Demonstrates modularity of Intellia’s in vivo liver insertion technology to durably restore protein, compared to traditional gene therapy Single-course administration of genome editing system provides ...

Beam Therapeutics (NASDAQ:BEAM) executives and clinical collaborators presented updated top-line data from the company’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, ...

BEAM-302 demonstrated safety and efficacy in increasing AAT levels and reducing mutant protein in alpha-1 antitrypsin deficiency patients. Beam Therapeutics announced promising initial results from ...

Achieved durable production of serum AAT protein at levels associated with a lower risk of AATD liver and lung diseases following repeat 200 mg doses of WVE-006 (11.9 µM total AAT, 7.2 µM M-AAT) First ...

Alpha-1 antitrypsin deficiency, AATD, is an inherited disease affecting the lung, liver, and, rarely, skin. Alpha-1 antitrypsin, AAT, is a protease inhibitor of the proteolytic enzyme elastase.

Treatment with 60 mg of BEAM-302 Led to Mean Steady-state Total AAT Level of 16.1 µM and All Patients Consistently and Durably Above the 11 µM Protective AAT Threshold with up to 12 Months of ...

Alpha-1 antitrypsin deficiency (AATD) is a recessive genetic disorder caused by single nucleotide variants (SNV) in the SERPINA1 gene encoding for alpha-1 antitrypsin (AAT), with the most common ...

Single dose of YOLT-202 was well tolerated with a favorable safety profile YolTech Therapeutics, a late clinical-stage biotechnology company developing in vivo gene editing therapies, today announced ...

Beam Therapeutics Inc. BEAM shares are up during Wednesday’s premarket session following the release of promising clinical data regarding its treatment BEAM-302. BEAM-302 is Beam’s lead genetic ...