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Home » Rare diseases » Expanding the cell and gene therapy treatment range

Matthew Durdy

Chief Executive, CGT Catapult

Learn about the potential of cell and gene therapies with expert insights. Discover how treatments offer hope for both rare and more common conditions.

A leading expert highlights how the cell and gene therapy industry stands at a pivotal moment in advancing patient care. With treatments available for rarer conditions, Matthew Durdy emphasises the potential to offer hope for patients with more common conditions.

Revolutionising disease treatment with enduring effects

Outlining the potential of cell and gene therapies to treat rare and more prevalent conditions, Durdy says: “The beauty of cell and gene therapy is that it starts from a different premise from the therapeutics of the last 100 years, with the focus on an intent to significantly change, modify or cure a disease. Very often, it is a single application that has an enduring effect, potentially for a lifetime, and that is what makes it extremely exciting.”

As Chief Executive of the Cell and Gene Therapy Catapult (CGT Catapult), a research and technology organisation focused on the advancement of the cell and gene therapy industry, he says products have already been shown to work, with appropriate safety, and have navigated the regulatory process.

Expanding gene therapy treatment options

In theory, any monogenetic disease with an identifiable mutation could be addressed with gene therapy. Current treatments cover spinal muscular atrophy (SMA), haemophilia and liver metabolic disorders. Now, sickle cell anaemia, diabetes, cardiac disease, Parkinson’s and cases where previous therapies failed are under consideration for treatment.

“In the past, there would be few treatment options, but some people are now being given lifesaving gene-modified cell therapies,” Durdy adds.

Working in the rare disease space has been
fundamental to the development of
the cell and gene therapy industry.

Pioneering rare disease treatments

Initially, developers prioritised rare conditions due to their clear treatment targets and clear unmet need. This focus has led to insights that are also applicable to more prevalent conditions.

“Working in the rare disease space has been fundamental to the development of the cell and gene therapy industry,” says Durdy. It provides a rationale to tackle risk and expense and explores options for broader conditions. Economic incentives further support the development of such treatments.

Advancing gene therapies across the industry

CGT Catapult works across the advanced therapies industry, collaborating with organisations developing cell and gene treatments for rare and common conditions, assisting health systems in adapting to intensive therapies and providing training to ensure there is a skilled workforce.

Durdy believes the healthcare industry faces a once-in-a-lifetime opportunity in cell and gene therapy development. “It is potentially industry-changing and is well worth taking the risk. In fact, if we don’t make that investment, we are going to fall behind the rest of the world.”

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