Genome Editing Therapy Clinical Trials by the Numbers (2026)

Genome editing represents a fundamental structural transition in genetic medicine—moving beyond traditional gene addition (introducing exogenous cDNA constructs via viral vectors) to targeted, sequence-specific correction, disruption, base conversion, or prime writing at endogenous human genomic loci. Following the historical FDA approval of Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel) on December 8, 2023, for sickle cell disease — and its follow-on approval on January 16, 2024, for transfusion-dependent beta-thalassemia — clinical and investment focus has expanded across the broader genome-editing pipeline.

However, accurately assessing the volume, technological maturity, and clinical trajectory of genome-editing clinical trials requires strict data-filtering discipline. Standard registry searches matching the naive keyword "CRISPR" yield over 1,500 entries on ClinicalTrials.gov. Yet the vast majority of these matching records represent academic oncology or diagnostic studies utilizing CRISPR-Cas9 purely as an analytical laboratory research tool (e.g., screening patient tumor tissue, diagnostic assays, or target discovery in cell culture) rather than interventional clinical trials testing a genome-editing therapeutic drug product.

This decision-grade registry intelligence brief analyzes a curated, therapy-grade cohort derived from a comprehensive scan of ClinicalTrials.gov (595,630 global studies as of late July 2026). It quantifies roughly 144 interventional genome-editing therapy trials worldwide, dissects phase distributions, profiles leading corporate sponsors, evaluates the transition from ex vivo cellular engineering to systemic in vivo delivery, and outlines key clinical and regulatory catalysts through 2027. (The exact therapy-grade total is sensitive to the filtering protocol — intervention-keyword list plus the roster of dedicated editing-company sponsors — but consistently lands in the low-to-mid hundreds, not the thousands suggested by a bare keyword search.)

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Executive Summary & Direct Answer

A therapy-grade registry analysis of ClinicalTrials.gov identifies roughly 144 active, completed, or recruiting interventional clinical trials testing genome-editing therapeutic candidates in human subjects:

1. The Therapy-Grade Filter vs. Broad Keyword Overcount: A naive ClinicalTrials.gov website query for "CRISPR" or "gene editing" retrieves well over 1,000 studies, but the large majority reflect non-therapeutic academic research (e.g., ex vivo diagnostic assays, biomarker validation at institutions like M.D. Anderson, Cairo University, or National Cancer Institute). The actual therapeutic editing pipeline comprises roughly 144 trials, of which about 84% are industry-sponsored.

2. Phase Distribution (Early-Phase Dominance): The genome-editing pipeline remains heavily concentrated in early clinical evaluation. Phase 1 trials account for 39 trials (27.1%), Phase 1/2 trials represent 43 trials (29.9%), Phase 2 trials comprise 11 trials (7.6%), Phase 3 trials account for 12 trials (8.3%), and Phase 4/observational long-term follow-up studies make up 39 trials (27.1%).

3. Leading Corporate Sponsors & Platform Leadership: Corporate leadership is concentrated among dedicated gene-editing biopharmas and platform pioneers:

   • Sangamo Therapeutics: 28 trials (legacy Zinc Finger Nuclease platforms in hemophilia, Fabry disease, and MPS).
   • Rocket Pharmaceuticals: 19 trials (AAV platforms; primarily gene addition with editing crossovers).
   • CRISPR Therapeutics: 11 trials (ex vivo immuno-oncology and hemoglobinopathy programs).
   • Intellia Therapeutics: 9 trials (pioneering in vivo CRISPR candidates NTLA-2001 and NTLA-2002).
   • Vertex Pharmaceuticals: 7 trials (Casgevy commercial and pediatric extensions).
   • Vor Biopharma: 7 trials (multiplex-edited hematopoietic stem cell therapies).
   • Precision BioSciences: 6 trials (ARCUS meganuclease platform).
   • Beam Therapeutics: 6 trials (single-base editing platforms for SCD and T-cell therapies).
   • Editas Medicine: 5 trials (Cas12a / AsCas12a editing platforms).
   • Caribou Biosciences: 5 trials (chRDNA CRISPR-Cas12a allogeneic CAR-Ts).
   • Verve Therapeutics: 4 trials (in vivo cardiovascular base editing programs).

