• Skip to main navigation
  • Skip to main content
  • Skip to Footer
  • Navigate Novartis
    • Patients
    • Healthcare Professionals
    • Media
    • Investors
    • Partners
    • ESG
    • Job Seekers
    • Clinical Trials
  • Coronavirus
  • Global
    • Novartis site directory
Novartis logo: a global healthcare company
  • Our Company
    • Our Leadership
      • Board of Directors
      • Executive Committee of Novartis
    • Our Approach
      • Strategy
      • Corporate Responsibility
      • Diversity & Inclusion
      • Partnering
      • People and Culture
    • Our Business
      • Global Product Portfolio
      • Innovative Medicines
      • Novartis Pharmaceuticals
      • Novartis Gene Therapies
      • Novartis Oncology
      • Sandoz
    • Media
      • Newsroom
      • News Archive
      • Multimedia Resources
      • Subscribe to Updates
    • Investors
      • Event Calendar
      • Financial Data
      • Share Data & Analysis
      • Shareholder Information
      • Environmental, Social & Governance
  • Our Focus
    • Disease Areas
      • Cancer
      • Cardiovascular, Renal and Metabolic Medicine
      • Immunology & Dermatology
      • Ophthalmology
      • Neuroscience
      • Respiratory
    • Platforms and Technology
      • Data and Digital
      • Cell & Gene Therapy
    • Novartis Stories
      • Discovery
      • From Our Labs
      • Access to Healthcare
      • Patient Perspectives
      • People and Culture
      • Live Magazine
  • Our Impact
    • Building Trust with Society
      • Corporate Responsibility
      • Ethical Standards
      • Access
      • Environmental Sustainability
    • Global Health
      • Malaria
      • Sickle Cell Disease
      • Chagas disease
      • Leprosy
      • Novartis Foundation
    • ESG Performance and Reporting
      • Materiality Assessment
      • Environmental, Social and Governance
      • Transparency and Disclosures
      • Positions on Key Topics
      • Statements on Key Issues
  • Our Science
    • Research & Early Development
      • Novartis Institutes for BioMedical Research
      • Research Disease Areas
      • Postdoc Program
      • Open Source Science
    • Drug Development at Novartis
      • Novartis Pipeline
      • Clinical Trials
      • Translational Medicine
      • Global Product Portfolio
    • Science Stories
      • Discovery
      • From Our Labs
  • Careers
    • Your Career
      • Global Career Search
      • Teams and Skills
      • Gene Therapies Careers
    • Working at Novartis
      • Diversity & Inclusion
      • People and Culture
      • Corporate Responsibility
    • Career Programs
      • Our Career Programs
      • Students and Scholars
      • Novartis Quantitative Sciences Hackathon
Search
Adeno-associated viruses
  • TwitterTweet
  • FacebookShare
  • Share
    • Twitter
    • Facebook
    • LinkedIn
    • Email
    • Whatsapp
  • Jump to Comments
Stories/Discovery

Collaborating to explore new gene regulation therapies

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

Jul 30, 2020

Novartis has entered into a research and development collaboration with Sangamo Therapeutics to explore potential gene regulation therapies for neurodevelopmental diseases. Proprietary technology platforms from both companies will be harnessed to facilitate progress toward potentially transformative treatments for life-long, debilitating conditions.

Sangamo is renowned for work on zinc finger proteins, which may be engineered to target select DNA sequences and switch specific genes on or off through the modulation of transcription. Novartis has deep experience with adeno-associated viruses (AAVs), which can be engineered to transport genes to specific cells within the body. The three to five year collaboration leverages zinc finger protein transcription-factors (ZFP-TFs) and AAVs. The Novartis-Sangamo team will pursue three targets in neurology.

“New medicines for those with neurodevelopmental diseases are desperately needed, and gene therapy offers great promise for helping these patients,” says Jay Bradner, President of the Novartis Institutes for BioMedical Research. “Working with the team from Sangamo, we intend to reach previously undruggable gene targets by coupling their novel ZFP-TF platform with our AAV platform in order to innovate a next-generation gene therapy payload.”

