In September 2023, The US Food and Drug Administration has approved Krystal Biotech’s Investigational New Drug (IND) ...

H.C. Wainwright analyst Patrick Trucchio reiterated a Buy rating and $80.00 price target on Beam Therapeutics Inc ...

A slew of pharma mergers and acquisitions in the first month of 2024 has continued with a $2.2 billion play by Sanofi for Inhibrx and its clinical-stage drug candidate for alpha-1 antitrypsin ...

Quantification of stool alpha-1 Antitrypsin (sAAT) excretion is established for estimation of enteric protein loss and assessment of disease activity. This study is to access the feasibility of ...

NTLA-3001 for alpha-1 antitrypsin deficiency-associated lung disease. Intellia joins a growing list of gene therapy companies that have had to make some hard decisions over the past several months ...

The therapy had initially pulled in support ... in geographic atrophy, and 4D-725, in alpha-1-antitrypsin deficiency lung disease. 4DMT has also decided not to invest anything into new preclinical ...

The discontinued programmes are headed by NTLA-3001, an in vivo CRISPR drug developed as a one-shot treatment for lung disease associated with alpha-1 antitrypsin deficiency (AATD). The inherited ...

CAMBRIDGE, Mass. - Wave Life Sciences Ltd . (NASDAQ:WVE), a clinical-stage biotechnology company with a market capitalization of $1.87 billion, has announced the commencement of its Phase 1 INLIGHT ...

Liver diseases can be inherited (such as Wilson disease and alpha 1-antitrypsin deficiency) or acquired (for example hepatitis and liver cancer). The performance of AAV gene-therapy vectors is ...