Voyager Therapeutics, Inc.

Ginkgo Bioworks, Inc.

David Dismuke

Garrett E Berry

Daniel Mccoy

Sylvain Cecchini

Luis Maranga

The present disclosure describes methods and systems for use in the production of recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system include engineered untranslated regions (UTR) which allow for the controlled expression of AAV capsid proteins, such as VP1, VP2 and VP3. In certain embodiments, the production process and system include engineered untranslated regions (UTR) which allow for the controlled expression of non-structural AAV replication proteins, such as Rep78 and Rep52.

Engineered untranslated regions (UTR) for AAV production

Provided herein are gene therapy compositions and methods for treating, preventing, and/or curing NPC1. More specifically, the disclosure provides Adeno-associated virus (AAV) vectors for delivery of nucleic acids and nucleic acids (including AAV transfer cassettes) for treating, preventing, and/or curing NPC1.

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Cary, NC, United States of America

David Dismuke