For years, cell and gene therapies were mainly associated with rare and orphan diseases , those that impact small patient groups and lack effective treatment options. However, recent developments in this space, including a high-profile acquisition and key regulatory changes, point to a paradigm shift. Cell and gene therapy is becoming increasingly commercially viable, with the potential to improve the lives of millions.

As these therapies reach larger populations and offer the promise of living longer and better , new market opportunities will likely emerge.

Aiming for widespread impact

In June 2025, Eli Lilly announced that it was acquiring Verve Therapeutics for $1 billion . Verve develops single-dose gene editing therapies designed to treat chronic diseases. The company’s leadi

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