4D Molecular Therapeutics Inc. (NASDAQ: (FDMT) stock surged by 1.7% at last close while the FDMT stock price gains by 5.74% in the after-hours trading session. 4DMT is a clinical-stage business developing targeted gene therapeutics using the ability of directed evolution. 4DMT’s Therapeutic Vector Evolution technology aims to harness the full potential of gene therapy.
FDMT stock’ Update
Preliminary clinical results from the Phase 1/2 clinical study of intravenous 4D-310 in people with Fabry disease using the unique C102 targeted and evolved vector created by Therapeutic Vector Evolution were released by 4D Molecular Therapeutics. The business also presented an insight on clinical data from the current Phase 1/2 clinical study of 4D-110 in choroideremia patients.
In all three cases, AGA enzyme activity was within or slightly above the normal range (up to 25-fold mean normal AGA activity). 4D-310 demonstrated a well-tolerated safety profile with no dose-limiting side effects. The drug 4D-110 was generally well tolerated, with early signs of therapeutic efficacy.
Fabry disease is a serious lysosomal storage disorder with significant unfulfilled needs, according to Dr. Jerry Vockley, Chief of Genetic and Genomic Medicine at the University Of Pittsburgh School Of Medicine and a key researcher on the 4D-310 Phase 1/2 research.
Dr. Raphael Schiffmann, Senior Vice President & Clinical Therapeutic Area Head, Lysosomal Storage Diseases and Cardiology, commented,
4D-310 was created with a unique dual mode of action in mind, allowing for the new cure of target tissues such as the heart, blood vessel walls, and kidneys via cross-correction from strong and sustainable serum AGA activity, as well as clear transduction and AGA expression in target cells. This dual MOA allows for the successful treatment of the widest possible range of Fabry patients. They plan to keep enlisting a broad patient group, including both anti-AGA antibody positive and negative individuals, as well as classic and late-onset individuals, based on the promising first clinical efficacy and safety profile of 4D-310.
