Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) marked a historic milestone this year by presenting the first case of treatment with base editors of a baby with a deadly metabolic disease.
Repair Biotechnologies Inc.’s REP-0003 has been awarded orphan drug designation by the FDA for the treatment of homozygous familial hypercholesterolemia (HoFH).
Cancer cachexia is a devastating condition that affects up to 80% of advanced cancer patients and causes approximately 2 million deaths worldwide annually. Cancer cachexia is characterized by uncontrolled weight loss and severe muscle wasting. Despite its significant impact, effective treatments remain elusive.
The search continues for effective treatments against metabolic syndrome and its related complication, diabetes. Current treatments fail in many patients to provide long-term glycemic control or lead to weight loss.
At the recent American Society of Gene and Cell Therapy (ASGCT) meeting, Wave Life Sciences Ltd. presented siRNAs designed to suppress expression of the liver gene inhibin subunit β E (INHBE). Human genetic data show that heterozygous INHBE loss-of-function carriers exhibit a healthy metabolic profile.
One of the main goals in the prevention of cardiovascular disorders is to maintain low-density lipoprotein cholesterol (LDL-C) at consistently low levels to ensure long-term cardiovascular protection. Investigators at Verve Therapeutics Inc. reported preclinical data on VERVE-102, a GalNAc base editing strategy designed to sustainably inactivate the PCSK9 gene and lower LDL-C in familial hypercholesterolemia.
Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances that the body can’t process. Current treatments mainly focus on managing symptoms and metabolite levels, and do not always prevent the progressive deterioration caused by mutations associated with the condition. However, emerging gene therapies hold promise for transforming these diseases by targeting their underlying causes, as presented in the oral abstract session, “Gene and cell therapy for metabolic diseases” of the ongoing 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) meeting in New Orleans.
Zhejiang Doer Biologics Co. Ltd. has presented data regarding their FGF21R/GCGR/GLP-1R triple agonist DR-10624 for the treatment of metabolic dysfunction-associated steatohepatitis (MASH).
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