Non-Viral Gene Editing Services

AmplifyBio’s non-viral gene editing platform has been validated in human trials, is precise, efficient, and can be used to knock in and knock out genes of interest simultaneously. The recently acquired technology platform was initially developed for the creation of highly personalized neoantigen TCR-T cell therapies and is now available to developers to be used for a wider range of editing.

The non-viral gene editing services available at AmplifyBio can be used to make efficient modifications, including simultaneous knock-in and knock-out of genes of interest in client cells, with exceptional accuracy and proven clinical safety, for discovery, optimization, and production of transformative therapeutics. The platform is extensible to more complex cell editing, including tunable and inducible gene expression, knockdown through shRNA, and edits to allow T cells to be local delivery agents for bioactive molecules and can accomplish dual knock-in at distinct locations in one step.

Advantages of AmplifyBio Gene Editing Capability

Targeted editing

  • Precise and efficient, with established clinical safety
  • Natural regulation

 

Non-viral

  • Inexpensive & rapid to produce & prototype
  • Consistency & fewer regulatory burdens



Single-Step & flexible

  • Multiple edit types impossible with virus
  • Various cell types
  • Retrofit existing vectors & swap out nucleases



Clinically Tested

  • Preclinical and release testing packages for development defined
  • Effective at GMP manufacturing scale
  • Safe in humans
non viral gene editing

Non-Viral Precision Genome Editing vs. Viral Insertion of Transgenes

AmplifyBio Precision Genome Engineering Services
Typical Viral-based Manufacturing Approach

Flexible, modular platform allows for knock-in, knock-out, surface secreted, and TCR-induced payloads and editing of endogenously regulated genes in a single step:

  •    Endogenous TCR knocked out
  •    Client TCR precisely knocked in
  •    Known copy numbers 
 
Final Product:
  •    Expression controlled by native TCR regulatory elements
  •    No competition for CD3 signaling complex
  •    Normal T cell function
  •    Superior safety profile

Platform limited by vector capacity & multi-step edits to achieve knockout of endogenous targets:

  • Endogenous TCR intact or knocked out sequentially
  • Random viral vector integration
  • No copy number control
  • Extra manufacturing steps and facilities
 
Final Product: 
  • Expression controlled of artificial/foreign regulatory elements
  • Competes with endogenous TCR components like CD3 and co-receptors such as CD8
  • Less functional T cell
  • Inferior safety profile
  • Potential mispairing of TCR chains when endogenous TCR is intact
 
AmplifyBio AAV Lenti/Retrovirus Transposon

Non-Viral

Y
N
N
Y

Site Specific Knock-Out

Single-Step
Multi-step to KO
Multi-step to KO
Single-Step

Site Specific Knock-In

Y
Y
N (Random)
N (Semi-Random)

Knock-In Size Limits

Limit undefined 
< 4.5 kb
~10 kb
Limit undefined 

Cost Effective

★★★
★★★

Speed

★★★
★★★

Safety

★★★
★★

Editing Efficiency

★★
★★

Functionality

★★★
★★★
★★★

Platform Flexibility

★★★
★★★

Contact Us

Comprehensive service programs for the development of Cell and Gene Therapy (CGT), mRNA, and other advanced therapies from concept to commercial. Offering expertise and support in discovery, characterization, preclinical CRO safety testing, and scalable manufacturing available in an integrated, innovation-rich environment that goes beyond the value proposition offered by traditional CDMO and PDMO contract models.