Looking toward 2019, our secular view of health care remains consistent with prior years: The sector's growth will continue to be driven by changing demographics, globalization, and innovation across all industries. Long-term investment opportunities in the sector remain abundant, and one area we are most excited about is gene therapy—a new class of biotechnology drugs. Biotech is a diverse industry that faced a particularly challenging 2018. However, we're optimistic about the extraordinary innovation to be found here, particularly gene therapy and its long-term value potential for both patients and investors.
The human genome was sequenced 15 years ago, but gene therapy has only gained serious traction as a potential game changer for the treatment of disease over the past several years. In fact, we've seen a marked uptick in gene therapy clinical trials since 2016. Currently, there are over 7,000 rare genetic diseases already well understood and affecting more than 350 million people globally. While half of these diseases affect children, only about 400 diseases have approved therapies. And of these approved therapies, most don't represent a cure—they only ameliorate symptoms.
The opportunity remains vast well into the foreseeable future. The National Institutes of Health projects that at the current rate of US Food and Drug Administration (FDA) approvals, it would take over 10,000 years to cure all of these known diseases, and new genetic drivers of disease are identified every week. Right now, there are about 250 gene programs in various stages of clinical trial. Only one gene therapy has recently been approved, but the FDA expects to clear about 40 for patient use by 2020 (see chart below).
Targeted methods can treat the once untreatable
Along with an accelerated understanding of genetic drivers of disease, we now have a wealth of novel modalities to target genetic diseases, rendering previously "undruggable" targets vulnerable to modern medicine. These technologies include gene therapy itself (supplying a normal copy of a missing or an errant gene), and other forms of genetic editing that entail modifying the genome directly or outside of the body and placing it back into the patient.
The implications for these treatments are wide ranging, and we're already seeing promising data for applications in a number of serious diseases. For example, patients with hemophilia are unable to properly clot blood and can suffer life-threatening bleeding. There are multiple gene therapy trials in advanced development across the different forms of hemophilia. Early results show the promise to completely cure the disease and eradicate the need for blood transfusions.
Elsewhere, strong test results were recently reported for a gene therapy approach in boys with Duchenne muscular dystrophy, a rare genetic disease that causes the wasting away of muscles. In preliminary tests, patients were able to regain significant muscle control—in some cases even running around and riding scooters. Lastly, the FDA recently approved the first gene therapy for Leber's congenital amaurosis—a rare form of pediatric hereditary blindness. While gene therapy is still in its relative infancy, we expect the field to grow exponentially, as technologies and treatments continue to evolve and new genetic drivers of disease continue to be identified.
Beyond gene therapy, growth and innovation across the sector remain as exciting as ever. As extraordinary change reshapes the health care landscape, we hope to uncover those companies driving disruption and making health care a better experience for all its many stakeholders.
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