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 ...

Sanofi’s efdoralprin alfa met all primary and key secondary endpoints in alpha-1 antitrypsin deficiency emphysema phase 2 studyEfdoralprin alfa ...

Alpha-1 antitrypsin deficiency (AATD) is a hereditary condition characterised by the misfolding of the alpha-1 antitrypsin (AAT) protein, resulting in its hepatic accumulation and compromised protease ...

Never-smokers with severe α1-antitrypsin deficiency can still develop lung disease, including emphysema, bronchiectasis, and airflow obstruction.

In a major new development in the quest to develop better gene therapies with which to treat a host of diseases, researchers at the University of Massachusetts Amherst and UMass Chan Medical School ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), a leading RNAi therapeutics company, announced today that it has filed a Clinical Trial Application (CTA) with the U.K.

Kamada shows strong Q2 2023 financials with 59% YoY revenue jump, led by KEDRAB sales, and solid liquidity. Despite growth, AAT deficiency project introduces uncertainty due to weak Phase 2 results, ...

Alpha-1 antitrypsin deficiency, or AATD, is an inherited disease that affects the lung, liver, and skin. The current treatment landscape involves smoking cessation, COPD treatment, and augmentation ...

LONDON — The novel liver-targeting agent fazirsiran has fared well in a small, but significant, study looking at its ability to improve liver histology in adults with alpha-1 antitrypsin (AAT) ...

Expanded Dose Exploration in Part A and Dose Escalation in Part B of BEAM-302 Phase 1/2 Study in Alpha-1 Antitrypsin ...

NESS ZIONA, Israel--(BUSINESS WIRE)--Kamada Ltd. (NASDAQ:KMDA) (TASE:KMDA), a plasma-derived protein therapeutics company focused on orphan indications, reports that the Israeli Patent Office (ILPO) ...