Related coverage linked through entity extraction aliases.
Apr 30, 2026
CRISPR therapies for alpha-1 antitrypsin deficiency advanced: BEAM-302 posted positive Phase 1/2 data, TSRA-196 gained FDA fast track/orphan status, and YOLT-202 raised AAT above the protective threshold.
Jun 08, 2026
Canaccord Genuity initiated Beam Therapeutics with a buy rating and $74 price target, highlighting near-term catalysts. Beam reached an agreement with the FDA on an accelerated approval pathway for its BEAM-302 AATD therapy, which showed early clinical proof-of-concept for in vivo base editing. The company plans a BLA for risto-cel and a pivotal plan for BEAM-302 in early 2026.
Mar 20, 2026
Krystal Biotech posted Q4 2025 net revenue of $107.1 million from Vyjuvek sales, with full-year revenue reaching $389.1 million. The company expanded to over 90 patients across Germany, France, and Japan while advancing its pipeline with new FDA designations.
Mar 14, 2026
YolTech Therapeutics received FDA approval for its IND application for YOLT-202, an in vivo gene-editing therapy for Alpha-1 Antitrypsin Deficiency. Interim data from an ongoing trial showed rapid increases in AAT levels to normal range with favorable safety.
