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Technology · Synbio & enabling tech / Genome engineering

Genome editing

Programmable genome editors — CRISPR-Cas9, base editors and prime editors — delivered ex vivo or in vivo via lipid nanoparticles to treat genetic disease, from the first approved CRISPR therapy Casgevy to in-vivo redosable CRISPR and single-dose cardiovascular base editing.

Risk: medium Research tools & R&D services US · EU · CN Science: why it works →Forums and policy: 48 verified 2026-07-28 · 11 Sources (0 independent)

1 · What it gives

Genome editing rewrites DNA in living cells with programmable nucleases — CRISPR-Cas9, base editors and prime editors — to treat genetic disease, engineer cells and build biological platforms. CRISPR Therapeutics’ Casgevy, the first approved CRISPR therapy, edits autologous CD34+ stem cells ex vivo to induce fetal hemoglobin, with Phase 3 CLIMB-151 showing 73% of treated children free of vaso-occlusive crises for at least 12 months (median about 19 months) and the FDA now reviewing expansion to children as young as 5. Intellia’s in-vivo lipid-nanoparticle-delivered CRISPR (NTLA-2001) achieved a median 52% reduction in serum TTR, and redosing reached a 90% median knockdown at day 28 — the first clinical redosing of an in-vivo CRISPR therapy. Base editing has moved into cardiovascular care: Lilly/Verve’s VERVE-102 cut PCSK9 by up to 88% and LDL-C by up to 62% from a single infusion (NEJM). Prime editing (“search-and-replace”) now reaches the liver in vivo via all-RNA LNP platforms, covering the vast majority of known disease-causing mutations.

The key directions of genome editing are:

  1. Ex-vivo CRISPR-Cas9 cell therapy (CRISPR-Cas9 Cell Therapy): Casgevy edits autologous hematopoietic stem cells to induce fetal hemoglobin for sickle cell disease and beta-thalassemia, now expanding to younger patients.
  2. In-vivo CRISPR via LNP delivery (In-vivo CRISPR): Intellia’s NTLA-2001 (ATTR amyloidosis) and lonvo-z (hereditary angioedema, rolling BLA in 2026) enable one-time, redosable outpatient editing.
  3. Base editing (Base Editing): Beam’s BEAM-304 (PKU), BEAM-302 (AATD) and BEAM-101 (SCD), plus Verve/Lilly’s VERVE-102, which cut LDL-C by up to 62% from a single PCSK9 edit.
  4. Prime editing & search-and-replace (Prime Editing): Prime Medicine and the Broad Institute advance precise substitutions and small indels, with all-RNA LNP delivery enabling in-vivo prime editing and an AI-redesigned reverse transcriptase.

Sectoral value chain#

[Target & guide design] ──> [Editor engineering] ──> [Delivery] ──> [Editing & selection]
                                  │
                          (LNP / viral / electroporation)
                                  │
                                  ▼
[Approved gene medicine] <─── [Clinical & regulatory] <─── [Manufacturing]

Value chain levels#

LevelDescriptionKey inputs/outputs
Target & guide designA disease gene is selected and a guide RNA / editor architecture is designed against itIn: Disease gene, reference genome.
Out: Guide RNA, editor design.
Editor & payload engineeringThe Cas9 / base / prime editor is optimized and packaged as mRNA + guideIn: Guide RNA, editor protein.
Out: mRNA editor + guide payload.
DeliveryPayload is delivered ex vivo (electroporation) or in vivo (LNP, viral)In: Payload, patient cells.
Out: Edited cells or dosed patient.
Editing & selectionCells are edited and, for ex-vivo therapies, the desired edit is selected/expandedIn: Delivered payload.
Out: Edited cell population.
ManufacturingClinical-grade drug substance and product are produced (viral/LNP/cell therapy)In: Edited cells or mRNA payload.
Out: Clinical-grade therapy.
Clinical & regulatoryTrials run under RMAT/ATMP pathways to a marketing authorizationIn: Clinical-grade therapy.
Out: FDA/EMA/NMPA-approved gene medicine.

Cross-cutting technologies of the sector:

  • CRISPR-Cas9 & base/prime editors (Editing Modalities): the three editor classes that define what DNA change is possible — cuts, single-base swaps, precise substitutions.
  • Lipid-nanoparticle & viral delivery (Delivery): LNP (mRNA + guide) and viral vectors that carry editors into cells and organs, especially the liver.
  • AI-assisted editor design (AI Editor Design): machine-learning redesign of editor enzymes (e.g. reverse transcriptase) to boost stability, abundance and potency.

2 · What it replaces 0 · matched by industry

No graded substitution linked yet.

