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 ...
YolTech Therapeutics, a late clinical-stage biotechnology company developing in vivo gene editing therapies, today announced ...
SAN DIEGO, Oct. 4, 2022 /PRNewswire/ -- Inhibrx, Inc. (Nasdaq: INBX), a clinical-stage biopharmaceutical company dedicated to the development of therapeutics for oncology and rare diseases, announced ...
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, ...
- Treatment with VX-864 led to a statistically significant increase from baseline in plasma functional AAT levels as compared to placebo and was generally well tolerated - - Results provide ...
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 ...
The FDA has approved Prolastin-C (alpha1-proteinase inhibitor [human], from Talecris Biotherapeutics), a more concentrated formulation of Prolastin, for the treatment of alpha1-antitrypsin (AAT) ...
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 ...
Vertex Pharmaceuticals Incorporated VRTX announced that it will not pursue late-stage development of its investigational oral small molecule corrector, VX-864, being evaluated for the treatment of ...
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