INDe sgRNAs Designed For Preclinical Safety, Toxicity, And Efficacy Studies
IND-enabling sgRNAs are high quality sgRNAs with increased quality oversight and documentation enabling comparability between reagents as you transition into clinical trials.
Synthego’s full spectrum of tiered quality, synthetic sgRNAs includes IND-enabling (INDe) sgRNAs, designed for the preclinical phase to provide highest efficiency and performance for IND-enabling safety, toxicity, and efficacy studies. INDe sgRNAs are manufactured in accordance with GLP guidelines for nonclinical studies with traceable quality documentation and oversight, enabling comparability between INDe and GMP sgRNA reagents. Built on Synthego’s proprietary Halo™ platform and our innovative gRNA design capabilities, Synthego’s INDe sgRNAs integrate seamlessly as an intermediary between RUO and GMP products, providing an efficient and convenient solution for researchers transitioning between discovery, preclinical, and clinical development stages.
Quality-First
IND-enabling sgRNAs are manufactured under relevant GLP quality standards to enable your preclinical GLP experiments for efficacy and safety.
Discovery to Therapy - Preclinical
Customizable INDe sgRNAs can be scaled up from RUO sgRNAs to build on performance and efficiency achieved from our innovative Halo™ platform, and provides comparability for the transition to GMP materials.
Regulatory Support
In addition to high quality INDe sgRNAs, Synthego offers in-depth support and expertise for pre-IND meetings and during the IND submission process.
| RUO | INDe | GMP | |
|---|---|---|---|
| Product Grade | Non-clinical | Non-clinical | Clinical (produced under cGMP) |
| Recommended applications | Discovery | IND-enabling studies | Phase 1 to Pivotal trials |
| Regulatory Compliance | N/A | Complies with equipment, facility, material controls in 21 CFR part 58 and provides comparability for nonclinical vs clinical materials | Compliant with FDA and EMA GMP regulations including Chemistry, Manufacturing, and Controls (CMC) for gene editing components used in cell and gene therapies |
| Raw Material Source | Sourced from the same vendor from RUO to GMP production. Material oversight by supply chain | Qualified and QA approved vendors traceable under QMS*. Material oversight by QA | Qualified and QA approved vendors traceable under QMS*. Material oversight by QA. QC Identity testing |
| Validated Cleaning Process | No | Yes | Yes |
| Manufacturing space |
Open Research Environments | Synthesis and Purification:
|
GMP suites segregated for manufacturing steps
Synthesis and Purification:
Final Processing:
|
| Delivered documentations | Certificate of Testing |
|
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Leverage Continuum-Grade Guides for Therapeutic Development
IND-enabling (INDe) sgRNAs are non-regulated synthetic sgRNAs that comply with the equipment, facility, and material controls in the relevant sections of 21 CFR part 58 on guidelines for Good Laboratory Practice (GLP) for nonclinical laboratory studies. INDe sgRNAs provide comparability for nonclinical to clinical materials meaning the ability to demonstrate product equivalence when scaling up to the GMP-grade sgRNAs necessary for successful clinical trials.
While INDe sgRNAs are research-grade sgRNAs, they are manufactured with enhanced quality oversight of raw materials and manufacturing space compared to RUO sgRNAs. Additionally, manufacturing INDe sgRNAs utilizes a fully digitized system for quality documentation creation and approval, increasing efficiency, accuracy, and consistency during the quality review process, similar to GMP sgRNAs.
In addition to INDe sgRNA manufacturing, Synthego provides full pre-IND/IMPD support including for the INTERACT meeting with the FDA, authoring relevant Briefing Package sections, and guiding discussions between the sponsor and the regulatory body. Then, Synthego assists authoring the relevant gRNA CMC sections, referencing our recently filed Drug Master File (DMF) for convenient and comprehensive IND/IMPD submission.