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 ...

The issue is that six years ago, Prime appeared to sign away its rights to create AATD treatments to another company it has long collaborated with, Beam Therapeutics.

Beam Therapeutics faces slow pipeline progress and high R&D costs despite a $500M cash boost. Learn why patience is key for ...

Patients with the condition Beam targeted, alpha-1 antitrypsin deficiency, or AATD, generally have a single misspelled letter in the gene that codes for AAT, a protein that helps protect the lungs.

BEAM-302 targets the liver and is designed to use CRISPR to precisely cut and fix the single errant letter of DNA that causes the majority of AATD cases, a mutation called PiZ. Patients with the ...

along with its BEAM-302 base-editing candidate for alpha-1 antitrypsin deficiency (AATD) and BEAM-301, an in vivo lipid nanoparticle (LNP) therapy for glycogen storage disease 1a. Roche has ...

BEAM-302 showed durable, dose-dependent correction of AATD mutation in 9 patients with effects seen as early as Day 7. Beam plans further dose escalation and will report new trial data in H2 2025 ...

Positive initial safety and efficacy data from the Phase 1/2 trial of BEAM-302 were previously reported in March 2025, establishing clinical proof of concept as a potential treatment for AATD and ...

We look forward to continuing dose escalation and accelerating the development of BEAM-302 for patients with AATD who urgently need more effective therapeutic options. Importantly, these data are ...