Future of AIAI

Transforming precision medicine: Artificial Intelligence and the genomics frontier

By Ajay Gannerkote, President, Integrated DNA Technologies

Artificial intelligence (AI) is rapidly reshaping how industries analyze complex datasets, forecast trends, and make critical decisions. Nowhere is this transformation more evident than in the life sciences, where AI is converging with genomics to accelerate precision medicine, uncover new drug targets, and pioneer cutting-edge therapies like RNA editing. At the heart of this evolution lies a new kind of scientific synergy—where algorithmic insights empower molecular biology to move faster, deeper, and more accurately to accelerate medical breakthroughs. 

Precision medicine, which tailors treatment based on a patient’s genetic profile, is heavily reliant on the capacity to interpret vast swaths of biological data. AI-powered analytics provide the computational strength and pattern recognition capabilities needed to extract meaningful signals from complex genomic information. This empowers clinicians and researchers to classify disease subtypes, predict individual treatment responses, and identify previously elusive biomarkers. 

AI meets genomic engineering: The power of integrated approaches 

One of the most promising applications of AI in genomics is its role in antisense oligonucleotide (ASO) development for rare and ultra-rare genetic conditions. These RNA-targeting therapies can alter gene expression in ways small-molecule drugs cannot. AI helps streamline the discovery pipeline by analyzing patient datasets to identify targetable mutations, significantly reducing the number of candidate compounds that need to be screened for efficacy. This is crucial for n-of-1 drugs, developed for individual patients, where time and cost constraints demand ultra-efficient development strategies.  

Integrated DNA Technologies (IDT) plays a multifaceted role in enabling such research. The company provides highly customizable nucleic acid synthesis, from simple oligonucleotides to highly complex chemical modifications. This flexibility is essential when optimizing candidate ASOs or designing CRISPR reagents for screening hundreds or thousands of genomic loci. In the context of early research for developing precision therapies, this rapid prototyping capacity accelerates progress from hypothesis to future tangible innovation. 

For example, a cutting-edge human phenome project is leveraging more than 100,000 IDT custom CRISPR guide RNAs (gRNAs) to conduct genome-wide knockout screens in human cells. These high-throughput experiments involve editing one gene at a time across the genome, followed by imaging-based analysis to assess cellular phenotypic outcomes. AI tools then interpret the complex image data, identifying gene-disease relationships. IDT contributes not only the reagents but also manufacturing expertise and reliability that ensure experimental consistency across such massive screens.  

The end-to-end workflow is further enhanced by sister companies under Danaher—Molecular Devices, Beckman Coulter, Leica Biosystems and Leica Microsystems—who provide automation and imaging capabilities. Together, this integrated system enables execution of AI-driven drug discovery at a scale that would be otherwise unmanageable. 

Next-generation CRISPR and sequence analysis tools 

CRISPR gene editing remains one of the most transformative technologies in modern bioscience, and AI is critical in making it more precise and clinically viable. The concern with off-target effects (OTEs)—unintended edits to the genome—has led to the development of high-fidelity enzymes like the Alt-R HiFi Cas9. Unlike earlier CRISPR variants, which often lacked precision in real-world biological settings, the Alt-R HiFi Cas9 is optimized for use in the ribonucleoprotein (RNP) format. This significantly reduces OTEs while maintaining high editing efficiency.  

At IDT, we developed this enzyme through an extensive screen of more than 250,000 Cas9 mutants. The final variant demonstrated superior performance in human hematopoietic stem and progenitor cells, effectively correcting the mutation responsible for sickle cell disease with minimal unintended edits. Developing mutants with unique functionality is an importantpart of translating CRISPR systems to clinical applications. 

Another breakthrough tool is the rhAmpSeq CRISPR Analysis System, which enables researchers to quantify OTEs with confidence, quickly. Scientists can multiplex the sequencing of their CRISPR editing outcomes across dozens or hundreds of genomic loci in a single run. Using blocked primers activated by RNase H2, the platform minimizes primer-dimer formation and improves amplification specificity. This highly multiplexed amplification protocol accelerates data generation and makes it especially effective for characterizing both on- and off-target editing events.  

These systems have been used in leading labs, including those working on developing CAR T-cell and CAR-NK therapies. Researchers from institutions like the University of Texas MD Anderson Cancer Center have applied Alt-R and rhAmpSeq systems to measure the specificity of gene-edited immune cells, confirming low off-target activity thanks to HiFi Cas9 and optimized gRNAs. 

