The proliferation of abnormal blood vessels in the retina is stimulated by VEGF family members. Wet AMD is a type of macular degeneration in which abnormal blood vessels grow into the macula and cause visual distortion, reduced acuity and, in some cases, blindness. In the non-human primate retinal laser-induced CNV model data presented in May 2021 at the annual conference of the American Society of Gene and Cell Therapy, at doses ranging from 1E11 to 1E12 vg/eye, 4D-150 intravitreal injection resulted in 100% suppression of high grade angiogenic lesions (grade 4). 4D-150 builds on the excellent tolerability to date of the proprietary R100 capsid in the 4D-125 XLRP clinical program at doses up to 1E12 vg/eye. Intravitreal delivery of biologics to the eye is routine, and a single dose intravitreal gene therapy that could provide long-term efficacy in patients would be an advantage for patients who struggle with treatment burden and/or treatment resistance. Efficient intravitreal delivery to the retina of a payload expressing two transgenes that inhibit four distinct angiogenic factors has the potential for greater efficacy and/or lower required doses versus therapies that target a single VEGF factor, including in patients refractory to currently approved anti-VEGF therapies. Secondary endpoints include the number of supplemental aflibercept injections received and change from baseline in best corrected visual acuity (BCVA) over time.ĤD-150 is a dual-transgene, intravitreal gene therapy designed to inhibit four distinct angiogenic factors to prevent angiogenesis and reduce vascular permeability for the treatment of wet AMD. The primary endpoints of the study are safety and tolerability. In dose expansion, patients (n=50) will be randomized 2:2:1 to receive one of 2 dose levels of 4D-150 (n=20 for each dose level) or aflibercept (n=10). In the dose-escalation phase, multiple dose levels of 4D-150 will be examined in an open-label, 3+3 design with an initial dose of 3E10 vg/eye. The Phase 1/2 clinical trial is a dose-escalation and randomized, controlled, masked expansion trial of intravitreal 4D-150 and is expected to enroll approximately 60 adults with wet AMD. “Utilizing the R100 capsid and a unique, dual-mechanism of action, 4D-150 has the potential to provide long-term benefits for patients with wet AMD after a single-dose of intravitreal anti-angiogenic gene therapy.” “While there have been significant advances in the treatment of wet AMD, the treatment burden remains significant, and as a result, many patients experience suboptimal vision outcomes,” said Arshad Khanani, M.D., M.A., Managing Partner, Director of Clinical Research at Sierra Eye Associates and a principal investigator on the 4D-150 Phase 1/2 clinical trial. “We believe that 4D-150’s design, which targets four distinct angiogenic factors with dual transgenes, has the potential for broad, robust and durable efficacy after a single low dose intravitreal administration in patients with wet AMD,” said Robert Kim, M.D., Senior Vice President and Ophthalmology Therapeutic Area Head of 4DMT. 4D-150 is a dual-transgene intravitreal gene therapy incorporating the R100 capsid which we invented through our proprietary Therapeutic Vector Evolution platform,” said David Kirn, M.D., Co-Founder and Chief Executive Officer of 4DMT. “The dosing of the first patient in the 4D-150 Phase 1/2 clinical trial in wet AMD marks an important milestone for our company and for the patients we aim to serve. (4DMT) (Nasdaq: FDMT), 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-150 for neovascular age-related macular degeneration (wet AMD). 06, 2022 (GLOBE NEWSWIRE) - 4D Molecular Therapeutics, Inc.
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