300 million people battle a disease without an approved treatment

In fact, 1 in 10 of us will develop a condition with no treatments, while many of these diseases could be treated with drugs sitting on the local pharmacy’s shelf.

The Problem

Today, less than 13% of the 22K recognized diseases in the world have FDA-approved treatments. About half of the diseases without cures are considered rare, meaning they impact fewer than 65 per 100K patients people worldwide, a category that includes Huntington’s disease, sickle cell disease and many forms of cancer.

While the combined magnitude of rare diseases is enormous, impacting 300-400M people globally, over 95% of rare diseases have no treatment and they receive a fraction of R&D dollars, just 14% of NIH funding in 2023. The severity is devastating: 50-80% of people with rare diseases are children, 30% of whom die before age 5. And then there are undertreated diseases — diseases with expensive, burdensome or inaccessible treatments that need better cures. For example, ALS has two approved treatments, but neither substantially extends lives.

But many existing drugs could be repurposed to treat these diseases. Inflammatory diseases, infectious diseases, cancers and other illnesses all share common mechanisms in the body (e.g., related proteins and genes responsible for the disease). Drugs known to treat one disease by targeting a specific mechanism could work on other diseases with similar mechanisms.

The gap between needed cures and existing drugs is driven by three market dynamics:

Most drugs that could be repurposed to save lives aren’t being studied, because it isn’t profitable.

No one is responsible for unlocking the full potential of every drug

Low-income and marginalized populations receive significantly less medical research investment overall, exacerbating these dynamics

Learn How We Plan to Solve this Problem

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