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4D Molecular Therapeutics Reports Interim Results from the 4D-310 Phase 1/2 Clinical Trial in Patients with Fabry Disease and Provides Clinical Data Update from the 4D-110 Phase 1/2 Clinical Trial in Patients with Choroideremia
- First-ever clinical activity data reported on 4D-310 Fabry disease product candidate utilizing the proprietary C102 targeted and evolved vector invented
About this update from 4d Molecular Therapeutics, Inc.
[{"type":"text","content":"- First-ever clinical activity data reported on 4D-310 Fabry disease product candidate utilizing the proprietary C102 targeted and evolved vector invented through Therapeutic Vector Evolution - AGA enzyme activity was within, or significantly above, the normal range in all three patients (up to 25-fold mean normal AGA activity) - 4D-310 had a manageable safety profile and no dose-limiting toxicities - 4D-110 was generally well-tolerated, with initial evidence of clinical activity observed - 4DMT to host conference call and webcast on Monday, October 25, 2021 at 2:00 p.m PDT EMERYVILLE, Calif., Oct. 25, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced interim clinical data from the Phase 1/2 clinical trial of intravenous 4D-310 in patients with Fabry disease. The company also provided a clinical data update from the on-going Phase 1/2 clinical trial of 4D-110 in patients with choroideremia. “Today we announced the first ever clinical data reported with an intravenous product invented from our Therapeutic Vector Evolution platform at 4DMT. These interim data demonstrate clinical proof-of-concept for tolerability and clinical activity,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. “These data support our belief that 4D-310 is well-tolerated, and has the potential to deliver patient benefit after a single intravenous injection. Importantly, the post-treatment AGA enzyme activity in patients’ blood was within, or significantly above, the normal range despite the presence of pre-existing anti-AGA antibodies, which are a result of prior ERT in these patients. To our knowledge, these initial clinical data are the first demonstration of durable serum AGA activity within the normal and above normal range following gene therapy in a difficult to treat classic Fabry disease patient population.” “We designed 4D-310 for a unique dual mechanism of action, enabling the potential treatment of target tissues such as heart, blood vessel walls and kidney through cross-correction from high and stable serum AGA activity, as well as through direct transduction and AGA expression in target cells,” said Dr. Raphael Schiffmann, M.D., Senior Vice President & Clinical Therapeutic Area Head, Lyso...