Global genomics leader Integrated DNA Technologies (IDT) is revolutionizing the field of translational gene editing with new additions to its end-to-end CRISPR portfolio. These expanded CRISPR translational research solutions are designed to help researchers accelerate more CRISPR-based therapies for patients like KJ Muldoon, an infant who suffered CPS1 deficiency and made history when he received the world’s first personalized CRISPR therapy manufactured jointly by IDT and Aldevron. IDT provided the guide RNA (gRNA), off-target analysis services and regulatory support for baby KJ’s treatment, which was delivered in a significantly compressed timeline.
Tailored gRNAs to Accelerate CRISPR Discoveries
As researchers advance from discovery to clinical applications, the demand for higher purity in CRISPR reagents increases. To meet this need, IDT’s chemically synthesized gRNAs are now available for online ordering in a range of modification and purity options. With high purity ideal for translational applications such as gene editing in primary cells and in vivo models, IDT’s high-performance liquid chromatography (HPLC)-purified gRNAs are orderable in 2 nmol and 10 nmol yields with larger quantities available by request and ship in as little as 12 business days. Formats include CRISPR-Cas9 gRNA and Custom Alt-R™ CRISPR gRNA. 2’ Fluoro, 2’ O-Methyl, and other modifications can be added to increase stability and specificity with IDT’s custom gRNA tool.
Supporting Scientists with Regulatory Filings and Ensuring Safety of Therapeutic Candidates
CRISPR-based genome editing allows for targeted editing at specific sites in the genome, but there is potential risk that off-target edits at other locations can occur. To enable scientists to understand where these off-target edits might happen and assess how they might impact safety early in the therapeutic development process, IDT launched UNCOVERseq, off-target nomination services which uses an enhanced GUIDE-seq™ methodology to identify off-target sites for its customers. When paired with IDT’s award-winning off-target confirmation services, rhAmpSeq™ CRISPR Analysis System, CRISPR pioneers can confidently accelerate their path to the clinic by obtaining a deeper understanding of editing risks.
The custom safety services provided in baby KJ’s treatment helped launch UNCOVERseq and represent a major step forward in ensuring the safety of CRISPR-based therapies.
Enhancing Safety, Quality and Efficiency: IDT’s Growing Pipeline and Future Collaborations
Future IDT launches, planned for late 2025, include complementary offerings such as the Alt-R HDR Enhancer Protein, which is designed specifically for therapeutic applications, and manufactured by Aldevron, to meet rigorous quality standards. The HDR Enhancer Protein improves HDR efficiency in difficult-to-edit cells, and maintains safety and cell health. IDT, in collaboration with Aldevron, will also be launching a novel Cas9 mRNA to support early discovery to clinical stage customers. IDT has been a long-time provider of CRISPR gRNA libraries for screening applications and will unveil a new design tool later this year for efficient ordering of custom, configured libraries.
“IDT’s rich history, deep expertise, and ability to manufacture bespoke products at the highest quality uniquely positions us to consistently innovate on our CRISPR portfolio and support customers at every stage of their gene editing journey,” said Sandy Ottensmann, VP/GM, Gene Writing & Editing at IDT. “On the heels of the world’s first personalized CRISPR-based therapy, we’re honored to bring more CRISPR tools that will enable researchers to make important discoveries, like the one designed for baby KJ, and progress science forward.”
Learn more about IDT’s CRISPR portfolio here.