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Gene Therapy 2.0: No Longer Science Fiction

Guest Author
June 4, 2019

Back in 1990, an experimental new treatment represented a bright light of hope for Ashanthi De Silva, who was just four at the time and became the first human to receive gene therapy. She was born with a crippled immune system due to inheriting two faulty copies of the gene responsible for manufacturing adenosine deaminase (ADA). By participating in the first human gene-therapy trial, she received new, working copies of the same gene.

“We’ve come a long way since the first gene therapy was announced in the 1990s and there were concerns about altering people’s fates,” said Ryan Cross, assistant editor of Chemical & Engineering, during the session, Gene Therapy 2.0: No Longer Science Fiction. “In the 2010s, a more positive outlook for gene therapies emerged, and today, many recognize that gene therapies might provide a one-and-done cure for some rare genetic diseases,” he added.


With tremendous progress on multiple fronts, the gene therapy race is only intensifying. In 2017, Spark Therapeutics Inc.’s FDA approval for Luxturna became the first gene therapy approved in the U.S. that targets an inherited disease caused by mutations of a specific gene. Then, just this past May, the FDA approved gene therapy Zolgensma, which alters the underlying genetic cause of spinal muscular atrophy, a rare disease that causes progressive muscle wasting and kills many babies before the age of two. The FDA anticipates more than 200 gene therapy Investigational New Drugs (INDs) per year by 2020.


“We are really, really early on with all of this, but the good news is that we have ample evidence that gene therapies can be safe and effective,” said Eric David, CEO of Aspa Therapeutics. During the session, panelists discussed topics including safety of gene therapies, overcoming obstacles like skyrocketing costs and science fiction-like concepts of using gene therapy to treat diseases in the womb.


One point was clear: To bring transformative gene therapies to patients faster, greater participation in clinical trials and a push to bring the cost of goods down and produce at scale should be at the forefront.


“Biologic products will always be more expensive, but the cost will come down—not in the next year or two, but the cost will come down, and it will come down fairly dramatically,” David said.


It’s certainly tricky with rare diseases. In cases where there may be, say, only 1,000 patients in the world to treat, companies may feel they need to charge more for a product. Then there’s efficacy and other unknowns to take into account. “We have companies saying, yes, we will admit to the payer that we don’t know what the efficacy will be, how many years it will last, so why don’t we look at pricing models that take that into account,” David said.


In terms of the timeline for sorting out pricing challenges, that’s still to be determined, but it certainly looks promising. “If there’s a means, there’s a way, and people are trying to get quite creative with business models,” said Irene Rombel, senior director, head of strategic analysis external innovation, discovery, product development and supply for Janssen Research & Development, Pharmaceutical Companies of Johnson and Johnson.


Beyond the obvious hurdles, gene therapy innovation is very quickly accelerating. “There are a number of really exciting companies spinning out of academia and doing some really elegant engineering to improve potency,” Rombel said.


“There will be bumps along the road, but this technology and this part of the industry is the most creative I have ever seen biotechnology to be,” David said.