominant optic atrophy (ADOA)
- Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children and Adolescents with Dravet Syndrome: Baseline Analysis of the BUTTERFLY Study
- Reducing the Time to Diagnosis and Increasing the Detection of Individuals with SCN1A-Related Disease Through a No-cost, Sponsored Epilepsy Genetic Testing Program
- Safety and Pharmacokinetics of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome: Single Ascending Dose Design for the Open-Label Phase 1/2a MONARCH Study
- Targeted Augmentation of Nuclear Gene Output (TANGO) of SCN1A Reduces Seizures and Rescues Parvalbumin Positive Interneuron Firing Frequency in a Mouse Model of Dravet Syndrome
- Targeted Augmentation of Nuclear Gene Output (TANGO) of SCN1A Reduces Seizures and Rescues Parvalbumin Positive Interneuron Firing Frequency in a Mouse Model of Dravet Syndrome – Oral Presentation
- Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome
- Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression
- Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for the treatment of autosomal dominant optic atrophy
- Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for treatment of autosomal dominant optic atrophy
- TANGO oligonucleotides for the treatment of Dravet Syndrome: Safety, biodistribution and pharmacology in the non-human primate
- Targeted Augmentation of Nuclear Gene Output (TANGO) of Scn1a Prevents SUDEP in a mouse model of Dravet Syndrome
