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Realizing the Promise of Gene Therapies for Patients Around the World

June 5, 2019
Gene Therapy, arguably the next great leap forward in medicine, may hold the promise of curing the incurable. The first successful gene therapy took place in 1990. Now almost thirty years later, the FDA has announced it expects to receive at least 200 new gene and cell therapy INDs in the next two years. While the potential is exciting and life-changing, the cost surrounding the breakthroughs is daunting.

The panel – Realizing the Promise of Gene Therapies for Patients Around the World- discussed the challenges that will unfold over the next several years.

Deborah Miller, Chief Executive Officer of CureDechenne, said what keeps her awake at night is access. “It’s a dream come true for people who qualify for clinical trials. I think we have to look at each case singularly. We have to look at Pfizer’s model. All of us are interested in gene therapy and how we can treat patients. But the real concern is 20 to 30 percent of patients are just not eligible.”

Miller is referring to Pfizer making a series of investments to build its presence in gene therapy. While Novartis AG and Roche have recently opted to acquire gene-therapy developers outright, Pfizer has truck partnerships that grant it the rights to specific programs at smaller companies. Pfizer is developing a gene therapy for hemophilia B with Spark Therapeutics, which was recently acquired by Roche, and another for hemophilia A with California based Sangamo Therapeutics.

Albert Hwang, Managing Director, Healthcare, Investment Banking, Morgan Stanley said, “I view gene therapy as the most important topic as the industry matures. Since 2015, there have been 16 or 17 IPOs in gene therapy alone. Twenty-nine if you include oncology names.”

The panelists agreed the industry most focus on the supply chain of therapies and pricing reimbursement. The system is not designed or prepared to allow people to pay overtime. They were impressed with steps Novartis made after the FDA recently approved Zolgensma, a one- time treatment for spinal muscular atrophy, a muscle-wasting disease and leading genetic cause of infant mortality that affects one in every 11,000 births. Novartis said the treatment will cost $2.1 million, or $425,000 a year spread out over five years. The company is working with insurers to create 5-year agreements based on success of the treatment as well as other novel pay-over-time options.

Matthew Patterson is the Chairman and Chief Executive Officer of Audentes, a company he founded in 2012. He noted, “When I founded Audentes, I thought I understood biologics manufacturing. And then during preclinical development we had a setback and had to re-invent the wheel. I learned it was a little more complicated than I thought.”

Dr. Katherine High, President and Head of Research and Development at Spark Therapeutics said, “I’m optimistic that the solution is out there. We have to work together through trial and error to find a solution we all can agree on.”

The panelists acknowledged the industry should be commended for tremendous strides made in manufacturing, but said faster advances are being made on the clinical side. Dr. Peter Marks, Director, Centers for Biologics Evaluation and Research, US Food & Drug Administration said,” It’s an exciting new area now with a robust pipeline that will be coming to market for a whole range of products. The challenge will be addressing how fast these products are manufactured.”

Deborah Miller said the system is set up with the burden on families trying to figure out how they’ll get their kids into clinical trials. She

added “it will be a tragedy to see drugs approved and to see families within reach of them, yet they can’t quite get that drug.”