Gene Therapies' Moment Of Reckoning

(MENAFN- Swissinfo) Gene therapies are a revolution in medicine but over the past few years, companies developing them for many serious hereditary diseases have faced various setbacks. What will it take for gene therapies to fulfill their promise? This content was published on April 4, 2025 - 09:00 10 minutes

I report on the Swiss pharmaceutical industry and healthcare topics such as access to medicine, biomedical innovation, and the impact of diseases like cancer. I grew up just outside San Francisco and studied international affairs with a focus on development economics and healthcare policy. Prior to joining SWI swissinfo in 2018, I was a freelance journalist and a researcher on business and human rights.

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Five years ago, gene therapy companies were flush with money. InvestmentsExternal link in cell and gene therapy topped $19.9 billion (CHF17.6 billion) globally in 2020, double the $9.8 billion raised from public offerings, venture capital and other financing in 2019.

Today, the industry looks different. Safety concerns, sceptical patients, hefty price tags and a host of manufacturing challenges have humbled the field. In 2022 and 2023 investments dropped below $13 billion, improving slightly in 2024 after some positive approvals and an uptick in clinical trials in Europe.

In the face of a cash crunch, some small gene therapy makers, once investor darlings, have slashed workforcesExternal link , collapsed, or abandoned markets entirely. This has prompted media reports of a crisisExternal link in the gene therapy industry.

“When one drug does well, people think that we've already won, that we've figured it out,” said Rodolphe Renac, an expert in healthcare innovation and president of the US affiliate of consulting company Alcimed.“Despite remarkable outcomes from some gene therapies, there are still improvements needed.”

Swiss companies like Roche and Novartis continue to invest in the field, and scientists remain optimistic about gene therapies' promise to cure diseases. But the tough reality of getting them to patients has led to mounting calls for changes to the way gene therapies are priced, paid for, and communicated to the public.

Cell and gene therapies are often grouped together because they are both treatments that aim to treat, prevent, or even cure diseases by modifying processes at the cellular or genetic level. Cell therapy introduces cells (that may be genetically altered) into a patient's body to restore function or fight disease.

In gene therapy, a functional gene is introduced or a faulty gene is corrected to restore normal cellular function. For gene addition or replacement, many therapies use a viral vector to transport genetic material into cells. Crispr-Cas9 is a technology used for gene editing.

Capturing the real costs

Gene therapies are widely considered a paradigm shift in medicine. By modifying or replacing genes or altering gene expression, often with a single infusion, gene therapies offer the prospect of not just treating but actually curing deadly hereditary diseases.

One of the biggest hurdles facing gene therapies, though, is how to price and pay for one-time“cures”. In January, the gene therapy Hemgenix sold by US firm CSL Behring for haemophilia B became the most expensive single therapy reimbursed by health insurers in Switzerland at CHF2.7 million ($3 million).

Most gene therapies on the market are for rare diseases – those that affect a small portion of the population (in some countries 1 in 2,000 people). Companies argue that the high price tags, often in the millions per patient, are needed to make up for the small volumes.

As more gene therapies are launched, payers, including both private insurers and national health authorities, are becoming more concerned about their ability to pay for them. Some payers, particularly in Europe and Latin America, have pushed backExternal link on the price tags, in some cases denying coverage entirely or negotiating the price downwards.

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