Warrior Families: Advancing Regenerative Medicine Through Science
This summary covers a webinar hosted by the U.S. Food and Drug Administration’s Office of Therapeutic Products, focusing on the role of ‘Warrior Families’ in advancing regenerative medicine for rare diseases. The webinar includes presentations from Dr. Allyson Berent, who will discuss her experience advocating for drug development for Angelman Syndrome, and panelists Erin Ward and Suzette James, who will share their experiences with myotubular myopathy and Batten Disease respectively. Key points include the importance of patient-centred approaches, the potential for transformative treatments for rare diseases, the need for investment in training clinical trialists, and the importance of collaboration, support, and investment in developing effective therapies for rare diseases. The webinar concludes with a Q&A session.
The webinar, hosted by the Office of Therapeutic Products within the Center for Biologics Evaluation and Research at the U.S. Food and Drug Administration, focuses on the role of passionate families in advancing regenerative medicine for rare diseases. The theme of the webinar is Warrior Families, highlighting the experiences of caregivers and their advocacy and research activities.
The webinar agenda includes a welcome from Dr. Nicole Verdun, Director of OTP, presentations from the panelists, and a Q&A session. The webinar is part of the RegenMedEd event series, which aims to provide educational resources on regenerative medicine therapies. The event will be recorded and posted on FDA’s website, and closed captioning is available.
Dr. Allyson Berent: Advocating for Angelman Syndrome
Dr. Allyson Berent, Chief Science Officer at the Foundation for Angelman Syndrome Therapeutics, will present on her experience advocating for drug development for Angelman Syndrome. The presentation will cover the journey of a patient, clinician, community, and industry in advancing regenerative medicine for Angelman Syndrome. Dr. Berent will discuss different therapeutic approaches, such as gene therapy and antisense oligonucleotides, being explored for Angelman Syndrome. The importance of collaboration, clinical trial readiness, and the development of endpoints and biomarkers will also be highlighted.
Panelists: Erin Ward and Suzette James
Erin Ward, founder of MTM-CNM Family Connection, will discuss her son’s experience with myotubular myopathy. Suzette James will share her experience with Batten Disease. Both panelists will emphasize the challenges and importance of natural history studies, data sharing, collaboration, transparency, and patient preferences in rare disease research and development.
- The webinar aims to empower patient and parent communities to connect with researchers, industries, and other partners to advance regenerative medicine.
- The webinar highlights the challenges and successes in advancing regenerative medicine for rare diseases and the importance of patient-centered approaches.
- The current landscape of regenerative medicine and the potential for transformative treatments for rare diseases will be discussed.
- The dedication and resilience of warrior families in driving progress in regenerative medicine will be showcased.
- The importance of making science-based decisions in drug development and the progress made in funding and advancing programs for rare diseases will be emphasized.
- The need for investment in training clinical trialists and the establishment of centers of excellence for gene therapy and regenerative medicine will be addressed.
- Collaboration, support, and investment are crucial for the development of effective therapies for rare diseases.
- The challenges and expenses of drug development will be discussed, along with the importance of advocacy and taking up space in the room.
- Natural history studies, data sharing, streamlined processes, risk-benefit analysis, and informed consent are essential in rare disease research.
- Pre-competitive collaboration and sharing information for the benefit of patients are encouraged.
- The webinar concludes with a discussion and Q&A session, allowing for audience engagement and further collaboration in the field.
- Title: Warrior Families: Advancing Regenerative Medicine Through Science
- Author(s): Anne Rowzee, Nicole Verdun, Allyson Berent, Erin Ward, Suzette James
- Author(s)’ affiliation: FDA; FAST; MTM-CNM Family Foundation; BDSRA Foundation
- Publication date: 2023-10-22