Science Digest: Biotechs Advancing Gene Therapy in North Carolina
- Karchem Consulting
- 3 days ago
- 3 min read
North Carolina has emerged in recent years as a biotech hub, specifically within the gene therapy space. As part of this Science Digest, a follow-up to Scaling Cell & Gene Therapies – which examines the state of cell and gene therapy, as well as the challenges that biotechs face entering 2026 – we delve into two companies that are utilizing innovative technologies and research techniques to push the possibilities of gene therapy forward.
Leveraging AI & Automation to Combat Antibiotic-Resistant Bacteria
Locus Biosciences, a Morrisville-based biotech, was recently awarded a $3.3 million contract to fund a proof-of-concept clinical trial of LBP-PA01, an AI-designed bacteriophage therapeutic. The bacteriophage therapeutic is designed to treat hospital-acquired pneumonia (HAP) and ventilator-associated pneumonia (VAP) caused by Pseudomonas aeruginosa, an antibiotic-resistant bacterium. HAP and VAP are common hospital-induced infections and are among the leading causes of mortality within intensive care units. The bacteria Pseudomonas aeruginosa has been identified by the World Health Organization as a significant public health threat as a result of its antibiotic-resistant properties.
As part of the Phase 1b clinical trial, Locus will research the safety, efficacy, and optimal dosing of patients whose infections can no longer be adequately treated using traditional antibiotics.
LBP-PA01 was created using Locus' AI-driven "discovery engine," which is capable of measuring millions of phage-bacteria interactions and generating more than a quadrillion potential phage combinations. Once the most promising candidates are identified, the discovery engine uses large language models to predict optimal genomic engineering configurations aimed at targeting HAP and VAP pathogens.
Clinical trials such as these used to take years to complete, a process comprising multiple rounds of research, each one increasing the chances of human error being introduced. Thanks to the utilization of AI and automation, technologies that are becoming increasingly essential for biotechs in today's industry, Locus can now perform this trial in just weeks, vastly accelerating the potential for scientific breakthroughs.
Treating Hepatitis B Through Direct Viral Gene Editing
Precision BioSciences, a Durham-based clinical-stage biotech, announced new data showing dose-dependent antiviral response for PBGENE-HBV, a cutting-edge in vivo gene editing therapy developed for treating chronic Hepatitis B. The announcement occurred during a late-breaking oral presentation at the American Association for the Study of Liver Diseases' The Liver Meeting in November 2025.
This Phase 1 study of PBGENE-HBV, which directly targets HBV cccDNA and integrated HBV DNA, found activity in all nine of its patients across 22 doses. According to Precision, the Phase 1 study suggests the presence of antiviral activity resulting from direct editing of viral genomes in patients. Precision believes that this has the potential to end viral transcription and antigen production, ultimately leading to a full cure for cccDNA.
Precision plans to finish dose administrations within the current cohort during early 2026. Once the exact dosage for halting nucleos(t)ide analogue therapy is identified, Precision will seek to initiate the Part 2 expansion phase, aimed at optimizing the PBGENE-HBV dose regimen in a larger number of patients for safety and efficacy.
Here At Karchem Consulting, We Keep Up With Your Science
Lab research is only as good as the data models that hold it. Biotechs leading the charge to develop groundbreaking gene therapies can't afford to let mismanaged and disorganized ecosystems slow them down.
Our team of scientists stays up-to-date on the latest in impactful research and emerging technologies so that we are always ready to help biotechs solve their data problems.
Want to learn more about our work designing laboratory software solutions for gene therapy companies? Contact us at Karchem Consulting today to get started.
