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.
#4d molecular therapeutics trial
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). Their novel Therapeutic Vector Evolution AAV platform holds huge promise for patients with inherited retinal dystrophies and related conditions.EMERYVILLE, CA, USA I JanuI 4D Molecular Therapeutics, Inc. 4DMT retains all patent and commercial rights to its proprietary AAV technology.ĭavid Wilson, MD, Director of OHSU’s Casey Eye Institute, commented, “We are excited to work with Peter Francis and his ophthalmology team at 4D. Casey Eye Institute and ONPRC will provide access to its researchers and resources for the inception, design and conduct of joint research programs. Under the terms of the agreement, 4DMT will provide research access to its proprietary vector and gene therapy product technology, development expertise and manufacturing capabilities. This collaboration will accelerate translation of new treatments for currently incurable retinal diseases,” Peter Francis, SVP Clinical, Translational R&D, Program Leader, Ophthalmology Therapeutic Area of 4D Molecular Therapeutics, said in a company news release. “I’m delighted with the potential of this exciting partnership between 4DMT and OHSU’s Casey Eye Institute and the Oregon National Primate Research Center. The collaboration will combine the complementary expertise of 4D Molecular Therapeutics with the Casey Eye Institute and the Oregon National Primate Research Center to identify and develop gene therapies for degenerative retinal diseases. 4D Molecular Therapeutics (4DMT) announced a research and development collaboration with the Casey Eye Institute and the Oregon National Primate Research Center at Oregon Health & Science University (OHSU).
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