11/8/2023 0 Comments Max yang 4d molecular therapeutics![]() “The unmet need for patients with Fabry disease is significant. 4D-310 is designed for both stable therapeutic AGA enzyme activity in the blood, as well as for intracellular production within affected tissues, including in cardiac muscle cells.” “4D-310 is designed with the goal of achieving a novel dual mechanism-of-action for these patients. Dosing the first patient in the Phase 1/2 clinical trial of 4D-310 marks the third product candidate to be administered to patients, all of which utilize proprietary vectors derived from our Therapeutic Vector Evolution platform,” said David Kirn, MD, chief executive officer, co-founder and president of 4DMT. “Through our Therapeutic Vector Evolution platform we apply the principles of directed evolution to invent targeted and evolved AAV vectors for the delivery of genes to specific tissue types. Fabry disease is an inherited lysosomal storage disease with high unmet medical need that results from loss of function mutations in the alpha-galactosidase (AGA) enzyme. EMERYVILLE, Calif., Ma(GLOBE NEWSWIRE) - 4D Molecular Therapeutics (Nasdaq: 4DMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced that the first patient has been dosed in the Phase 1/2 clinical trial of 4D-310 for the treatment of Fabry disease.
0 Comments
Leave a Reply. |
AuthorWrite something about yourself. No need to be fancy, just an overview. ArchivesCategories |