Researchers from Neumirna Therapeutics ApS have presented an anti-miR-134 ASO approach named NMT.001 for the potential treatment of drug-resistant epilepsy.
Amphista Therapeutics Ltd. has developed and presented data for AMX-883, a novel orally bioavailable bromodomain-containing protein 9 (BRD9) degradation inducer for acute myeloid leukemia (AML)
treatment.
Mutations in the KCNT1 gene produce gain-of-function effects that lead to overactivation of the potassium channel and consequent disruption of normal neuronal electrical signaling. These alterations give rise to a severe, early-onset developmental and epileptic encephalopathy that is typically associated with a high seizure burden and resistance to standard antiseizure medications.
ENL-YEATS is an epigenetic reader that sustains transcriptional programs essential for AML, whereas FLT3 mutations, present in approximately 30% of patients, drive malignant proliferation. Dual inhibition of ENL-YEATS and FLT3 may therefore more effectively disrupt complementary drivers of leukemogenesis than FLT3 targeting alone.
Investigators from Secarna Pharmaceuticals GmbH & Co. KG recently presented data for their antisense oligonucleotide (ASO) SECN-15 that targets and downregulates the expression of neuropilin-1 (NRP1), a transmembrane co-receptor that promotes tumor progression in several tumor types, including breast and gastric cancers.
Evidence suggests the composition of the gut microbiome modulates a tumor’s response to therapy. Maat Pharma SA has thus developed Microbiome Ecosystem Therapies (METs) that replicate, at a large scale, the effects of the gut microbiome in people who respond to immune checkpoint inhibitor (ICI) therapy.
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.
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.
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