Biotech product

CRISPR therapies (Casgevy + base/prime editing)

Casgevy approval (December 2023) marked first FDA-approved CRISPR therapy; 23+ clinical trials now active with base/prime editing showing efficacy in rare genetic diseases and potential for broader indications.

What to watch next

Intellia's FDA approval filing 2026, Beam and Prime Medicine accelerated approvals late 2026-2027, expanded in vivo CRISPR programs, and next-generation base/prime editors with improved efficiency and safety profiles.

Key sub-ideas & techniques

• Ex vivo CRISPR/Cas9 (Casgevy) — CRISPR Therapeutics + Vertex's Casgevy was the first FDA-approved CRISPR therapy (Dec 2023) — ex vivo edit of HSCs to reactivate fetal hemoglobin in sickle-cell disease and beta-thalassemia. [source]
• Base editing — David Liu's base editors (cytosine and adenine) make single-letter DNA changes without double-strand breaks — Beam (BEAM-101 for sickle cell, BEAM-302 for AATD) and Verve (PCSK9 for cardiovascular) are the lead programs. [source]
• Prime editing — Liu lab's prime editing, the 'search-and-replace' of genome editing, achieved its first-ever human evidence in 2025 with PM359 in chronic granulomatous disease, on an FDA accelerated-approval path. [source]
• In vivo CRISPR via LNP — Intellia's NTLA-2002 (lonvoguran ziclumeran) for hereditary angioedema cut attacks ~87% in Phase 3 — the first Phase-3-positive in-body CRISPR therapy and a template for the rest of the genome. [source]
• Epigenome editing — dCas9 fused to silencing or activating domains can switch genes off or on without cutting DNA — Tune Therapeutics, Chroma Medicine, and Epic Bio are running the first programs (Tune's TUNE-401 for HBV in trials). [source]

Current frontier

• Casgevy (exagamglogene autotemcel) FDA-approved December 8, 2023, for sickle cell disease in patients ≥12 years; uses CRISPR/Cas9 to induce fetal hemoglobin, eliminating vaso-occlusive crises in clinical trials. [source]
• Intellia's lonvoguran ziclumeran (in vivo CRISPR therapy) reduced hereditary angioedema attacks by 87% vs. placebo in Phase 3 HAELO trial (announced April 27, 2026); rolling FDA BLA submission initiated. [source]
• Beam Therapeutics' BEAM-302 (base editing for alpha-1 antitrypsin deficiency) dosed 29 patients with well-tolerated safety profile and no dose-limiting toxicities; FDA RMAT designation granted; optimal 60 mg dose selected for pivotal development. [source]
• Prime Medicine's PM359 (prime editing ex vivo therapy for chronic granulomatous disease) shows first human safety/efficacy data with FDA 'plausible mechanism' pathway grant; company pursuing accelerated approval with potential 2026 alignment meeting. [source]
• As of 2026, 23+ clinical trials are active using base editing or prime editing for leukemias, hypercholesterolemia, AATD, sickle cell disease, beta-thalassemia, and chronic granulomatous disease. [source]

Key people

• Feng Zhang James and Patricia Poitras Professor of Neuroscience; Core Member Broad Institute · MIT McGovern Institute; Broad Institute of MIT and Harvard [source]
• David Liu Richard Merkin Professor; Director Merkin Institute; HHMI Investigator · Harvard University; Broad Institute of MIT and Harvard [source]
• Editas Medicine/CRISPR Therapeutics Leadership CEO (Casgevy development) · CRISPR Therapeutics; Vertex Pharmaceuticals (co-developer) [source]
• John Leonard Chief Executive Officer · Intellia Therapeutics [source]
• Keith Gottesdiener Chief Executive Officer · Prime Medicine [source]

Startups & labs to watch

• Prime Medicine (prime editing platform) Prime Medicine Inc. · STARTUP · Series B: $200M+ (2022); backed by Jeff Bezos and others — First-ever prime editing human data (PM359 for CGD); pursuing accelerated approval pathway 2026; expects clinical data may support approval with FDA alignment meeting ongoing. [source]
• Verve Therapeutics (base editing for cardiovascular disease) Verve Therapeutics Inc. · STARTUP · Public company; venture-backed with multiple funding rounds — VERVE-102 Phase 1b data showed ~50% LDL reduction in highest-dose patients; shifted to improved LNP formulation after VERVE-101 liver enzyme abnormalities; targeting PCSK9 base editing. [source]
• Intellia Therapeutics (in vivo CRISPR for rare diseases) Intellia Therapeutics Inc. · STARTUP · Public company; multiple funding rounds and partnerships — First Phase 3 in vivo CRISPR data (hereditary angioedema); rolling FDA submission 2026; expects U.S. launch H1 2027 if approved; expanding to other rare genetic diseases. [source]
• Beam Therapeutics (base editing for genetic disease) Beam Therapeutics Inc. · STARTUP · Public company; founded by Liu, Barrangou, and others with $300M+ raised — BEAM-302 advancing to pivotal development for AATD with 60 mg optimal dose; BEAM-301 (GSD-Ia) data expected 2026; RMAT designation supporting accelerated pathway. [source]
• EDIT-401 (LDLR upregulation, in vivo gene editing) Editas Medicine · STARTUP · Public (NASDAQ: EDIT) — Lead in vivo program targeting hyperlipidemia/HeFH; >=90% LDL-C lowering in NHPs; FIH expected 2026 with human PoC by year-end; positions Editas as direct competitor to Verve/Lilly in cardiovascular gene editing. [source]