Oxford BioMedica Announces Six-Month Efficacy Results With Low Dose of ProSavin in Phase I/II Trial in Parkinson’s Disease
Oxford BioMedica announced promising additional results from the low-dose cohort of patients in the Phase I/II trial of its novel gene therapy, ProSavin, for the treatment of Parkinson’s disease. The three patients in this cohort showed improved motor function, as measured by the Unified Parkinson’s Disease Rating Score (UPDRS) in the ‘off’ state, of an average of 30% at six months.
The robust safety profile of ProSavin has been maintained at six months with no evidence of adverse events or immunologic reactions to the treatment. In addition, all patients showed improvement in Quality of Life, as measured by the industry-standard PDQ-39 questionnaire, at six months.
The three patients that received the low dose of ProSavin demonstrated improvements in their UPDRS motor ‘off’ scores in the range of 10-50% after six months. This score measures the degree of mobility in the absence of standard of care dopaminergic therapies. One patient showed an improvement of up to 50% at six months, which was an increase from the three-month assessment, and another patient maintained an improvement of 30%. One patient showed an improvement of 10% at the six-month assessment compared to 23% at three months, although this patient’s score may have been affected by adjustments to his L-DOPA ‘equivalent’ therapy.
The clinical evaluation of the high dose of ProSavin is progressing. There have been no safety issues to date and the preliminary data from the first patient treated at the high dose are promising.
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Topic world Gene therapy
Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.
Topic world Gene therapy
Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.
