IDT Launches HDR Enhancer Protein to Boost CRISPR Gene Editing Efficiency in Therapeutic Applications
核心洞察
Integrated DNA Technologies (搜索) has launched the Alt-R HDR Enhancer Protein (搜索), a research-grade recombinant protein that increases homology-directed repair efficiency by up to two-fold in challenging cells including iPSCs and HSPCs.
The protein-based enhancer maintains cell viability and genomic integrity while promoting more precise genome modifications without increasing off-target edits or translocations.
This launch expands IDT's CRISPR portfolio alongside new high-purity gRNAs and UNCOVERseq off-target analysis services, supporting the acceleration of CRISPR-based therapies from research to clinical applications.
Integrated DNA Technologies (搜索) (IDT) has launched the Alt-R HDR Enhancer Protein (搜索), a novel recombinant protein designed to significantly improve the efficiency of homology-directed repair (HDR (搜索)) in CRISPR gene editing applications. The protein-based solution addresses a critical challenge in therapeutic genome editing by facilitating up to a two-fold increase in HDR efficiency in difficult-to-edit cells, including induced pluripotent stem cells (iPSCs) and hematopoietic stem and progenitor cells (HSPCs).
Addressing CRISPR's HDR Challenge
While CRISPR-Cas systems demonstrate high efficiency in generating gene knockouts through non-homologous end joining (NHEJ (搜索)), achieving precise knock-ins via HDR (搜索) has remained challenging in primary and difficult-to-edit cells. The Alt-R HDR Enhancer Protein (搜索) specifically targets this limitation by shifting the DNA repair pathway balance toward HDR, enabling more precise genome modifications.
"IDT's legacy of innovation, market leadership in CRISPR and strong collaborations with gene editing pioneers enabled us to develop a first-to-market Alt-R HDR Enhancer Protein (搜索) that delivers on our customers therapeutic research needs and desire to fast-track breakthroughs," said Sandy Ottensmann, VP/GM, Gene Writing & Editing at IDT.
Enhanced Safety and Efficacy Profile
Studies conducted with the Alt-R HDR Enhancer Protein (搜索) demonstrate significant improvements in HDR (搜索) efficiency across multiple loci and cell types. Critically, the protein maintains cell viability and genomic integrity while preserving editing specificity, with no increase in off-target edits or translocations. This safety profile makes it well-suited for high-fidelity editing workflows required in therapeutic development.
The enhancer protein seamlessly integrates into existing CRISPR workflows and is compatible with different Cas systems and common delivery methods. Manufactured by Aldevron (搜索), a fellow Danaher (搜索) operating company, the protein meets rigorous quality standards required for therapeutic applications.
Expanding Translational CRISPR Portfolio
The HDR (搜索) Enhancer Protein launch is part of IDT's broader expansion of translational CRISPR solutions designed to bridge the gap from discovery research to clinical applications. The company has also introduced high-purity guide RNAs (gRNAs) available through high-performance liquid chromatography (HPLC) purification, orderable in 2 nmol and 10 nmol yields with shipping in as little as 12 business days.
Additionally, IDT has launched UNCOVERseq, an off-target nomination service using enhanced GUIDE-seq methodology to identify potential off-target editing sites. When combined with IDT's rhAmpSeq CRISPR Analysis System for off-target confirmation, these services enable researchers to assess editing risks early in therapeutic development.
Clinical Translation Success
These developments follow IDT's contribution to a landmark clinical achievement - the world's first personalized CRISPR therapy delivered to infant KJ Muldoon, who suffered from CPS1 deficiency (搜索). IDT provided the guide RNA (搜索), off-target analysis services, and regulatory support for this treatment, which was delivered in a significantly compressed timeline through collaboration with Aldevron (搜索).
"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," Ottensmann noted.
Product Availability and Future Pipeline
The Alt-R HDR Enhancer Protein (搜索) is currently available in research use only (RUO) format at 500 μg and 5 mg quantities, with current good manufacturing practice (CGMP) grade to follow. IDT plans additional launches for late 2025, including a novel Cas9 mRNA (搜索) developed in collaboration with Aldevron (搜索) to support customers from early discovery through clinical stages.
The company continues to expand its end-to-end CRISPR workflow capabilities, from design to analysis, supporting gene editing pioneers with optimized Cas9 (搜索) and Cas12a (搜索) systems, customizable gRNAs, HDR (搜索) solutions, and analysis tools backed by expert support.