Homozygous familial hypercholesterolemia (HoFH) is a genetic condition caused by mutations in the LDLR gene and characterized by severe hypercholesterolemia and premature cardiovascular disease. Repair Biotechnologies Inc. has developed REP-0003, a therapeutic based on the Cholesterol Degrading Platform (CDP) that degrades excessive intracellular free cholesterol into a non-toxic and excretable catabolite to safely reverse cholesterol-rich vulnerable plaque.
Though immune checkpoint inhibitors have improved the outcomes of patients with hepatocellular carcinoma (HCC), the response rates remain limited. At the annual meeting of the American Association for the Study of Liver Diseases, researchers highlighted N-lysine methyltransferase SMYD2 as an oncogenic protein overexpressed in several tumor types.
Human endogenous retroviruses (HERVs) are residues from ancient viral infections that have become integrated into the human genome. One of them – HERV-K – is aberrantly reactivated in solid tumors but silent in healthy tissues.
Researchers at Tubulis GmbH reported preclinical efficacy data on TUB-030, a novel 5T4-targeting antibody-drug conjugate (ADC), in several sarcoma subtypes.
Heart failure with preserved ejection fraction (HFpEF) is a complex disease with limited therapeutic options. Protein histone deacetylase 6 (HDAC6) is known to be involved in several biological processes, such as autophagy or inflammation, among others, but its impact on HFpEF has not been well evaluated to date.
METTL3, the enzyme that adds the m6A RNA modification, is a key regulator of RNA processing and protein synthesis. In cancer, METTL3 is often overexpressed, driving tumor growth, invasion and therapy resistance across multiple malignancies, including lung, pancreatic, ovarian, colorectal cancers and acute myeloid leukemia.
Schizophrenia is a complex psychiatric disorder marked by positive, negative and cognitive symptoms, many of which are poorly addressed by current dopamine-blocking antipsychotics. Preclinical studies show that muscarinic acetylcholine M4 receptor positive allosteric modulators (M4 PAMs) can reduce hyperdopaminergic activity and improve both positive and negative symptom-related behaviors, suggesting they may offer enhanced antipsychotic efficacy with improved tolerability.
The limited efficacy of cancer immunotherapy is usually attributed to suboptimal antitumor T-cell generation, as well as impaired immunological memory development. MDX-2004 is a trispecific antibody-fusion protein developed by Modex Therapeutics Inc. that targets CD3, CD28 and CD137 on human T cells, which was designed to overcome these limitations by rejuvenating the immune system and enhancing antitumor immunity.
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