Vertex may have pivoted away from the space, but candidates in development by Arrowhead/Takeda, Wave, Korro and others could ...

【参考文献】 [1] Wave Life Sciences Announces First-Ever Therapeutic RNA Editing in Humans Achieved in RestorAATion-2 Trial of ...

Background and aims Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder characterized by the misfolding and accumulation of the mutant variant of alpha-1 antitrypsin (AAT) within ...

for alpha-1 antitrypsin deficiency (AATD), a type of genetic disorder. WVE-006 is a GalNAc-conjugated oligonucleotide that enables A-to-I RNA editing and is administered subcutaneously.

Wave Life Sciences (NASDAQ:WVE) announced positive results from its phase 1b/2a RestorAAtion-2 study using WVE-006 for the treatment of patients with alpha-1 antitrypsin deficiency [AATD].

Regulatory filing of Phase 1/2 clinical study for Alpha-1 Antitrypsin Deficiency (AATD) submitted to Australian Bellberry Human Research Ethics ...

Initial Clinical Data for BEAM-101 and Preclinical Non-human Primate Data for ESCAPE Accepted for Presentation at American Society of Hematology ...

Achieved first-ever RNA editing in humans in RestorAATion-2 trial of WVE-006 (GalNAc-AIMer) in alpha-1 antitrypsin deficiency and announced three wholly owned GalNAc-AIMer preclinical programs that ...