NEWPORT BEACH, Calif.–(BUSINESS WIRE)–CureDuchenne, a leading global nonprofit dedicated to finding and funding a cure for Duchenne muscular dystrophy (DMD), celebrates Pfizer Inc’s (NYSE: PFE) announcement surrounding the first dosing of a patient with Duchenne in their Phase III gene therapy clinical trial. Pfizer has showcased tremendous progress over the past year in pushing forward PF-06939926, its rAAV9 mini-dystrophin gene therapy for DMD.
“Our mission as an organization is to help each family and every child affected by Duchenne. Pfizer’s first dosing of a patient with Duchenne in their Phase III clinical trial is encouraging progress and an exciting acceleration of their mini-dystrophin treatment for the Duchenne community.”
PF-06939926 was initially developed by Jude Samulski, PhD and Xiao Xiao, PhD at Bamboo Therapeutics, Inc. (Bamboo). CureDuchenne Ventures, the investment arm of CureDuchenne, provided critical support to Bamboo during the pre-clinical drug development stage, enabling them to proceed with early vector manufacturing and to expedite critical pre-clinical experiments. Bamboo was later acquired by Pfizer in August 2016.
“Pfizer’s announcement is a significant step forward for everyone affected by Duchenne,” said Debra Miller, CureDuchenne founder and CEO. “Our mission as an organization is to help each family and every child affected by Duchenne. Pfizer’s first dosing of a patient with Duchenne in their Phase III clinical trial is encouraging progress and an exciting acceleration of their mini-dystrophin treatment for the Duchenne community.”
Duchenne gene-replacement therapies such as Pfizer’s aim to deliver a functioning copy of a gene to a patient’s cells in order to compensate for the defective DMD gene. The newly added DMD mini-gene makes a small form of dystrophin protein to compensate for the dystrophin protein missing in Duchenne. This lack of dystrophin is the cause of debilitating decline in the muscles in a Duchenne patient.
Pfizer’s acquisition of Bamboo is another example how CureDuchenne’s venture philanthropy support can help foster programs at critical stages, allowing them to grow and mature for larger impact. CureDuchenne Ventures provides funding and domain expertise to accelerate critical research to early and mid-stage Duchenne companies, enabling them greater likelihood for success.
For more information about the clinical trial, please check out https://www.cureduchenne.org/partner-news/pfizer-doses-first-participant-in-phase-3-study-for-duchenne-muscular-dystrophy-investigational-gene-therapy/
About Duchenne Muscular Dystrophy (DMD)
DMD is a severe X-linked form of muscular dystrophy that affects approximately 1 in 5,000 males born each year. DMD is caused by the absence of the dystrophin protein. Dystrophin is a large protein that provides multiple cellular functions and helps to protect skeletal and cardiac muscle against injury, inflammation and fibrosis. Individuals with DMD show progressive muscular degeneration, lose the ability to walk by early to mid-teens, and progress to full loss of upper body function. Cardiopulmonary complications are the primary cause of death.
CureDuchenne is recognized as a global leader in research, patient care, and innovation for improving and extending the lives of those with Duchenne muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 individuals living today. CureDuchenne is dedicated to finding and funding a cure for Duchenne by breaking the traditional charitable mold through an innovative venture philanthropy model that funds groundbreaking research, early diagnosis, and community education. For more information on how to help raise awareness and funds needed for research, please visit www.cureduchenne.org.
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