Guillain-Barré syndrome (GBS) is an immune-driven inflammatory disorder of the peripheral nervous system characterized by muscle weakness and paralysis. Despite treatment options, GBS stays severe, with a mortality rate of 3%-10%. The mechanisms behind GBS are poorly understood and new therapeutic options are needed.
Aminoglycoside antibiotics are essential for treating some severe bacterial infections but are notorious for causing irreversible hearing loss in 20%-47% of patients.
RAS G12D is one of the most frequent mutations in RAS, and when it occurs, it leaves RAS in a permanently active state, causing the cell to proliferate uncontrollably. Examples of the so-called RAS-addicted cancers are colorectal cancer or pancreatic ductal adenocarcinoma.
Immunostimulant therapy using agonistic cytokines or activating antibodies has been associated with off-target side effects, failure to preferentially activate cytotoxic lymphocytes (CTLs) over regulatory T cells (Treg), and the development of T-cell exhaustion. With the aim of overcoming these issues, researchers from Recourse Biologics Inc. designed a potentially first-in-class immunostimulatory fusion protein.
Dimicare Biotech and affiliated organizations have presented the discovery and preclinical characterization of DCB-001, a trichloroacetimidamide compound being developed as a novel precision antibiotic against multidrug-resistant strains.
The new gene therapy aims to address the root cause of prion disease by using the CHARM epigenetic editing platform from the Whitehead Institute to target and silence the gene that codes for the disease-causing protein.
Avirmax Biopharma Inc. has begun IND-enabling studies of ABI-201, a gene therapy for dry age-related macular degeneration (AMD). ABI-201 is an AAV vector that delivers three genes to correct the dysregulation of complement activation, to protect retinal pigment epithelia and photoreceptors, as well as to block retinal neovascularization.
Human antigen R (HuR) controls the stability and translation of several transcripts that are key for metabolism, inflammation and cancer, including TNF-α or MYC. Previous findings have shown the pro-tumorigenic role of HuR in hepatocellular carcinoma (HCC), and its inhibition to be involved in metabolic dysfunction-associated steatohepatitis (MASH).
Multiple endogenous retroviruses (ERVs) in human DNA may be programmed to activate as cancer therapy. A recent study, led by scientists at the Dana-Farber Cancer Institute, expanded on a previously reported case of kidney cancer cure after hematopoietic stem cell transplantation attributed to the expression of an ERV driven by the hypoxia-inducible factor 2 (HIF2). The question was whether this finding might play out with different ERVs and different types of cancer through HIF.
Taichengsi (Shanghai) Biomedical Co. Ltd. has described antibody-drug conjugates comprising an antibody covalently bound to a cytotoxic drug through a bivalent olefin linker optionally consisting of an isotope-labeled compound reported to be useful for the treatment of cancer.
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