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Key Advances in Gene Therapy and the Promise for Patients

Recent scientific and clinical breakthroughs in gene therapy offer hope to patients who live with cancer, retinal disease, hemophilia, neurodegenerative conditions, and other diseases. Gene therapy is the introduction, removal, or change in the content of a person’s genetic code with the goal of treating or curing a disease.

Following the passage of the 21st Century Cures Act in December 2016, both the Food and Drug Administration (FDA) and the National Institutes of Health (NIH) have made significant progress in advancing the science and helping to bring safe and effective gene therapies to patients. Over the last year, three gene therapy products have been approved by the FDA to treat certain types of cancer and vision loss. Additionally, at least eight gene therapy product candidates have received the Regenerative Medicine Advanced Therapy (RMAT) designation, with many more in the pipeline.

Last week, the Bipartisan Policy Center, the Alliance for Regenerative Medicine (ARM), and the American Society of Gene and Cell Therapy hosted a roundtable discussion on gene therapy with leaders in the field representing patients, industry, scientists, payers, the investment community, as well as federal government officials, including FDA’s CBER Director Peter Marks.

We explored the current state of research and science, the considerable progress made by federal agencies to advance the field, the barriers to the development and access to safe and effective therapies, and a range of policies and strategies needed to address those barriers. The group also discussed the need for and promise of gene therapies for patients with neurodegenerative diseases which currently affect millions of Americans and for which more innovation and progress is needed.

According to ARM, nearly 700 gene therapies or gene-modified cell therapies are in clinical trials, more than half of which are in Phase II or Phase III. These trials represent immense promise for those with neurodegenerative conditions such as Parkinson’s disease, Alzheimer’s disease, ALS, and Huntington’s disease.

Attendees identified six key areas that need to be addressed moving forward:

  • New value-based payment models that take into account the unique nature of gene therapies (a potentially one-time durable therapy vs. ongoing treatment of symptoms);
  • Mechanisms to capture and assess both short- and long-term outcomes to advance the science and assess the durability of treatments;
  • Increased international standards and requirements convergence;
  • Greater collaboration and coordination among academic scientists and industry to facilitate bringing new discoveries to patients more rapidly;
  • Increased public education on the promise of gene therapies; and
  • Further exploration of ways to support the development and delivery of gene-based therapies for those with neurodegenerative diseases.

Next month, our organizations plan to release a report based on the insights gained during the meeting and additional research.

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