PTB-101 SDF Alpha

The FDA has approved 4- and 5-gram vial sizes of Takeda's GLASSIA (Alpha-1 Proteinase Inhibitor [Human]), expanding beyond the original 1-gram vial approved in 2010, with availability beginning July 2025.

Beam Therapeutics' BEAM-302 demonstrated successful DNA correction in alpha-1 antitrypsin deficiency patients, marking the first clinical proof of concept for direct mutation correction using base editing technology.

Beam Therapeutics' BEAM-302 demonstrated the first-ever clinical genetic correction of the disease-causing PiZ mutation in Alpha-1 Antitrypsin Deficiency patients, with a single dose producing durable increases in functional AAT protein.

Axatilimab, a CSF1R-blocking antibody, received FDA approval in August 2024 for chronic graft-versus-host disease treatment, demonstrating a 74% overall response rate in clinical trials.

Wave Life Sciences achieves first-ever RNA editing results in humans for alpha-1 antitrypsin deficiency, demonstrating therapeutic protein level increases with a single dose treatment.