Encoded Therapeutics to Present Initial Data from the ENVISION Natural History Study in Children with Dravet Syndrome SCN1A + at the 2021 American Epilepsy Society Annual Meeting

The results of a preliminary observational study revealed a high use of antiepileptic drugs, a significant delay in adaptive functioning and a consequent impact on communication and socialization in young children with Dravet syndrome, worsening with age.

SUD SAN FRANCISCO, California, November 23, 2021– (BUSINESS WIRE) – Encoded Therapeutics today announced the upcoming presentation of initial data from the prospective ENVISION study on the natural history of children with Dravet syndrome SCN1A +, a rare epileptic and developmental encephalopathy, at the occasion of the 2021 American Epilepsy Society Annual Meeting. in Chicago from December 3-7, 2021. Additionally, Encoded will present its ongoing DRAVET ENGAGE initiative, which aims to capture the experiences and perspectives of the Dravet syndrome patient community, as well as a summary of molecular diagnosis and phenotypic results in people with Dravet syndrome SCN1A + identified by genetic testing.

Title: ENVISION: An international prospective natural history study in young children with Dravet syndrome SCN1A +
Abstract number: 2,222
Dated: Sunday 5 December
Platform presentation details: # B.07 | 10 am-10.15am HC | W475, level 4
Poster session details: 12 p.m. to 2 p.m. HC | Hall F1, level 3
Speaker: Andreas Brunklaus, MD, consultant pediatric neurologist at the Royal Hospital for Children, Glasgow, UK

Title: A patient-centered approach to integrate family experiences into clinical trial design and clinical care for Dravet syndrome
Abstract number: 3.073
Date hour : Monday December 6 | 12 p.m. to 1:45 p.m. HC
Site: Hall F1, level 3
Speaker: Emma James, Ph.D., MFPM (Hon), Vice President, Medical and Patient Affairs at Encoded Therapeutics

Title: Molecular diagnosis and phenotypic results in people with SCN1A-related disease identified through a sponsored and free epilepsy genetic testing program
Abstract number: 3.351
Date hour : Monday December 6 | 12 p.m. to 1:45 p.m. HC
Site: Hall F1, level 3
Co-presenter author: Jennifer Gorzelany, Head of Strategy at Encoded Therapeutics

About ETX101

Encoded develops ETX101 as a unique potential disease-modifying gene regulation therapy targeting the underlying cause of SCN1A+ Dravet syndrome in the hope of treating the full range of manifestations of the disease. In ETX101, a selective cell regulatory element and a transgene encoding a modified transcription factor are delivered in an adeno-associated virus (AAV) capsid clinically validated to upregulate, or increase, endogenous expression. SCN1A gene in the brain. This approach is expected to increase the production of NaV1.1 protein and sodium channel density in target neurons, leading to restored function. ETX101 has received Orphan Drug Designation and Rare Pediatric Disease Designation by the United States Food and Drug Administration. Encoded plans to treat the first participant of the ENDEAVOR interventional trial in 2022.

About the ENVISION natural history study

ENVISION is an ongoing observational study in infants and children with Dravet syndrome SCN1A +. This prospective natural history study is designed to better define the epileptic, neurodevelopmental, motor and behavioral manifestations of Dravet syndrome SCN1A + in children aged 6 to 60 months with SCN1A mutations. The study will examine these characteristics over two years using standardized assessments and will also explore the impact of the disease on parents / guardians and the use of health resources. These results will help identify appropriate scales and endpoints to assess the safety and efficacy of ETX101 in clinical trials. For more information on TO CONSIDER (NCT04537832), visit clinicaltrials.gov and search for “Encoded Therapeutics”.

About Dravet syndrome

Dravet syndrome is a serious and lifelong disease of the central nervous system that occurs in approximately 1 in 16,000 births worldwide, the majority of cases resulting from loss-of-function mutations in the SCN1A uncomfortable. This rare developmental and epileptic encephalopathy affects people of both sexes and all races equally, manifesting in a wide range of symptoms. Frequent, prolonged, and difficult to treat seizures usually begin in the first year of a previously healthy baby. Additional symptoms, such as cognitive delays, sleep disturbances, motor disturbances and behavioral disturbances, worsen the effects of the disease when they appear, often in the second or third year of life. ‘a child. More information on Dravet syndrome can be found at www.dravetfoundation.org.

About coded therapies

Encoded Therapeutics creates one-time disease-modifying gene therapies for pediatric central nervous system (CNS) disorders through its platform for selective cell targeting and regulation. The Encoded approach offers unprecedented genetic specificity and cell selectivity to open up new opportunities by targeting a range of disease mechanisms. Encoded’s technology is compatible with any delivery system to control where and when therapeutic transgenes are expressed, thereby shaping the functionality of target cells and holding broader therapeutic potential beyond CNS disorders. For more information, please visit www.encoded.com and follow us on LinkedIn, Twitter @EncodedTx and YouTube.

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Lynnea olivarez
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