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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.
Targeted editing
Non-viral
Single-Step & flexible
Clinically Tested
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:
Platform limited by vector capacity & multi-step edits to achieve knockout of endogenous targets:
| 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 |
★★★ |
★ |
★ |
★★★ |
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.