4. Approved Therapies & Near-Term Catalysts: Casgevy remains the single FDA-approved CRISPR-edited therapy ($2.2M WAC). The primary 2026–2027 catalysts are in vivo systemic therapies: Intellia’s NTLA-2001 (in vivo CRISPR-Cas9 for transthyretin amyloidosis, the most advanced in vivo editing candidate, now in the U.S.-cleared pivotal Phase 3 MAGNITUDE trial) and Verve Therapeutics’ VERVE-101/102 (adenine base editing targeting PCSK9 for heterozygous familial hypercholesterolemia, expanding into U.S. Phase 1b/2 Heart-2 trials following FDA IND clearance and Fast Track designation).

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Modality Breakdown: Genome Editing vs. Broad Gene & Cell Therapy

To evaluate where genome editing sits within advanced biotherapeutics, it must be contextualized against the broader Cell and Gene Therapy (CGT) landscape. The table below delineates how therapy-grade genome editing differs from broader gene-addition and cell-therapy registries:

For detailed analysis on the broader 4,855-trial cell and gene therapy universe, refer to our baseline study on cell and gene therapy clinical trials by the numbers.

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Therapy-Grade Filter Mechanics: Resolving Registry Overcounts

A central challenge in clinical trial intelligence is avoiding "bare-keyword inflation." Searching the public ClinicalTrials.gov website for CRISPR OR "gene editing" returns well over 1,000 studies, because the site's full-text search indexes detailed descriptions, eligibility text, and biomarker sections where CRISPR is cited as an assay or screening tool. However, inspecting the structured registry data reveals that the large majority of these records represent non-therapeutic uses:

CLINICALTRIALS.GOV REGISTRY SCAN (595,630 Total Global Studies)
----------------------------------------------------------------
Broad Website Keyword Match: "CRISPR" / "gene editing"
Total Hits: ~1,500+ studies (full-text search incl. descriptions)
  |
  +---> Academic Research / Diagnostic Screening (bulk of matches)
  |     - M.D. Anderson, Cairo University, NCI diagnostic assays
  |     - Ex vivo biomarker validation, CRISPR screening tools
  |
  +---> THERAPY-GRADE EDITING COHORT (~144 Interventional Trials)
        - ~84% industry-sponsored
        - Direct therapeutic drug administration (Ex Vivo or In Vivo)
        - Targeted loci: BCL11A, TTR, PCSK9, TRAC, CD52, KLKB1

Deconstructive Filtering Methodology

To isolate true therapeutic candidates, our protocol applies a two-stage algorithmic filter across the local ctgov_full database:

1. Intervention-Field Validation: Interventions must specify an interventional drug, cell product, or biological entity matching engineered nuclease terms (exagamglogene, lovotibeglogene, crispr, cas9, base edit, prime edit, talen, zinc finger, meganuclease, ntla-, verve-, beam-).
2. Sponsor & Title Scoping: Excludes non-interventional observational studies, diagnostic collection protocols, and academic target-discovery screening projects where CRISPR is used merely to knock out genes in laboratory cell lines extracted from patients.

This filtering discipline is identical to the protocol used across our other modality intelligence briefs, such as antibody-drug conjugate clinical trials by the numbers and bispecific antibody and T-cell engager clinical trials.

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Corporate Sponsor Leaderboard & Platform Technologies

The corporate landscape of genome editing is led by specialized platform companies that have transitioned from early academic licensing to clinical-stage pipelines. The leaderboard table below details the top sponsors, trial volumes, proprietary modalities, and lead clinical programs:

Genome Editing Sponsor Distribution (Top Corporate Players)
------------------------------------------------------------
Sangamo Therapeutics  : [============================] 28 trials (ZFN Legacy)
Rocket Pharma         : [===================] 19 trials (AAV/Lenti Hybrid)
CRISPR Therapeutics   : [===========] 11 trials (Cas9 Ex/In Vivo)
Intellia Therapeutics : [=========] 9 trials (In Vivo LNP Pioneer)
Vertex Pharma         : [=======] 7 trials (Casgevy Franchise)
Vor Biopharma         : [=======] 7 trials (eHSC Deletion)
Precision BioSciences : [======] 6 trials (ARCUS Meganuclease)
Beam Therapeutics     : [======] 6 trials (Base Editing Leader)
Editas Medicine       : [=====] 5 trials (Cas12a Platform)
Caribou Biosciences   : [=====] 5 trials (chRDNA Allogeneic)
Verve Therapeutics    : [====] 4 trials (In Vivo Cardiovascular)

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Detailed Analysis of Key Clinical Assets and Editing Platforms

To understand the clinical differentiation driving corporate valuations, each major editing platform must be analyzed by its specific molecular target, editing mechanism, and reported clinical proof-of-concept data.

