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AstraZeneca, PTC Therapeutics team up in research collaboration
by Kelsey Kaustinen  |  Email the author


ALDERLEY PARK, England and SOUTH PLAINFIELD, N.J.AstraZeneca and PTC Therapeutics, Inc. have entered into an exclusive research collaboration and license agreement for the application of PTC's proprietary GEMS (Gene Expression Modulation by Small-molecules) technology for the discovery and development of potential new therapies for cancer, as well as other diseases that represent a great unmet medical need. Per the terms of the agreement, AstraZeneca will make an up-front cash payment of an undisclosed amount to PTC for the initiation of the first target in the collaboration, plus committed research funding. The agreement's first efforts will be focused on oncology with the potential to pursue up to eight targets in different therapeutic areas.  
"We are delighted to enter into this collaboration with PTC Therapeutics. PTC's RNA biology expertise and the uniqueness of the GEMS technology are complementary to our internal efforts," said Dr. Susan Galbraith, Vice President and Head of the Oncology Innovative Medicines Research Unit at AstraZeneca, in press release about the agreement. "We believe that the GEMS technology will enable AstraZeneca to address important disease mechanisms that were intractable with our existing approaches."
PTC will also qualify for future milestone payments, dependent upon the achievement of certain research, development regulatory and commercial milestones. AstraZeneca will retain the global commercialization rights, but PTC will have the option of participating in the development of certain product candidates that result from the collaboration. PTC will also receive tiered royalties on worldwide net sales from AstraZeneca.  
"We are honored to enter into this partnership with AstraZeneca," said Stuart Peltz, Ph.D., President and CEO of PTC Therapeutics, in a press release regarding the collaboration. "AstraZeneca is widely recognized for its high scientific standards and track record in developing novel therapies for the world's most serious illnesses."  
The collaboration has plenty to work with within its objectives, as there are over 7 million cancer-related deaths globally every year, and the World Health Organization estimates that number will rise to 11 million by 2030. Additionally, PTC's GEMS technology offers the opportunity for different drug discovery approaches. The technology enables the discovery of drugs that can increase or decrease the expression of particular proteins by targeting the regulatory mechanisms. GEMS can even deliver small molecules that can be taken orally. The platform works to identify small molecules that modulate post-transcriptional control mechanism. Those compounds target specific processes that act through the regulatory regions of messenger RNA molecules.  
PTC, a biopharmaceutical company focusing on discovering, developing and commercializing small-molecule drugs that target post-transcriptional control processes, is located in South Plainfield, N.J.
Code: E06291101



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