##  [Gene Therapy](/research-and-development/technology-platforms/gene-therapy "Gene Therapy") 

 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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 ![adeno associated viruses 3d hero](/sites/novartis_com/files/styles/cta_image/public/adeno-associated-viruses-3d-hero.jpg.webp?itok=S-N3T3bV "adeno associated viruses 3d hero") 

 

##  Collaborating to explore new gene regulation therapies 

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



 

 [Learn More](/stories/collaborating-explore-new-gene-regulation-therapies "Learn More") 



 

 



 





 ![Dr. Botond Roska shares his thoughts on the future of gene therapy.](/sites/novartis_com/files/styles/cta_image/public/2021-07/bringing-gene-therapy-focus-roska-hero-image.jpg.webp?itok=BUDYZBTK "Gene therapy pioneer Dr. Botond Roska has studied the retina for the past two decades.") 

 

##  Bringing gene therapy into focus 

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



 

 [Learn More](/stories/bringing-gene-therapy-focus "Learn More") 



 

 



 





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## 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.







 



     ![boy playing puddle flip](/sites/novartis_com/files/styles/banner_image_1920/public/boy-playing-puddle-flip.jpg?h=501e7db7&itok=xt7kC-gR "boy playing puddle flip") 

 



##  AveXis is now Novartis Gene Therapies 

Novartis Gene Therapies is focused on Spinal Muscular Atrophy (SMA)

 [Learn More](/about/novartis-gene-therapies "Learn More")