Mouse models for hemophilia A are commonly generated by factor VIII (FVIII) knockout, however, these mice rapidly develop anti-FVIII antibodies during repetitive FVIII administration. To overcome this preclinical research challenge, investigators from Octapharma AG and affiliated organizations developed a new model unable to produce antibodies but otherwise not immunosuppressed.
Cognition Therapeutics Inc. evolved from the work of a neuroscientist and a chemist working in the San Francisco Bay area, seeking out targets to block the effects of Alzheimer’s disease. Since the company’s 2007 inception, it has received close to $200 million in U.S. NIH grant funding. Investors often tell CEO Lisa Ricciardi, who joined the company in 2020: “’That’s because you have a relationship with the FDA.’ Well, no. It’s because it’s competitive” and the company’s research has met the muster. “You have to apply two or three times. … It’s with rigor that these results are generated and that we’re able to get more funding.”
Raising money to advance promising science is a constant struggle, bringing biopharma executive leadership together to learn about investment strategies in the opening session at the Biotechnology Innovation Organization’s CEO and Investor Conference in New York. “We’re bottoms-up investors. If we like the technology, we like the product, we think it’s going to work, we want to find a way to invest,” said Chris Garabedian, chairman and CEO of Xontogeny.
Researchers at E-Therapeutics plc recently presented efficacy and safety data on ETX-148, a pan-hemophilia agent in murine models of hemophilia A and B.
Fabry disease is an X-linked lysosomal storage disorder caused by a deficiency of the enzyme α-galactosidase A (GLA), which leads to complex multisystemic involvement, with the majority of patients developing hypertrophic cardiomyopathy.
Early dysfunction of the endothelial/blood-brain barrier (BBB) is involved in the pathogenesis of cerebral small vessel disease (SVD), which is a contributor to about 50% of dementias. Sphingosine-1-phosphate (S1P) is a sphingolipid that regulates the BBB integrity when bound to its receptor S1P1 on endothelial cells.
Tumor immune evasion mechanisms could be reversed by activating intracellular antiviral immune responses. It has been reported that the use of DNA methyltransferase (DNMT) inhibitors combined with poly(ADP ribose) polymerase (PARP) inhibitors activated stimulator of interferon genes (STING) signaling pathway in a process named pathogen mimicry response.
Hepatocellular carcinoma (HCC) is an aggressive disease that accounts for 80%-90% of all primary liver cancers. Previous findings have shown fibroblast growth factor 19 (FGF-19) to be overexpressed in up to 30% of HCC cases, exerting its oncogenic effect through its receptors fibroblast growth factor receptor 3 (FGFR3) and FGFR4.
Researchers from Nanjing Drum Tower Hospital reported findings from their evaluation of the role of NLR family CARD domain containing 5 (NLRC5) in post-stroke neuroinflammation. Immunofluorescence staining of mouse brains revealed that NLRC5 was mainly expressed in microglia.
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