Encoded Therapeutics Announces U.S. FDA Breakthrough Therapy Designation Granted to ETX101 for the Treatment of Dravet Syndrome

Encoded Therapeutics, Inc. ("Encoded"), a clinical-stage biotechnology company developing precision genetic medicines for severe neurological disorders, today announced that the U.S. Food and Drug ...
The American Journal of Managed Care

Researchers Find Genetic Variant Linked With Earlier-Onset Childhood Epilepsy

A specific genetic variant of SCN1A may be associated with earlier-onset epilepsy. SCN1A variants are most commonly associated with Dravet syndrome, the most common genetic epilepsy, which is ...
Morningstar

Encoded Therapeutics Announces Regenerative Medicine Advanced Therapy (RMAT) Designation Granted by U.S. FDA for ETX101 in SCN1A+ Dravet Syndrome

Designation based on preliminary clinical evidence indicating the potential for ETX101 to address unmet medical needs associated with SCN1A+ Dravet syndrome Completion of enrollment in POLARIS ...
Business Wire

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

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Encoded Therapeutics today announced the upcoming presentation of initial data from the ENVISION prospective natural history study of children with SCN1A+ ...
GEN

New Gene Therapy May Potentially Treat Severe Form of Epilepsy

Scientists from the University of Virginia (UVA) School of Medicine describe in a study involving mouse models how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, ...
Business Wire

CAMP4 Presents In Vivo Data Supporting Novel Therapeutic Strategy for Dravet Syndrome at American Epilepsy Society Annual Meeting

CAMBRIDGE, Mass.--(BUSINESS WIRE)--CAMP4 Therapeutics, a biotechnology company harnessing the power of RNA to restore healthy gene expression, today announced the presentation of in vivo ...
Science Daily

Researchers discover genetic variant associated with earlier onset childhood epilepsy

Researchers have discovered a specific genetic variant in SCN1A, the most common genetic epilepsy, that leads to an earlier onset of epilepsy, with clinical features distinct from other epilepsies.
EurekAlert!

The Split Intein of the SCN1A gene (IMAGE)

The SCN1A gene is too large to fit into aconventional viral delivery systems. Scientists overcame this hurdle with a pioneering "split-intein" mechanism, which is like two separate trucks, delivering ...