Currently, cancer therapy trial-and-error methodology is inefficient and unsustainable. Oncology is the worst therapeutic area for drug trial success; only 3.4% of drugs that enter phase I end up being FDA approved, and 57% fail due to poor drug efficacy in trials. Building tools that may aid in predicting an individual’s response to a specific therapy may help in reducing costs, guesswork, and importantly improve the outcome of patients and accelerate new drug development.
Researchers from Affinia Therapeutics Inc. have described the development and preclinical evaluation of a new AAV-based gene therapy, designed using a novel cardiotropic capsid, for the potential treatment of MYBPC3-associated hypertrophic cardiomyopathy (HCM).
Fibroblast activation protein (FAP) is absent or expressed at a very low level in normal tissues. However, it is overexpressed in around 80% of solid tumor stroma.
At the European Association of Nuclear Medicine meeting, Monopar Therapeutics Inc. presented the preclinical characterization of MNPR-101, a first-in-class urokinase-type plasminogen activator receptor (uPAR)-targeting radiopharmaceutical agent with high stability in vivo and more than 1 week of shelf-life in its labeled form, [177Lu]-MNPR-101.
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).
FMS‐like 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.
The Japanese government, industry and academia are deliberating health care policies and initiatives to boost Japan’s role in the future of regenerative medicine, experts at Bio Japan 2024 said, as the fruits of cell and gene therapy research come to fruition with new approvals.
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.
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