NIH, Gates Foundation invest $200M to develop gene therapies for developing countries

The partnership will have a particular focus on therapies to treat sickle cell disease and HIV, with each party committing $100 million to the effort.

Although touted as having potential to cure some of the most intractable of human illnesses, gene therapies also carry some eye-popping price tags that have threatened to limit their availability to wealthy countries. However, a new public-private partnership aims to make them available to people around the world, including in developing countries.

The National Institutes and Health and the Bill & Melinda Gates Foundation plan to each invest $100 million in an effort to develop affordable gene therapies to treat sickle cell disease and HIV and make them available around the world, including in low-resource settings. The goal is to advance product candidates into the clinic in 10 years.

“This unprecedented collaboration focuses from the get-go on access, scalability and affordability of advanced gene-based strategies for sickle cell disease and HIV to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries,” NIH Director Francis Collins said in a statement, adding that the plan was to “go big or go home.”

The U.S. Food and Drug Administration and European Medicines Agency have approved three gene therapies between them in the last two years. In December 2017, the FDA approved Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl), for biallelic RPE65 mutation-associated retinal dystrophy, which has a list price of $850,000, followed by EMA approval in November 2018. Most recently, in May, the FDA approved Novartis’ Zolgensma (onasemnogene abeparvovec-xioi) for spinal muscular atrophy, which carries a list price of $2.1 million. On Tuesday, Novartis reported in its third-quarter earnings that Zolgensma had sales of $160 million.

And in June, bluebird bio secured conditional EMA approval for Zynteglo (autologous CD34-positive cells encoding beta-A-T87Q-globin gene) for the blood disorder beta-thalassemia, pricing it at about $1.78 million. In addition to Zynteglo for beta-thalassemia, for which the company has also sought FDA approval, bluebird’s gene therapy LentiGlobin is in development for sickle cell disease.

Although they carry high list prices, those prices do not necessarily reflect what payers end up paying, let alone what patients pay out of pocket. Nevertheless, those prices have still been deemed cost-effective in the context of Western healthcare systems because they represent one-time treatments rather than treatments taken over the patient’s lifespan, with costs adding up over time. However, that is not the case for developing countries.

“In recent years, gene-based treatments have been groundbreaking for rare genetic disorders and infectious diseases,” Bill & Melinda Gates Foundation Global Health Program President Trevor Mundel said in a statement. “While these treatments are exciting, people in low- and middle-income countries do not have access to these breakthroughs.”

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