Parkinson’s disease (PD) is a progressive disease characterized by loss of dopaminergic neurons in the substantia nigra, which lead to motor symptoms. Aromatic-L-amino-acid decarboxylase (AADC) converts levodopa into dopamine and glial cell line-derived neurotrophic factor (GDNF) promotes the survival, growth and regeneration of dopaminergic neurons. VGN-R09b is an adeno-associated viral vector (AAV)-based AADC and GDNF combination gene therapy that is delivered to the striatum for the treatment of PD.
Polo-like kinase 4 (PLK4), which regulates centriole duplication and mitotic progression, is overexpressed in several cancers, including neuroblastoma as well as breast, lung and colorectal cancers, making it an attractive target. Several PLK4 inhibitors have been developed, but only one has entered clinical trials, which involve patients with acute myeloid or chronic myelomonocytic leukemia.
While immunotherapy targeting PD-1 or its ligand PD-L1 has transformed cancer treatment by overcoming T-cell exhaustion, only a minority of patients achieves durable responses after treatment.
Antimicrobial peptides such as human β-defensin 3 (hBD3) have shown promise as a treatment for periodontitis; however, their poor stability and high production costs have hindered their clinical translation until now.
In their efforts to develop next-generation antibiotics, researchers from Hunan University and collaborators designed amidated oligopyridinium peptidomimetics.
Primary sclerosing cholangitis is an autoimmune disease affecting the liver in which there is reduced expression and function of the bile acid receptor GPBAR1 in the cholangiocytes.
IKZF2, a transcription factor in the Ikaros family, plays a key role in stabilizing regulatory T (Treg) cells and promoting immune suppression within the tumor microenvironment (TME). Selective targeting of IKZF2 in intratumoral Tregs has the potential to enhance antitumor immunity and improve the efficacy of immunotherapy, while minimizing immune-related toxicities associated with systemic Treg depletion.
Recurv Pharma Inc. has developed a novel taxoid compound formulated in an oil-in-water nanoemulsion, named RP-001, as a potential therapeutic approach for the management of pancreatic ductal adenocarcinoma (PDAC), either as a single agent or combined with immune checkpoint inhibitors.
Allterum Therapeutics Inc. has obtained IND clearance from the FDA for 4A10, paving the way for initiation of a first-in-human phase I trial in patients with relapsed or refractory acute lymphoblastic leukemia (ALL). 4A10 is a monoclonal antibody with a human immunoglobulin G subclass 1 (IgG1) backbone that specifically binds to CD127 (IL-7Rα).
Basal-like breast cancer is a particularly aggressive subtype of the malignancy, for which it has been difficult to identify good therapeutic targets. In collaboration, European researchers mined public transcriptome datasets to show that the aldehyde dehydrogenase 1A isoform ALDH1A3 was predominantly expressed in epithelial cells within basal-like tumors, whereas the isoforms ALDH1A1 and ALDH1A2 were predominantly expressed in stromal and immune-associated cells.
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