Ptc Therapeutics, Inc.

Minakshi B Jani

Anuradha Bhattacharyya

Nadiya Sydorenko

Young-Choon Moon

Seongwoo Hwang

Suresh D Babu

Nikolai A Naryshkin

Matthew G Woll

Amal Dakka

Christopher Robert Trotta

Kari Wiedinger

Wencheng Li

Vijayalakshmi Gabbeta

Kerstin Effenberger

The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.  In particular, the present description relates to substituted monocyclic heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.

Methods for treating Huntington's disease

Described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a small molecule splicing modifier compound of Formula (I) provided herein or a form thereof, wherein W, X, A and B are as defined herein. In one aspect, methods for modifying RNA splicing to modulate the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene that contains an intronic REMS is modified utilizing a splicing modifier compound of Formula (I), are described herein. In another aspect, methods for modifying RNA splicing to modulate the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene is modified to comprise an intronic REMS utilizing a splicing modifier compound of Formula (I), are described herein.

Methods for modifying RNA splicing

The present description relates to a method or use of a compound for treating or ameliorating HD (Huntington's Disease) in a subject in need thereof comprising administering to the subject an effective amount of a compound of Formula (I): or a form thereof, wherein R, R, R, Rand Rare as defined herein. In particular, the present description relates to a method of use of a compound of Formula (I) or a form or composition thereof for treating or ameliorating HD in a subject in need thereof comprising, administering an effective amount of the compound or a form or composition thereof to the subject.

The present description relates to a method or use of a compound for treating or ameliorating HD (Huntington's Disease) in a subject in need thereof comprising administering to the subject an effective amount of a compound of Formula (I): or a form thereof, wherein w, w, w, w, w, wand ware as defined herein. In particular, the present description relates to a method of use of a compound of Formula (I) or a form or composition thereof for treating or ameliorating HD in a subject in need thereof comprising, administering an effective amount of the compound or a form or composition thereof to the subject.

Methods for treating Huntington's Disease

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Iselin, NJ, United States of America

Minakshi B Jani