Jörn Aldag, who has over 20 years of leadership experience in the life sciences industry, including CEO of gene therapy pioneer uniQure, tells Scrip that the research carried at the world-class Italian research institute SR-Tiget.

Pharma and the US biotech will target key regulators of cellular metabolism with focus on immune and neurological disorders. Royalty Pharma buys portion of PTC’s royalty rights to risdiplam, under development at Roche.

Gene and cellular therapies that can replace decades of expensive therapy do not come cheap. Even the initial remedy of payment by installments could be irrelevant when not all patients respond in the same way.

Recent panel review of biologics center's intramural research programs had its share of technical glitches and involved agency speakers blindly presenting to committee members they could not see; the experience suggests the difficulties an industry sponsor with an application hanging in the balance may face if a virtual advisory committee were to be held.