Dive Insight:

Sickle cell affects millions of people. In the United States, it’s the most common inherited blood disorder. And yet, until 2017, there hadn’t been a new drug to treat it in 20 years.

Two additional medicines, Adakveo from Novartis and Oxbryta from Global Blood Therapeutics, gained approval in late 2019. Still, the relatively few treatment options for a such a widespread and devastating disease has fueled criticism of the health disparities and racism that patients of color — and, in particular, Black patients — face all too often.

“Even with today’s best available care, [the disease] continues to drive premature deaths and disability,” Novartis said in a statement Wednesday.

The Gates Foundation, one of the world’s largest private foundations, with over $51 billion in total assets, has set its sights on sickle cell and another health crisis disproportionately affecting people of color, HIV. Since 2018, the foundation has funded multiple efforts to discover gene-based cures for these diseases that can be delivered via a single shot.

Prior to its agreement with the foundation, Novartis had already been working on a cell therapy for sickle cell anemia with partner Intellia Therapeutics, a gene-editing company.

That therapy, called ADPT03, is what’s known as an ex vivo treatment. Cells are extracted from the patient, sent to a lab where they’re engineered using CRISPR to fix the genetic problems, and then shipped back and reinfused. Other companies, including Bluebird bio, are using a similar approach for their own sickle cell gene therapies.

On Tuesday, however, Bluebird reported two participants in studies of its therapy, which uses a different type of gene engineering to modify cells, later developed blood cancer, a major safety setback that will likely raise concerns and scrutiny around ex vivo gene therapy.

While the ex vivo process holds some advantages that make it an attractive choice for diseases like sickle cell, it is complicated and requires a certain amount of lab, manufacturing and hospital infrastructure that can be hard to find in many parts of the world. Novartis noted that “such procedures often do not exist in the areas where [sickle cell disease] is most prevalent, excluding the vast majority of those with the disease from these life-changing gene therapies.”

Now, with the backing of the Gates Foundation, Novartis will have a team dedicated to developing a gene therapy that can be infused directly into patients and, hopefully, overcome some of the barriers to access.

“Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings,” Trevor Mundel, president of global health at the Gates Foundation, said in the Wednesday statement. “What’s exciting about this project is that it brings ambitious science to bear on that challenge.”

Novartis said that, with this new work, a main focus of the early drug design strategy will be addressing access and distribution challenges in low- and middle-income countries where healthcare infrastructure is limited. The funding agreement also includes “specific provisions to support global access to any resulting innovations,” according to the company.