3 · How to implement 6 steps

  1. 1
    Target & guide design
    A disease-causing gene is selected and a guide RNA (plus editor architecture) is designed against the target locus, using the reference genome and predictive models of editing efficiency and off-target risk.
  2. 2
    Editor engineering
    The Cas9, base or prime editor is optimized — including AI-assisted redesign of components such as the reverse transcriptase — and packaged as mRNA plus chemically modified guide RNA.
  3. 3
    Delivery
    The payload is delivered ex vivo into harvested cells by electroporation, or in vivo to the patient via lipid nanoparticles (often GalNAc-conjugated for liver targeting) or viral vectors.
  4. 4
    Editing & selection
    For ex-vivo therapies the desired edit is confirmed and the edited cell population (e.g. CD34+ stem cells) is selected and expanded; for in-vivo therapies, editing is confirmed via circulating biomarkers (TTR, PCSK9, kallikrein).
  5. 5
    ManufacturingContract manufacturing (CMO/CDMO)
    Clinical-grade drug substance and product are manufactured — autologous edited cell products under GMP cell-processing, or LNP-mRNA drug product under aseptic fill-finish.
    Who can do this step matched automatically
    Cell AgriTech Malaysia
    5,000 L industrial scale
    “At Cell AgriTech, we position ourselves as Asia’s first dedicated CDMO for cultivated meat and plant cell culture.”
    Ajinomoto Japan
    industrial scale
    “It remains highly active, focusing on its 2030 roadmap through business model transformation and expansion in CDMO services and high-value-added materials.”
    Space F South Korea
    “Space Rintec's Blueprint for Space CRO and CDMO - EDAILY — Space Rintech (Space Lintech) plans full-scale commercialization of its space CRO/CDMO business next year, targeting 10 …”
    Taiwan Sugar Corporation (TSC) Taiwan
    500,000 tons pilot scale
    “It refines sugar (including refined white sugar, golden brown sugar, fine granulated sugar and invert sugar syrup), produces bio-based chemicals/biotech outputs (alcohol, …”
  6. 6
    Clinical & regulatoryRegulatory & certification
    Trials run under accelerated pathways (FDA RMAT, EMA ATMP, NMPA gene-therapy review) toward a marketing authorization, exemplified by Casgevy’s approvals and Intellia’s rolling BLA for lonvo-z in 2026.
    Registration in Russia / EAEU: Regulation

4 · Who delivers

Matched suppliers (matched automatically)

ToolGen (KOSDAQ)South KoreaNot classifiedactive
PairwiseUnited StatesResearch / RTOactive
Sanatech SeedJapanResearch / RTOactive
KWS SaatGermanyServices / consultingactive
Origin Agritech (NASDAQ: SEED)ChinaResearch / RTOactive
Shandong Shunfeng BiotechnologyChinaProduceractive
Inari AgricultureUnited StatesProduceractive
CibusUnited StatesResearch / RTOactive

In the services and supply catalogue 1 · paid service

Reagents & kits

Leading companies and institutes (from the brief)

Company / InstituteCountryKey products / platformsTech featuresStatus 2026
CRISPR Therapeutics🇨🇭 SwitzerlandCasgevy (CRISPR-Cas9)First approved CRISPR therapy; SCD/TDTcommercial
Intellia Therapeutics🇺🇸 USAIn-vivo CRISPR (LNP)NTLA-2001 ATTR; lonvo-z HAE BLA 2026commercial
Beam Therapeutics🇺🇸 USABase-editing platformBEAM-304 PKU; BEAM-302 AATDclinical
Prime Medicine🇺🇸 USAPrime editing (search-and-replace)In-vivo LNP prime editingclinical
Verve Therapeutics🇺🇸 USACardiovascular base editingVERVE-102 PCSK9 −62% LDL-C (Lilly)clinical
EdiGene🇨🇳 ChinaChina CRISPR therapeuticsET-01 β-thalassemia (first NMPA-cleared)clinical
Editas Medicine🇺🇸 USAin-vivo CRISPR therapeuticsEDIT-401 LDLR upregulation; IND mid-2026preclinical
Caribou Biosciences🇺🇸 USAchRDNA CRISPRCB-010 allogeneic CAR-Tclinical
Tessera Therapeutics🇺🇸 USAGene Writing platformmobile-element insertional editingclinical
Scribe Therapeutics🇺🇸 USACasX X-edit platformengineered CasX, Sanofi partnershipclinical

5 · Rules & route to market

United States

The United States concentrates the genome-editing pipeline, spanning in-vivo CRISPR, base editing, prime editing and cardiovascular base editing, under the FDA’s Regenerative Medicine Advanced Therapy (RMAT) pathway.

