-- High levels of editing at the target site in preclinical studies with full restoration of circulating wild-type AAT protein (M-AAT) to normal human range -- -- Expects to file IND and/or CTA in mid ...

Alpha-1 antitrypsin deficiency, or AATD, is an inherited disease that affects the lung, liver, and skin. The current treatment landscape involves smoking cessation, COPD treatment, and augmentation ...

Sanofi is buying Inhibrx in a $1.7 billion deal as the French pharmaceutical giant expands its pipeline of drugs that can treat rare diseases. The agreement will give Sanofi access to Inhibrx's ...

Alpha-1 antitrypsin (AAT) deficiency is a genetic disease where your body doesn’t make enough normal functioning AAT. AAT is a protein your liver makes. AAT has many important roles in your body, ...

CAMBRIDGE, Mass., March 14, 2025 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on ...

(RTTNews) - Beam Therapeutics Inc. (BEAM) announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-302, a ...

CRISPR Therapeutics has developed SyNTase editing, a proprietary, next-generation, site-specific gene correction platform.

DALLAS – Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 antitrypsin deficiency — a promising leap toward treating humans with this ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Least-squares mean percent differences in serum Z-AAT ...

Alpha-1 antitrypsin deficiency (AATD) is a hereditary condition characterised by the misfolding of the alpha-1 antitrypsin (AAT) protein, resulting in its hepatic accumulation and compromised protease ...