But an FDA nod, which analysts see as likely, doesn’t mean it will be smooth sailing for the therapy. A one-time therapy that can deliver a copy of the gene that is faulty in people with hemophilia offers a major advance for patients. And, because the viral makeup of the therapy essentially primes the immune system to attack it next time it sees the same therapy, patients often can receive a treatment only once, raising the stakes of a decision. “You’re transitioning patients from an existing therapy to gene therapy that may not work past five years," he said. But for long-term investors, strong patient uptake, rather than an FDA approval, could be a better signal for an entry.

Source: Wall Street Journal June 30, 2023 05:17 UTC