With the EMA approving the first gene therapy to treat a rare disease, it is a signal to the healthcare industry as a whole that truly innovative and pioneering therapies are inevitably going to reach the market and transform the health of patients in need. The gene and cell therapy industry's route to market may resemble an un-kept wilderness with processes and guidelines yet to be well trodden by industry sponsors, but lessons learned by the pioneers trying to commercialise their first-in-kind products are massive success stories and beneficial for the industry as a whole.
A traditional stumbling block for therapy developers is satisfying the rigour of the regulatory agencies. Paula Salmikangas, Chair of the Cell-Based Products Working Party at the European Medicines Agency spoke at the World Stem Cells & Regenerative Medicine Congress 2012 to provide her regulatory experience and perspectives in this area. Paula defines a gene therapy medicinal product as a biological medicinal product which has the following characteristics:
(a) it contains an active substance which contains or consists of a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence;
(b) its therapeutic, prophylactic or diagnostic effect relates directly to the recombinant nucleic acid sequence it contains, or to the product of genetic expression of this sequence. Gene therapy medicinal products shall not include vaccines
against infectious diseases.
Just released is Paula's presentation entitled "Specific regulatory challenges of genetically modified cells" so make sure you download it and take a look.
Production for next year's World Stem Cells & Regenerative Medicine Congress 2013 is already underway so if you want to be involved, message me below or find out more here.