Switzerland-based Ferring Pharmaceuticals and Blackstone Life Sciences, a private investment platform, are jointly investing more than USD570m in an investigational gene therapy called nadofaragene firadenovec (rAd-IFN/Syn3) intended for bladder cancer patients, it was reported yesterday.

This step has resulted in Ferring Pharmaceuticals introducing a new company called FerGene that is to concentrate on the global development and United States commercialisation of nadofaragene firadenovec. The gene therapy is being produced for patients having high-grade, Bacillus Calmette-Guérin (BCG) unresponsive, non-muscle invasive bladder cancer (NMIBC). Presently, the gene therapy is in late phase three development.

The United States Food and Drug Administration (FDA) has granted Nadofaragene firadenovec breakthrough therapy designation and its biologics license application has been accepted for filing by the regulator and given priority review. It is an adenovirus vector-based gene therapy having the gene interferon alfa-2b and is administered using a catheter into the bladder every three months.

The gene therapy's phase 3 clinical trial data will be released in early December by Colin Dinney from the University of Texas MD Anderson Cancer Center (MDACC), who has led its development. If approved by the FDA, the marketing authorisation of nadofaragene firadenovec will be held by FerGene. The new gene therapy company will receive an investment of USD400m from Blackstone Life Sciences and up to USD170m from Ferring Pharmaceuticals.