in-vivo CRISPR, base editing, prime editing, cardiovascular base editing#

  • Intellia Therapeutics (Cambridge, MA): in-vivo LNP-delivered CRISPR — NTLA-2001 for ATTR amyloidosis (first clinical redosing of an in-vivo CRISPR therapy) and lonvo-z for hereditary angioedema, with a rolling BLA submission underway in 2026.
  • Beam Therapeutics (Cambridge, MA): base editing platform — BEAM-304 (PKU, IND clearance June 2026), BEAM-302 (AATD) and BEAM-101 (SCD, all patients reaching HbF >60%).
  • Prime Medicine & Verve Therapeutics: prime editing moving in vivo via all-RNA LNP platforms, and Verve’s VERVE-102 (now under Eli Lilly) cutting LDL-C by up to 62% from a single PCSK9 base edit.
  • Editas Medicine (Cambridge, MA): transitioned in December 2024 from ex-vivo CRISPR-Cas12a (reni-cel, discontinued) to in-vivo gene editing; lead candidate EDIT-401 upregulates LDLR and cut LDL-C, Lp(a) and ApoB by ~90% in non-human primates, IND/CTA targeted for mid-2026.
  • Caribou Biosciences (Berkeley, CA): chRDNA (hybrid DNA/RNA guide) CRISPR for off-the-shelf allogeneic cell therapy — CB-010 anti-CD19 CAR-T.
  • Tessera Therapeutics: Gene Writing platform using engineered mobile genetic elements to write therapeutic sequences into the genome (preclinical platform).
  • Scribe Therapeutics (Berkeley, CA): engineered CasX (X-edit) CRISPR platform, partnered with Sanofi on in-vivo editing.
European Union

Europe anchors the field’s commercial milestone — CRISPR Therapeutics’ Casgevy, the first approved CRISPR therapy — and pushes CRISPR beyond human therapy into microbiome editing under the EMA’s Advanced Therapy Medicinal Products (ATMP) framework.

approved CRISPR therapy, microbiome editing#

  • CRISPR Therapeutics (Zug, Switzerland): Casgevy (exagamglogene autotemcel), the first approved CRISPR-Cas9 therapy, for sickle cell disease and transfusion-dependent beta-thalassemia; the FDA is reviewing expansion to children as young as 5 after Phase 3 CLIMB-151 data (73% VOC-free ≥12 months).
  • Eligo Bioscience (Paris): CRISPR-engineered phage and phage-derived capsid particles that deliver base editors and CRISPR payloads to gut bacteria in situ, enabling in-vivo microbiome editing.
  • EMA ATMP pathway: the advanced-therapy regulatory route governing gene-editing medicines across the EU.
China

China is building a domestic genome-editing therapy pipeline under the NMPA, with EdiGene’s beta-thalassemia program the first gene-editing therapy cleared for a clinical trial in the country.

China CRISPR therapeutics, beta-thalassemia, NMPA-cleared trials#

  • EdiGene (博雅辑因, Beijing): ET-01, a gene-editing therapy for beta-thalassemia, was the first gene-editing therapy cleared for a clinical trial by the NMPA’s Center for Drug Evaluation.
  • CorrectSequence Therapeutics (Shanghai): CS-101, a base-editing therapy for beta-thalassemia reported in Nature, reduced patients’ need for blood transfusions within weeks.
  • NMPA regulatory framework: the Center for Drug Evaluation’s gene-therapy review pathway anchors a growing pipeline of domestic CRISPR and base-editing candidates.

6 · Money: tenders & grants

TenderGreece – Biochemical analysers – ΗΛΕΚΤΡΟΝΙΚΟΣ ΑΝΟΙΚΤΟΣ ΔΙΑΓΩΝΙΣΜΟΣ ΑΝΩ ΤΩΝ ΟΡΙΩΝ ΣΕ ΕΥΡΩ ΓΙΑ ΤΗΝ ΠΡΟΜΗΘΕΙΑ …
ΓΕΝΙΚΟ ΑΝΤΙΚΑΡΚΙΝΙΚΟ ΟΓΚΟΛΟΓΙΚΟ ΝΟΣΟΚΟΜΕΙΟ ΑΘΗΝΩΝ "Ο ΑΓΙΟΣ ΣΑΒΒΑΣ" · Greece
Dec 11, 2026
56 days
TenderNetherlands – Blood-testing reagents – Gevalideerde Totaaloplossing voor de hielprikscreening op …
Rijksinstituut voor Volksgezondheid en Milieu (RIVM) · Netherlands
Nov 10, 2026
25 days
GrantFirst in Human and Early Stage Clinical Trials of Novel Investigational Drugs or Neuromodulatory Device-based …
National Institutes of Health · United States
Oct 15, 2027
364 days

