Prime Medicine, Inc. announced the launch of a preclinical program targeting alpha-1 antitrypsin deficiency (AATD), a genetic disorder affecting lung and liver function, utilizing their ...

today unveiled a preclinical program for the treatment of alpha-1 antitrypsin deficiency (AATD), the next program within its liver franchise. Prime Medicine expects to file an investigational new ...

BEAM-302 demonstrated safety and efficacy in increasing AAT levels and reducing mutant protein in alpha-1 antitrypsin deficiency patients. Beam Therapeutics announced promising initial results ...

Korro Bio’s (KRRO) treatment of alpha-1 antitrypsin deficiency received FDA orphan designation, according to a post on the agency’s stie. Published first on TheFly – the ultimate source for ...

AIRNA Raises Oversubscribed $155 Million Series B Financing to Fund Phase 1/2 Clinical Trial for Alpha-1 Antitrypsin Deficiency and Future Pipeline Financing led by Venrock Healthcare Capital ...

Beam Therapeutics Inc. BEAM announced that the FDA has cleared the investigational new drug (IND) application to begin clinical studies on its pipeline candidate, BEAM-302, for the treatment of ...

Beam Therapeutics Inc. (NASDAQ:BEAM) on Saturday presented additional data from the Phase 1/2 clinical trial of BEAM-302 in ...

has granted orphan drug designation to the investigational medicine KRRO-110 for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). “Receiving orphan drug designation from the FDA ...

Korro Bio's OPERA platform offers precise RNA editing for genetic diseases like Alpha-1 antitrypsin deficiency. Learn more on ...

Beam Therapeutics Inc. (BEAM) presented additional data from the Phase 1/2 clinical trial of BEAM-302 in patients with alpha-1 antitrypsin deficiency (AATD) at the 2025 Alpha-1 ...

Financing led by Venrock Healthcare Capital Partners with co-lead Forbion Growth, and participation from RTW Investments, Nextech Invest, and other new ...