Avrobio Offloads Gene Therapy Program For Rare Genetic Disorder To Novartis

Avrobio Inc AVRO agreed to sell its investigational hematopoietic stem cell (HSC) gene therapy program for cystinosis to Novartis AG NVS for $87.5 million in cash

Cystinosis is characterized by the accumulation of the amino acid cystine (a building block of proteins) within cells. Excess cystine damages cells and often forms crystals that can build up and cause problems in many organs and tissues.

Also Read: Analyst Says Gene Therapy Data Update Offers Insights into Potential Pivotal Endpoints.

Avrobio retained full rights to its portfolio of first-in-class HSC gene therapies for Gaucher disease type 1 and type 3, Hunter syndrome and Pompe disease. 

Proceeds from this transaction are expected to extend the company's cash runway into the fourth quarter of 2024. 

In addition, Avrobio exclusively licensed to Novartis certain other assets, know-how and other intellectual property related to Avrobio's gene therapy platform for use in cystinosis. 

Avrobio Price Action: AVRO shares are up 65.14% at $1.28 Monday at publication

Read Next: NeuBase Releases Promising Preclinical Data From Stealth Editors To Achieve Gene Editing

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