Charcot-Marie-Tooth (CMT) disease is a group of clinically and genetically heterogeneous sensorimotor peripheral neuropathies. It is the most frequent inherited neuromuscular disorder affecting 9.7-82.3 patients per 100,000 individuals. Over 100 genes with all patterns of inheritance have been linked to CMT. These genes encode proteins involved in nerve-specific processes, such as axonal transport, myelination and synaptic transmission, and in general housekeeping pathways. However, the reason underlying why defects in such ubiquitous proteins predominantly affect peripheral nerves remains unclear.
About one-third of patients with major depressive disorder (MDD) are treatment resistant. Ketamine is very effective in treatment-resistant depression, but it is associated with psychotomimetic effects. Metabotropic glutamate mGlu2 and mGlu3 receptors negatively regulate the release of glutamate and are involved in the pathogenesis of depression.
Adolore Biotherapeutics Inc. has announced that the FDA has granted orphan drug designation to the company’s Kv7-activating rdHSV-CA8* gene therapy for treatment of primary and secondary erythromelalgia.
Triple-negative breast cancer (TNBC) lacks hormone receptors and HER2 amplification, limiting the effectiveness of targeted therapies and contributing to its aggressive clinical behavior. As aberrant activation of STAT3 is a key driver of TNBC growth, strategies aimed at suppressing STAT3 signaling are emerging as a potential treatment approach.
GT Biopharma Inc. has filed an IND application with the FDA for GTB-5550 TriKE, a B7-H3-targeted natural killer (NK) cell engager for the treatment of B7-H3-expressing solid tumor cancers. Pending approval, the planned phase I basket trial will evaluate GTB-5550 administered subcutaneously in solid tumors.
Interax Biotech Ltd. and Alveus Therapeutics Inc. have signed a strategic research collaboration and licensing agreement to develop a differentiated small-molecule candidate for metabolic disease. Designed to overcome the limitations of current obesity medications, the candidate is expected to deliver durable weight loss with superior tolerability.
Medicenna Therapeutics Corp. is advancing MDNA-113, a first-in-class, IL-13-directed and conditionally activated PD-1 x IL-2 bifunctional superkine, through preclinical development toward a planned IND filing in the second half of this year.
About 10% of acute hepatitis cases and 50% of acute liver failure are caused by drug induction, where treatment remains largely limited. The SLIT/ROBO signaling axis is composed of the secretory SLIT proteins (SLIT1, SLIT2 and SLIT3) and their corresponding receptor ROBO. This axis is known to be involved in organ development, angiogenesis and leukocyte migration, as well as cancer metastasis, and has shown protective effects against organ damage.
Once it was considered to be more or less a passive energy-storing device that could double as a cushion. But increasingly, fat is conceptualized as an endocrine organ as much as a tissue type. Now, separate research groups have reported new insights into the functional roles of different fats based on their anatomical location and functional characteristics.
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