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2. ProSavin®

ProSavin is Oxford BioMedica’s novel gene-based therapeutic for the treatment of Parkinson's disease. The product is administered directly to the striatum in the brain. It delivers three genes required to convert cells that normally do not produce dopamine into cells that do, thereby replacing the dopamine synthesising cells lost during the course of the disease. ProSavin utilises Oxford BioMedica’s proprietary LentiVector® system to deliver the genes AADC (aromatic amino acid decarboxylase), TH (tyrosine hydroxylase) and CH1 (GTP-cyclohydrolase 1). These genes reprogramme transduced cells to manufacture and secrete dopamine.

3. Phase I/II trial of ProSavin

The Phase I/II trial is being conducted at the Henri Mondor Hospital in Créteil, which is a European centre of excellence for neurosurgery and a member of the Assistance Publique Hôpitaux de Paris (APHP) in France. The study is designed to assess the safety and efficacy of ProSavin in patients with Parkinson’s disease who are failing on current treatment with L-DOPA but have not progressed to experiencing marked drug-induced movement disorders called dyskinesias. The first stage is an open-label dose escalation to evaluate at least two dose levels of ProSavin in cohorts of three patients each. In the second stage of the trial, a further 12 patients will be recruited, some of which will act as a control group and only receive “sham” surgery. Efficacy is assessed at regular intervals using the Unified Parkinson’s Disease Rating Score (UPDRS). The primary endpoints of the study are: 1) the number and severity of any adverse events associated with the administration of ProSavin, including the incidence of dyskinesias; and 2) efficacy based on the UPDRS assessment at six months after treatment. The secondary objectives of the trial include the extent to which patients’ current L-DOPA therapy can be reduced or removed following administration of ProSavin.

4. LentiVector®

LentiVector is a highly effective gene delivery system. It is based on an engineered lentiviral vector, which is harmless to humans. The LentiVector system has been shown to express its genetic payload efficiently and stably in multiple tissue types, and is particularly effective in targeting non-dividing cells, such as neurons in the brain. In addition, genes delivered with a LentiVector system have shown long-term stable expression and, hence, offer long-term therapeutic benefit.