Long-term potential revealed for novel modifier gene therapy
Posted: 14 January 2025 | Catherine Eckford (European Pharmaceutical Review) | No comments yet
The positive two-year findings from the Phase I/II gene therapy trial in retinitis pigmentosa reiterate the potential of mutation-agnostic therapies.
New long-term data reveals that a modifier gene therapy delivered meaningful visual function improvements for retinitis pigmentosa participants in a Phase I/II clinical trial.
A two-year safety and efficacy update from the OCU400 clinical trial showed that 100 percent of treated evaluable participants improved or preserved their visual function compared to untreated eyes at both one and two years.
These statistically significant results were reported regardless of mutation at two years. According to Ocugen, this validates the gene-agnostic mechanism of action for OCU400.
Dr Arun Upadhyay, Chief Scientific Officer and Head of Research & Development at Ocugen explained in an EPR’s interview in 2024: “unlike conventional methods that typically focus on replacing a mutated gene with a functional copy, modifier gene therapy modifies gene expression using master gene regulators. These master regulators work in a “gene-agnostic” way and open the possibility for the treatment of diseases caused by different gene mutations”.
Impact of the long-term results for the modifier gene therapy
“These two year low light visual acuity (LLVA) findings, which are the most sensitive measure of visual function, are consistent with the results observed at one year [for the modifier gene therapy OCU400]”
“It is truly remarkable to see the significant improvements in visual acuity in patients treated with OCU400 sustained at two years,” stated Dr Syed Shah, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin. “OCU400’s demonstrated effectiveness across multiple mutations not only offers hope to [retinitis pigmentosa] patients but also opens new possibilities for treating other retinal diseases.”
“Establishing the long-term safety and efficacy of OCU400 demonstrates the durability of this novel gene therapy,” shared Dr Huma Qamar, MPH, Chief Medical Officer at Ocugen. “These two year low light visual acuity (LLVA) findings, which are the most sensitive measure of visual function, are consistent with the results observed at one year.”
The Phase III liMeliGhT clinical trial for Ocugen’s modifier gene therapy product OCU400 is ongoing.
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Biologics, Biopharmaceuticals, Clinical Development, Clinical Trials, Data Analysis, Dosage, Drug Development, Drug Safety, Gene therapy, Industry Insight, Research & Development (R&D), Technology, Therapeutics
