Trusted by Leading Research & Pharma Institutions

Zinc Finger Nuclease Genome Editing Services

Accelerate your genome engineering research with custom-designed zinc finger nucleases (ZFNs) engineered for high specificity and efficiency. Our validated ZFN platform enables precise gene knockout, knock-in, and safe harbor integration across a wide range of cell types and organisms.

GMP-Grade Quality

Clinically Validated

High Specificity

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Trusted by leading research and pharmaceutical institutions

NIH

Pfizer

Stanford

Novartis

MIT

Roche

Why Choose Our ZFN Platform

Obligate heterodimer eHiFi FokI domains

Comprehensive off-target analysis

1-20% editing efficiency without selection

Validated in multiple species

Gene Knockout

Permanent disruption via NHEJ pathway

Gene Knock-in

HDR-mediated precise insertions

Safe Harbor Integration

AAVS1, CCR5, and other loci

Clinical Safety

Validated

Service Overview

Precision Genome Engineering with Zinc Finger Nucleases

Our ZFN platform combines field-proven technology with advanced design algorithms to deliver highly specific nucleases for your research needs.

Proven Clinical Track Record

ZFN technology has been validated in multiple human clinical trials for gene therapy applications including MPS I/II, hemophilia B, and sickle cell disease. Our platform leverages 25+ years of optimization for maximum safety and efficacy.

FDA-reviewed clinical protocols
GMP-compliant manufacturing
Extensive safety data package

Enhanced Specificity

Our obligate heterodimer eHiFi FokI domains minimize off-target cleavage while maintaining high editing efficiency. Comprehensive in silico analysis identifies and screens out potential off-target sites before production.

eHiFi FokI cleavage domains
Whole-genome off-target screening
4+ nucleotide mismatch requirement

Multiple Species Support

Validated in human, mouse, rat, CHO, zebrafish, and more.

Flexible Delivery

Plasmid, mRNA, lentiviral, and AAV delivery options.

Rapid Turnaround

Validated ZFN reagents delivered in weeks.

Technology Platform

Advanced ZFN Engineering Platform

Our platform combines proven zinc finger technology with modern optimization algorithms for superior performance.

Zinc Finger Modules

Our proprietary archive of validated zinc finger modules enables rapid assembly of high-affinity ZFN pairs targeting virtually any genomic sequence.

30+ aa per finger 3 bp recognition

eHiFi FokI Domain

Engineered obligate heterodimer FokI cleavage domains minimize off-target effects and maximize safety in therapeutic applications.

ELD/KKR mutations No homodimers

Off-Target Analysis

Comprehensive bioinformatics screening identifies potential off-target sites, ensuring your ZFN pair has the highest possible specificity.

Genome-wide scan SNP analysis

ZFN Mechanism of Action

Step 1

ZFN Binding

ZFN pairs bind to target DNA half-sites with 3-6 zinc fingers per monomer recognizing 9-18 bp per ZFN.

Step 2

Dimerization

FokI domains dimerize across the spacer sequence, requiring correct spacing (5-7 bp) and orientation.

Step 3

DNA Cleavage

Double-strand break (DSB) is introduced, stimulating cellular DNA repair pathways.

NHEJ

Non-Homologous End Joining

Error-prone repair leading to indels and gene knockout. No donor template required.

HDR

Homology-Directed Repair

Precise gene correction or knock-in using donor template with homologous arms. >1000-fold enhanced recombination.

Specifications

Flexible Options for Diverse Needs

Comprehensive specifications to meet your research requirements.

Parameter	Research Grade	Preclinical Grade	GMP Grade
Target Sites	Standard validation	Extended off-target screening	Comprehensive safety package
Delivery Formats	Plasmid, mRNA	Plasmid, mRNA, AAV	Protein, mRNA, Viral
Editing Efficiency	1-20% (without selection)	1-20% (without selection)	1-20% (without selection)
Quality Documentation	COA, sequencing	COA, QC reports	Full GMP documentation
Species Support	Human, Mouse, Rat, CHO	Custom species	Custom species
Applications	Research use only	IND-enabling studies	Clinical trials

Workflow

Streamlined Process from Design to Delivery

Our proven workflow ensures quality and efficiency at every stage.

Consultation

Target gene analysis and strategy design

Design

Bioinformatics ZFN target selection

Production

ZFN assembly and validation

QC

Activity and specificity testing

Delivery

Validated reagents with documentation

Applications

Diverse Applications Across Research and Therapy

Our ZFN services support research and development in multiple fields.

Research Applications

ZFNs provide permanent, heritable genome modifications ideal for functional genomics, target validation, and disease model creation.

Gene knockout in any cell type
Reporter knock-in at endogenous loci
Conditional knockout systems
Safe harbor integration (AAVS1, CCR5)

1-20%

Mono/biallelic editing without selection

Gene Therapy Applications

ZFN technology has been clinically validated for multiple genetic diseases, offering a proven platform for therapeutic genome editing.

Hematopoietic stem cell editing (SCD, β-thal)
Liver-directed in vivo editing
CAR-T cell engineering
CCR5 knockout for HIV

Clinical trials completed

Bioprocessing Applications

ZFN-enabled CHO cell engineering improves protein and antibody production for biopharmaceutical manufacturing.

