FerGene

FerGene was established in November 2019 as a joint venture between Ferring Pharmaceuticals and Blackstone Life Sciences, launching with a substantial investment of over \$570 million. This strategic collaboration was singularly focused on advancing and commercializing a late-stage gene therapy, nadofaragene firadenovec, for a specific subset of bladder cancer patients. The company was formed as a subsidiary of Ferring to specifically handle the U.S. commercialization and global clinical development of this potential treatment. Blackstone Life Sciences contributed \$400 million, while Ferring invested up to \$170 million, creating a well-resourced entity to tackle a significant unmet medical need.

The business operated in the highly specialized market of oncological gene therapy, targeting patients with high-grade, Bacillus Calmette-Guérin (BCG) unresponsive, non-muscle invasive bladder cancer (NMIBC). This patient population has seen limited therapeutic advancements over the past two decades and often faces bladder removal as the next step after failing BCG therapy. FerGene's business model was centered on bringing nadofaragene firadenovec through the final stages of clinical trials, securing regulatory approval, and marketing it in the United States, while Ferring would handle potential commercialization outside the U.S. The company's leadership included CEO David Meek, appointed in late 2019, who brought extensive experience in the US oncology market.

The core product, nadofaragene firadenovec (brand name ADSTILADRIN®), is an adenovirus vector-based gene therapy. Administered directly into the bladder via a catheter every three months, the therapy uses a non-replicating adenovirus to deliver the gene for interferon alfa-2b into the bladder wall cells. This process effectively turns the patient's own cells into microfactories that produce high quantities of interferon alfa-2b, a naturally occurring protein that helps the body fight cancer. The treatment received Breakthrough Therapy designation from the FDA, and Phase 3 trials showed promising results, with 53% of certain patients achieving a complete response at three months. Despite the initial promise and significant financial backing, FerGene faced challenges, receiving a complete response letter from the FDA regarding chemistry, manufacturing, and control (CMC) issues. Ultimately, the collaboration between Ferring and Blackstone ended in October 2022, and the program was brought back under Ferring's direct control. Ferring continued the development, eventually securing FDA approval for ADSTILADRIN® in December 2022.

Keywords: gene therapy, bladder cancer, oncology, nadofaragene firadenovec, Ferring Pharmaceuticals, Blackstone Life Sciences, non-muscle invasive bladder cancer, BCG unresponsive, ADSTILADRIN®, urology