Events and Presentations Events and Presentations Upcoming Events There are no upcoming events available at this time. Past Events April 15, 2021 at 12:45 PM EDT 20th Annual Needham Virtual Healthcare Conference Click here for webcast March 31, 2021 at 2:00 PM EDT Stifel 3rd Annual CNS Day Click here for webcast March 11, 2021 at 3:35 PM EST Barclays Global Healthcare Conference Click here for webcast January 11, 2021 at 4:30 PM EST 39th Annual J.P. Morgan Healthcare Conference Click here for webcast December 10, 2020 at 12:45 PM EST Needham Virtual Epilepsy & Pain - Specialty CNS Therapeutics Conference Click here for webcast Corporate Presentation Corporate Presentation – May 2021 Key Publications August 26, 2020 Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome Source Science Translational Medicine July 9, 2020 Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression Source Nature Communications Genetic Epilepsies Presentations December 4, 2020 Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children and Adolescents with Dravet Syndrome: Baseline Analysis of the BUTTERFLY Study Source AES 2020 December 4, 2020 Reducing the Time to Diagnosis and Increasing the Detection of Individuals with SCN1A-Related Disease Through a No-cost, Sponsored Epilepsy Genetic Testing Program Source AES 2020 December 4, 2020 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 Source AES 2020 December 4, 2020 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 Source AES 2020 December 4, 2020 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 Source AES 2020 December 8, 2019 TANGO oligonucleotides for the treatment of Dravet Syndrome: Safety, biodistribution and pharmacology in the non-human primate Source AES 2019 December 7, 2019 Targeted Augmentation of Nuclear Gene Output (TANGO) of Scn1a Prevents SUDEP in a mouse model of Dravet Syndrome Source AES 2019 Neuro-Ophthalmology Presentations May 4, 2021 Antisense oligonucleotide mediated increase in OPA1 improves mitochondrial function in fibroblasts derived from patients with autosomal dominant optic atrophy (ADOA) June 12, 2020 Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for the treatment of autosomal dominant optic atrophy Source ARVO 2020 May 12, 2020 POSTER: Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for treatment of autosomal dominant optic atrophy Source ASGCT 2020 Toolkit Print Page Email Alerts Contact IR FAQ
Events and Presentations Upcoming Events There are no upcoming events available at this time. Past Events April 15, 2021 at 12:45 PM EDT 20th Annual Needham Virtual Healthcare Conference Click here for webcast March 31, 2021 at 2:00 PM EDT Stifel 3rd Annual CNS Day Click here for webcast March 11, 2021 at 3:35 PM EST Barclays Global Healthcare Conference Click here for webcast January 11, 2021 at 4:30 PM EST 39th Annual J.P. Morgan Healthcare Conference Click here for webcast December 10, 2020 at 12:45 PM EST Needham Virtual Epilepsy & Pain - Specialty CNS Therapeutics Conference Click here for webcast Corporate Presentation Corporate Presentation – May 2021 Key Publications August 26, 2020 Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome Source Science Translational Medicine July 9, 2020 Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression Source Nature Communications Genetic Epilepsies Presentations December 4, 2020 Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children and Adolescents with Dravet Syndrome: Baseline Analysis of the BUTTERFLY Study Source AES 2020 December 4, 2020 Reducing the Time to Diagnosis and Increasing the Detection of Individuals with SCN1A-Related Disease Through a No-cost, Sponsored Epilepsy Genetic Testing Program Source AES 2020 December 4, 2020 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 Source AES 2020 December 4, 2020 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 Source AES 2020 December 4, 2020 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 Source AES 2020 December 8, 2019 TANGO oligonucleotides for the treatment of Dravet Syndrome: Safety, biodistribution and pharmacology in the non-human primate Source AES 2019 December 7, 2019 Targeted Augmentation of Nuclear Gene Output (TANGO) of Scn1a Prevents SUDEP in a mouse model of Dravet Syndrome Source AES 2019 Neuro-Ophthalmology Presentations May 4, 2021 Antisense oligonucleotide mediated increase in OPA1 improves mitochondrial function in fibroblasts derived from patients with autosomal dominant optic atrophy (ADOA) June 12, 2020 Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for the treatment of autosomal dominant optic atrophy Source ARVO 2020 May 12, 2020 POSTER: Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for treatment of autosomal dominant optic atrophy Source ASGCT 2020
April 15, 2021 at 12:45 PM EDT 20th Annual Needham Virtual Healthcare Conference Click here for webcast
December 10, 2020 at 12:45 PM EST Needham Virtual Epilepsy & Pain - Specialty CNS Therapeutics Conference Click here for webcast
August 26, 2020 Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome Source Science Translational Medicine
July 9, 2020 Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression Source Nature Communications
December 4, 2020 Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children and Adolescents with Dravet Syndrome: Baseline Analysis of the BUTTERFLY Study Source AES 2020
December 4, 2020 Reducing the Time to Diagnosis and Increasing the Detection of Individuals with SCN1A-Related Disease Through a No-cost, Sponsored Epilepsy Genetic Testing Program Source AES 2020
December 4, 2020 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 Source AES 2020
December 4, 2020 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 Source AES 2020
December 4, 2020 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 Source AES 2020
December 8, 2019 TANGO oligonucleotides for the treatment of Dravet Syndrome: Safety, biodistribution and pharmacology in the non-human primate Source AES 2019
December 7, 2019 Targeted Augmentation of Nuclear Gene Output (TANGO) of Scn1a Prevents SUDEP in a mouse model of Dravet Syndrome Source AES 2019
May 4, 2021 Antisense oligonucleotide mediated increase in OPA1 improves mitochondrial function in fibroblasts derived from patients with autosomal dominant optic atrophy (ADOA)
June 12, 2020 Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for the treatment of autosomal dominant optic atrophy Source ARVO 2020
May 12, 2020 POSTER: Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for treatment of autosomal dominant optic atrophy Source ASGCT 2020