At GSK we are on a mission. A mission to help people to do more, feel better, live longer.

GSK develops and commercialises medicines to treat a broad range of acute and chronic diseases across 170 countries, with leading global positions in respiratory and HIV (ViiV). The business is focused across a wide range of therapy area including respiratory, vaccines, cardiovascular, metabolic, immuno-inflammation, infectious diseases, neurosciences, oncology, dermatology and HIV. 

Our Rare Diseases unit consists of dedicated individuals who represent some of the world’s leading talent in gene therapy and rare conditions, working to harness our scientific expertise, partnerships, global reach and manufacturing capability to develop and deliver much needed medicines. Less than 5% of known rare diseases have approved medicines to treat them. Our aim is to change this.

GSK currently has active research programmes across a range of rare diseases including primary immune deficiencies, amyloidosis, lysosomal storage disorders and pulmonary arterial hypertension.

Collaboration is key to success. We know we won’t discover everything inside our own labs and to make progress requires a concerted effort from companies, regulators and academic institutions. Since October 2010, we have been part of a strategic alliance with TIGET, a world-leading research centre for stem cell gene therapy based in Italy.  This alliance involves autologous gene therapy for primary immune deficiencies (such as ADA-SCID or adenosine deaminase severe combined immunodeficiency), lysosomal storage disorders and blood disorders.

More recently we have entered into collaboration with Adaptimmune, a specialist research company exploring the potential of gene therapy to improve the ability of the immune system to fight cancer.

Patients and their families are at the heart of everything we do. They are experts in their own disease with an extraordinary determination to overcome multiple challenges against all odds. That’s why we actively seek their advice. We are working hard alongside other institutions, scientific and patient experts so that one day our medicines will hopefully make a positive difference to the lives of rare disease patients and their families.

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