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.
Current anticancer approaches, such as antibody or CAR T-cell therapies, rely on targeting tumor-associated antigens rather than tumor-specific antigens, with the consequent on-target, off-tumor effects.
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).
Researchers from Protagonist Therapeutics Inc. reported the preclinical characterization of PN-881, an oral macrocyclic peptide that inhibits the dimeric forms of IL-17 – AA, AF, and FF.
Aberrant activation of β-catenin, often due to mutations in its encoding gene or loss-of-function mutations in APC, contributes to tumor progression and therapy resistance, as seen in advanced hepatocellular carcinoma (HCC), where approximately 30% of cases exhibit β-catenin activation.
Radiopharmaceuticals can offer a targeted approach for cancers that have limited therapeutical options. Abdera Therapeutics Inc. recently presented results of their novel 5T4-targeted radiopharmaceutical.
Epidermal growth factor receptor (EGFR), when overactive or overexpressed, may lead to tumor growth and spread, and is thus a robust target for therapy.
Current therapies for chronic hepatitis B virus (HBV) infection can effectively suppress viral replication but do not achieve a functional or complete cure. Capsid assembly modulators inhibit the assembly of viral capsids, prevent the encapsidation of pregenomic RNA, and interfere with both the formation and amplification of covalently closed circular DNA, which is essential for viral persistence.
Among the most significant genetic risk factors for age-related macular degeneration is the Y402H variant of complement factor H, which, along with its splice isoform FHL-1, impairs binding to the retinal surface.
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