OTL-204 / Kyowa Kirin - LARVOL DELTA

Orchard Therapeutics Announces New Clinical and Pre-clinical Data at ASGCT 2023 (GlobeNewswire) - "Orchard Therapeutics...today announced six presentations from across its hematopoietic stem cell (HSC) gene therapy platform will be featured at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) taking place May 16-20, 2023, in Los Angeles....'Taken together, the data from our MPS programs continues to show the potential of a one-time HSC gene therapy to address some of the most severe manifestations of this complex group of multi-system diseases. In addition, we continue to apply the differentiated delivery mechanism of our platform to advance a strategic research pipeline in difficult-to-treat diseases such as NOD2-Crohn’s disease, where we will share updated pre-clinical PoC results. With our research collaborators, we also look forward to providing a first-look at pre-clinical results from OTL-204, which is being developed for the potential treatment of FTD caused by heterozygous mutations in the progranulin gene.'"

Preclinical • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration