Only a couple of years since the first sickle cell disease (SCD) gene therapies gained U.S. FDA approval, researchers are working to expand access for younger children, and to improve manufacturing and commercialization to reach patients faster.
The overexpression of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) is tied to poor prognosis in several cancer types, including osteosarcoma and Ewing sarcoma, and is therefore a potential therapeutic target in these cancers. In this regard, Avammune Therapeutics Inc. is developing an ENPP1 inhibitor – AVA-NP-695.
A little over a week after announcing that the Evoke and Evoke+ studies failed to show that oral semaglutide could slow cognition decline in patients with Alzheimer’s disease, investors and researchers got the first look at the actual data from the studies, which were presented at the Clinical Trials on Alzheimer’s Disease 2025 meeting.
At the Clinical Trials on Alzheimer’s Disease 2025 meeting, a panel of experts discussed the need for developing combination therapies for the complex diseases that result in Alzheimer's disease and other dementias.
Abilita Therapeutics Inc. has developed a novel approach targeting CCR8 – ABT-863 – that works as a potent inverse agonist to block CCL1-dependent and basal receptor signaling.
LRRK2 plays a key role in the biology of multiple neurodegenerative disorders, including Parkinson’s disease (PD) and progressive supranuclear palsy (PSP), where mutations or altered activity are associated with impaired cellular signaling and neuronal decline.
Previous findings have shown that the bisteric mTORC1 inhibitor Rapalink-1 exerts superior efficacy than the parent inhibitors rapamycin and sapanisertib in orthotopic xenograft models of glioblastoma. The bitopic clinical derivative of this compound is RMC-5552 from Revolution Medicines Inc., which is currently in phase I/II clinical studies for glioblastoma.
At the Clinical Trials on Alzheimer’s Disease 2025 meeting, a panel of experts discussed the need for developing combination therapies for the complex diseases that result in Alzheimer's disease and other dementias.
Abion Inc. has presented data for ABN-202, a hyperglycosylated interferon (IFN) β mutein fused to a monoclonal antibody targeting tumor-associated calcium signal transducer 2 (TROP2) that retains activity under acidic conditions.
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