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BioWorld - Sunday, May 3, 2026
Home » AATD

Articles Tagged with ''AATD''

Single strand RNA

Backtrack to GalNAc: Korro AATD strategy Rewrite post-fizzle

Nov. 13, 2025
By Randy Osborne
No Comments
Korro Bio Inc.’s latest update on RNA editing prospect KRRO-110 may mean one less competitor in alpha-1 antitrypsin deficiency (AATD), and shares of the firm (NASDAQ:KRRO) closed Nov. 13 at $6.50, down $24.92, or 79%. As part of the third-quarter earnings report, Korro said KRRO-110 produced functional protein in AATD patients but fell short of projected levels of functional protein after a single administration.
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Single strand RNA
Genetic/congenital

Airna’s $155M series B advances RNA editing drug AIR-001 for AATD

April 2, 2025
By Karen Carey
As it prepares to advance its lead RNA editing candidate, AIR-001, into a phase I/II trial for alpha-1 antitrypsin deficiency, Airna Corp. Inc. closed an oversubscribed $155 million series B financing less than a year after completing its series A round. The company, based in Cambridge, Mass., with research operations in Tübingen, Germany, focuses not only on repairing harmful genetic variants found in rare genetic disorders, but also on introducing beneficial variants that improve health in common conditions.
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Single strand RNA

Airna’s $155M series B advances RNA editing drug AIR-001 for AATD

April 1, 2025
By Karen Carey
As it prepares to advance its lead RNA editing candidate, AIR-001, into a phase I/II trial for alpha-1 antitrypsin deficiency, Airna Corp. Inc. closed an oversubscribed $155 million series B financing less than a year after completing its series A round. The company, based in Cambridge, Mass., with research operations in Tübingen, Germany, focuses not only on repairing harmful genetic variants found in rare genetic disorders, but also on introducing beneficial variants that improve health in common conditions.
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Scientist, microscope and dropper
Endocrine/metabolic

Prime Medicine announces new α1-antitrypsin deficiency program

March 19, 2025
Prime Medicine Inc. has announced its new preclinical program for the treatment of α1-antitrypsin deficiency (AATD). The program is advancing through its final stages of lead optimization, with an IND and/or clinical trial application (CTA) filing anticipated around the middle of next year.
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Liver and lungs
Endocrine/metabolic

Korro Bio files in Australia to begin first-in-human study of KRRO-110 for AATD

Nov. 5, 2024
Korro Bio Inc. has announced a submission to the Australian Bellberry Human Research Ethics Committee (HREC) for a phase I/II study of KRRO-110 for α-1 antitrypsin deficiency (AATD).
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Dollar sign droplet above test tube
Genetic/congenital

Airna’s series A to bring lead project into clinic, develop pipeline

Aug. 2, 2024
Airna Corp. Inc., of Cambridge, Mass., announced it has closed an oversubscribed $60 million financing round, bringing its total series A funding to $90 million.
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Scissors and gold DNA on black blackground
Genetic/congenital

Gene-editing therapeutic YOLT-202 shown to restore α1-antitrypsin deficiency

May 22, 2024
α1-Antitrypsin deficiency is a hereditary disorder that affects the liver in children and adults, as well as the lungs in adults. The disease is mostly caused by the Z allele mutation in the SERPINA1 gene, where a glutamic acid amino acid is substituted by lysine (E342K) leading to protein misfolding and aggregation.
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mRNA on digital background
Drug Design, Drug Delivery & Technologies

Circio establishes in vivo proof of concept for Circvec circular RNA platform

April 18, 2024
Circio Holding ASA has established technical in vivo proof of concept for its proprietary Circvec circular RNA platform by demonstrating statistically significant improvement in durability over mRNA-based expression.
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Microscope
Endocrine/Metabolic

UK clearance for phase I/II trial of Beam’s BEAM-302 for α1-antitrypsin deficiency

March 27, 2024
Beam Therapeutics Inc. has received clearance of its clinical trial authorization (CTA) application by the U.K.’s Medicines and Healthcare Products Regulatory Agency for BEAM-302, an in vivo base editor, as a potential treatment for patients with α1-antitrypsin deficiency (AATD).
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Endocrine/Metabolic

Korro presents data for KRRO-110 for α-1 antitrypsin deficiency

Jan. 19, 2024
Korro Bio Inc. has highlighted new data for KRRO-110, its first development candidate for the potential treatment of α-1 antitrypsin deficiency (AATD), an inherited genetic disorder caused by single nucleotide variants in the SERPINA1 gene.
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