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 ...
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Alpha-1 antitrypsin deficiency: Study reveals why some patients with the hereditary disease remain healthy
Alpha-1-antitrypsin is a so-called protease inhibitor, a type of enzyme inhibitor. It is produced in the liver but exerts its effects in the lungs, where it regulates immune cell activity. This ...
Sanofi’s efdoralprin alfa met all primary and key secondary endpoints in alpha-1 antitrypsin deficiency emphysema phase 2 studyEfdoralprin alfa ...
Korro Bio, Inc. announced that its investigational drug KRRO-110 has received orphan drug designation from the FDA for treating Alpha-1 Antitrypsin Deficiency (AATD). This designation highlights the ...
Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...
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Rescued by fat bubbles: UTSW scientists treat rare genetic disease with designer molecule
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 ...
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 ...
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 Partners ...
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