CureDuchenne Shares Pfizer’s Update Regarding Initiation of Gene Therapy Trial

Today, Pfizer announced that the first patient was dosed in their mini-dystrophin gene therapy trial.  This is an exciting day for the Duchenne community.  Pfizer has the experience to conduct this trial, and manufacturing capacity to see it through to success.  Of course, this is an early stage safety trial and there is a long way to go until we know if this therapy will be safe and effective.  We applaud Pfizer’s leadership for addressing our community and the desperate need we have for a meaningful therapy for our loved ones.

Our prayers, best wishes and gratitude go out to the boys and the families that participate in all of the clinical trials that are so necessary to bring treatments to this disease.

CureDuchenne is proud to have contributed to this progress with our funding of Bamboo Therapeutics, which was acquired by Pfizer in 2016.  CureDuchenne has a successful record of funding early stage science and clinical trials which have moved into the clinic…and approval.  We count on the Duchenne community to support our efforts to make sure we keep the drug development pipeline full.

Please read the update from Pfizer:

Pfizer Biomedical Institute

April 12, 2018

Dear Debra,

Pfizer is pleased to provide the following update regarding the Phase 1b, Multicenter, Open-label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of PF-06939926 in Ambulatory Boys with Duchenne Muscular Dystrophy (DMD).  The first boy received an infusion of the mini-dystrophin gene on March 22nd, administered under the supervision of the principal investigator at the study site.  This first participant continues to be monitored.

The study will enroll approximately 12 ambulatory boys aged 5 to 12 years with DMD.  In addition to evaluating safety and tolerability, the study will evaluate measurements of dystrophin expression and distribution, as well as assessments of muscle strength, quality, and function. As part of the screening process, potential candidates invited by the study principal investigator will be tested to confirm a negative result for antibodies against the adeno-associated virus, serotype 9 (AAV9) capsid and for a T-cell (immune) response to dystrophin. Screening and enrollment of patients is expected to continue at up to four clinical research sites in the United States.   Early data from this trial are expected in the first half of 2019, once the first patient completes one full year post-treatment. More information about the trial and participating sites may be found at (NCT03362502).

We recognize the commitment that the DMD community has shown for this study and for Pfizer’s research and development efforts related thereto.   We further recognize the potential impact innovative therapies, like gene therapy, may have in transforming the lives of individuals and families affected by DMD and we share with you that hope and vision.  It is with heartfelt gratitude that we extend our sincerest ‘thank you’ to all the boys and families who continue to express interest in and who participate in clinical research.  We are furthermore indebted to the advocacy associations and advocates who provide the tools and support needed for families to engage in clinical research, who continue to care for the Duchenne community and who lend their expertise in the research and development process.

Additional public-facing information about this milestone achievement can be found at:

Kind regards,

Katherine Beaverson

Patient Advocacy Lead

Cc:  Tara Moorehead, Michael Binks, MD


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