Gene therapy and rare diseases topic of Life Sciences Caucus

The General Assembly's Life Sciences Caucus met Tuesday, June 28, to learn about the promise of gene therapies as potential treatments for rare diseases.

Nearly two dozen legislators and staff members heard from Priya Kishnani, M.D., and Vandana Shashi, M.D., of the Duke Undiagnosed Diseases Network and Marianne Hamilton Lopez, M.D., of the Duke Margolis Center for Health Policy along with Charlene Cowell, executive director of Bleeding Disorders of North Carolina. Caucus Co-Chairs Sen. Paul Newton, Sen. Mike Woodard and Rep. Donna White were in attendance.

Hamilton Lopez told legislators that gene therapies will often differ from traditional treatment in that the therapies may only be administered once as opposed to conventional treatments that are given as multiple or continual doses of medication. Gene therapies are currently much more expensive than conventional therapies, which makes them a challenge for our current system to pay. The upfront cost of these breakthrough therapies must be weighed against the impressive benefits they offer compared to conventional therapies.

Presentation

Story of Wade Hosey, child with spinal muscular atrophy

Caucus Co-Chairs Sen. Paul Newton and Rep. Donna White

Caucus Co-Chair Sen. Mike Woodard

Life Sciences Caucus Meeting, June 27, 2022