University of Miami receives USD 3.15m NIH grant for neonatal genome sequencing antisense therapy

The University of Miami School of Medicine has received a USD 3.15 million NIH R01 grant from the National Human Genome Research Institute to build a clinical framework linking rapid genome sequencing to patient-customized antisense oligonucleotide therapies for infants admitted to neonatal intensive care units. The four-year award, project number 1R01HG014337-01A1, runs through March 2030 and is led by principal investigators Timothy Wei-Wen Yu and Pankaj B. Agrawal in the university's Department of Pediatrics.

The BEGIN program — Beginning Genetic Interventions in Infants — targets a population where genetic conditions account for roughly 20% of NICU admissions. The research integrates three parallel workstreams: rapid whole-genome sequencing to identify pathogenic variants, a biorepository with RNA sequencing to assess which variants are amenable to antisense oligonucleotide intervention, and surveys of families and clinicians to evaluate the care model's acceptability.

Antisense oligonucleotides are short synthetic DNA or RNA molecules, typically 15 to 30 nucleotides in length, that can be designed to alter gene-splicing patterns or direct a target gene toward degradation. Because their sequence can be customized to a specific mutation, they represent a modular therapeutic platform for rare diseases where conventional drug development is constrained by small patient numbers. The grant positions ASO design as a scalable response to the diagnostic yield expected from NICU genome sequencing programs.

The award is administered under funding opportunity RFA-HG-25-002 and reviewed by a Special Emphasis Panel within NHGRI's molecular genetics and genomics portfolio. The HG014337 grant award reflects NHGRI's sustained interest in closing the gap between genomic diagnosis and therapeutic action, a priority that has gained traction as sequencing costs have declined and turnaround times for clinical genome analysis have shortened to days or hours in some hospital systems.

The translational structure of the BEGIN program distinguishes it from earlier NICU genomics efforts that focused primarily on diagnostic yield. By building a biorepository and establishing referral pathways to external laboratories and nonprofit organizations capable of manufacturing individualized ASOs, the project creates infrastructure that could persist beyond the grant period and serve patients identified through sequencing but not yet matched to a therapy. The VIGOR network, referenced in the project description as a collaborating resource, and GeneDx, named as a sequencing partner, extend the reach of the University of Miami platform into a broader case-identification system.

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