PPMD is excited to announce that this year’s action-packed Advocacy Conference, March 3-5 2019 will include a special Gene Therapy Policy Forum on Monday, March 4 from 9am to 3pm at The Mayflower Hotel in D.C.

Hosted by PPMD, this forum boasts an incredible guest list of experts, including representatives from companies involved in gene therapy and the FDA’s Center for Biologics Research and Evaluation (CBER), the agency division responsible for reviewing gene therapies.

With Duchenne gene therapy clinical trials currently underway, the community is rightfully consumed with questions on a range of issues, including:

• Timelines for learning about results from the first studies
• The design of future trials and when broadening the inclusion criteria may occur
• Regulatory pathways for gene therapy
• Understanding pre-screening for antibodies and the issues around testing
• Exploring alternative gene therapy delivery methods
• Will redosing ever be possible?

This policy forum will bring together regulators, companies, researchers, and most importantly our patient community to start the process of sharing information on the current status of gene therapies in Duchenne, understanding areas of collaboration, building relationships, and setting expectations. We aim for a day of meaningful dialogue and promotion of cross-sectional collaboration to ensure we are all moving in the same direction together and that the patient community remains front and center to address questions and provide the best possible information on gene therapy today in Duchenne.

A report from the forum will prepared and posted for the community.

Gene Therapy Webinar Series

Following the forum, we plan to host an educational webinar series on gene therapy covering a number of these topics so that the entire community can also get the benefit of the discussion and information that will come from the forum. We believe the forum will help to inform and shape the planning for the series.

Register to Attend PPMD’s Advocacy Conference & Gene Therapy Policy Forum

Click here to register for PPMD’s 2019 Advocacy Conference which includes the Gene Therapy Policy Forum. This registration is for Duchenne families who plan to also attend Hill meetings.

Register Now

Hotel deadline is February 11th – be sure to reserve your room asap.

Interested in attending only the Policy Forum?

If you are just interested in only attending the Gene Therapy Policy Forum on Monday, and will not be participating in the other Advocacy Conference activities, please click here to register.