AstraZeneca, PTC Therapeutics team up in research collaboration

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.

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ALDERLEY PARK, England and SOUTH PLAINFIELD,N.J.—AstraZeneca and PTC Therapeutics, Inc. have entered into an exclusiveresearch collaboration and license agreement for the application of PTC'sproprietary GEMS (Gene Expression Modulation by Small-molecules) technology forthe discovery and development of potential new therapies for cancer, as well asother diseases that represent a great unmet medical need. Per the terms of theagreement, AstraZeneca will make an up-front cash payment of an undisclosed amountto PTC for the initiation of the first target in the collaboration, pluscommitted research funding. The agreement's first efforts will be focused ononcology with the potential to pursue up to eight targets in differenttherapeutic areas.
 
 
"We are delighted to enter into this collaboration with PTCTherapeutics. PTC's RNA biology expertise and the uniqueness of the GEMStechnology are complementary to our internal efforts," said Dr. SusanGalbraith, Vice President and Head of the Oncology Innovative MedicinesResearch Unit at AstraZeneca, in press release about the agreement. "We believethat the GEMS technology will enable AstraZeneca to address important diseasemechanisms that were intractable with our existing approaches."
 
PTC will also qualify for future milestone payments,dependent upon the achievement of certain research, development regulatory andcommercial milestones. AstraZeneca will retain the global commercializationrights, but PTC will have the option of participating in the development ofcertain product candidates that result from the collaboration. PTC will alsoreceive tiered royalties on worldwide net sales from AstraZeneca.
 
 
"We are honored to enter into this partnership withAstraZeneca," said Stuart Peltz, Ph.D., President and CEO of PTC Therapeutics,in a press release regarding the collaboration. "AstraZeneca is widelyrecognized for its high scientific standards and track record in developingnovel therapies for the world's most serious illnesses."
 
 
The collaboration has plenty to work with within itsobjectives, as there are over 7 million cancer-related deaths globally everyyear, and the World Health Organization estimates that number will rise to 11million by 2030. Additionally, PTC's GEMS technology offers the opportunity fordifferent drug discovery approaches. The technology enables the discovery ofdrugs that can increase or decrease the expression of particular proteins bytargeting the regulatory mechanisms. GEMS can even deliver small molecules thatcan be taken orally. The platform works to identify small molecules thatmodulate post-transcriptional control mechanism. Those compounds targetspecific processes that act through the regulatory regions of messenger RNAmolecules.
 
 
PTC, a biopharmaceutical company focusing on discovering,developing and commercializing small-molecule drugs that targetpost-transcriptional control processes, is located in South Plainfield, N.J.



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