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An Inside Look at CAR-T, Gene Therapy Launches at ‘What’s Hot’ May 16

Xconomy Boston — 

Getting a new drugmaking technology through clinical development and to market is a major milestone, the culmination of years of scientific work. But the true test of how important that technology is comes afterwards, when the real world gets its hands on it.

This type of story is playing out either right now, or soon will, in biopharma for multiple new forms of cutting edge medicine. Gene therapy, and CAR-T cell therapy, two genetic modification techniques that promise long-lasting results with a single treatment, are in the early days of their commercial launch in the U.S. Another drugmaking method, RNA interference, should follow in a matter of months.

All of them are entering an uncertain phase in their scientific stories.—and key leaders in all these fields will be telling their stories on May 16 at “What’s Hot in Boston Biotech” at The Broad Institute. You can check out the full lineup and grab your tickets here. And read on for a bit more of what is in store.

All the emerging drugmaking approaches mentioned above are confronting a fragmented and complex U.S. healthcare system that hasn’t yet seen anything like them. In the case of gene therapy and CAR-T, the commercial launches are underway, and just handfuls of patients have been treated. Spark Therapeutics’s (NASDAQ: ONCE) gene therapy for genetic vision loss, voretigene neparvovec (Luxturna), has only been given to three patients. The two approved CAR-T therapies, from Novartis (NYSE: NVS) and Gilead Sciences (NASDAQ: GILD), generated $52 million in their last quarters combined.

What have these launches looked like on the front lines, from the doctors that are administering them to patients? How are they being handled by payers and healthcare institutions? What deals are being struck between companies and insurers to adapt the U.S. healthcare system to these new types of treatments, and what important work has to be done to broaden their use? How do the first movers in the field plan to pave the way for others?

We’ll tackles these questions and more on May 16 through a series of candid, interactive chats in a variety of formats. We’re featuring a slew of new and emerging drugmaking methods at various different points of their scientific journeys, from the early human testing (CRISPR gene editing and messenger RNA therapeutics) on forward.

Some of the speakers you can expect to see include:

Feng Zhang: Core Member, Broad Institute of MIT and Harvard; Investigator, McGovern Institute for Brain Research at MIT

Craig Mello: Nobel Laureate; Professor of Molecular Medicine, University of Massachusetts Medical School, RNA Therapeutics Institute

Caron Jacobson: Medical Director, Immune Effector Cell Therapy Program, Dana-Farber Cancer Institute

Michael Sherman: Senior Vice President & Chief Medical Officer, Harvard Pilgrim Health Care

Again, you can grab your tickets here, and check out the agenda and full speaker lineup here. See you all next week at the Broad.