# Accelerating Gene Therapy and Editing with Advanced MS-Based Data Analysis for Nonstandard and Hybrid Nucleotide Sequences

> **NIH NIH R44** · MASSMATRIX, INC. · 2024 · $860,633

## Abstract

PROJECT SUMMARY / ABSTRACT
Title: Accelerating Gene Therapy and Editing with Advanced MS-Based Data Analysis for Nonstandard and
Hybrid Nucleotide Sequences
This project seeks to improve reliability and speed up the development of life-saving and life-enhancing
oligonucleotide-based therapeutics and magnify the positive impact of biomedical research and education
worldwide, leading to a quantum leap in our understanding of the molecular and cellular pathways and
mechanisms involved in healthy and diseased biological systems. Direct sequencing via high-resolution mass
spectrometry is an essential assay in the development and QA/ manufacturing of these therapeutics but
manual intervention in the data analysis pipeline due to inadequate software has caused a bottleneck in the
process of getting these life-saving therapeutics to those in need. Similarly, in-depth characterization of RNA
and their modifications needed for improving biomarker identification is hindered in the RNA research
community.
To address these challenges, semi-automated direct sequencing of oligonucleotides by mass spectrometry will
be implemented and delivered on MassMatrix’s high-throughput, enterprise cloud platform (mmCloud).
Enhancements to MassMatrix’s oligonucleotide sequencing engine will be made for characterizing nonstandard,
hybrid and conjugate oligonucleotides. A unified data repository on mmCloud will enable assay comparisons,
knowledge capture and management, and collaboration within and across research teams. Together, these
aims are expected to improve the reliability and accuracy of results as well as to significantly reduce the
oligonucleotide characterization bottleneck for the pharmaceutical industry and the research community. Deeper
understanding and better decision making will follow, both having a potentially dramatic positive impact on
downstream processes and resource deployment, including improved drug safety, efficacy and availability of life-
saving and enhancing therapeutics.

## Key facts

- **NIH application ID:** 10881890
- **Project number:** 5R44GM152984-02
- **Recipient organization:** MASSMATRIX, INC.
- **Principal Investigator:** George Hall Johnson
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $860,633
- **Award type:** 5
- **Project period:** 2023-07-05 → 2025-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10881890

## Citation

> US National Institutes of Health, RePORTER application 10881890, Accelerating Gene Therapy and Editing with Advanced MS-Based Data Analysis for Nonstandard and Hybrid Nucleotide Sequences (5R44GM152984-02). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10881890. Licensed CC0.

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