4D Molecular Therapeutics 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.”
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