Gene therapy aims to treat diseases by replacing, inactivating or introducing genes into cells — either inside the body (in vivo) or outside of the body (ex vivo).

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Collaborating to explore new gene regulation therapies

Novartis and Sangamo Therapeutics plan to pursue potential treatments for neurodevelopmental diseases.

Dr. Botond Roska shares his thoughts on the future of gene therapy.

Bringing gene therapy into focus

Dr. Botond Roska says we should keep our eyes on gene therapy.

Novartis gene therapy R&D currently focuses on AAV-based therapies and CRISPR-based technologies




AAV (adeno-associated virus)-based therapies have the power to deliver new or working copies of a missing or nonworking gene to human cells. As AAV seems not to be associated with any disease, it is considered a safe and attractive vector for gene delivery. Novartis is exploring the use of AAV-based therapies across neurology and ophthalmology by delivering new genes into patient cells.

CRISPR (clustered regularly interspaced short palindromic repeats)-based technologies create double-stranded breaks in DNA, acting like a pair of molecular scissors that can be used to insert, remove, or replace specific pieces of a person’s existing DNA. Novartis is conducting early research on the use of CRISPR-based technologies across hematology and ophthalmology to potentially treat diseases by correcting genetic defects.