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, ...
Sanofi’s efdoralprin alfa met all primary and key secondary endpoints in alpha-1 antitrypsin deficiency emphysema phase 2 studyEfdoralprin alfa ...
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 ...
Topline results from the Phase 1 study showed a favorable safety and tolerability profile with no drug-related severe or serious adverse events. Topline data from the multiple ascending dose cohorts ...
PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that the first patient has been dosed in Takeda’s (TSE:4502/NYSE:TAK) Phase 3 REDWOOD clinical study of ...
Hosted on MSN
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 ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback