Biotechnology company Beam Therapeutics has reported encouraging data for its treatment for the genetic disease Alpha-1 Antitrypsin Deficiency (AATD). Beam announced BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation designed to directly correct the underlying genetic mutation that causes AATD, has shown a well-tolerated safety profile and efficacy in a… Read More
Biotech Tessera Therapeutics has presented updates across its in vivo genetic medicine programmes for Alpha-1 antitrypsin deficiency (AATD), Phenylketonuria (PKU), and Sickle Cell Disease (SCD), as well as advances in in vivo T cell therapies. These data were shared across four oral presentations and three poster presentations at the American Society of Gene and… Read More
Korro Bio, a biopharmaceutical company focused on developing a new class of genetic medicines for both rare and highly prevalent diseases, has nominated its first development candidate, KRRO-110, for the potential treatment of alpha-1 antitrypsin deficiency (AATD). KRRO-110 is an RNA editing oligonucleotide delivered to liver cells… Read More
