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).
Inhibrx Inc. and Sanofi SA have agreed to a deal worth up to $2.2 billion, whereby the latter’s Aventis Inc. subsidiary will acquire INBRX-101, an optimized, recombinant alpha-1 antitrypsin (AAT) augmentation therapy undergoing a registrational trial for AAT deficiency (AATD), an inherited genetic disorder caused by single nucleotide variants in the SERPINA1 gene.
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.
Beam Therapeutics Inc. has offered a progress update on its genetic disease franchise. BEAM-302, the company’s priority genetic disease program, is a potential treatment for α1-antitrypsin deficiency (AATD).
Korro Bio Inc. has nominated its first development candidate, KRRO-110, for the potential treatment of α1-antitrypsin deficiency. KRRO-110 is a proprietary RNA editing oligonucleotide delivered to liver cells using clinically validated LNP technology licensed from Genevant.
Arriving on the gene therapy scene with an undisclosed seed funding sum, Alveogene is tackling respiratory diseases with high unmet need via a next-generation lentiviral delivery platform to advance into the clinic a candidate for rare inherited disorder alpha-1 antitrypsin deficiency.
Krystal Biotech Inc. has received IND clearance from the FDA for KB-408 for the treatment of α1-antitrypsin deficiency (AATD). KB-408 is a modified, replication-defective, nonintegrating HSV-1-derived vector carrying two full-length copies of the serpin family A member 1 gene (SERPINA1) to enable expression of α1-antitrypsin (AAT).
Airna Corp. Inc. has launched through a $30 million financing to develop a pipeline of RNA editing therapeutics for treating rare diseases and prevalent diseases. It’s a wide spectrum of diseases made potentially more easily treatable because of the relatively recent revolution in RNA editing.
Though data won’t be available for a few years, the disclosure in mid-July that Grifols SA completed enrollment in the phase III study called Sparta caused some ears to perk up in the alpha-1-antitrypsin deficiency (AATD) space, where a number of contenders are busy.
Alveogene Ltd. has launched with a focus on inhaled gene therapies for respiratory disorders. The company has been created by Oxford Science Enterprises, Harrington Discovery Institute at University Hospitals, and Old College Capital in partnership with six scientists from the UK Respiratory Gene Therapy Consortium (GTC).
