Researchers from the Sino-American Cancer Foundation and Taipei Medical University have developed a novel nanobody-based CAR T-cell platform directed against C-type lectin-like molecule-1 (CLL-1) for the possible treatment of acute myeloid leukemia (AML).

Exon skipping therapies based on antisense phosphorodiamidate morpholino oligomer (PMO) have great potential to restore dystrophin in the skeletal muscle and treat Duchenne muscular dystrophy (DMD). Entrada Therapeutics Inc. has developed an endosomal escape vehicle conjugated to DMD exon skipping PMOs (exon 51 skipping), ENTR-601-51, for the potential treatment of DMD.

Liminatus Pharma Inc. has announced plans to conduct a phase I trial of IBA-101, a next-generation CD47-blockade antibody designed to work alongside PD-1/PD-L1 checkpoint inhibitors across a range of solid tumors.

Glioblastoma multiforme (GBM) is an aggressive and highly invasive intracranial tumor arising from the malignant transformation of brain and spinal cord cells. To date, surgery followed by adjuvant chemotherapy is the standard therapy for treating GBM, where temozolomide is the only first-line FDA-approved drug for GBM treatment. The aim of this study from Shenzhen University was to test the effect of a novel chloroethyl nitrosourea analog, HJ-03, in the treatment of GBM, which might overcome temozolomide resistance.

VST Bio Corp. has closed its series A financing designed to support the company’s work in vascular diseases with the development of first-in-class antibodies targeting edema and inflammation for patients with ischemic stroke.

Neurodegenerative disease and cognitive decline cannot be explained by a single process. Beta-amyloid plaques, hyperphosphorylated tau, alpha-synuclein, activated microglia and astrocytes, altered receptors such as TREM2, mitochondrial dysfunction, epigenetic changes and cerebrovascular alterations all seem to contribute to the development of dementia in Alzheimer’s disease (AD). While scientists attempt to address each of these elements, prevention is growing as a primary goal.

Enodia Therapeutics Inc. was spun out of The Institut Pasteur by Argobio Studio based on the work of Caroline Demangel, co-founder of Enodia and head of the immunobiology and therapy unit at The Institut Pasteur. Demangel’s lab discovered the mechanism of action of mycolactone, a natural Sec61 inhibitor that causes Buruli ulcers.