By Renata Gomes, Biocant Center for Innovation in Biotechnology (Research group of Lino Ferreira, MIT-Portugal bioengineering faculty) Gene and stem cell therapies have been a growing topic of discussion and research. Legislation has changed, regulatory bodies have been formed and re-structured, intellectual property has become the exchange currency between academia and industry, and regenerative medicine has developed into a universe of opportunities and challenges. All of these developments were explored at the Third Annual Workshop on the Business-Government Interface: Challenges in Cell and Gene Therapies, which was held at Biocant Park in Cantanhede, Portugal, on June 2, 2009. The event was organized by the MIT Portugal Program with the support of Biocant and the Centre for Neurosciences and Cell Biology (CNC) in Coimbra. It focused on cellular and gene therapies; intellectual property, regulatory issues and funding; the funding and commercialization of cellular and gene therapies; and stem cell banking and the clinical impact of cellular therapies.  Image A question from the audience for the Biocant workshop panelists Promise in the Marketplace In his presentation on the status of cellular and gene therapies, Dr. Miguel Santos, Director of Project and IP Management at ECBio, said that the use of stem cells for therapeutic ends offers a great deal of promise in the marketplace. And while most of the companies doing this work are privately held and are located mostly in the United States and the UK, he said, such companies are emerging elsewhere as well, including in Portugal, where there is a growing market. According to Dr. Santos, clinicians tend to draw on cell-based therapies only as a last line of treatment, which has meant that there has been limited commercial use of stem cell-based therapeutic products and difficulty in creating economically stable companies in this area. Nevertheless, he said, it is projected that this could change; in fact, revenues for such therapies could reach US$82 billion by 2012. Dr. Santos recommended that those who are thinking of starting stem cell-related ventures “find a market niche and focus on quality.” In his talk, Professor Sérgio Simões of CNC (and an MIT Portugal faculty member) said that gene therapy offers tremendous opportunities for the development of new technologies for the creation of “smart artificial viruses.” This will involve the interaction of various fields, he said, and could lead to growth in employment as well as revenue. A number of gene therapy products will reach the market in a near future, he added, including Cerepro® by Ark Therapeutics. Professor Simões also noted that the market value of gene therapy is estimated to be US$5.34 billion in 2011. “This is a great achievement, considering that these therapies are mainly been developed for the treatment of rare diseases that have a small target population,” he said, noting that applications are now emerging that will help address cardiovascular diseases, which have a huge target population. Professor Simões recommended that most gene therapy resources be invested in the development of adhesion genes, saying that they are safer and better controlled than others used in gene therapy. Intellectual Property, Regulation and Funding Given the promising future for gene and cell-based therapies, several workshop speakers emphasized the need for those who participate to be fluent in related intellectual property, regulation and funding issues. In her presentation, Margarida Menezes Ferreira from Infarmed Portugal provided an overview of the regulatory requirements that apply to medicines using advanced gene and somatic therapies. It is imperative, she said, for companies to show that each therapy under development will provide added benefit – both for commercial viability and clinical acceptance. She also said that gene-based therapies must be produced in a highly reliable manner, with no deviations from the standard procedures that are the focus of most regulatory guidelines that affect product quality and safety. She stressed that all of those pondering product development must not forget that a “heterelogous use [of somatic cells, genetic material, etc.] equals medicinal use; thus it must comply with all regulations.” Duncan Mackay from Genzyme Europe addressed the future of regenerative medicine from industry’s point of view. He said that the public’s trust regarding the use of gene and cell-based therapies must be gained and reinforced if there is to be a stable and sustainable job market in this area. “Regulation brings certainty,” he said, “and certainty brings investment.” He called on the European Union to create and implement standardized policies so as to guarantee the quality and uniformity of therapies – as well as to cut costs for companies that currently must comply with a variety of different regulations. Public Policy and Business Models Brock Reeve, Executive Director of the Harvard Stem Cell Institute, addressed stem cell regulation in the United States. The Obama administration has overturned the previous restrictions on stem cell research imposed by former president George W. Bush, he said, and federal funds – not only private funds – are now allowed, although under new guidelines. The guidelines, he explained, were adapted from those in effect in countries such as the UK and Canada, and only refer to stem cell research, not clinical trials using stem cells, given that those fall under the direction of the U.S. Food and Drug Administration. Mr. Reeve sees the overturning of the Bush administration restrictions as a victory, because “private funding cannot overcome or equal the NIH [National Institutes of Health] and federal funding.” Nonetheless, he added, “we are not yet out of the woods,” as there are still hurdles to overcome. In his presentation, MIT Professor Charles Cooney emphasized that the current worldwide economic downturn is hindering pharmaceutical industry growth, which is predicted to be only 3 percent this year. “Money is available, but it doesn’t come cheap,” he said, especially when there is no sense of economic security. Professor Cooney also discussed suitable business models given the economic climate. In his remarks, Manuel Heitor, Portugal’s Secretary of State for Science, Technology and Higher Education, expressed Portugal’s interest in learning from the United States’ experience. Partnerships and Intellectual Property The workshop also included remarks from En-Ju Lin from Neurologix, a clinical-stage biotech company dedicated to the development and commercialization of gene-transfer therapies for brain and central nervous system disorders. She told the workshop audience that her company is currently experiencing an inflow of capital from other biotech companies, and emphasized the importance of seeking out partnerships to cover costs. She also said that Neurologix has a promising group of gene-based therapies that are near the end stage of clinical trials. Although gene therapy is gaining wider acceptance, she said, “it is not yet fully accepted as a therapeutic modality, because there are no established regulatory pathways.” FIDIA Advanced Biopolymers was represented at the workshop by Elisa Figallo. FIDIA, an Italian biomaterials-based company, holds more than 15 patents and has a number of products on the market; it is actively seeking collaboration with academic researchers. MIT Professor Kenneth Oye spoke about intellectual property, the complex regulatory issues behind it, and the centrality for biotech development of intellectual property and the patents that arise from it. The workshop also included presentations by Luso Cord and Crioestaminal, Portugal’s public and private entities for the preservation of stem cells for future therapeutic use, both of which are also seeking partnerships with academic and industrial researchers. Other key workshop participants included MIT Portugal faculty members Stan Finkelstein (MIT), Manuel Nunes da Ponte (FCT/UNL), Manuel Carrondo (FCT/UNL) and Lino Ferreira (CNC, Biocant); João Nuno Moieira, Luis Almeida, and Catarina Oliviera of CNC; Rui Sousa (Stemmatters); AndréFaustino (Gene PreDiT), and Miguel Prazeres (IST). | 
