One Treatment, Two Targets: BEAM-302 in AATD: John Hurst, MD, PhD

BEAM-302, an in vivo base-editor therapy, was well tolerated in patients with PiZZ-alpha-1 antitrypsin deficiency (AATD)–associated lung disease and those with liver and/or lung disease, according to a late-breaking abstract presented at the 2026 European Respiratory Society (ERS) International Congress.¹

AATD is an autosomal codominant disorder that can cause severe lung and liver disease due to the homozygous Z (1096G>A) mutation. BEAM-302 is comprised of gRNA targeting the SERPINA1 g.1096G>A allele.¹ What makes this therapy unique is that it takes a single approach to treating the 2 most common manifestations of AATD, lung and liver disease, John R. Hurst, MD, PhD, lead study author and pro vice provost of international health at University College London, said in an interview with The American Journal of Managed Care®.

“By reducing the Z alpha-1 antitrypsin production in the liver and the circulating Z polymers, they're reducing the risk of liver disease, and by providing replacement of M alpha-1 antitrypsin in their blood and therefore in the lung, they're preventing the development of the lung disease side of things,” Hurst explained. “So we have both bases covered with just one treatment.”

In addition to targeting both diseases, BEAM-302 is also expanding treatment options for patients with AATD, which Hurst says are limited and often burdensome. Currently, there are no licensed treatments for liver disease in patients with AATD. Alpha-1 augmentation, another treatment option, has been shown to reduce the rate of change in lung density but requires frequent injections and infusions, Hurst said.

Although the treatment is still being investigated, Hurst emphasized an “exciting future” for patients based on current data.

“Preventing that from happening with a new treatment offers a whole host of new opportunities for people in the future living with alpha-1,” he said. “That's why this is so important.”

References

1. Hurst JR, Van't Wout E, Ashdown T, et al. First-in-human in vivo gene editing for severe alpha-1 antitrypsin deficiency (AATD): initial data from the Phase 1/2 study of BEAM-302. Presented at the 2026 European Respiratory Society International Congress; Barcelona, Spain; September 5-9, 2026.