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John F. Crowley: Discussing the state of Cell and Gene Therapy research at The White House

John F. Crowley/LinkedIn

Jan 12, 2025, 15:19

John F. Crowley: Discussing the state of Cell and Gene Therapy research at The White House

John F. Crowley, President and CEO of Biotechnology Innovation Organization, shared a post on LinkedIn:

“AT THE WHITE HOUSE…yesterday I moderated a discussion at an event at The White House to discuss the state of Cell and Gene Therapy research.

If you don’t know what this is or why you should care, this is a picture of Emily Whitehead. In 2012 as a small child she was dying from an aggressive form of Leukemia.

Out of desperation to save her life, Emily’s parents Tom and Kari took her to U Penn and she became the first child with cancer to receive a Cell-based therapy known as CART-T in a study by Carl June.

The process involved collecting Emily’s T-cells (a type of white blood cell), genetically reprogramming them to recognize and attack cancer cells, and infusing the modified cells back into Emily’s blood.

The treatment was highly experimental, but it was Emily’s only chance for a cure. Incredibly, the T-cell therapy worked, and Emily has remained cancer-free to this day. Yes, Emily was literally CURED of cancer.

This miracle of medicine has now been repeated thousands of times.

And likewise, Gene Therapies are now effectively being used to treat and often cure rare inherited diseases. But tragically, the entire system is broken and we are failing millions of patients who could benefit.

The research takes too long. The manufacturing costs are still too high and the processes too complex.

The regulatory burdens are immense. And after all that, insurance and payment systems deny coverage for the vast majority of patients who could benefit.

I am grateful to the Biden White House for hosting yesterday session.

And I am very optimistic that fixing this broken system will be a high priority for the incoming Trump Administration. We have the science at hand, we just need a system that works- and works for all.

And works now. This is one of my highest priorities for BIO. The world cannot wait….”

John F. Crowley