Will the US FDA grant full approval for an in vivo (inside the body) CRISPR gene editing therapy before the end of 2028?

CRISPR In Vivo: FDA Approved by 2028?

While the first CRISPR therapy (Casgevy) was approved in late 2023, it is an *ex vivo* treatment (editing cells outside the body). The next major technical leap is *in vivo* editing, where the gene editing components are delivered directly into the patient's body. Leading candidates, such as those targeting ATTR amyloidosis, are currently in late-stage trials with BLA submissions expected in the 2027-2028 timeframe.

First to Market Advantage

The strong 'Yes' vote is warranted. Data from Phase 2/3 trials for the leading candidates have shown promising safety and efficacy profiles. Given the FDA's priority review status for breakthrough therapies, and barring unforeseen long-term safety issues, the regulatory approval of the first true in vivo CRISPR therapy by 2028 is highly likely.