Inclusion body myositis (IBM) is the most common acquired myopathy in people over 50 years of age, characterized by chronic and progressive muscle weakness, where its pathogenesis involves inflammatory and degenerative pathways that are not well understood to date.
Bardet-Biedl syndrome (BBS) is a group of rare autosomal recessive ciliopathies characterized by dysfunction of primary cilia, which affects multiple organ systems and leads to early-onset obesity, progressive retinal degeneration resulting in vision loss or blindness, and renal abnormalities that may progress to renal failure. Mutations in the BBS10 gene are the second most prevalent cause of BBS, accounting for over 20% of cases.
Hepatocellular carcinoma (HCC) is a fatal cancer and the third cause of cancer-related deaths worldwide. Current therapies have focused on CAR T cells for treating HCC. Glypican-3 (GPC3) is a membrane protein that is overexpressed in HCC but not in healthy adult liver tissue, thus becoming a promising therapeutic target for HCC management.
Rhinovirus (RV) infection is among the most common respiratory infections causing exacerbations of chronic obstructive pulmonary disease and asthma. The high antigenic diversity of RV is a barrier to the development of effective cross-protective vaccines for RV infection.
Neuroblastoma is a pediatric solid tumor characterized by dysregulation of the CDK-RB-E2F axis, leading to elevated E2F activity, uncontrolled progression through the G1/S transition and an aggressive tumor phenotype. Researchers from Circle Pharma Inc. and collaborators presented preclinical data on CID-078, a first-in-class oral macrocycle with dual cyclin A and B RxL inhibitory activity, in models of neuroblastoma.
To address the various limitations of traditional CAR T therapy in autoimmune disease, Sail Biomedicines Inc. has developed an in vivo CAR T platform that enables in vivo transient programming of patient immune cells.
Frontera Therapeutics Inc. has developed FT-017, an adenovirus-associated vector(AAV)-based gene therapy that carries a human MYBPC3 optimized codon, for the treatment of hypertrophic cardiomyopathy (HCM).
Aging and chronic conditions are risk factors for mortality in patients with pneumonia. Lipoprotein-associated phospholipase A2 (Lp-PLA2) is a recognized biomarker of vascular inflammation, where high levels of Lp-PLA2 have been found in the plasma of patients with several chronic conditions such as chronic kidney disease or diabetes.
Latus Bio Inc. is developing a new gene therapy, LTS-101, for the treatment of neuronal ceroid lipofuscinosis type2 (CLN2), a form of Batten disease characterized by deficiency in the tripeptidyl peptidase 1 (TPP1) protein that leads to lysosomal dysfunction and neurodegeneration.
Tissue factor (TF), a transmembrane protein overexpressed in tumors such as cervical, head and neck, NSCLC, and pancreatic cancers, initiates the extrinsic coagulation pathway under normal subendothelial expression. TF-targeted vedotin antibody-drug conjugates (ADCs) are in clinical evaluation for solid tumors, but toxicities, including peripheral neuropathy, ocular effects and epistaxis, limit their use.
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