Across the entire sector of for-profit companies building gene therapy medicines, a sobering process is playing out. Even when they create a treatment that works, companies price it exorbitantly (around $2 million to $3 million per patient), arguing that a one-time cure saves the health care system years of costly supportive care.
For diseases with fewer than 100 patients, such prices are still not enough for these efforts to make commercial sense, as demonstrated by a biotechnology company, bluebird bio. After 20 years of engineering such gene therapies and significant success in the clinic, bluebird bio now struggles to remain solvent — even as it priced its most recent approved medicine at $3 million. Another gene-editing biotechnology company recently halted clinical trials for a rare disease with 300 patients in the United States, saying it couldn’t make the economic case to continue the experiments. For a disease that affects one person, the current for-profit system thus makes building a gene therapy or a gene editing cure a daunting challenge.
To make CRISPR cures a reality, the biomedical community needs to start with regulation. For treatments developed for genetic diseases that affect tens of thousands of people (or, say, if a company tries to take on heart disease, which affects millions), the Food and Drug Administration has a well-established, yearslong review process. But the F.D.A. needs to consider a new regulatory process that could create a more streamlined path for bringing much-needed CRISPR medicine tailored to patients with a one-of-a-kind genetic typo. There is precedent for this: Starting in the late 1990s, the F.D.A. facilitated regulatory pathways for innovation of a then-new class of genomic medicines for cancer — CAR-T therapy — which is now widely used clinically. The same can be done for CRISPR.
Streamlining regulation won’t be enough, though. Where will the funding for developing cures for single patients come from? Biotechnology companies are unlikely to voluntarily take this on, given the financial cost, though the for-profit sector could make a significant contribution by sharing technologies and resources that would accelerate this effort. Tapping into federal and state funding could provide a path forward. Recently, the F.D.A. greenlighted a clinical trial collaboration between experts at U.C.S.F. and U.C.L.A. and my colleagues at U.C. Berkeley’s Innovative Genomics Institute, for a gene editing approach to sickle cell disease. My sense of pride in this achievement is diluted by the realization that ours is the only such all-academic trial in the entirety of the gene editing space. To truly realize the potential of this technology, there should be dozens of such efforts underway.
This approach poses a difficult but essential question: Why should the average taxpayer contribute to building medicines for rare diseases? Would the money be better spent on finding treatments for common ailments?
Investing public funding in CRISPR cures for rare diseases not only will help us treat people with uncommon mutations (a global community numbering hundreds of millions of people) but also can provide insights that can be infused into CRISPR clinical innovation for common diseases.
But for the next few years, devastating genetic ailments and cancer are where CRISPR clinical trials must remain; ethical considerations over the safety of patients being exposed to new technology dictate that. Today’s tools are also the cognate of the first iPod — at the time, an exhilarating advance but still low tech compared with present-day smartphones. Everything we learn about how to gene-edit people from this work, coupled with continued CRISPR innovation in the academic and for-profit sector, will provide a foundation for more deeply understanding how to safely edit DNA to treat and potentially prevent dire common diseases.
Fyodor Urnov
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