NEW YORK – Hemogenyx Pharmaceuticals said recently that it has received institutional review board approval for its first clinical site, allowing it to begin studying the CAR T-cell therapy candidate HEMO-CAR-T (HG-CT-1) in a Phase I clinical trial in relapsed or refractory acute myeloid leukemia patients. The cell therapy is designed to target AML cells that express the FLT3 protein.
Cambridge, Massachusetts-based Intellia Therapeutics this week said the US Food and Drug Administration has granted it regenerative medicine advanced therapy designation for nexiguran ziclumeran, also known as nex-z or NTLA-2001, an investigational in vivo CRISPR-based therapeutic it is developing as a treatment for hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN). RMAT designation is for treatments that meet the FDA's definition of a cell or gene therapy intended to treat, modify, or cure a serious or life-threatening disease and that may address unmet needs in that disease setting. Intellia leads development and commercialization of nex-z as part of a multitarget collaboration with Regeneron.
Health and research company Care Access this week said it will offer free health screenings in Birmingham, Alabama, and Palo Alto, California, next month to identify heart and kidney health risks for patients. The free health screening includes testing for lipoprotein(a), a cardiovascular risk factor, as well as tests for other metabolism and kidney risks. The effort is part of the organization's Future of Medicine program, in which it provides free testing and health education and connects participants with clinical research opportunities.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.