Further, the analyst believes investor attention should now shift to the company's emerging pipeline, where he sees a "significant opportunity" in the Phase 2-ready alpha-1 antitrypsin deficiency
"Additionally, we have rapidly grown our pipeline beyond CF, advancing seven new potential medicines across five disease areas, including beta thalassemia, sickle cell disease, alpha-1 antitrypsin deficiency
, APOL1-mediated kidney diseases and pain.
(NASDAQ: ARWR) today announced that it has dosed the first patient in SEQUOIA (AROAAT2001), a potentially pivotal Phase 2/3 clinical study of ARO-AAT, the company's second generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency
Its current pipeline focuses on new and effective treatments for Alpha-1 Antitrypsin Deficiency
(Alpha-1, or AATD) and genetic Amyotrophic Lateral Sclerosis (ALS.)
Beyond CF, Vertex has a pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency
, Duchenne muscular dystrophy and APOL1-mediated kidney disease.
INBRX-101 is a modified recombinant version of human alpha-1 antitrypsin, or AAT, for the treatment of patients with alpha-1 antitrypsin deficiency
, or AATD.
Summary: Looking at the current market trends as well as the promising demand status of the "Alpha 1 Antitrypsin Deficiency
Treatment Market", it can be projected that the future years will bring out positive outcomes.
[USPRwire, Fri May 17 2019] Looking at the current market trends as well as the promising demand status of the " Alpha 1 Antitrypsin Deficiency
Treatment Market ", it can be projected that the future years will bring out positive outcomes.
Alpha-1 or Alpha-1 Antitrypsin Deficiency
is a lack of the protein alpha-1 antitrypsin (AAT) which is produced in the liver.
for POL6014, a clinical stage selective inhibitor of human neutrophil elastase with the potential to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases such as non-cystic fibrosis bronchiectasis (NCFB), alpha-1 antitrypsin deficiency
(AATD) and primary ciliary dyskinesia (PCD).
The tot, from Longbenton, North Tyneside, suffers from a rare genetic condition called alpha 1 antitrypsin deficiency