The drug’s success in a Phase 2 study in alpha-1 antitrypsin deficiency “validates” Sanofi’s 2024 acquisition of Inhibrx, ...
Sanofi’s $1.7 billion rare disease bet from last year has delivered a phase 2 win. | Sanofi’s $1.7 billion rare disease bet ...
Sanofi’s efdoralprin alfa met all primary and key secondary endpoints in alpha-1 antitrypsin deficiency emphysema phase 2 studyEfdoralprin alfa ...
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SNY's Efdoralprin Alfa Meets Key Endpoints in Rare Disease Study
Sanofi's efdoralprin alfa meets all key endpoints in a global phase II study, showing potential as a first restorative ...
Alpha-1 antitrypsin deficiency (AATD) is a monogenic disease caused by mutations in the SERPINA1 gene, which encodes alpha-1 antitrypsin (AAT), a serine protease inhibitor mainly produced by ...
Beam Therapeutics recently announced that its BEAM-302 program received FDA orphan drug designation, alongside encouraging updates for its gene-editing therapies targeting rare diseases such as sickle ...
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