AATD News Briefs

liver scan

Transient Elastography Useful in Identifying AATD-Related Liver Disease

Transient elastography seems to be more useful than ultrasonography in identifying liver disease related to alpha-1 antitrypsin deficiency (AATD), according to a study presented at the American Thoracic Society (ATS) 2022 International Conference. The authors of the study said that larger prospective studies are needed to establish the use of the technique in staging liver…

high risk pregnancy

Term Pregnancy and Successful Delivery in a Patient With AATD

Pregnancy for patients with severe alpha-1 antitrypsin deficiency (AATD) should be considered on a case-by-case basis, a case report published in Archivos de Bronconeumonología suggests. Patients with AATD typically have high-risk pregnancies due to several possible complications. Although scarce studies describing pregnancy outcomes in patients with AATD exist, previous literature has suggested that due to the high…

Neutrophil

AATD Treatment Benefits Neutrophil Adhesion

Treatment for alpha-1 antitrypsin deficiency (AATD) could deliver additional cellular benefits that modulate other factors in the physiopathology of the disease, according to an experimental study recently published in the American Journal of Respiratory Cell and Molecular Biology. Alpha-1 antitrypsin (AAT) augmentation therapy demonstrated in vitro antiinflammatory activity by diminishing controlled neutrophil adhesion, as reported…

bronchial tree

INBRX-101 Maintains Functional AAT Levels in Phase 1 Trial

Inhibrx Incorporated announced favorable initial results of its phase 1 clinical trial on the safety, pharmacokinetics, and pharmacodynamics of the optimized recombinant human alpha-1 antitrypsin (AAT)-Fc protein therapy INBRX-101 for patients with alpha-1 antitrypsin deficiency (AATD). The results, announced via news release, showed favorable safety and tolerability, and no drug-related severe or serious adverse events.…

blood test tube

Are C-Reactive Protein Levels Required for Diagnosing AATD?

C-reactive protein measurement may not be necessary to diagnose alpha-1 antitrypsin deficiency (AATD) if patients are evaluated for alpha-1 antitrypsin (AAT) levels and undergo genotyping at the same time, according to a prospective observational analytic study published in the Clinical Respiratory Journal. For many years, it has been suggested to measure C-reactive protein levels in…

Emphysema of the lungs, CT scan

Alvelestat Shows Effectiveness Against Lung Disease Related to AATD in Phase 2 Trial

Mereo BioPharma released positive results from its phase 2 clinical trial of alvelestat, an investigational oral neutrophil elastase (NE) inhibitor being developed to treat patients with severe alpha-1 antitrypsin deficiency-associated lung disease (AATD-LD). “These positive top-line results demonstrate the clear impact of alvelestat on key known biomarkers along the pathogenic pathway of AATD-LD,” Denise Scots-Knight,…

experimental AATD therapies

Development of AIMers on Track for the Potential Treatment of AATD

New preclinical data confirming the effects of GalNAc-conjugated SERPINA1 AIMers in restoring the production of functional alpha-1 antitrypsin (AAT) protein in the serum of a mouse model of AAT deficiency (AATD) were recently presented at the TIDES USA: Oligonucleotide & Peptide Therapeutics Conference.  Previously, researchers had reported that the SERPINA1 AIMers led to around 60%…

infusion

Novel Recombinant AAT May Be Superior to Human-Derived AAT for AATD

A novel recombinant alpha-1 antitrypsin (AAT) protein showed promising results as a potential treatment for patients with alpha-1 antitrypsin deficiency (AATD), according to an article recently published in Biotechnology and Bioengineering.  During in vitro experiments, recombinant AAT exhibited inhibitory activity comparable to human plasma derivatives with higher oxidation resistance, as reported by Koyuturk and colleagues. …

genetic testing

Rare New Variant Identified in Alpha-1 Antitrypsin Deficiency

Researchers recently discovered a Q0parma mutation in 3 patients with alpha-1 antitrypsin deficiency (AATD) presenting with pulmonary manifestations and published their results in Respirology Case Reports. This finding helps expand the list of null alleles known to occur within the serpin family A member 1 (SERPINA1) gene, underlining the importance of genetic screening and implementation…

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