Thursday 8th - Wednesday 14th December
Sponsors
Platinum Sponsor
Established in 1997, BioMarin is a world leader in developing and commercializing first- or best-in-class therapies for rare genetic diseases. We take pride in going where the science leads us, pioneering breakthrough treatments for debilitating and life-threatening conditions where we can significantly improve upon the current standard of care. Our culture revolves around the ethos that no disease should go untreated, and our people are driven to discover, develop, and commercialize medicines that give patients, their families, and their caregivers hope where there was little or none. We fuel our R&D engine by looking for opportunities that align with our strengths and competencies. And we relentlessly pursue exciting, early-stage science that has the potential to change the course of disease. Voted one of America’s Best Midsize Employer by Forbes in 2019, our employees feel connected to their work at BioMarin because they believe in our purpose: the patient. The passion and dedication that our employees bring to work each day is a testament to the inspiration our patients provide, and the knowledge of the impact we can make in their lives.
Gold Sponsor
PTC has been successful over the years because our team is deeply committed to a single purpose: Extending life’s moments for patients with rare diseases and their loved ones. Our biggest accomplishments have always been measured by units of time – or “measured by moments.” Whether our therapies grant hours, days, months or years, every small moment is an enormous win.
Silver Sponsors
Synlogic™ is pioneering the development of a novel class of living Synthetic Biotic™ medicines based on its proprietary drug discovery and development platform. Synlogic’s pipeline includes Synthetic Biotic medicines for the treatment of Phenylketonuria (PKU), Enteric hyperoxaluria, and solid tumors. In addition, the company is leveraging the broad potential of its platform to create Synthetic Biotic medicines for the treatment of other diseases, such as inflammatory and immune disorders. For more information, please visit synlogictx.com.
Homology Medicines, Inc. is a clinical-stage genetic medicines company dedicated to transforming the lives of patients suffering from rare diseases by targeting the underlying cause of the disease. The Company’s lead clinical program, HMI-102, is a gene therapy for adults with phenylketonuria (PKU) and additional programs focus on gene editing in PKU, lysosomal storage disorders including Hunter syndrome, paroxysmal nocturnal hemoglobinuria (PNH) and other diseases. Homology’s proprietary platform is designed to utilize its family of 15 human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo through a gene therapy or nuclease-free gene editing modality, as well as to deliver one-time gene therapy to produce antibodies throughout the body through the GTx-mAb platform. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a focus on rare diseases and believes that its data, internal manufacturing capabilities and broad intellectual property position the Company as a leader in genetic medicines.