Beam Therapeutics

Type: Company (clinical-stage base editing) Ticker: BEAM (Nasdaq) Founded by: David Liu, Feng Zhang, J. Keith Joung IP: Owns the foundational base-editing IP; licenses to Verve (cardiovascular).

Beam is the company built on base editing — single-base DNA conversion (A→G / C→T) with no double-strand break, the "safer CRISPR." By 2026 it has clinical or IND-stage programs across three indications, almost all via LNP delivery to the liver for in vivo correction (plus an ex vivo sickle-cell program). Its differentiator vs Intellia: base editing corrects a disease-causing letter rather than knocking a gene out.

Key Contributions

• BEAM-302 — first-ever genetic correction in AATD patients — In vivo ABE correcting the PiZ (Z-AAT) mutation; single IV doses (15–60 mg) gave dose-dependent, durable AAT increases; top cohort reached disease-halting levels. (Beam clinical 2026)
• BEAM-304 IND cleared for PKU (Jun 18 2026) — In vivo ABE correcting PAH (R408W first); preclinical mice showed robust liver editing and normalized plasma phenylalanine; Phase 1/2 planned to test dietary liberalization. (Beam clinical 2026)
• BEAM-101 — >90% disease-protein reduction in sickle cell — Ex vivo, sustained ≥36 weeks, eliminates busulfan conditioning; a treatment-related death is under monitoring. (Beam clinical 2026)

Mentioned In

• Base Editing — Beam is the flagship base-editing company.
• CRISPR Clinical Translation — part of the in vivo clinical wave.

Related Entities

• Intellia Therapeutics — in vivo knockout counterpart; first to Phase 3.
• David Liu — invented base editing (and prime editing); Beam co-founder.