Brochure
For customers looking to accelerate their therapeutic gene editing program from discovery to clinical trials, our Engineering Run and full CGMP compliant guide RNA (gRNA) manufacturing and sequencing services offer a streamlined solution. Backed by comprehensive documentation, our services simplify regulatory filings, offering a straightforward path to clinical success.
Leverage advanced CRISPR technology to empower and streamline genomics research and therapeutic development. Our specialized CGMP gRNA manufacturing and sequencing services ensure regulatory compliance while supporting complex gene editing projects. We are committed to delivering the highest manufacturing standards, ensuring that our gRNA products can enhance efficiency of CRISPR genome editing projects across every stage of therapeutic development. Bring your genomic research to the forefront of personalized medicine innovation.
With nearly 40 years of experience, Integrated DNA Technologies (IDT) leads with a proprietary synthesis process unlike any other in the industry supporting your journey from research to clinical applications with unmatched expertise.
Our 41,000 sq. ft. Therapeutic Oligonucleotide Manufacturing Facility provides CGMP CRISPR manufacturing services tailored to your development—from preclinical studies to clinical trials.
We prioritize quality with thorough release testing, including advanced identity assays such as gRNA sequencing. This ensures every CRISPR gRNA meets the highest standards of quality and consistency.
IDT provides regulatory information tailored to your clinical phase, helping you meet requirements confidently and efficiently.
Your project is in the hands of a dedicated, cross-functional team of professionals at IDT. Each team member is an expert in their field, working diligently to ensure the success of your therapeutic CRISPR program.
IDT and Aldevron partnered with the Children’s Hospital of Philadelphia (CHOP) to manufacture the world’s first mRNA-based personalized CRISPR therapy for an infant with urea cycle disorder (UCD). Delivered in just six months—three times faster than standard timelines for gene editing drug products.
IDT’s gRNAs produced at its Therapeutic Oligonucleotide Manufacturing Facility deliver the high-quality current Good Manufacturing Practices (CGMP) material required for successful completion of your preclinical studies and clinical trials. To further support regulatory compliance and accelerate IND/CTA filings, IDT offers gRNA sequencing services for customers manufacturing their CGMP gRNAs with us.
Engineering Run: Synthetic gRNA produced by the same manufacturing process as CGMP products but with limited Quality Assurance documentation.
CGMP: Synthetic gRNA manufactured under CGMP-compliant conditions and includes full Quality Assurance Documentation.
gRNA Sequencing Services
Sequence-level contaminants during gRNA manufacturing can potentially compromise therapeutic efficacy and delay regulatory approvals. IDT can help by integrating gRNA sequencing into your Engineering Run or CGMP manufacturing workflow. Our orthogonal identity assay provides full-length sequence verification to support regulatory filings and accelerate your therapeutic development timelines, without the added complexity of third-party vendors.
| Engineering run | CGMP | |
|---|---|---|
| Cleanroom | Certification not required | ISO 8 Clean Room – Certified |
| Changeover | In-place with cleaning validation | In-place with cleaning validation |
| Materials | QA release on raw materials | QA release on raw materials |
| Batch records | Draft based on customer’s specifications | Based on customer specification |
| Release testing | Qualified methods | Validated methods |
Through comprehensive quality control and analytical testing, we are committed to ensure that our therapeutic oligonucleotides consistently meet the regulatory requirements to accelerate your project and bring your discoveries to life.
| Category | Attribute | Method |
|---|---|---|
| Identity | Molecular weight | ESI-MS |
| Sequence identity | NGS-based gRNA sequencing | |
| Purity | Purity | Single-channel CE or LC-MS |
| Process related impurities | Elemental impurities Residual solvents | USP <233> USP <467> |
| Safety | Endotoxin Bioburden | USP <85> limulus amebocyte lysate (LAL) USP <61/62> |
| Yield | UV/Vis | Optical density at 260nm |
| General | Appearance | Visual inspection |
The functional performance of 100 nt Cas9 sgRNAs targeting HPRT1 was studied in K562 cells using cell-based editing activity and in vitro cleavage assays. As shown in Figure 1, pilot run gRNAs performed better than the RUO gRNAs with the same sequence but purified using the standard desalt method. However, similar in cellulo % editing and in vitro % cleavage performance were observed with pilot runs and HPLC-purified gRNAs. Moreover, different pilot runs exhibited similar functional activity, demonstrating batch-to-batch consistency.
To validate gRNA identity and detect contaminants, IDT performs complete sequencing of Engineering Run and CGMP oligos. Our method provides >500x read depth across the spacer region and implements stringent purity thresholds, ensuring compliance with regulatory expectations.