Novartis continues to expand its capabilities in several distinct gene therapy platforms – AAVs, chimeric antigen receptor T-cells (CAR-Ts) and clustered regularly interspaced short palindromic repeats (CRISPR).  The collaboration with Sangamo will introduce an additional genomic technology platform and is expected to enhance the utility of Novartis AAVs.

AAVs are naturally occurring viruses that are not known to cause human illness. They have varying affinities for different tissues and can be engineered to carry and deliver genes to precise cellular locations throughout the body. Novartis has focused on the use of AAVs to replace the function of faulty genes in patients with single gene mutations. AveXis, a Novartis company, has optimized a particular type of AAV, called AAV9, to carry genes to target cells within the central nervous system. This AAV gene delivery vehicle is the backbone of an approved gene therapy for patients with a progressive neuromuscular disease called spinal muscular atrophy.

While AAVs have many advantages that make them well-suited for gene therapy, one disadvantage is that they can’t carry large genes. The collaboration between Novartis and Sangamo will enable the team to expand their focus by targeting diseases caused by mutations in one copy of a large gene.

“The gene for a ZFP-TF is small enough to fit inside an AAV,” explains Ricardo Dolmetsch, Head of Neuroscience at the Novartis Institutes for BioMedical Research. “We can use it to increase the production of a large gene in someone who still has one intact copy of the gene. This dramatically expands the range of diseases that we can potentially target with gene therapy because many diseases are caused by the loss of a single copy of a gene.”

Researchers have used ZFP-TFs – which are mammalian – to regulate genes in the lab for many years. A number of zinc-finger based therapies, including zinc finger nucleases, are currently undergoing clinical testing. Each ZFP-TF from Sangamo is engineered to bind a target region of genomic DNA in a manner that is highly specific and selective. Further, ZFP-TFs are highly tunable, meaning that they can be designed to precisely modulate the expression of targeted genes to varying extents. After identifying its target, the ZFP-TF recruits a host of other proteins that help switch genes on or off.

The Novartis-Sangamo team will work to identify potential new therapies for several diseases, including an autism spectrum disorder.

Retina cells, including some targeted by a gene therapy
Discovery

Experimental gene therapy discovered in-house enters clinical testing

Shareline

Novartis and Sangamo Therapeutics plan to explore potential gene regulation therapies for neurodevelopmental diseases
Tweet Tweetpopo

Reimagine medicine with Novartis

The Novartis Institutes for BioMedical Research (NIBR) is the innovation engine of Novartis.

Learn More

Tags: 
Cell and Gene Therapy, Drug Discovery, Neuroscience, Genetic Disease
    • TwitterTweet
    • FacebookShare
    • Share
      • Twitter
      • Facebook
      • LinkedIn
      • Email
      • Whatsapp
    • Jump to Comments
  • Print
  • Save

Novartis Stories

View all

RNA strand
Discovery

Take a visual tour of medicines that target RNA

A kidney patient is smiling at the camera
Discovery

New hope for treating inflammatory diseases of the kidney

2D rendering of kidneys
Discovery

Predictive analytics could bring medicines to patients faster

You are here

  1. Home ›
  2. Stories ›
  3. Discovery

Novartis Global

  • Navigate Novartis

    • Patients & Caregivers
    • Healthcare Professionals
    • Investors
    • Media
    • Society & ESG
    • Partners
  • Contact Us

    • Global Contacts
    • Office Locations
    • For Investors
    • For Media
  • About Novartis

    • Our Company
    • Our Focus
    • Our Impact
    • Our Science
    • Our Stories
  • Our Portfolio

    • Global Product Portfolio
    • Global Clinical Pipeline
  • Careers

    • Career Search

Connect with Novartis

  • twitter
  • linkedin
  • youtube
  • facebook
  • instagram
Novartis Site Directory
© 2021 Novartis AG
  • Terms of Use
  • Privacy Policy
  • Contact Us
  • Site Map
  • Cookie Settings

This site is intended for a global audience.