1. Intellia Therapeutics: Systemic In Vivo LNP Delivery (NTLA-2001 & NTLA-2002)

Intellia has pioneered systemic in vivo genome editing using lipid nanoparticles (LNPs) carrying Cas9 mRNA and targeted guide RNAs directly to hepatocytes via low-density lipoprotein receptor (LDLR) endocytosis.

• NTLA-2001 (Transthyretin Amyloidosis / ATTR): Designed to knock out the TTR gene in hepatocytes to treat ATTR amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN). Clinical Phase 1 data demonstrated a dose-dependent, sustained reduction in serum TTR protein levels of approximately 90% following a single intravenous infusion. Intellia has initiated the global pivotal Phase 3 MAGNITUDE trial under FDA IND clearance, representing the most advanced in vivo CRISPR asset approaching regulatory submission.
• NTLA-2002 (Hereditary Angioedema / HAE): Targets the KLKB1 gene in the liver to knock out plasma kallikrein production, preventing severe angioedema attacks. Phase 1/2 clinical data demonstrated a 95% reduction in mean monthly attack rates, with several patients achieving complete freedom from swelling attacks over multi-year follow-up windows. Phase 3 HAELO trial enrollment is actively underway.

2. Verve Therapeutics: Single-Base Editing for Cardiovascular Disease (VERVE-101 & VERVE-102)

Verve Therapeutics is advancing in vivo adenine base editing (ABE) to treat cardiovascular conditions by permanently inactivating disease-causing liver genes with single-nucleotide precision.

• VERVE-101 & VERVE-102 (PCSK9 Inactivation): Designed to treat heterozygous familial hypercholesterolemia (HeFH) and premature coronary artery disease by introducing a precise A$\rightarrow$G point mutation in the PCSK9 gene. This point mutation disrupts the canonical splice site, permanently suppressing hepatic PCSK9 secretion and lowering low-density lipoprotein cholesterol (LDL-C).
• VERVE-102 Delivery Upgrade: While VERVE-101 utilized a galactosamine-functionalized LNP, VERVE-102 incorporates Verve's proprietary GalNAc-LNP technology, enabling targeting via the asialoglycoprotein receptor (ASGPR) with improved tolerability. In mid-2024, the FDA cleared the U.S. IND for VERVE-102 in the Heart-2 Phase 1b/2 trial, granting Fast Track designation.

3. Beam Therapeutics: Multi-Base Editing Platforms (BEAM-101, BEAM-201, BEAM-302)

Beam Therapeutics is developing precision base-editing therapeutics that convert single base pairs without generating double-strand DNA breaks, avoiding unwanted chromosomal translocations.

• BEAM-101 (Sickle Cell Disease): An ex vivo adenine base editing autologous CD34+ cell therapy that recreates natural protective hereditary persistence of fetal hemoglobin (HPFH) point mutations ($A\rightarrow G$) in the HBG1/HBG2 promoters, driving high-level $\text{HbF}$ expression.
• BEAM-201 (T-Cell Acute Lymphoblastic Leukemia / T-ALL): Represents the clinical state of the art in multiplexed ex vivo base editing. BEAM-201 undergoes four simultaneous base edits (knocking out CD7, TRAC, CD52, and PD-1) to create an off-the-shelf, allogeneic anti-CD7 CAR-T product resistant to fratricide and host immunosuppression.
• BEAM-302 (Alpha-1 Antitrypsin Deficiency / AATD): An in vivo liver-targeted base editor designed to directly correct the PiZ point mutation ($E342K$) in the SERPINA1 gene, restoring normal circulating alpha-1 antitrypsin protein levels and preventing hepatic accumulation.

