On the heels of its role in a historic medical milestone, Coralville-based Integrated DNA Technologies (IDT) announced the expansion of its end-to-end CRISPR portfolio to support faster development of gene-editing therapies. The company’s enhanced tools and services played a key role in the world’s first personalized CRISPR therapy for an infant with CPS1 deficiency.
IDT, which partnered with Aldevron on the treatment, supplied guide RNA (gRNA), off-target analysis services and regulatory support for the patient, KJ Muldoon. The therapy was developed and delivered in an accelerated timeline, a release stated.
Tailored gRNAs aim to speed discoveries
As research progresses from discovery to clinical application, the demand for high-purity CRISPR reagents continues to grow, the release stated. In response, IDT now offers online ordering of chemically synthesized gRNAs in a variety of modification and purity options.
IDT’s high-performance liquid chromatography (HPLC)-purified gRNAs – suited for use in gene editing of primary cells and in vivo models – are available in 2 nmol and 10 nmol yields, with larger quantities available upon request. Orders can ship in as few as 12 business days. Formats include CRISPR-Cas9 gRNA and Custom Alt-R CRISPR gRNA, with available modifications such as 2’ Fluoro and 2’ O-Methyl to enhance stability and specificity, the release stated.
Tools to support safety and regulatory progress
CRISPR-based gene editing enables targeted modifications in the genome but carries the risk of off-target effects. To help scientists assess potential off-target edits early in therapeutic development, IDT developed UNCOVERseq, an off-target nomination service based on an enhanced GUIDE-seq method.
Paired with IDT’s rhAmpSeq CRISPR Analysis System, which confirms off-target sites, the service helps researchers evaluate gene-editing risks and move more confidently toward clinical applications. The same custom safety services used in KJ Muldoon’s treatment informed the development of UNCOVERseq.
Planned launches build on growing pipeline
IDT plans to introduce additional products in late 2025, including the Alt-R HDR Enhancer Protein, manufactured by Aldevron, to improve homology-directed repair (HDR) efficiency in challenging cell types. The protein is designed to meet therapeutic-grade quality and safety standards.
The company also plans to launch a novel Cas9 mRNA product, aimed at serving customers from discovery through clinical development. Later this year, IDT will release a new design tool to streamline custom gRNA library ordering for CRISPR screening applications.
“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, vice president and general manager of gene writing and 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.”
