Voyager Therapeutics, Inc.

Centre National De La Recherche Scientifique

Institut National De La Sante Et De La Recherche Medicale

UniversitÉde Strasbourg

Martin Goulet

Holger Patzke

Jinzhao Hou

Dinah Wen-Yee Sah

Mathieu E Nonnenmacher

Todd Carter

Yanqun Shu

Hélène Puccio

Hongxing Wang

The invention provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.

Compositions and methods for delivery of AAV

The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of frataxin (FXN), whether in vitro and/or in vivo including, but not limited to, the exploitation of engineered promoters. Such compositions include delivery via administration of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Friedreich's ataxia or another neuromuscular or neurological condition resulting from a deficiency in the quantity and/or function of frataxin or associated with decreased expression or protein levels of frataxin.

Frataxin expression constructs having engineered promoters and methods of use thereof

Described herein are compositions and methods for treating Friedreich's Ataxia (FA) using adeno-associated virus (AAV) to deliver therapeutics agents.

Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery

Growing community of inventors

Weston, MA, United States of America

Martin Goulet