Stoke Therapeutics, Inc.

Cold Spring Harbor Laboratory

Isabel Aznarez

Adrian R Krainer

Zhou Han

Huw M Nash

Enxuan Jing

Gene Liau

Tomoki Nomakuchi

Aditya Venkatesh

Baruch Ticho

Juergen Scharner

Adrian Krainer

Jacob Kach

Alternative splicing events in genes can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in genes can modulate the expression level of functional proteins in patients and/or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition or disease caused by protein deficiency and/or mitochondrial function deficit.

OPA1 antisense oligomers for treatment of conditions and diseases

Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient LAL protein expression or a subject having Cholesteryl Ester Storage Disease.

Methods and compositions for treatment of cholesteryl ester storage disease

Alternative splicing events in SCN1A gene can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in SCN1A gene can modulate the expression level of functional proteins in Dravet Syndrome patients and/or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition caused by SCN1A, SCN8A or SCN5A protein deficiency.

Antisense oligomers for treatment of conditions and diseases

The present disclosure relates to compositions and methods for inhibiting nonsense-mediated mRNA decay in a gene-specific manner, for example in the treatment of diseases or disorders caused by nonsense mutations.

Reducing nonsense-mediated MRNA decay

Provided herein are methods and compositions for increasing the expression of a protein, and for treating a subject in need thereof, e.g., a subject with deficient protein expression or a subject having a disease described herein.

Antisense oligomers and uses thereof

Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.

Antisense oligomers for treatment of autosomal dominant mental retardation-5 and Dravet Syndrome

Provided herein are methods and compositions for increasing production of a target protein or functional RNA by a cell.

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Queens, NY, United States of America

Isabel Aznarez