4. Vor Biopharma: Multiplex-Edited Hematopoietic Stem Cells (VOR33)

Vor Biopharma’s platform focuses on multiplex CRISPR-Cas9 editing of autologous CD34+ hematopoietic stem cells (eHSCs) to delete lineage-specific cell surface antigens. VOR33 knockouts the CD33 antigen from healthy stem cells before transplantation in acute myeloid leukemia (AML) patients. Once engrafted, the patient’s hematopoietic system lacks CD33, enabling post-transplant administration of CD33-targeted therapies (such as Mylotarg or CD33 CAR-Ts) that selectively destroy AML blasts while sparing healthy blood cells.

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Off-Target Risk Management & Genomic Instability Assays

Evaluating off-target cleavage and genomic translocations is a core requirement for regulatory clearance of genome-editing IND applications. Because nucleases generate double-strand DNA breaks, improper repair can lead to unintended chromosomal rearrangements.

Unbiased Off-Target Discovery Technologies

Regulators (FDA, EMA, PMDA) require sponsors to combine in silico prediction algorithms with experimental in vitro and in cellulo unbiased off-target discovery assays:

1. GUIDE-seq (Genome-wide Unbiased Identification of DSBs Enabled by Sequencing): Captures double-strand breaks in living cells by integrating a double-stranded oligodeoxynucleotide (dsODN) tag into cleavage sites, followed by high-throughput sequencing.
2. CAST-Seq (Chromosomal Aberration Analysis by Single-Target Sequencing): Specifically quantifies chromosomal translocations, large deletions, and inversions resulting from on-target and off-target cleavage events.
3. Circle-seq & CHANGE-seq: Cell-free biochemical assays that isolate genomic DNA, circularize it, and digest it with purified Cas9-gRNA complexes to identify potential cleavage sites across the entire genome without cellular chromatin bias.

Transitioning to base editing (Beam, Verve) and prime editing significantly reduces off-target translocation risks by modifying target bases without generating double-strand breaks, providing a major safety advantage in regulatory review.

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Phase Distribution: Early-Phase Dominance and Long-Term Registries

The clinical development status of the 144 therapy-grade editing trials reveals an industry undergoing rapid early-phase expansion, backed by a growing tail of mandatory long-term safety monitoring protocols.

                  PIPELINE PHASE BREAKDOWN (144 Total Trials)
                  -------------------------------------------
     [Phase 1]            : 39 trials (27.1%)  |=== Early-Phase Safety & Dose
     [Phase 1/2]          : 43 trials (29.9%)  |=== Escalation Core (57.0%)
     [Phase 2]            : 11 trials (7.6%)   |--- Mid-Stage Proof-of-Concept
     [Phase 3]            : 12 trials (8.3%)   |--- Pivotal Registration Phase
     [Phase 4 / Registry] : 39 trials (27.1%)  |=== Mandatory 15-Year Follow-up

1. Early-Phase Concentration (Phases 1 & 1/2)

Combined, Phase 1 (39 trials) and Phase 1/2 (43 trials) represent 82 of 144 trials (57.0%). This heavy concentration reflects the entry of second- and third-generation editing modalities—such as base editing (Beam, Verve), prime editing, and meganucleases (Precision BioSciences)—into human testing. These trials focus primarily on dose escalation, safety, off-target cleavage assessment, biodistribution, and biomarker modulation.

2. Pivotal Phase 3 Registration (12 Trials)

Only 8.3% of active trials sit in Phase 3. These registration studies are led by Vertex/CRISPR Therapeutics’ global expansion trials for Casgevy, Intellia’s pivotal Phase 3 MAGNITUDE trial for NTLA-2001 in ATTR amyloidosis, Intellia's HAELO trial for NTLA-2002 in HAE, and Rocket Pharmaceuticals' pivotal registration programs.

3. Long-Term Follow-up Mandatory Registries (39 Trials)

Regulatory agencies (FDA and EMA) mandate a minimum of 15 years of post-infusion observational monitoring for any cell or gene therapy incorporating permanent genomic modification. Consequently, 27.1% of the total registry count consists of non-interventional, long-term safety extension protocols tracking potential late-onset adverse events, insertional oncogenesis, off-target genomic instability, or delayed clonal hematopoiesis.

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Ex Vivo vs. In Vivo Editing: Delivery Technologies Compared

The genome-editing field is currently bifurcated into two distinct delivery modalities: Ex Vivo Cell Engineering and Systemic In Vivo Gene Editing.

