This year’s already action-packed Advocacy Conference, February 21-23 welcomes a special Gene Therapy Forum on Monday, February 22 from 9am to 1pm at The Mayflower Hotel in D.C. Hosted by PPMD, this forum boasts an incredible guest list of experts including representatives from the FDA’s Center for Biologics Research and Evaluation (CBER), the agency division responsible for reviewing gene therapies.  


Many efforts have started in bringing a Duchenne gene therapy forward, but these efforts have focused on single muscle and limb delivery. Our next hurdle is to deliver these therapies systemically (body-wide delivery), while maintaining safety and efficacy. This policy forum will bring together regulators, researchers and our patient community to start the process of sharing information on the current status of (systemic) gene therapies in Duchenne, understanding areas of collaboration, building relationships, and setting expectations. 


The day will start with an overview of where current Duchenne gene therapies are in development. We will then move to a panel discussion on existing hurdles to moving gene therapies into the clinic, what innovations are being applied to overcome them, what challenges continue to exist, and potential paths forward.

Participating panelists include*: 

  • Carrie Miceli, PhD
  • Dongshen Duan, PhD
  • Jim Wilson, MD PhD
  • Lee Sweeney, PhD
  • Jerry Mendell, MD, PhD
  • Kevin Flanigan, MD


PPMD staff will be live tweeting from the forum (follow us @ParentProjectMD) and we will provide a full report shortly after. This half day forum is designed to encourage our community to come together to set the stage for gene therapy development and work together to develop a successful approach to bringing these therapies to patients. 


Register to Attend the Gene Therapy Forum

Click here to register for PPMD's 2016 Advocacy Conference which includes the Gene Therapy Forum.


If you are just interested in attending the forum, but will not be participating in the other Advocacy Conference activities, please click here to register.

*While panelists are confirmed, the list of participants is subject to change dependent on travel conditions and scheduling.


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Comment by Jay Griffin on February 16, 2016 at 8:13am
Thank you very much
Comment by Alpa Khushalani on February 10, 2016 at 2:27pm

Thank you so much to PPMD for keeping our community always at the forefront of the next battle to win the war against Duchenne. Looking forward to the report from the discussion

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