German researchers welcome national, EU support for gene therapy research

National and EU-level support goes a long way towards supporting research into gene therapies against rare diseases, according to German researchers, but Europe still lags behind North American countries in innovation.

An estimated four million people in Germany suffer from rare diseases – a term used to refer to a group of more than 6,000 different diseases, with each one affecting less than five in 10,000 people.

The symptoms and course of different rare diseases vary widely, and due to the small number of patients and often complex clinical picture, many patients find themselves without an available cure.

But as most rare diseases are genetic, gene therapies are increasingly viewed as a potential silver lining for those affected.

“For rare genetic diseases, we often have the situation that there are none or almost no therapy options available – and this is where gene therapies could become a game changer,” Sarah Hedtrich, professor at Charité’s Berlin Institute of Health, told Euractiv.

Gene therapies are innovative treatments that address the genetic cause of a disease by replacing the malfunctioning gene, inactivating it, or introducing a new or modified gene into the body to help fight the disease.

“This way, you can not only effectively treat a patient, but potentially also heal them, because by re-introducing the healthy gene sequence, the cause of the disease is eliminated”, explained Hedtrich, whose current research focuses on treatments for serious rare diseases affecting patients’ skin or lungs.

Research funding on offer

Between the public funding available from different levels, Hedtrich feels there is generally sufficient support for academic research into gene therapies.

“All in all, I think that, in Germany, we cannot complain about the available support, also because the EU, for instance, offers funding opportunities for projects in rare diseases,” she said.

The German government funds “research into diagnostic and therapeutic measures for rare diseases,” according to the Health Ministry.

Since 2003, the Research Ministry also supports research networks on rare diseases with a total funding of up to €141 million earmarked for 2003 to 2025, Meanwhile, research on gene therapies can also receive support from other funding pools.

EU needs to step up to unleash gene therapies potential, stakeholders say

While gene therapies have the potential to transform the approach to rare diseases and improve the quality of life for patients, stakeholders and patients complain that they are very expensive to develop and difficult to access.

The EU has also addressed rare diseases via various pieces of legislation, including pharmaceutical legislation, which is currently being revised by co-legislators based on the Commission’s proposal or a directive on non-communicable diseases.

Once it comes to finding investors and getting a newly developed therapy onto the market, things can get a bit more complicated. Typically, developing a treatment for rare diseases can be expensive, while the business case is limited due to the small number of patients.

“I do think this is a problem,” Hedtrich said. “But it has been detected, and there is already a lot of support and funding.”

According to the researcher, one example is the so-called orphan drug designation, through which the EU grants special property rights for treatments against rare diseases, also known as orphan drugs, thus making their development more profitable.

More targeted, timely support

However, according to Hedtrich, more targeted government initiatives for gene therapies would help move the issue forward.

“In Germany, one is often in this ‘wait and see’ position, waiting to see what happens first, and thus clear, targeted programmes for gene therapies are missing,” she said.

In the researcher’s view, this results from a more hesitant approach displayed by many European countries when it comes to new scientific developments such as gene editing, leading to Europe lagging behind countries like the US or Canada.

“I think Germany risks falling behind,” she warned.

So far, new developments on gene therapies against rare diseases “mostly come from America,” Holger Lerche, medical director at Tübingen University Hospital, also told BR earlier this year, adding the challenge is now to bring the new therapies “from blueprints to the patient.”

However, Hedtrich is optimistic that this development will proceed swiftly.

“I do think that, over the next five to ten years, we will see many developments – there is such an enthusiasm and drive at the moment,” she said. “In ten years, we will be able to treat many diseases completely differently and better than we currently do – of this, I am sure.”

[Edited by Giedrė Peseckytė/Alice Taylor]

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