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4D Molecular Therapeutics Announces FDA Fast Track Designation Granted to 4D-125 for the Treatment of X-linked Retinitis Pigmentosa
EMERYVILLE, Calif., Jan. 10, 2022 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (NASDAQ: FDMT), a clinical-stage gene therapy company harnessing the power of
About this update from 4d Molecular Therapeutics, Inc.
[{"type":"text","content":"EMERYVILLE, Calif., Jan. 10, 2022 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (NASDAQ: FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for 4D-125 for treatment of patients with inherited retinal dystrophies due to defects in the RPGR gene, including X-linked Retinitis Pigmentosa (XLRP). 4D-125 is a targeted and evolved R100-based product candidate, which was invented at 4DMT for efficient intravitreal delivery, and is designed to deliver a functional copy of the RPGR gene to photoreceptors in the retina. “Patients living with XLRP currently have no approved treatments, and they suffer from progressive vision loss and blindness that reduces their quality of life and independence,” said Robert Kim, M.D., Senior Vice President and Ophthalmology Therapeutic Area Head of 4DMT. “Fast Track Designation is a landmark event for the program and underscores the potential of 4D-125 to address a significant unmet need for those living with XLRP.” About Fast Track Designation The FDA’s Fast Track process is designed to accelerate the development and review of treatments for serious and life-threatening diseases where no treatment exists or where the treatment in discovery may provide advantages over what is currently available. A drug candidate that receives Fast Track designation is eligible for more frequent communication with the FDA throughout the drug development process and a rolling and/or priority review of its marketing application if relevant criteria are met. About 4D-125 and XLRP 4D-125 is 4DMT’s targeted and evolved R100-based product candidate for XLRP and is designed to deliver a functional copy of the RPGR gene to photoreceptors in the retina. 4DMT is currently enrolling patients in an on-going Phase 1/2 clinical trial. The study employed a standard 3+3 dose-escalation design, followed by dose expansion. In dose-escalation, patients were enrolled in one of two dose cohorts: 3E11 vg/eye and 1E12 vg/eye. The dose expansion phase of the study is enrolling patients at the 1E12 vg/eye dose. The primary objectives of this trial are to evaluate the safety and maximum tolerated dose of 4D-125. Secondary endpoints include assessments of clinical activity, including both vis...