Akouos Inc. has received IND clearance from the FDA to initiate a first-in-human phase I/II pediatric trial of AK-OTOF, a gene therapy intended for the treatment of patients with otoferlin gene (OTOF)-mediated hearing loss.
Boehringer Ingelheim Pharma GmbH & Co KG has presented data on the identification of several potent and selective tyrosine kinase inhibitors (TKIs) of HER2 that are highly active against its oncogenic exon 20 YVMA insertion mutant (HER2 YVMA), which is the most prevalent HER2 exon 20 mutation in non-small-cell lung cancer (NSCLC).
Transcenta Holding Ltd. has received IND clearance from the FDA for TST-003, its first-in-class, high-affinity, humanized monoclonal antibody targeting gremlin-1.
The design of genetically modified exosomes that combine multiple targets killed cancer cells and conferred immunity against them. Scientists at the University of Southern California (USC) applied bioengineering techniques to introduce up to four antitumor functions in the same type of extracellular vesicles and destroy EGFR-positive triple-negative breast cancer (TNBC) tumor cells.
Age-related diseases have been explained as due in part to the excessive generation and accumulation of waste products like the various insoluble protein aggregates observed in nondividing neurons of Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis and Huntington’s disease.
Aliad Biopharma Co. Ltd. has presented compounds acting as autophagy inducer, mTOR complex 1 (mTORC1) and mTORC2 inhibitors reported to be useful for the treatment of autism spectrum disorders, Alzheimer’s disease, epilepsy, fragile-X syndrome, macrocephaly and tuberous sclerosis.
Wigen Biomedicine Technology (Shanghai) Co. Ltd. has identified Wee1-like protein kinase (Wee1) inhibitors reported to be useful for the treatment of cancer.
Marvel Biotechnology Inc. has synthesized purine compounds acting as adenosine A2A receptor (ADORA2A) antagonists reported to be useful for the treatment of cancer, depression, multiple sclerosis, nonalcoholic steatohepatitis (NASH), scleroderma, attention deficit hyperactivity disorder (ADHD), and Parkinson's and Alzheimer's diseases.
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