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Rare Disease & Regenerative Medicine 2019

This report provides industry specific statistics compiled from cell therapy, gene therapy, tissue engineering, and other regenerative medicine companies active in developing therapies for rare disease and provides context for the potential of regenerative medicine to improve upon the current standard of care.

As of the end of 2019:

There are 420 companies worldwide working on regenerative medicine therapies in rare disease.

There are currently 647 ongoing clinical trials utilizing regenerative medicines to treat rare disease.

There are an estimated 300 million patients suffering from rare diseases worldwide.

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A rare disease typically affects fewer than five in 10,000 people worldwide. Despite the rarity of each disease individually, when considered collectively, rare diseases affect a significant population – there are roughly 30 million people in the US and more than 300 million people worldwide living with a rare disease. There are nearly 7,000 different rare diseases currently identified, while only about 5% have an FDA-approved treatment. In addition, these diseases are often debilitating and potentially fatal; about 30% of children with a rare disease will die before their fifth birthday. Approximately 80% of rare diseases have identified genetic origins.

647+

Regenerative medicine clinical
trials in rare disease underway
worldwide by the end of 2019