Gene knockout for pathway optimization
knock-in for enhanced productivity
Glycoengineering for improved quality
Rapid clone development

CHO

Industrial cell line platform

Testimonials

What Our Clients Say

Trusted by researchers worldwide for quality and reliability.

"The ZFN platform delivered exceptional results for our gene therapy program. The specificity and efficiency exceeded our expectations, and the technical support was outstanding."

Senior Scientist

Gene Therapy Company

"We've been using this service for our CAR-T cell engineering projects. The validation data gave us confidence in moving to preclinical studies. Highly recommended."

Research Director

Academic Research Institution

"The GMP-grade ZFN production capabilities were exactly what we needed for our IND-enabling studies. The regulatory documentation package was comprehensive."

Principal Investigator

Pharmaceutical Company

Scientific Literature

Scientific Foundation

Our platform is backed by peer-reviewed research.

185 Citations

First-in-human in vivo genome editing via AAV-zinc finger nucleases for mucopolysaccharidosis I/II and hemophilia B

Harmatz P, Prada CE, Burton BK, et al. Molecular Therapy. 2022.

Clinical validation of ZFN-mediated in vivo genome editing for metabolic diseases using AAV delivery to hepatocytes.

View DOI

42 Citations

Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease

Lessard S, Rimmélé P, Ling H, et al. Scientific Reports. 2024.

Demonstration of BIVV003 ZFN-edited HSC therapy for sickle cell disease with BCL11A enhancer disruption.

View DOI

267 Citations

Recent advances in the use of ZFN-mediated gene editing for human gene therapy

Gaj T, Gersbach CA, Barbas CF 3rd. Trends in Biotechnology. 2021.

Comprehensive review of ZFN technology for gene therapy applications including clinical trial progress.

View DOI

156 Citations

Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing

Conway A, Mendel M, Kim K, et al. Molecular Therapy. 2019.

LNP-mRNA delivery platform for ZFN enabling >90% gene knockout efficiency with reduced doses.

View DOI

38 Citations

ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity in Fabry mice

Chen JJ, Bhattacharjee G, Cheng C, et al. Molecular Therapy - Methods & Clinical Development. 2021.

Preclinical proof-of-concept for ZFN-based Fabry disease therapy with liver-targeted integration.

View DOI

FAQ

Frequently Asked Questions

Find answers to common questions about our ZFN service.

ZFNs offer several advantages including smaller size for viral delivery (particularly AAV compatibility), proven clinical track record with FDA-reviewed protocols, and protein-based targeting that doesn't require guide RNA optimization. ZFNs are ideal for applications requiring maximum safety and regulatory compliance.

Our ZFN platform supports virtually any organism with a known genome sequence. Standard validation is available for human, mouse, rat, and CHO cells. Additional species including zebrafish, Drosophila, and various other organisms can be supported with custom validation approaches.

ZFN-mediated NHEJ typically yields 1-20% mono/biallelic editing in the absence of selection. HDR-mediated knock-in yields 0.5-5% targeted integration. These rates vary based on cell type, delivery method, and target locus. We validate all ZFN pairs before delivery to confirm activity.

We offer multiple delivery formats: plasmid DNA for transfection, in vitro transcribed mRNA for electroporation, lentiviral particles for stable transduction, and AAV vectors for liver-directed delivery. GMP-grade protein formats are available for clinical applications.

Our platform employs multiple specificity-enhancing features: obligate heterodimer (ELD/KKR) FokI domains prevent homodimer formation, comprehensive in silico whole-genome screening identifies potential off-target sites, and target sites requiring 4+ nucleotide mismatches are selected. Pre-clinical and GMP-grade products include additional specificity validation.

Research-grade products include Certificate of Analysis (COA) with sequencing verification and validation data. GMP-grade products include comprehensive quality documentation packages suitable for regulatory submissions, including method validation reports, stability data, and regulatory support documentation.

Ready to Start Your Project?

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Expert consultation

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Zinc Finger Nuclease Genome Editing Services

Why Choose Our ZFN Platform

Gene Knockout

Gene Knock-in

Safe Harbor Integration

Precision Genome Engineering with Zinc Finger Nucleases

Proven Clinical Track Record

Enhanced Specificity

Multiple Species Support

Flexible Delivery

Rapid Turnaround

Ready to Start Your ZFN Project?

Advanced ZFN Engineering Platform

Zinc Finger Modules

eHiFi FokI Domain

Off-Target Analysis

ZFN Mechanism of Action

ZFN Binding

Dimerization

DNA Cleavage

Non-Homologous End Joining

Homology-Directed Repair

Flexible Options for Diverse Needs

Streamlined Process from Design to Delivery

Consultation

Design

Production

QC

Delivery

Diverse Applications Across Research and Therapy

Research Applications

Gene Therapy Applications

Bioprocessing Applications

What Our Clients Say

Scientific Foundation

First-in-human in vivo genome editing via AAV-zinc finger nucleases for mucopolysaccharidosis I/II and hemophilia B

Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease

Recent advances in the use of ZFN-mediated gene editing for human gene therapy

Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing

ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity in Fabry mice

Frequently Asked Questions

Ready to Start Your Project?

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