Novartis adding cystinosis gene therapy candidate from AvroBio

Novartis will pay $87.5 million in cash to add the Phase I/II hematopoietic stem cell (HSC) gene therapy program to its advanced therapy pipeline.

Novartis, through its acquisition of AveXis in 2018, is one of a small but growing number of players to bring a gene therapy to market: Zolgensma (onasemnogene abeparvovec) was approved in 2019 for spinal muscular atrophy (SMA).

The Swiss pharma giant says it has over 20 gene therapy project in its pipeline and this week is adding another through the deal with AvroBio. AVR-RD-04 is an investigational gene therapy for cystinosis being studied in a Phase I/II clinical trial sponsored by the University of California, San Diego.

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Along with the gene therapy candidate, Novartis has licensed certain other assets, know-how and other intellectual property from AvroBio for use in cystinosis – a genetic condition present from birth that leads to the build-up of cystine crystals in the body.

“This transaction strengthens AvroBio’s balance sheet, focuses our pipeline strategy and is a strong endorsement of our HSC gene therapy approach and plato gene therapy platform,” said Erik Ostrowski, AvroBio’s interim CEO and current CFO.

This is the latest exit from a gene therapy program for AvroBio, which in January 2022 deprioritized its Fabry disease program – its then lead gene therapy candidate – following disappointing trial results.

AvroBio will now concentrate on HSC gene therapies for Gaucher disease type 1 and type 3, Hunter syndrome and Pompe disease.