Posts Tagged Biomarin
Neglected and Rare Diseases Panel
Posted by Darshana V. Nadkarni, Ph.D. in Biotech - Medical Device - Life Science - Healthcare on February 24, 2014
EPPICon 2014 (www.eppicglobal.org) will address the important topic of developing therapeutics for rare and neglected diseases. So what are rare and neglected diseases? Rare diseases are diseases that affect a small percentage of the population, most of them are genetic, and may be present throughout a person’s life. In the United States, rare diseases are defined as those that affect fewer than 200,000 people. About 7,000 diseases have been designated as rare and as many as one in ten Americans may suffer from a rare disease. More rare diseases are discovered ongoingly. Despite the growing numbers of rare diseases, the overall numbers are small and therefore they had not been a focus of research and many do not have treatments available.
Neglected diseases have also not been a focus of great deal of research and many of these diseases also lack viable treatment options. These include tropical diseases like Tuberculosis and Malaria and affect over 1.4 B people, worldwide. Often these affect the most vulnerable populations in the developing world, who lack access to basic sanitation, healthcare, and clean water.
A panel moderated by Roopa Ramamoorthi will discuss how companies and researchers can engage and bring forward cures for these diseases. Rmamoorthi is an experienced scientist with extensive background in global health, drug development, biotechnology, bacteriology, and engineering. As an Associate Director for Partnering and Scientific Affairs, at BioVentures for Global Health, Ramamoorthi leads the efforts to match researchers with pharma and other contributors, with an aim to accelerate product development for neglected tropical diseases like TB and Malaria.
Panelists include David Swinney, CEO of iRND3 (Institute for Rare and Neglected Diseases Drug Discovery). Swinney has 20+ years of broad experience in preclinical drug discovery. He founded his current non profit, in 2010, with a mission to help discover new medicines for rare and neglected diseases. The equipment for its lab in Mountain View was donated by Roche, and three early stage drug discovery programs at iRND3 have focused on pediatric cancers and parasitic diseases.
Eric Easom is the VP of neglected diseases at Anacor Pharmaceuticals, and Vimal Srivastava, is VP or Product Development, at Ultragenyx Pharmaceuticals. Anacor is focused on discovering, developing and commercializing novel small molecule therapeutics derived from its novel boron chemistry platform. Ultragenyx is committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with an initial focus on serious and debilitating, metabolic genetic diseases. On the panel, they are likely to add the big pharma perspective regarding focusing on neglected and rare diseases.
Ponni Subbiah, MD has an extensive experience in global medical affairs and clinical development across multiple therapeutic areas in the pharmaceutical sector. Currently, in the role of Global Program Leader, Subbiah is leading drug development efforts, at PATH, a nonprofit organization, with a bold vision for improving health, worldwide. PATH seeks to blend the entrepreneurial side of the business and the scientific expertise of a research institution with on-the-ground experience of an international NGO.
Other interesting panels at EPPICon include, “Innovations in Clinical Development of Novel Agents” and “Point of Care Gold Rush – Hype versus Reality”. Besides keynotes and networking opportunity, the conference will also feature a Speed Pitch session where entrepreneurs of early stage companies in the life sciences, are invited to give a five minute pitch about their technology and receive a quick feedback from a distinguished panel of VCs.
EPPIC is a volunteer driven organization, with a mission to promote networking, entrepreneurship, and mentoring for life science professionals. All day EPPIC conference will be held at The Westin, San Francisco Airport, in Milbrae, CA, on March 29, 2014. For more details and to register for the conference, go to www.eppicglobal.org . Please note: Early bird registration is extended to March, 7. EPPICon has an excellent lineup of great speakers and panels. This is not a conference to miss for any life science industry professional. Hope to see you there.