Mark Curtis & Richard Philipson
Providing a critical overview of the sector’s commercial developments – M&As, licensing agreements & collaborations, financial results, IPOs and clinical/regulatory updates, with commentary from our Expert Contributors.
This month sees plenty of activity in the start-up arena, with new companies being formed to advance gene therapy treatments for alpha-1 antitrypsin deficiency (Apic Bio), Parkinson’s disease (Prevail Therapeutics) and complement-mediated diseases (Aevitas Therapeutics), and an IPO filed by Krystal to support its epidermolysis bullosa gene therapy; a joint venture between Agilis Biotherapeutics and Gene Therapy Research Institution Company Ltd completes the picture, although the disease targets for this collaboration are not disclosed. There certainly seems to be no lack of investment funds, even for the most challenging of targets like Parkinson’s disease, or for targets like alpha-1 antitrypsin deficiency, where an enzyme replacement therapy is already available.
It was a celebratory month for the cell therapy industry. Novartis received the world’s first approval for a CAR-T therapy. Ctl019, which will be marketed under the tradename kymriah (tisagenlecleucel), it was approved for the treatment of B-cell acute lymphoblastic leukemia in children and young adults. As the body of clinical evidence grew over the last few years, it became increasingly clear that the therapy would offer patients a giant leap in standard-of-care. The decision from the FDA was a resounding – yes. The therapy will initially be priced at US$475,000. Just days later, in one of the most significant deals in recent times in the biotech industry, Novartis’ rival Kite pharma was acquired by Gilead, in an all-cash deal, for a staggering US$11.9 billion. The deal is positive for patients overall, as the deep pipeline of car and tcr candidates assembled by Kite is now backed by the financial might needed to bring them to market.
Citation: Cell Gene Therapy Insights 2017; 3(7), 583-595.