Jennifer Kwon, PhD, on Reducing LDL Cholesterol in NHPs With Epigenome Editing
The senior scientist at Tune Therapeutics discussed preclinical research presented at the ASGCT 2023 meeting.
“As we learn more about sequencing technologies, we reveal what the side effects are from actual genome editing. And so, epigenome editing does offer an alternative, where, instead of cutting, we can actually just tune the DNA expression.”
Epigenome editing may work as a therapeutic modality for robust and durable gene silencing, according to recent preclinical research from Tune Therapeutics. The company investigated transient lipid nanoparticle delivery of an RNA encoding an epi-repressor targeted specifically to the cynomolgus macaque PCSK9 promoter to non-human primates. Researchers found that it led to epigenetic remodeling of target loci and repressed PCSK9 expression, which was accompanied by a stable reduction in low-density lipoprotein cholesterol of about 50%.
These data were presented in a late-breaking session at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, held May 16-20, in Los Angeles, California, by Jennifer Kwon, PhD, senior scientist, Tune Therapeutics. CGTLive spoke with Kwon to learn more about the potential of epigenome editing to modify diseases in a variety of cell types and tissues by repressing or promoting genes. She stressed the advantages of this modality of gene therapy as compared to gene editing, which yields permanent editing of the genome and a higher risk of adverse events with the cutting and editing technologies involved. She discussed the data presented at ASGCT and proof-of-concept efficacy seen in both human cell lines and non-human primates.
Click here to read more coverage of the ASGCT 2023 meeting.
REFERENCE
Kwon JB, Knapp A, Hill A, et al. Transient delivery of epigenome editors stably represses PCSK9 and lowers LDL cholesterol in non-human primates. Presented at: ASGCT 2023 Annual Meeting; May 16-20; Los Angeles, California. Late-breaking abstract.
Considerations for Administering Hemoglobinopathy Gene Therapies From a Pharmacist’s Perspective
April 20th 2024Alexis Kuhn, PharmD, BCOP, a pediatric oncology pharmacist at Mayo Clinic, discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.
