Voyager Therapeutics, Inc.

Holger Patzke

Dinah Wen-Yee Sah

Jinzhao Hou

Qingmin Chen

Martin Goulet

Mathieu E Nonnenmacher

Todd Carter

Yanqun Shu

Joan R Coates

Jenna Carroll Soper

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

The present invention relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and/or canine degenerative myelopathy (DM).

Treatment of amyotrophic lateral sclerosis (ALS)

The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.

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Cambridge, MA, United States of America

Holger Patzke