Biotechs Race to Cure AATD With Gene Editing
Get the Health newsletter
Daily health & science — research, biotech, public health, the studies worth knowing. Free.
- Beam Therapeutics has treated 29 AATD patients with its base-editing therapy, with high-dose patients showing mutant Z-AAT nearly disappearing and total AAT more than doubling; the company could file for FDA approval in 2028.
- AATD affects an estimated 200,000 people in North America and Europe, almost all sharing the same single-letter DNA mutation, making the potential market more than twice the size of Vertex's $12 billion-a-year cystic fibrosis franchise.
- Wave Life Sciences and Korro Bio are pursuing RNA-editing approaches for AATD, but clinical data from both has so far been "lackluster" compared with Beam's results, according to the article.
- Prime Medicine and Beam struck an agreement giving Beam rights to use prime editing for mutations correctable by base editing — including AATD — but the article describes the broader race as featuring "a broken alliance" and potential legal battles.
- Grifols currently provides the only AATD treatment, weekly protein replacement infusions costing around $100,000 per year, which requires lifelong dosing, fails to fully protect the lungs, and does nothing for the liver.
- David Liu's base-editing technology at the Broad Institute was identified by Beam CEO John Evans within two days of learning about it as the tool to fix AATD's single DNA-letter error; Beam launched in 2018 and dosed its first patient in June 2024.
- A Shanghai-based biotech co-founded by a former Beam scientist is competing alongside Yoltech and others, intensifying U.S.-China biotech tensions that the article frames as part of the contentious race.
Why it matters: For roughly 200,000 AATD patients stuck on $100,000-per-year weekly infusions that don't protect the liver, Beam's one-time gene edit could be transformative — and analysts peg the market at more than double Vertex's $12 billion cystic fibrosis franchise. But Beam's lead is contested by RNA editors, a fractured prime-editing alliance, and Chinese rivals, meaning the multibillion-dollar prize is far from settled.
