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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]
• In vivo prime editing reaches the clinic — Prime Medicine's PM577a, an LNP-formulated prime editor delivered IV to hepatocytes, is the first in vivo prime editing therapy authorized for human trials, correcting the ATP7B H1069Q mutation in Wilson Disease. [source]
• Caribou CB-011 allogeneic CRISPR CAR-T — CRISPR-edited allogeneic anti-BCMA CAR-T immune-cloaked via B2M-knockout/HLA-E-fusion; 92% ORR, 83% CR/sCR, 91% MRD-negativity in a 12-patient r/r multiple myeloma cohort, 50% still in CR at 15 months (CaMMouflage phase 1, EHA 2026). [source]
• AstraZeneca/Neogene NT-175 CRISPR TCR-T (solid tumors) — CRISPR-edited (TCR + TGFβR2 knockout) autologous T-cell therapy targeting the shared TP53 R175H neoantigen; acceptable safety and early anti-tumor efficacy signals in a phase 1 across pancreatic, colorectal and breast tumors (ASCO 2026, Abstract 2506). [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]
• Intellia's lonvo-z became the first in vivo CRISPR therapy to win a positive pivotal Phase 3 (HAELO), cutting hereditary angioedema attacks 87% vs placebo (p<0.0001) with 62% attack-free over 6 months; NEJM-published, rolling BLA underway for 1H 2027 launch. [source]
• Autolus' fast-off-rate CD19 CAR-T obe-cel showed 83% (5/6) remission (per standard lupus criteria) at target dose, with several complete/partial renal responses, in severe refractory lupus in the Phase 1 CARLYSLE trial, presented at EULAR 2026. [source]
• Beam BEAM-304 (in vivo LNP adenine base editor for PKU/PAH) cleared FDA IND June 18 2026; preclinical normalization of plasma Phe. [source]
• Dual CD19/BCMA CAR-T (Beijing GoBroad Boren) induced sustained remission in 11 refractory systemic-sclerosis patients (EULAR 2026). [source]
• VERVE-102 (Verve/Lilly) in vivo base editing cut PCSK9 ~88% and LDL-C ~62% (n=35, Heart-2), durable >1yr, no dose-limiting toxicities (NEJM). [source]
• bioRxiv preprint: in vivo epigenome editing gave the most durable PCSK9 silencing vs nuclease/deaminase editing and siRNA (mRNA editors in Arcturus LUNAR LNPs, mice). [source]
• CASTLE basket trial (EULAR 2026): CD19 CAR-T zorpocabtagene autoleucel — 9/10 SLE patients in DORIS remission at 6 mo, drug-free through 24 wk; 1/28 SLE relapse at median 13 mo. [source]
• EHA 2026: CARsgen allogeneic CAR-T CT0596 (BCMA, 8 pts) and CT1190B (CD19/CD20, 13 lymphoma pts); Phase Ib planned 2026. [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]
• Autolus Therapeutics Autolus Therapeutics plc · STARTUP · Public (Nasdaq: AUTL) — Commercial-stage CAR-T company (obe-cel/Aucatzyl approved in B-ALL) extending its fast-off-rate CD19 platform into autoimmune disease (lupus), a fast-emerging high-value indication for cell therapy. [source]
• Caribou Biosciences Caribou Biosciences (allogeneic CRISPR CAR-T) · COMPANY — Entity reconciliation: developer of CB-011 (off-the-shelf, immune-cloaked anti-BCMA CAR-T); a lead player in the allogeneic/in-vivo cell-engineering gap theme. [source]