The regulation of type I interferon (IFN-1) signaling is crucial for fine-tuning the innate immune response to combat pathogens, fight cancer and prevent autoimmune diseases. Disrupted IFN-1 signaling plays a key role in autoimmune and inflammatory diseases. Therefore, understanding how IFN-1 production and signaling are regulated could reveal new approaches for treating conditions driven by this pathway.
Neucore Bio Inc. has received a $350,000 STTR phase I grant from the National Center for Advancing Translational Sciences (NCATS) at the U.S. National Institutes of Health (NIH) to evaluate the company’s targeted exosome platform to deliver an RNA-based therapy to treat Charcot-Marie-Tooth disease type 1a (CMT1A).
Researchers have identified a potential therapeutic target for muscular dystrophies, a group of genetic disorders characterized by progressive muscle weakness and degeneration. The study reveals that inhibiting the microRNA (miRNA) known as miRNA-33 can significantly improve muscle regeneration and ameliorate the dystrophic phenotype in animal models.
Researchers at the University of California, Los Angeles and collaborators have analyzed what cell types are infected by hantaviruses and how infection alters gene expression.
Avenzo Therapeutics Inc. has obtained IND clearance by the FDA for AVZO-103 (formerly VAC-103), a nectin-4/TROP2 bispecific antibody-drug conjugate (ADC).
In an effort to exploit the advantages of therapeutic bacteria and viruses while avoiding their disadvantages, researchers at Columbia and Rockefeller Universities have developed an anticancer platform called CAPPSID (Coordinated Activity of Prokaryote and Picornavirus for Safe Intracellular Delivery).
Vyriad Inc. and Yeda Research and Development Co. Ltd., the commercial arm of the Weizmann Institute of Science, have signed an exclusive license agreement for a novel viral vector targeting approach.
Arrowhead Pharmaceuticals Inc. has announced a global licensing and collaboration agreement with Novartis AG for ARO-SNCA, Arrowhead’s preclinical stage siRNA therapy for the treatment of synucleinopathies such as Parkinson’s disease, and for additional targets.
Jynneos, a current vaccine against orthopoxviruses such as smallpox and mpox virus, elicits only modest antibody responses, highlighting the need to search for more immunogenic vaccines or treatments. The global outbreak of mpox virus infections in 2022 lent urgency to this challenge.
Shanghai Huilun Pharmaceutical Co. Ltd. has described sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain, multiple sclerosis, Charcot-Marie-tooth disease and arrhythmia.
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