Cutting-edge science for transformative gene therapies


We believe that patients deserve more than incremental improvements in treatments or symptom management. We are in pursuit of long-lasting, transformative treatments that address the underlying biology of disease. Our understanding of the genetic basis of many diseases, developments in viral vector design, and progress in vector manufacturing methods places us on the cusp of a new era of gene therapies. We have built the capabilities to deliver on the promise of this era.

Our team of world-class scientific, clinical and product development experts and our world-renowned partners are working together to develop gene therapies that harness the latest scientific advancements. Our teams combine cutting-edge gene transfer techniques, innovative vector design, and tailored delivery methods to optimize the safety, efficacy and durability of the clinical effects of our gene therapies.

MECHANISM

 

  • Well-validated genetic targets and gene-transfer techniques

  • Effective and efficient approaches to address the genetic defect

  • Optimization to increase expression

VECTOR

 

  • Appropriate packaging capacity

  • Effectively transfers genes

  • Targets the appropriate cells

  • Minimizing immune response

ROUTE OF DELIVERY

  • Delivers therapy to relevant tissue

  • Minimize off-target effects

  • Minimize invasiveness for patient

  • Optimize vector volume to the condition

Exquisitely designed for each unique disease


We tailor our approaches to the unique features of each specific disease and patient population with a thoughtful focus on optimizing mechanism, vector and administration.

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AXOVANT GENE THERAPIES

Eleven Times Square, 33rd Floor
New York, NY 10036
United States

+1-833-AXOVANT (1-833-296-8268)

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AXOVANT SCIENCES GMBH

Viaduktstrasse 8
4051 Basel
Switzerland