Blackstone and Huiling jointly raised US $570 million to focus on the commercialization of new gene therapy

On November 25, ferring pharmaceuticals announced the establishment of a new company, fergene, focusing on the global development and commercialization of gene therapy nadofaragene flamenovec (RAD IFN / syn3) in the United States, and will receive investment and expertise of 400 million dollars from Blackstone life sciences and investment of up to 170 million dollars from ferring Bladder cancer is one of the most common cancers About 430000 new cases are reported every year all over the world It becomes the ninth most common cancer in the world, and it is the most expensive cancer in the whole life It brings heavy burden to patients, their relatives and medical system In advanced NMIBC patients, BCG is the gold standard treatment Although it is effective, more than 60% of patients will relapse For such relapsed patients, the next treatment option is usually total cystectomy (complete cystectomy) Therefore, the unresponsive population of Bacillus Calmette Guerin (BCG) is one of the high unmet clinical needs ▲ bladder cancer information chart (picture source: ferring) Nadofaragene fireadenovec is a novel gene therapy for non muscle invasive bladder cancer (NMIBC) patients who have no response to BCG Nadofaragene firedenovec is composed of adenovirus containing interferon α - 2b gene After the virus is introduced into bladder, it enters bladder wall cells Inside the cells, the virus will decompose, leaving active genes to complete its work The internal gene / DNA mechanism of the cell picks up the gene and translates its DNA sequence, resulting in the production of anti-cancer interferon α - 2b protein, which is the natural protein used by the human body to resist cancer This novel gene therapy can transform patients' own bladder wall cells into a variety of interferon micro factories, thus enhancing the body's natural defense against cancer In 2017, the company published in the Journal of clinical oncology the results of two earlier trials of nadofaragene firetenovec (RAD IFN / syn3) showed that the treatment was well tolerated For patients with high level (Hg) NMIBC after BCG treatment who are unable or unwilling to undergo radical cystectomy, nadofaragene firedanovec has shown promising results In this study, 40 patients received rad-ifn α / syn3 between November 5, 2012 and April 8, 2015 At 12 months after the initial treatment, 14 patients (35.0%) had no recurrence of Hg Of these 14 patients, 2 experienced relapse 21 months and 28 months after the start of treatment, and 1 died of the upper tract tumor at 17 months without relapse RADS – IFN α / syn3 were well tolerated, no grade IV or V adverse events (AE) occurred, and no patients interrupted treatment due to adverse events The most frequently reported drug-related AEs were acute urination, dysuria, fatigue, frequency of urination, hematuria and nocturia ▲ image source: ascopubs At present, nadofaragene firedanovec is in the later stage of development, and has been awarded the title of breakthrough therapy, and has obtained the application of its biological preparation licensing application (BLA), and has obtained the FDA's priority review The data of phase 3 trial of nadofaragene firedanovec on 150 patients will be published at the 20th annual meeting of the society of Urology and Oncology (Suo) in Washington, D.C on December 5, 2019, reported by Dr Colin dinney, professor and head of Department of Urology, MD Anderson, University of Texas Dr dinney is the founder and former chairman of the cancer center (MDACC), the Association for clinical trials of urological oncology (suo-ctc) He took the lead in developing nadofaragene firedenovec, and co led the development of the program with Dr Nigel Parker of FKD therapies oy (FKD) Blackstone life sciences is a private investment platform that invests throughout the life cycle of companies and products in key life sciences areas By combining large-scale investment and hands-on leadership, Blackstone Life Sciences helps bring new drugs to market that are expected to improve the lives of patients ▲ image source: finance commerce Ferring pharmaceuticals is a research-oriented biopharmaceutical company dedicated to helping people around the world build families and lead better lives Headquartered in St praxes, Switzerland, ferring is a leader in reproductive medicine and maternal health, as well as Gastroenterology and urology Founded in 1950, ferring now has about 6500 employees around the world, operating subsidiaries in nearly 60 countries and selling products in 110 countries ▲ image source: dailyrecord As a subsidiary of ferring, if nadofaragene firetenovec is approved by FDA, fergene will be authorized to sell the gene therapy.