GlaxoSmithKline Forms Alliance for Rare Disease Research

GlaxoSmithKline has announced a new alliance with Italy-based Fondazione Telethon and Fondazione San Raffaele in order to boost its research on treatments for rare diseases.

The two Italian firms focus their work on ADA Seyer Combined Immune Deficiency, a rare genetic disorder that affects about 350 children around the world. Through the partnership GlaxoSmithKline intends to bolster its rare disease research unit. This unit will focus on diseases to which research can "realistically" make a significant contribution, says unit head Marc Dunoyer.

"With less than 10% of patients with rare diseases currently being treated worldwide, we recognise the size of the challenge, but also the opportunity to deliver new medicines to patients."

The unit aims to target a list of about 200 "priority" diseases, which is expected to change over time as their scientific knowledge and understanding change.

GlaxoSmithKline is the UK's largest drug maker. As such companies' drugs face generic competition it is common to expand into rare diseases as they seek new ways to generate revenue. Therapies for rare conditions can win an 'orphan' status from regulators, allowing companies to charge more and giving them a seven-year monopoly on production in the US.

The partnership also plans to develop six additional applications, which will use gene therapy on stem cells taken from patients' bone marrow to potentially treat a range of rare disorders. According to Mr. Dunoyer, GlaxoSmithKline will continue seeking collaboration on rare disease research.

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