Krystal Biotech said IOLITE enrollment for KB803 is complete and it remains on track for KB803 and KB801 registrational data readouts in 2026. The company also reported FDA platform technology designations for KB407 and KB111 and continued VYJUVEK expansion.
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.
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.
