Novartis Gene Therapy Update

We have received this message from Novartis regarding their gene therapy program for cystinosis:

“Dear Cystinosis Community,

In May 2023, Novartis acquired the investigational autologous hematopoietic stem cell (HSC) gene therapy program for the treatment of cystinosis from AVROBIO. We look forward to continuing the development of this program and bringing new hope to patients with cystinosis.

We thank you for your patience as we work quickly to transfer the knowledge base built by AVROBIO and build the infrastructure to support this new clinical program. This process is well underway but will mean that previously communicated timelines by AVROBIO for the next phases of the program will change.

Novartis Gene Therapies is committed to working with our partners in the cystinosis community by collaborating with each advocacy group to hear the community voice and further understand what is important to you as we develop this new therapy. The cystinosis advocacy community are strong allies who we are partnering with to deepen our understanding of this disease as we advance promising science with transformative potential for cystinosis.

We look forward to continuing our close collaboration with the cystinosis community and commit to providing you with updates on a regular basis as we have more information available to share.


The Novartis Gene Therapies Cystinosis Development Team

Medical Information (US/LACAN):, 1-833-828-3947

Medical Information (Europe, International):, +353 (1) 566-2364″

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