CRISPR-Mediated Gene Therapy- One lab, three paths to faster outcomes

1

LABORATORY AUTOMATION | DIGITAL SOLUTIONS | ANALYTICAL TOOLS

Preliminary Screening

Select the target, develop the proper gene editing strategy, and screen libraries to select the gRNA.

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2

LABORATORY AUTOMATION | DIGITAL SOLUTIONS | ANALYTICAL TOOLS

Isolate & Enrich

Obtain patient samples to isolate and enrich the desired cell population.

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3

LABORATORY AUTOMATION | DIGITAL SOLUTIONS | ANALYTICAL TOOLS

Gene Modification and Analysis

Edit the target gene via a CRISPR-Cas approach and analyze for expected effect.

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4

ANALYTICAL TOOLS | DIGITAL SOLUTIONS

Expansion

Expand CRISPR-edited cells for downstream characterization.

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5

LABORATORY AUTOMATION | ANALYTICAL TOOLS | DIGITAL SOLUTIONS

Characterization and Optimization

Characterize molecular consequences of CRISPR edits and optimize as needed.

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6

ANALYTICAL TOOLS | DIGITAL SOLUTIONS

Functional Assessment

In vitro and in vivo functional activity assessment is performed to inform on risk.

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7

LABORATORY AUTOMATION | DIGITAL SOLUTIONS

Off-Target Analysis

Chromosomal, sequencing and in silico analyses help uncover and understand off-target effects.

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8

DIGITAL SOLUTIONS

Regulatory Interaction

Prepare data and reports for regulatory filings in support of the CRISPR-mediated gene therapy.

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