Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder marked by progressive degeneration of upper and lower motor neurons, resulting in paralysis and death typically within 3-5 years of symptom onset. Historically, treatment options have been extremely limited. However, the identification of genetic contributors to ALS pathogenesis has enabled the application of antisense oligonucleotides (ASOs) to selectively modify or reduce the expression of disease-associated genes at the RNA level.
Apollo Therapeutics Ltd. has developed APL-4098, a small-molecule general control nonderepressible 2 (GCN2) inhibitor for the potential treatment of AML.
Astrazeneca plc has provided data for their CD22-targeting antibody-drug conjugate (ADC) AZD-4512 under development for the treatment of B-cell malignancies, which still have significant rates of disease resistance and relapse, as well as treatment-related toxicities.
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.
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