Early-stage gene therapy shows potential against Parkinson’s

An early-stage gene therapy has shown potential for the treatment of Parkinson’s disease in a study published in The Lancet.

The gene-based biopharma, Oxford BioMedica, is behind the research to develop ProSavin. Parkinson’s disease is typically treated with oral dopamine replacement therapies, whereas the investigational gene therapy aims to restore dopamine production.

 
“ProSavin has demonstrated a favourable safety profile and a statistically significant improvement in motor function relative to baseline at six and 12 months post-treatment.”
Statement from Oxford BioMedica
“[Our study suggests that this] therapeutic approach that provides continuous and stable dopamine replacement, restricted to the dopamine-depleted striatum, might provide an effective long-term treatment without the onset of behavioural complications.”
Prof Stéphane Palfi and others, authors of the paper in The Lancet.

 

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Related news:

Gene therapy might offer hope in Parkinson’s (OnMedica).

Reference links:

Oxford BioMedica announces publication of ProSavin phase I/II study in The Lancet (BioMedica press release).

Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson’s disease: a dose escalation, open-label, phase 1/2 trial (The Lancet)

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