AI is increasingly being integrated into CRISPR workflows to enhance guide RNA (gRNA) design and genome editing precision. Across the field, AI-driven platforms and algorithms are used to predict off-target effects, optimize gRNA sequences, and rank candidates based on predicted activity and safety. These advancements empower researchers to make data informed decisions, which streamlines the design process and improves the likelihood of successful genome editing. As AI continues to evolve, its role in supporting CRISPR applications is expected to expand, driving greater efficiency and accuracy in gene editing research. 

Synthetic biology and DNA assembly: Innovating from design to delivery 

Synthetic biology continues to benefit from improvements in AI and automated sequence design. DNA cloning, a foundational technique for gene function studies, protein production, and synthetic circuit engineering, requires thoughtful sequence planning and assembly strategy. This process can be streamlined by a wide and well-integrated portfolio of tools and services, including gBlocks Gene Fragments and customizable synthetic DNA, supported by online design software that streamline complex projects. 

One common challenge in DNA synthesis is managing sequence complexity, such as high guanine and cytosine content, repetitive elements, and secondary structures. Across the field, researchers are leveraging AI to identify problematic regions within DNA sequences and to suggest design adjustments. These strategies are especially important when assembling large constructs, such as multi-gene cassettes or regulatory pathways, where sequence complexity can present significant obstacles. While a variety of online design platforms and software tools exist to support these efforts, the integration of AI into sequence design and analysis is an evolving area, with many organizations exploring how best to harness these technologies to streamline synthetic biology workflows. 

Researchers using Golden Gate or seamless cloning strategies benefit from these tools by reducing trial-and-error during vector construction. The combination of scarless assembly, predictive sequence modeling, and robust synthesis ensures high success rates even in challenging applications. Whether for metabolic engineering, vaccine development, or synthetic biosensors, a good selection of tools and support infrastructure gives scientists confidence in their constructs. 

IDT’s DNA fragments also play a crucial role in agricultural genomics, where researchers decode genomes of diverse crop species to identify traits like drought resistance or pest tolerance. The ability to synthesize precise DNA sequences that correspond to specific alleles or genetic variants accelerates marker-assisted breeding programs. These efforts are further enhanced by AI, which correlates genotype and phenotype data to predict the success of selective breeding strategies. 

Building the future of genomics through quality and scalability 

As AI continues to expand its reach across genomic science, the importance of consistent, high-quality reagents and services grows in parallel. Our global manufacturing network, which includes sites in the US, Belgium, and Singapore, provides researchers with fast turnaround times and scalable production—from research-grade materials to CGMP-compliant manufacturing. 

The company’s commitment to supporting researchers is evident in our collaborative approach. We routinely engage with academic consortia, biopharma developers, and public health institutions, offering not only products but also expert consultation and technical support, resulting in dozens of formal research collaborations and thousands of support cases. In areas such as pandemic preparedness, our PCR and NGS tools were instrumental in rapidly identifying and tracking viral variants, helping facilitate agility within the biotech infrastructure to meet urgent global needs. 

As new challenges and opportunities emerge—whether in the form supporting mRNA therapeutics, RNA editing, or real-time disease surveillance—companies like IDT are helping to translate the potential of AI and genomics into real-world impact. The convergence of computational intelligence and molecular precision is not only enabling science at an unprecedented scale but also redefining what is possible in medicine, agriculture, and beyond. 

Accelerating discovery with intelligence and innovation 

From disease detection to drug design and agricultural innovation, the unifying thread is AI’s ability to handle and interpret massive datasets, and the capacity of genomics to generate them. AI-driven analytics will not replace the need for rigorous experimentation; instead, they supercharge it, reducing the time between hypothesis and validation. Our suite of innovative solutions—from high-fidelity enzymes and multiplexed analysis tools to customizable gene fragments and AI-enhanced design platforms—illustrates how technological depth and scientific rigor can empower discovery at every stage. The synergy between AI and molecular biology represents a paradigm shift in how we approach health, disease, and biological discovery.  

As more AI capabilities are integrated into genomic research pipelines, we can expect continued acceleration in drug discovery, greater customization in therapeutic development, and broader applications across fields from oncology to virology. The tools and services we provide are enabling this transformation—not through promotion, but by supporting the infrastructure of progress. The intersection of AI and genomics is not a futuristic concept: it is the reality of today’s biomedical innovation landscape. Through AI-enabled workflows and high-quality molecular tools, researchers are charting new territory in understanding, diagnosing, and treating disease with a level of precision that was not previously possible. 

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