Be Biopharma Inc. has developed a CRISPR/Cas9-based precision B-cell gene therapy to deliver active tissue non-specific alkaline phosphatase (ALP) for the potential treatment of hypophosphatasia (HPP).
FMSâlike tyrosine kinase 3 (FLT3) is a type III receptor tyrosine kinase validated as a therapeutic target for acute myeloid leukemia (AML) and regarded as an indicator of poor prognosis. Unfortunately, current FLT3 inhibitors, such as midostaurin, quizartinib or gilteritinib, often lead to myelosuppression or cardiovascular toxicity.
Scientists from different laboratories around the world have presented the latest advances in research into malignant brain tumors at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), which is being held Oct. 22 to 25 in Rome.
Mutations in the BBS10 gene are the second most common cause of Bardet-Biedl syndrome (BBS). Researchers from Meiragtx Ltd. aimed to optimize and identify an AAV vector carrying the human (h)BBS10 gene, obtaining sustained efficacy as well as good safety for clinical translation for the treatment of BBS.
Studies evaluating the in vitro and in vivo antifungal activity, hemolytic activity and cytotoxicity of COT-832, a novel polyene macrolide antifungal that is a chemical modification of amphotericin B (AmB), were presented at IDWeek by Shionogi & Co. Ltd.
At the ongoing European Society of Gene & Cell Therapy meeting in Rome, Aviadobio Ltd. presented preclinical data for a novel ATXN2-targeting miRNA-containing vector, AVB-205, developed based on previous research that has shown the potential of ATXN2 silencing as a promising therapeutic strategy for amyotrophic lateral sclerosis (ALS) and tau-negative frontotemporal dementia (FTD).
The influenza virus undergoes rapid and frequent antigen shifts that usually force seasonal vaccine updating. The mismatch between vaccine strains and circulating viruses leads to limited vaccine effectiveness, mainly in immunocompromised and older individuals.
Researchers from Phoenixlab, The Scripps Research Institute and affiliated organizations presented the discovery of a novel bivalent protease-activated receptor PAR1- and PAR3-derived peptide, named biparetide, being developed for the treatment of inflammatory bowel disease (IBD).
Patients receiving a kidney transplant and who are positive for BK virus (BKV) are at risk of losing their transplant due to BKV reactivation. At this time, there are no antiviral options available for the prevention of BKV-associated nephropathy. Researchers from Aicuris Anti-infective Cures GmbH presented preclinical data on AIC-468, an antisense RNA therapy that inhibits the splicing process of a viral mRNA encoding a protein critical for BKV replication.
Clostridioides difficile, a spore-forming and anaerobic gram-positive bacterium, causes a wide-spectrum diarrheal disease that can ultimately lead to life-threatening conditions such as toxic megacolon or colonic perforation.
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