Public R&D projects 3

Publicly funded research projects (US NSF, and EU, UK and other funders via OpenAIRE) whose title names this technology — matched by title, not reviewed; amounts as awarded, in the funder's currency. all funded projects →

2026EDGE-FGT: Genotype-independent Transformation and Genome Editing in Diploid Wheat via Shoot Apical Meristem (SAM)-based Delivery
NSF · 2619473
890,706 USD
2021Towards lice-resistant salmon: functional genetics and genome editing to enhance disease resistance in aquaculture
UKRI · BB/V009990/1
435,673 GBP
2018Improving resistance to infectious salmon anaemia using genome editing: Novel approaches to tackling viral disease in aquaculture
UKRI · BB/R008973/1
205,539 GBP

7 · Tech stack & innovations

The genome-editing stack spans three editor classes and a delivery toolbox that is moving the field from one-time ex-vivo cures toward redosable, in-vivo medicines.

  1. CRISPR-Cas9 & ex-vivo cell editing (CRISPR-Cas9):
    • Casgevy edits autologous CD34+ hematopoietic stem cells ex vivo, disrupting the BCL11A erythroid enhancer to reactivate fetal hemoglobin; Phase 3 CLIMB-151 shows 73% of children VOC-free for ≥12 months.
    • Electroporation delivers the editor into harvested cells, which are reinfused after editing and expansion.
  2. Base & prime editing (Base & Prime Editing):
    • Adenine/cytosine base editors swap single bases without double-strand breaks — Beam’s BEAM-304 (PKU) and Lilly/Verve’s VERVE-102, which cut PCSK9 by up to 88% and LDL-C by up to 62% from one infusion.
    • Prime editors write precise substitutions and small indels (“search-and-replace”); all-RNA LNP platforms and an AI-redesigned reverse transcriptase now enable in-vivo prime editing in the liver.
  3. In-vivo delivery & microbiome editing (Delivery & Microbiome):
    • Lipid-nanoparticle delivery carries mRNA editors plus guides in vivo — Intellia’s NTLA-2001 achieved the first clinical redosing of an in-vivo CRISPR therapy (90% median TTR knockdown on redose).
    • Eligo’s CRISPR-engineered phage capsid particles deliver base editors to gut bacteria in situ, extending genome editing to the microbiome.

Where this appears 48 sources

Appears in 48 distinct sources across 23 regions, 2012–2026.

  • directory listings 3
  • forum programmes 2
  • policy documents 37
  • reports 6

Regions: Belgium · Brazil · Central and Eastern Europe · Chile · China · European Union · Germany · Global · Hungary · India · Ireland · Japan · Kenya · Malaysia · Netherlands · New Zealand · Pakistan · Russia · Spain · Sri Lanka · Taiwan · United Kingdom · United States

Distinct sources per year
  1. 2021: 4
  2. 2022: 3
  3. 2023: 10
  4. 2024: 6
  5. 2025: 8
  6. 2026: 5
  7. 2027: 0

Matched themes 25

CRISPR CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) CRISPR Technology CRISPR and Gene Editing CRISPR gene editing CRISPR-Cas CRISPR-Cas9 technology Expanding gene editing technology Gene Editing Gene Editing (GEd) Gene editing techniques like Crispr-Cas9 Genetic editing Genome Editing Genome editing tools Innovative Platform & Tools - Gene Editing Precision genome editing genetic editing technologies Геномное редактирование ゲノム編集 ゲノム編集ツール 基因編輯 基因组编辑 基因编辑 基因编辑技术 遺伝子編集技術

Themes from forum programmes, policy documents and reports are mapped to technologies automatically and then reviewed; counts are distinct sources, not mentions, and are not a ranking. data sources and method → · Trending, not yet covered →

Sources 11 · 0 independent · 11 producer sites

Producers' own sites 11

  1. Crispr therapeutics producer checked 2026-06-16 · link not kept
  2. Intellia therapeutics producer checked 2026-05-11 · link not kept
  3. Beam therapeutics producer checked 2026-06-18 · link not kept
  4. Prime medicine producer checked 2026-06-15 · link not kept
  5. Verve therapeutics producer checked 2026-06-01 · link not kept
  6. Eligo bioscience producer checked 2026-06-15 · link not kept
  7. Edigene producer checked 2026-06-24 · link not kept
  8. Caribou biosciences producer checked 2026-07-01 · link not kept
  9. Tessera therapeutics producer checked 2026-05-14 · link not kept
  10. Scribe therapeutics producer checked 2026-05-19 · link not kept
  11. Editas medicine producer checked 2026-07-29 · link not kept