Thanks to our sponsors for their invaluable support in enabling us to deliver our annual summit and helping us to ‘Re-imagine the Rare Disease Patient Journey’
Prometic is a biopharmaceutical corporation with globally recognized expertise in bioseparation and uses its own affinity technology for the development of best in class plasma-derived therapeutics and orphan drugs. ProMetic is also active in developing its own novel small molecule therapeutics products targeting unmet medical needs in the field of fibrosis. Headquartered in Laval, Canada, Prometic has a global presence through its various sites in the UK and USA, providing both manufacturing and R&D capability http://prometic.com/
Pfizer, a global innovative biopharmaceutical company, provides over 140 different medicines, vaccines and consumer healthcare products worldwide each year. Putting patients at the heart of everything they do, they believe collaboration is important, working closely with the NHS, patients and healthcare professionals to improve healthcare delivery.
Pfizer has a broad portfolio, providing medicines for many common illnesses and conditions such as cardiovascular disease and pain through to oncology and rare diseases. With more than 20 years’ experience in rare disease research and treatments approved in 81 countries, Pfizer focuses on two main areas of research in rare disease: hematologic diseases and neuromuscular disease.
For more information please visit: https://www.pfizer.co.uk/
Abcam is a global life sciences company specialising in producing highly validated antibodies to both research and clinical communities. Their mission is to help life scientists achieve their goal quicker by providing products of the highest quality with increased specificity, reproducibility and sensitivity. Research areas include oncology, epigenetics, and immunology.
Abcams headquarters are in Cambridge, UK, and currently have over 1000 employees across twelve locations worldwide.
For more information please visit: http://www.abcam.com/
Our mission at Healx is to transform the lives of rare disease patients by repurposing existing drugs as potential treatments for those living with rare conditions.
At Healx, we believe patient groups play a central role in our development process in finding new treatments. We use our innovative technology platform to predict which drugs could have potential therapeutic benefits and should be taken forward for experimental testing and progression to clinical trials.
This has been exemplified through our work with a number of charities covering diseases such as Fragile X syndrome, CDKL5 syndrome and several cancer-related disorders. In each case, we’ve successfully identified promising drug candidates for further investigation.
For more information, please visit: www.healx.io
Vertex is a global biotechnology company that aims to discover, develop and commercialize innovative medicines so people with serious diseases can lead better lives. In addition to our clinical development programs focused on cystic fibrosis, Vertex has more than a dozen on-going research programs aimed at other serious and life-threatening diseases.
Founded in 1989 in Cambridge, Mass., Vertex today has research and development sites and commercial offices in the United States, Europe, Canada and Australia. For seven years in a row, Science magazine has named Vertex one of its Top Employers in the life sciences. For additional information and the latest updates from the company, please visit www.vrtx.com.
Sobi is an international specialty healthcare company dedicated to rare diseases. Our mission is to develop and deliver innovative therapies and services to improve the lives of patients. The product portfolio is primary focused on Haemophilia, Inflammation and Genetic diseases. We also market a portfolio of specialty and rare disease products across Europe, the Middle East, North Africa and Russia for partner companies.
Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2016, Sobi had total revenues of SEK 5.2 billion (USD 608 M) and approximately 760 employees. The share (STO:SOBI) is listed on NASDAQ OMX Stockholm. More information is available at www.sobi.com.
LifeArc is the new name for MRC Technology, a medical research charity with a 25 year legacy of helping scientists and organisations turn their research into treatments and diagnostics for patients.
The new name reflects the charity’s purpose: to be the arc or bridge between research and improving patients’ lives.
LifeArc is pioneering new ways to turn great science into greater patient impact. It brings together a network of partners to tackle specific diseases and directly funds academic and early stage research.
So far, LifeArc’s work has helped to develop four drugs (Keytruda®, Actemra®, Tysabri® and Entyvio®) and a test for antimicrobial resistance.
www.lifearc.org Twitter @lifearc1.
Sanofi Genzyme, the specialty care business unit of Sanofi, focuses on rare diseases, multiple sclerosis, oncology, and immunology. We help people with debilitating and complex conditions that are often difficult to diagnose and treat. Our approach is shaped by our experience developing highly specialised treatments and working collaboratively with healthcare providers and patient groups. We are dedicated to discovering and advancing new therapies, providing hope to patients and their families around the world.
Bronze level educational grant
BioMarin is a world leader in developing innovative biopharmaceuticals for rare genetic diseases mostly affecting children. Their mission is the same now as when the company was founded in 1997; to bring new treatments to market that will make a big impact on small patient populations. Currently BioMarin has six products on the market treating lysosomal storage disorders (MPS I, MPS IVA, MPS VI, CLN2 disease), phenylketonuria (PKU) and Lambert-Eaton myasthenic syndrome (LEMS). With a growing product pipeline, BioMarin’s portfolio is expanding to include treatments for conditions such as Duchenne Muscular Dystrophy and Hemophilia A.
Headquartered in San Rafael, California, BioMarin now employs over 2,200 people. In 2016, total revenues reached $1bn for the first time, representing a 26% increase over 2015, with revenue growth expecting to continue in 2017.
For more information please visit: http://www.biomarin.com/