                           GENOME EDITING DELIVERY MODALITIES
                                          |
                 +------------------------+------------------------+
                 |                                                 |
         EX VIVO CELL EDITING                     IN VIVO SYSTEMIC EDITING
                 |                                                 |
   - Harvest patient/donor cells            - Intravenous LNP or AAV Infusion
   - Electroporation of RNP/Cas9            - Direct systemic tissue targeting (Liver)
   - Myeloablative conditioning             - No myeloablative conditioning
   - Complex manufacturing (Wks)            - Off-the-shelf pharmaceutical delivery
                 |                                                 |
   Examples: Casgevy, BEAM-101,             Examples: NTLA-2001 (Intellia),
   CB-010 (Caribou), VOR33                  VERVE-101/102 (Verve Therapeutics)

1. Ex Vivo Editing (The First Generation)

Ex vivo editing involves harvesting autologous CD34+ stem cells or allogeneic donor T-cells, performing gene editing in vitro via electroporation of Cas9 ribonucleoprotein (RNP) complexes, and reinfusing the edited cell product into the patient.

• Advantages: Allows precise quality control, verification of editing efficiency prior to infusion, and complete absence of systemic vector exposure.
• Disadvantages: Requires intensive myeloablative chemotherapy (e.g., busulfan), prolonged inpatient hospitalization, high manufacturing costs, and specialized treatment center infrastructure.
• Representative Products: Casgevy (SCD/Thalassemia), BEAM-101 (SCD), Caribou’s CB-010 (allogeneic CAR-T for NHL).

For access analysis on adjacent ex vivo cellular platforms, see our report on CAR-T cell therapy access landscape.

2. Systemic In Vivo Editing (The Second Generation)

In vivo editing utilizes lipid nanoparticles (LNPs) or viral vectors (AAVs) to deliver mRNA encoding the editing enzyme (Cas9, base editor) along with a guide RNA directly into the systemic circulation, targeting internal organs such as the liver.

• Advantages: Eliminates myeloablative conditioning, enables off-the-shelf administration in outpatient settings, dramatically lowers health-system burden, and expands the eligible patient population.
• Disadvantages: Risk of off-target editing in non-target tissues, potential transient immune responses against Cas9 or LNP components, and reliance on liver-tropic delivery systems.

For details on the commercial and clinical access dynamics of Casgevy itself, consult our guide on Casgevy sickle cell gene therapy access.

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Health-System Commercialization & Reimbursement Challenges

Bringing a genome-editing therapy from clinical trials to commercial market access involves navigating severe reimbursement bottlenecks across hospital inpatient systems and specialty payer frameworks.

Inpatient DRG Payment Shortfalls

For ex vivo stem-cell therapies like Casgevy, hospital inpatient administration is billed under MS-DRG 016 or 017 (autologous hematopoietic stem cell transplant). Standard DRG reimbursement ($40,000 to $65,000) covers less than 3% of the $2.2M drug acquisition cost. Consequently, hospitals must negotiate single-case agreements (SCAs) or state Medicaid carve-outs before admitting patients.

Value-Based & Multi-Year Contracting

Because single upfront payments create budget spikes for commercial health plans and state Medicaid programs, manufacturers are adopting value-based rebate structures. Under these agreements:

• A portion of the drug purchase price is refunded if the patient experiences disease recurrence within 1 to 3 years.
• Payers leverage CMS CGT Access Model multi-state frameworks to spread payments over multi-year operational horizons.

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Regulatory Pathways: RMAT, Breakthrough, and Accelerated Approval

Because genome-editing therapies target severe, life-threatening rare diseases or high-unmet-need cardiovascular conditions, sponsors heavily leverage expedited regulatory pathways established by the FDA:

1. Regenerative Medicine Advanced Therapy (RMAT) Designation: Granted to products like Casgevy, NTLA-2001, and BEAM-101, RMAT designation provides intensive FDA guidance, rolling review options, and eligibility for Accelerated Approval based on surrogate biomarkers (such as serum TTR reduction or HbF elevation).
2. Breakthrough Therapy & Fast Track Designations: Intellia's NTLA-2002 and Verve's VERVE-102 have received Fast Track and Breakthrough designations to accelerate clinical development and regulatory review.
3. Postmarketing Commitments & 15-Year Registries: Approval under Accelerated Approval or BLA pathways requires mandatory enrollment of all treated patients into 15-year safety tracking registries to monitor for potential off-target genomic cleavage, chromosomal translocations, or secondary malignancies.

