GlaxoSmithKline (NYSE: GSK) is investing $16 million in an upfront payment to work with an Italian firm to battle “bubble baby” disease.
The alliance with Fondazione Telethon will also team researchers in efforts to use gene therapy to combat other genetic disorders.
Patients’ bone marrow will be used to provide stem cells as part of the effort.
The research underlying the effort comes from the Raffaele Telethon Institute for Gene Therapy.
(Read here for other news from GSK’s efforts to combat rare diseases )
GSK receives a license to develop and commercialize any treatment for ADA Severe Combined Immune Deficiency, which affects some 350 children around the world. Phase I and II clinical trials have already been conducted.
The joint effort also will target six other stem cell-related therapies.
Fondazione Telephone also will receive additional payments based on milestones.
“This alliance is an important addition to GSK’s growing portfolio in rare diseases and advances the work our stem cell researchers have been doing to deliver transformative regenerative therapies,” said Dr Philippe Monteyne, head of development and Chief Medical Officer for GSK Rare Diseases, in a statement. “Not only does the lead programme for ADA-SCID represent an opportunity to provide a treatment option for an under-served patient population, by combining our experience in manufacturing complex biological products with the pioneering research of world-leaders in gene and stem cell therapies, we have the chance to dramatically advance this field.”
GSK operates its U.S. headquarters in RTP.
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