First discussion of the novel production approach used for ProSavin, a LentiVector-based product, to be made at Bio 2003 this week in Washington DC.
Oxford BioMedica has said that details of its novel manufacturing process for ProSavin, for the treatment of Parkinson's disease, will be presented at the Bio 2003 Annual Convention in Washington DC on 25 June.
The new manufacturing process has the capacity to satisfy all of the company's requirements up to Phase II clinical trials and the team is currently working on a refinement of the process to scale-up for Phase III trials and commercial production.
This will be the first time that Oxford BioMedica has disclosed details of its novel production approach for LentiVector-based products.
LentiVector is able to deliver genes to non-dividing cells, such as brain cells, and ProSavin delivers the genes for dopamine synthesis directly to the striatum of the brain, thereby creating endogenous production of dopamine, the neurotransmitter that is lost in Parkinson1s patients.
The target market is late stage disease where ProSavin could offer significant advantages over existing therapy.
Carlos Ibanez, director of process and product development at Oxford BioMedica's US subsidiary BioMedica Inc, will give a presentation during a panel discussion on innovative manufacturing strategies for viral and non-viral gene based therapies.
The establishment of effective manufacturing processes represents a key milestone on the path to the IND submission that is planned for ProSavin later in 2003 following the completion of preclinical efficacy and toxicity studies.
Phase I trials are planned for 2004.
Commenting on the development, Doug Jolly, CEO of BioMedica Inc, said: "We are delighted to have brought the LentiVector production system successfully from laboratory bench to manufacturing for clinical trials.
The process has been developed following constructive dialogue with the FDA and UK regulatory authorities and through close collaboration with the technical team in Oxford.
Gene therapy has been limited in the past by production problems.
This will not be the case for Oxford BioMedica1s products." During the past 18 months the technical team at BioMedica Inc has been developing a cost effective, GMP-compatible manufacturing process that can be used for ProSavin and all of the company1s other LentiVector-based products.
The vector production process is at the 40 litre scale by transient transfection.
The clinical trial material is processed by chromatographic methods and packaged in single use glass vials.
This yields 2-5ml of high titre product (>109TU/ml), which is sufficient for 15-40 human dose equivalents.
It can be transported and stored at -70C with an expected shelf life in excess of 12 months.
The obvious regulatory issues for a novel gene therapy such as appropriate safety and product release assays, including those for replication-competent virus, have been discussed with the FDA. Based on these discussions, relevant assays have been developed.
