In Vivo Gene Editing Goes Clinical: Funding, AI, and Delivery Breakthroughs

In Vivo Gene Editing Goes Clinical: Funding, AI, and Delivery Breakthroughs

Published Nov 16, 2025

Biotech funding and deals in late 2025 highlight accelerating in vivo gene editing: Azalea Therapeutics, co‐founded by CRISPR pioneer Jennifer Doudna, closed an $82M Series A to develop a single‐dose dual‐vector in vivo CAR‐T for B‐cell malignancies and plans clinical trials within 12–18 months; Editas Medicine has formally shifted to a fully in vivo CRISPR focus after preclinical data showing ~40% editing of the HBG1/2 promoter in HSCs, ~20% fetal hemoglobin expression in humanized mice at one month, and efficient liver editing in non‐human primates, targeting human proof‐of‐concept by 2027 with runway into Q2 2027; AstraZeneca agreed to spend up to $555M with Algen for AI‐driven target discovery. These developments affect patient impact, market opportunity and partnership activity, but regulatory, safety (off‐target/immunogenicity), durability and manufacturing hurdles remain; near‐term milestones include IND/CTA filings in 2026 and clinical readouts in 2026–2027.