Historic fist: CRISPR therapy gets regulatory approval

What's the news: In a historic first, a CRISPR therapy gets regulatory approval.

Why should we believe it: This news comes via the UK's Medicines and Healthcare products Regulatory Agency. The approved treatment is Casgevy, a CRISPR-based treatment for sickle cell and beta-thalassemia.

Casgevy is developed by Vertex Pharmaceuticals and CRISPR Therapeutics. The first is a large Boston-based biotech, the second is a Swiss biotech founded by Nobel laureate Emmanuelle Charpentier.

Why this is a big deal: Charpentier, along with Jennifer Doudna, co-wrote the first paper proposing a therapeutic use of CRISPR only in 2012. This is the work that the two shared the Nobel Prize for in 2020.

It's amazing that a little more than a decade later from its original discovery, this revolutionary approach is being rolled out as a standard of care for a progressive, painful disease that had no widely available cure until now.

So what's next: Approval of Casgevy by the FDA is expected in December. Looking past that, who knows? Lots of CRISPR-based therapies are in the works. The coming months and years are sure to see many new CRISPR therapies approved for previously complicated or incurable conditions.