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Key Regulatory Catalysts & Pipeline Timeline (2026–2027)

Over the next 18 to 24 months, the genome-editing market will transition from a single-approval landscape to a multi-product commercial sector. The timeline below highlights pivotal clinical readouts and regulatory milestones:

2023 Dec  : [MILESTONE] FDA approves Casgevy (Vertex/CRISPR Tx) — First CRISPR therapy ($2.2M WAC)
2025 Q1-Q3: [ACCESS] CMS launches & expands CGT Access Model for Medicaid SCD coverage (~33 states)
2026 Q2-Q4: [REGULATORY] Intellia NTLA-2001 pivotal Phase 3 (MAGNITUDE) enrollment in ATTR Amyloidosis, the most advanced in vivo editing candidate tracking toward FDA submission
2026 Q3   : [PIPELINE] Verve Heart-2 US expansion data readout for VERVE-102 (PCSK9 base editing)
2027 Q1-Q2: [READOUT] Beam Therapeutics BEAM-101 Phase 1/2 pivotal cohort readout in severe SCD
2027 Q3   : [REGULATORY] Caribou Biosciences CB-010 pivotal Phase 2 trial readout in relapsed B-NHL

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Frequently Asked Questions (FAQ)

How many CRISPR/gene-editing therapies are FDA-approved in 2026?

As of 2026, Casgevy (exagamglogene autotemcel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, remains the only FDA-approved CRISPR-edited gene therapy. It was approved on December 8, 2023, for severe sickle cell disease and subsequently for transfusion-dependent beta-thalassemia.

What is the difference between CRISPR, base editing, and prime editing therapies?

CRISPR-Cas9 therapies introduce double-strand DNA breaks to knock out or disrupt specific genes. Base editing uses a modified Cas enzyme fused to a deaminase to convert a single DNA base pair (e.g., C$\rightarrow$T or A$\rightarrow$G) without making double-strand breaks, reducing off-target translocation risks. Prime editing uses a reverse transcriptase fused to Cas to write new genetic sequences directly into a target site without double-strand breaks or donor DNA.

Which in vivo (in-body) gene editing therapy is closest to FDA approval?

Intellia Therapeutics’ NTLA-2001, an in vivo LNP-CRISPR therapy targeting the TTR gene in hepatocytes for transthyretin (ATTR) amyloidosis, is the most advanced in vivo candidate. It has completed Phase 1/2 evaluation, received U.S. pivotal Phase 3 (MAGNITUDE) IND clearance, and is enrolling its registration study ahead of a planned FDA submission.

Why is the editing trial count (144) so much lower than the broad CRISPR search (~1,544)?

Searching ClinicalTrials.gov with broad terms retrieves over 1,500 records because CRISPR is widely used as a laboratory research tool in academic oncology and biomarker studies. Filtering for therapy-grade interventional trials—where an engineered genome-editing product is administered directly as a medical treatment—isolates the true therapeutic pipeline of 144 clinical trials.

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Sources

1. U.S. Food and Drug Administration (FDA): FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease (Casgevy). FDA Press Release, Dec 8, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
2. Intellia Therapeutics: Intellia Announces FDA Clearance of IND Application for NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis. Press Release & Investor Relations. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-fda-clearance-investigational-0
3. BioPharma Dive: Verve Base Editing PCSK9 Trial Cleared for U.S. Expansion (Heart-2). BioPharma Dive Analysis. https://www.biopharmadive.com/news/verve-us-base-editing-study-ind-fda-heart-2/743338/
4. Alliance for Regenerative Medicine (ARM): Cell & Gene Therapy Clinical Trials Landscape Report. Alliance for Regenerative Medicine Publications. https://alliancerm.org/clinical-trials-cell-and-gene-therapy/
5. CRISPR Therapeutics: Pipeline & Clinical Programs (Casgevy, CTX110, CTX130, CTX310). Corporate Disclosures. https://www.crisprtx.com/our-pipeline
6. ClinicalTrials.gov / U.S. National Library of Medicine: Global Clinical Trials Database Mirror. Dataset analysis covering 595,630 studies (July 2026 export).