Ferring Pharmaceuticals and Blackstone Life Sciences have announced a joint investment of more than $570m in nadofaragene firadenovec, an investigational therapy for bladder cancer.

Nadofaragene firadenovec (rAd-IFN/Syn3) is being developed as a treatment for patients with non-muscle invasive bladder cancer (NMIBC), and unresponsive to Bacillus Calmette-Guérin (BCG).

Ferring Pharmaceuticals chairman Frederik Paulsen said: “Bringing a novel gene therapy to the market requires dedicated focus and capabilities, and FerGene, a Ferring company, will have the resources and team needed to help us potentially bring nadofaragene firadenovec to patients.

“Through this new joint financing model between Ferring and Blackstone Life Sciences, we aim to ensure more people with high-grade, BGC unresponsive, non-muscle invasive bladder cancer may benefit from this novel gene therapy if approved.”


FerGene will market nadofaragene firadenovec, following FDA approval

FerGene, a new gene therapy firm, will be established as a subsidiary of Ferring, to commercialise nadofaragene firadenovec in the US and advance the global clinical development.

Blackstone will invest $400m and Ferring will invest up to $170m in FerGene, and would possibly launch and commercialise rAd-IFN/Syn3 outside of the US.

The therapy, which is currently in late Phase 3 development, has secured FDA breakthrough therapy designation, in addition to the acceptance of Biologics License Application (BLA) and receipt of FDA Priority Review.

rAd-IFN/Syn3 is an adenovirus vector-based gene therapy that contains the gene interferon alfa-2b, administered by catheter into the bladder every three months.

The virus breaks down itself to release the active gene into the cells of the bladder wall, which is picked up by the internal gene/DNA machinery of the cells.

The DNA sequence of the gene is translated inside the body, enabling the cells to secrete high quantities of interferon alfa-2b protein, a protein that occurs naturally in the body to fight cancer.

Blackstone Life Sciences head Nick Galakatos said: “This innovative partnership with Ferring illustrates the unique value of Blackstone Life Sciences in bringing transformative therapies to market.

“Our expertise and experience in hands-on clinical development and early commercialization will help further advance this promising therapy for bladder cancer patients in the US and around the world.”