Children’s Hospital Boston, Pfizer announce DMD agreement

Pfizer Inc. and Children’s Hospital Boston have announced a new collaborative program geared towards identifying potential drug therapies for Duchenne muscular dystrophy (DMD).

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BOSTON—Pfizer Inc. and Children's Hospital Boston haveannounced a new collaborative program geared towards identifying potential drugtherapies for Duchenne muscular dystrophy (DMD). The research agreement willlast one year, combining the organizations' academic and industry strengths.
"This agreement brings together the best of two worlds,namely Pfizer's ability to develop promising new compounds and our ability totest them in physiologically relevant models," Louis Kunkel, Ph.D., Director ofthe Program in Genomics at Children's Hospital Boston, said in a press release.Kunkel discovered the dystrophin gene underlying the biology of DMD. "To havePfizer show this level of interest in DMD is very exciting, and I am pleased tohave this opportunity for us to put our complementary resources to worktogether."
The agreement will focus on the work in Kunkel's laboratory.In March of this year, Kunkel and his colleagues announced the results of theirefforts to screen 1,200 chemicals approved for human use in a zebrafish modelof DMD in search of any that might present a restorative effect on muscle team.The team discovered that several demonstrated the desired effect, includingsome compounds owned by Pfizer.
"We see this as a truly novel collaboration that combinesthe efforts of a world-class academic DMD research laboratory with our chemicaland preclinical expertise," Ed Mascioli, M.D., Vice President and ChiefScientific Office of Pfizer's Orphan and Genetic Diseases Research Unit, saidin a press release. "We are proud to be working with someone of Dr. Kunkel'sstature in the DMD research arena."
Under the agreement, Pfizer will provide Kunkel with accessto select proprietary compounds via its Orphan and Genetic Diseases ResearchUnit, as well as relevant data regarding the compounds. Pfizer will also commitinternal resources, such as medicinal chemistry, to the collaboration. Kunkel'slaboratory, in collaboration with Carl Morris, Ph.D., from Pfizer, will testthe compounds in the DMD zebrafish model and aim at identifying candidates forfurther preclinical development.
"Here at Children's, we have the expertise in basic scienceand clinical care," said Kunkel, "but not the crucial chemical and developmentbackground that Pfizer has brought to the table."
"This agreement helps line up the pieces of the drugdevelopment pipeline for the benefit of children and adults with DMD," ErikHalvorsen, director of technology and business development for Children's, saidin a press release. "It is rare to see an industrial partner allow this kind ofaccess, and we are very pleased to have established this novel arrangement withPfizer."
Pfizer and Children's established another research agreementin June of this year, when Pfizer announced that Children's would be one ofseven hospitals in the Boston are to join its Centers for TherapeuticInnovation, which seek to facilitate and support joint drug discovery anddevelopment through early clinical trials. The DMD agreement is a separatearrangement between the organizations.
SOURCE: Children's Hospital Boston press release

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