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CureDuchenne congratulates Pfizer on their progress to a Phase III clinical trial for #PF-06939926, its AAV9 mini-dystrophin gene therapy for Duchenne muscular dystrophy. We’re grateful to Pfizer for their dedication to the Duchenne community. Pfizer’s announcement is a big step forward for gene therapy efforts in Duchenne, an innovative field where there is still so much to learn. There is still more work to do, but this trial will enable much more knowledge as to the efficacy and viability of this kind of treatment for our Duchenne boys. We need to remember that clinical trials are experiments, not treatments, as we celebrate this next step in development.

How to get more info:

More information about the trial’s criteria that can be found here:

Who’s this for and how will it work:

Pfizer’s gene therapy works by delivering a functioning copy of a gene to a patient’s cells to compensate for the defective DMD gene in Duchenne muscular dystrophy. This newly added DMD mini-gene makes a small form of dystrophin protein, to compensate for the dystrophin protein missing in Duchenne. The lack of dystrophin is what causes the debilitating decline to the muscles in a patient with Duchenne. Gene therapy is not mutation-specific, but the initial trials are limited to patients between the ages of 4-7 years old.


Pfizer’s #PF-06939926 was initially developed at Bamboo Therapeutics, with the great work of Dr. Jude Samulski and Dr. Xiao Xiao. They developed a platform technology that uses adeno-associated virus (AAV) to deliver a mini-dystrophin gene sequence to the muscles. CureDuchenne Ventures provided support to Bamboo at a crucial time during their drug development process, through a $1.5 million investment to enable them to proceed with vector manufacturing and critical experiments. Eight months after this investment, Pfizer acquired Bamboo and its manufacturing facility in a deal worth up to $645 million, ultimately building and growing a state-of-the-art facility in Sanford, NC. Therefore, CureDuchenne is proud to have directly contributed to this great advancement and grateful to see the work of Bamboo and Pfizer progress to Pfizer’s Phase III clinical trial.


The CureDuchenne venture philanthropy model has a proven track record of coming through with an infusion of funding at a critical time to get important research off the ground, to bridge a moment that is ‘make or break’, or to ensure that a platform or therapy has a fighting chance. Two other recent investments fueled by CureDuchenne have brought momentum to the Duchenne community.

  • Exonics is a company that was co-founded in 2017 by CureDuchenne Ventures and Dr. Eric Olson, focused on CRISPR/Cas-9 technologies to treat Duchenne. The initial funding didn’t only start up the company, it also allowed for advancements that attracted others to invest. As a result, Exonics and its promising gene editing therapies were acquired by Vertex in 2019 for $245 million upfront, with the potential for $750 million more for achieving development and regulatory milestones.
  • When CureDuchenne recognized Dr. Kevin Flanagan’s early research at Nationwide Children’s Hospital for the potential of skipping of exon 2, a rare mutation in an already rare disease, he said, “…we wouldn’t be here today without their support. CureDuchenne plays a vital role in funding early phase research projects to help accelerate their development.” CureDuchenne has provided more than $1 million since 2010 to support Nationwide’s pre-clinical studies. Audentes Therapeutics has licensed this technology and will begin clinical trials shortly.

On days like today, when we’re celebrating progress, we know that there are still a lot of unknowns, and a lot of hurdles to get over. But the only way to get there is to advance research and support the scientific innovators out there who keep asking the hard questions and are working hard to find the answers. This work is breaking ground for Duchenne and giving us hope that we will find a cure for this generation. The technological advances also pave the way for other rare diseases. Pfizer’s Phase III clinical trial is an important indication of discovery and progress.

#LetsCureDuchenne #genetherapy

The trial sites are not yet recruiting, for more information:

Contact: Pfizer Call Center


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