Novartis' pipeline tacks on another gene therapy, adding Avrobio's cystinosis programme

22 May 2023
firstwordpharma.com
Gene TherapyLicense out/in
Novartis moved to add another experimental gene therapy to its pipeline under an agreement for Avrobio's programme for the treatment of cystinosis. The Swiss drugmaker will pay $87.5 million in cash as part of the deal.
Avrobio recently reported that the haematopoietic stem cell (HSC) gene therapy demonstrated a durable treatment effect out to 36 months in a Phase I/II study. The trial includes six patients whose own HSCs are genetically modified to express a functional version of cystinosin. Preliminary data suggest that post-gene therapy, functional cystinosin is produced throughout the body as evidenced by clinical measures in multiple tissues.
At the time, Avrobio's chief medical officer Essra Ridha said the results lay "the foundation for a registration-enabling clinical trial." Under the agreement, Novartis will receive certain assets related to the cystinosis program, whilst also licensing other assets, know-how and intellectual property related to Avrobio's gene therapy platform for use in cystinosis.
Avrobio's interim CEO Erik Ostrowski – who was appointed earlier this month - noted that the transaction "strengthens [our] balance sheet," extending the company's cash runway into the fourth quarter of 2024. The drugmaker is planning to move its investigational HSC gene therapy AVR-RD-02 into a registrational Phase II/III for Gaucher disease type 3 later this year after reporting promising interim data.
More to follow.
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