Krystal Biotech, Inc. KRYS announced initial clinical data updates on its two inhaled genetic medicine pipeline candidates, ...

Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder associated with liver disease, ranging from fibrosis to ...

Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder associated with liver disease, ranging from fibrosis to hepatocellular carcinoma. The disease remains asymptomatic until its final ...

Shares in Wave Life Sciences rose by 74% after it reported clinical data with a pioneering new RNA-editing therapy for alpha-1 antitrypsin deficiency (AATD). Wave claims this is the first clinical ...

The new Inhibrx will also start with $200 million in funding from the French drugmaker. AATD is an inherited disorder whose primary consequence is life-threatening pulmonary emphysema, resulting ...

Korro Bio has secured approvals in Australia to commence the two-part Phase I/IIa REWRITE trial of its investigational product, KRRO-110, aimed at treating alpha-1 antitrypsin deficiency (AATD).

Korro Bio Inc. has announced a submission to the Australian Bellberry Human Research Ethics Committee (HREC) for a phase I/II study of KRRO-110 for α-1 antitrypsin deficiency (AATD). BioWorld Science ...

WVE-006 was developed using Wave’s oligonucleotide chemistry platform and is designed to address AATD-related lung disease and/or liver disease. Wave said the proof-of-mechanism data are “the ...

Alvelestat is another candidate in its pipeline, which is being developed for the treatment of patients with alpha-1 antitrypsin deficiency-associated lung disease [AATD-LD]. However, the company ...

Net Loss: Korro's net loss was $21.0 million for the three months ended September 30, 2024, as compared to $18.5 million for the same period in 2023. AATD is a genetic disorder most commonly caused by ...

Beam Therapeutics Begins Dosing in Phase I/II AATD Study Beam Therapeutics Inc. (NASDAQ: BEAM) announced that it has dosed the first patient in the phase I/II study, evaluating its investigational ...