Neurogene Inc.’s stock sank 43% on news that its phase I/II gene therapy, NGN-401, resulted in a serious adverse event in a pediatric Rett syndrome patient receiving the highest dose.
Scientists at Gachon University and their collaborators investigated the neuroprotective effects of the GPR40 agonist TUG-469 in models of Parkinson’s disease (PD).
The EMA has changed its mind about an earlier decision that the risks of Leqembi (lecanemab) outweigh the benefits and is now recommending the Alzheimer’s disease drug is approved for a subgroup of patients. That follows an appeal by Eisai Co. Ltd. and a re-examination of the data, after details relating to 274 patients with two copies of the ApoE4 gene were removed from the file.
Despite government efforts to prop up biopharma and med-tech research toward creating women’s health products, companies must eventually reach out to the private markets to bring their inventions to the next stage of development. Anna Zornosa-Heymann, a women’s health investor, serves as a part-time contractor with the U.S. NIH’s SEED (Small business Education & Entrepreneurial Development) office, where she helps companies move from government to external funding. Government funds are “excellent to pay for research … but those funds don’t allow you to build a first-class team and to develop a sales apparatus,” she told BioWorld.
While women make up half the world’s population and own two out of every five businesses, there are substantial knowledge gaps about conditions affecting their health – mostly due to decades of research excluding women from clinical trials and investment decisions.
DEAD-box helicase 17 (DDX17) is an RNA helicase involved in the early phases of neuronal differentiation. Researchers have identified a total of 13 patients presenting with neurodevelopmental phenotypes and who harbored de novo monoallelic variants in the DDX17 gene. The phenotype was characterized by intellectual disability, delayed speech and language, as well as motor delay.
Poseida Therapeutics Inc. has updated progress made in its early-stage pipeline of differentiated T stem cell memory cell-rich allogeneic CAR T therapies in oncology and autoimmune diseases.
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