Insilico Medicine Cayman Topco has obtained IND approval from the FDA for ISM-8969 for the treatment of Parkinson’s disease, enabling initiation of a phase I trial in healthy volunteers.
Researchers from Leiden University Medical Center previously demonstrated that the junction adhesion molecule-independent mutant (jin-3) reovirus, which is a double-stranded RNA virus not associated with severe human disease, presents extended tumor tropism compared to wild-type reoviruses.
The prevalence of asthma differs between men and women, and furthermore, the difference changes over the lifespan. “Asthma is more common in boys than girls, but more common in women than men,” Clare Lloyd told BioWorld. Females are particularly susceptible to asthma during developmental periods of hormonal changes, also known as puberty, pregnancy and menopause.
Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant skeletal muscle disorder with a prevalence of approximately 1 in 8,000. The disorder is driven by aberrant expression of double homeodomain protein 4 (DUX4) within the D4Z4 macrosatellite array. Currently, effective treatments for FSHD are lacking. Strategies aimed at reducing DUX4 expression could hold promise as potential therapeutic approaches for FSHD.
Recent evidence has suggested threonine tyrosine kinase (TTK) as a crucial element of the mitotic checkpoint for the correct functioning of spindle assembly checkpoint (SAC), making it a potential therapeutic target in cancer.
Scientists from the Smidt Heart Institute, Cedars-Sinai Medical Center (Los Angeles, CA, USA) recently explored adeno-associated virus (AAV) vectors carrying the T-box transcription factor 18 (TBX18) transgene to produce a physiologically responsive biological pacemaker activity for the treatment of bradycardia.
Hyperactivity of the transmembrane adaptor protein STING, which normally helps the cell respond to infection and injury, has been implicated in several autoimmune or inflammatory diseases, including systemic lupus erythematosus and amyotrophic lateral sclerosis.
Researchers from Lanzhou University and Southern Medical University (China) have presented the discovery of a structurally distinct pyrimidine-based chemotype for the inhibition of KRAS G12C.
MicroRNAs (miRNAs) are small noncoding RNAs gaining increasing attention due to their crucial role in gene expression regulation and influence in various cellular processes and diseases. miRNAs can be encapsulated in extracellular vesicles (EVs), which are released by most cell types to modulate gene expression in recipient cells.
B-cell precursor acute lymphoblastic leukemia (BCP-ALL) is the most common pediatric cancer. Although pediatric ALL is cured in over 90% of cases, children with high-risk BCP-ALL present increased chance of treatment failure and relapse.
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