pharmaphorum March 10, 2025
Armed with new proof-of-concept data showing that its in vivo base-editing therapy BEAM-302 can correct the gene mutation in alpha-1 antitrypsin deficiency (AATD), Beam Therapeutics is seeking to raise $500 million from investors to take the programme forward.
The Cambridge, Massachusetts biotech said this morning that a single dose of BEAM-302 given by an intravenous infusion was able to raise the total and functional levels of the AAT enzyme deficient in the disease and reduce the levels of the mutant, non-functional form.
Shares in the biotech on the Nasdaq were up around 15% after the news emerged, which Beam said is the first-ever clinical genetic correction of a disease-causing mutation with a single therapeutic administration, as well as an important...
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