Studying a monogenic condition like Alpha-1 antitrypsin deficiency provides a unique opportunity to better understand disease progression. "This work deepens our insight into protein folding ...

for the treatment of alpha-1 antitrypsin deficiency (AATD) in the United States. BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct the ...

today announced that the United States (U.S.) Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for BEAM-302 for the treatment of alpha-1 antitrypsin ...

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 ...

Korro Bio, Inc. has completed dosing for the first two cohorts in its Phase 1/2a REWRITE clinical study of KRRO-110, aimed at treating Alpha-1 Antitrypsin Deficiency (AATD), with interim results ...

Korro Bio, Inc. announced that its investigational drug KRRO-110 has received orphan drug designation from the FDA for treating Alpha-1 Antitrypsin ... people in the United States.

Studying a monogenic condition like Alpha-1 Antitrypsin Deficiency provides a unique opportunity to better understand disease progression. This work deepens our insight into protein folding disorders ...

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 ...

which is in Phase 1 clinical trials for treating Alpha-1 antitrypsin deficiency; and KB301 that is in Phase 2 clinical trials for treating aesthetic skin conditions, as well as in open label study ...

In a nutshell: • Hereditary disease alpha-1-antitrypsin deficiency: researchers have discovered why some patients remain healthy despite a genetic defect, while others develop severe liver ...