American Society of Gene and Cell Therapy

Newborn Screening SPOTlight Podcast

Advances in gene and cell therapies are enabling researchers, clinicians, families, and regulators to work together in incredible new ways to treat previously untreatable conditions.

Listen to Christina Mayer share her efforts to advance policies that help to realize a future where gene and cell therapies are available to all individuals for all diseases. Christina is currently the Senior Manager of Government Affairs at the American Society of Gene and Cell Therapy in Milwaukee, Wisconsin. She works with federal government agencies and decision-makers to impact key components of gene and cell therapies like NIH research funding, genetic testing and screening, payment policy and patient access to approved therapies. She also contributes to the Society’s work on other policy priorities, such as regulatory oversight and the responsible use of new genetic technologies. Christina has a Master of Public Administration from the University of Nebraska-Omaha.

Listen with us as we imagine a future where the availability and equitable use of gene and cell therapies helps to realize the promise of a healthy future for all.

Podcast Interview Questions with Christina Mayer:

1. You are currently the Senior Manager of Government Affairs at the American Society of Gene and Cell Therapy. Can you tell us about the mission of the American Society of Gene and Cell Therapy? What is your role there?

2. What an exciting and rewarding time to advocate for gene and cell therapies! ASGCT reminds us all that there are literally 1000s of clinical trials for novel therapies and over the next decade there will be about 30 approved therapies for genetic disease, not counting cancer. What can our listeners do to become more aware of these efforts and to become advocates for continued advancement and access to these life-saving and in many cases disease curing therapies?

3. In its 2020 – 2022 strategic plan, ASGCT identified access to genetic testing and screening as one of its core patient access priorities. What efforts have been made by ASGCT to advance access to genetic testing and screening in newborns? Can you describe ways that the current approach to newborn screening in the United States could be improved to enable the use of gene and cell therapies? What are ways that our listeners can get connected to your organization?

4. In Feb 2021, ASGCT provided a public comment to Advisory Committee on Heritable Diseases in Newborns and Children (ACHDNC) on the newborn screening process.In this letter, ASGCT stated its support of the Newborn Screening Saves Lives Act. Can you tell us listeners more about the history of this act? Why is it important that it get passed? What would happen if it doesn’t?

5. Also, in the letter, ASGCT offers three recommendations to the ACHDNC. They are: 1) Ensure the RUSP keeps pace with treatment approvals, 2) Collaborate with and rely upon the FDA, and 3) Ensure the process to advance a disorder through the ACHDNC is transparent, predictable,and timely. Would you mind sharing the evidence that supported each of recommendations? (Perhaps, discuss the problem and why this recommendation would solve that problem)

6. ASGCT has worked partner organizations to support other NBS and you are hosting a workgroup and symposium in May to discuss advancements in NBS. Thank you for inviting Dr. Brower and NBSTRN to present during the workshop. Please tell us more about these important events and how they can participate.

7. You and Dr. Brower serve on the planning committee for an effort by EveryLife Foundation to develop actionable policy solutions aimed at ensuring newborn screening continues to advance. This includes the research facilitated by NBSTRN and ASGCT, as well as the policies that you and your team champion. Can you describe why these types of efforts to build coalitions and collaborations across different stakeholder groups are so important?

8. You have a very interesting career path and your work inspires many of us to achieve meaningful change and work to advance discoveries that save and improve lives. Can you share with our listeners what sparked your interest in the revolution that is gene and cell therapy as well as newborn screening research?

9. What does NBS research mean to you?

Learn more about ASGCT annual meeting at https://annualmeeting.asgct.org

To learn more about newborn screening research data tools and resources, visit www.nbstrn.org