Daniel Hart, PhD, on CRISPR-Mediated In Vivo Epigenomic Activation


The Senior Director and Head of Technology Development at Epic Bio discussed potential applications of the new technology.

“We've become interested in this other side of epigenetic regulation, which is that you can turn genes on. So, I think people are familiar with the idea you can turn genes off with epigenetic editing but switching them on is something that has not really been demonstrated before. So, we set up screens to identify these factors, [were] successful in doing so. Now that we have them, I think the challenge is understanding what the best applications would be for them, but I think it's easy to imagine deploying them in diseases known as haploinsufficiencies.”

CRISPR-mediated novel epigenetic activators demonstrated durable gene activation in both immortalized and primary cells, with 2-fold activation of LDLR sustained for up to 5 weeks in primary human hepatocytes within a humanized mouse liver model. LDLR haploinsufficiency constitutes 85 to 90% of genetically confirmed cases of familial hypercholesterolemia and these data validate the potential of epigenetic activation of disease-modifying genes in vivo.

Daniel Hart, PhD, Senior Director and Head of Technology Development, Epic Bio, presented these data at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland. CGTLive spoke with Hart to learn more about the findings and their significance in the field. He shared further research that Epic is conducting with epigenomic activation and potential applications of the technology in the future. He also shared his excitement for the growing prominence of epigenomic editing and nonviral strategies for gene editing.

Carosso G, Yeo R, Gainous TB, et al. Compact Epigenetic Modulators for CRISPR Mediated Persistent Gene Activation. Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract #56
Related Videos
Luke Roberts, MBBS, PhD, on Challenges in Developing Gene Therapy for Heart Failure
Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Lisa Nieland on Slowing Tumor Growth in Glioblastoma With Novel AAV Therapy
Manali Kamdar, MD, on Acclimating to Routine CAR T Practice in the Field
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Jennifer Taylor-Cousar, MD, MSCS, on Early Signals of Efficacy With Cystic Fibrosis Gene Therapy
Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Manali Kamdar, MD, on Evaluating Liso-Cel in Mantle Cell Lymphoma by Lines of Therapy, Prior BTKi
© 2024 MJH Life Sciences

All rights reserved.