Korro disclosed striking mouse pharmacology for a redosable GalNAc RNA editor in alpha-1 antitrypsin deficiency. The dual functional-protein restoration and mutant-protein reduction thesis is biologically attractive, but prior translation failure and absent human data…
The FDA cleared Prime Medicine’s IND for PM647, a one-time liver-directed prime editor intended to correct the SERPINA1 E342K PiZ mutation. A global open-label Phase 1/2 study may now begin in adults with lung-predominant…
Beam Therapeutics presented updated Phase I/II data for BEAM-302, an LNP-delivered adenine base editor intended to correct the SERPINA1 PiZ mutation in alpha-1 antitrypsin deficiency. At 60 mg, six lung-disease and five liver-disease participants…
Sentynl Therapeutics and Mereo BioPharma entered an option and license agreement for alvelestat in alpha-1 antitrypsin deficiency-associated lung disease. Sentynl receives an option for U.S. commercialization and global manufacturing rights; Mereo retains rest-of-world commercial…
Beam Therapeutics dosed the first participant in the global pivotal cohort of BEAM-302, an in vivo liver-directed base-editing therapy for alpha-1 antitrypsin deficiency. The cohort is embedded in the ongoing open-label Phase I/II study…
Tessera Therapeutics appointed Joseph Romanelli as president and chief executive officer, succeeding Michael Severino, who had led the Flagship-founded Gene Writing company since June 2022. Romanelli also joins Tessera’s board and Flagship Pioneering as…
Beam Therapeutics announced that updated Phase 1/2 BEAM-302 data will be presented in a late-breaking oral session at the European Respiratory Society Congress on September 8, 2026. The July 23 announcement is a catalyst…
A binding arbitration tribunal ruled that PM647, Prime Medicine’s investigational prime-editing therapy for Alpha-1 Antitrypsin Deficiency, falls within Prime Medicine’s contractual ‘Field’ as defined under the parties’ 2019 Collaboration and License Agreement with Beam…
Wave Life Sciences reported positive updated data from the RestorAATion-2 study of WVE-006, demonstrating substantial restoration of functional alpha-1 antitrypsin protein and significant reduction of toxic mutant protein in Alpha-1 Antitrypsin Deficiency (AATD).
Beam Therapeutics presented updated Phase 1/2 data for BEAM-302 at ATS 2026, reinforcing the company’s in vivo base-editing platform and supporting plans for an accelerated approval strategy in Alpha-1 Antitrypsin Deficiency (AATD).
