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Collaborating to explore new gene regulation therapies
Novartis and Sangamo Therapeutics plan to pursue potential treatments for neurodevelopmental diseases.
![Gene therapy pioneer Dr. Botond Roska has studied the retina for the past two decades. Dr. Botond Roska shares his thoughts on the future of gene therapy.](/sites/novartiscom/files/styles/cta_image/public/2021-07/bringing-gene-therapy-focus-roska-hero-image.jpg.webp?itok=1jiCejo5)
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-Based
CRISPR-based
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.