Pulmonary arterial hypertension (PAH) is a life-threatening disease; vasodilators may aid in managing this condition, but their impact on prognosis is still limited, potentially due to a lack of biomarkers to guide therapy. Japanese researchers have presented results of their efforts to discover potential molecular markers that may predict response to pulmonary vasodilators.
Researchers from Mount Sinai Center for Translational Medicine and Pharmacology at Icahn School of Medicine at Mount Sinai and colleagues have developed a therapeutic humanized antibody that blocks the action of follicle-stimulating hormone (FSH), a pituitary hormone previously thought to only play a role in fertility.
Adeno-associated virus (AAV)-based gene therapy is considered a promising strategy to treat hearing loss. However, its clinical application is limited by the genetic heterogeneity of hereditary hearing loss, requiring gene-specific analysis and approach optimization for broader treatment applications.
Rallybio Corp. has received an equity milestone payment of $12.5 million from Recursion Pharmaceuticals Inc. triggered by the initiation of additional preclinical studies for REV-102, an investigational oral ENPP1 inhibitor.
The mechanisms behind diabetic cardiomyopathy (DCM) have been deeply studied but still not well-established within the scientific community. Mutations in cardiac junction proteins may result in heart failure and arrhythmia. ER degradation enhancing α-mannosidase like protein 2 (EDEM2) is involved in the degradation of misfolded N-glycosylated proteins, but its role in the heart is not clear and was investigated.
Researchers from the CUNY Advanced Science Research Center and their collaborators recently published a paper in Science Advances on Aug. 27, 2025, about synthetic carbohydrate receptors (SCRs) and their potential as broad-spectrum antivirals by targeting the viral envelope N-glycans. They described the antiviral activity of a series of tetrapodal SCRs both in vitro and in vivo, showing their potential as broad-spectrum inhibitors of viral infection.
Enlaza Therapeutics Inc. will take charge of research through nominating candidates in its potentially $2 billion-plus deal with Vertex Pharmaceuticals Inc. For its efforts, Enlaza is getting $45 million in an up-front payment and equity investment, plus the opportunity to bring in more than $2 billion in research, development, regulatory and commercial milestones and tiered royalties on net sales.
Aglaeapharma Inc. has described compounds acting as α2-adrenoceptor agonists reported to be useful for the treatment of rhinitis, pain, insomnia, inflammatory disorders, glaucoma, cancer, anesthesia and acute kidney injury, among others.
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