Did you know that globally, around 320 million people live with a rare genetic disease? In fact, an estimated 4,000 medical conditions are a result of gene disorders.

Consequently, over recent years, gene therapy and its immense potential have grown to be one of the most active areas of research in Life Sciences. So much so, the majority of the world’s biggest pharmaceutical companies have invested in cell and gene therapies as well as heaps of new BioTechs opening their doors to find new treatment possibilities from breakthroughs in gene editing science.

As today is Jeans For Genes Day, we thought what better time to spotlight this extraordinary area of research and development we’re fortunate to work so closely alongside...

So, what is gene therapy?

In each and every one of us, all our genes carry DNA material that’s needed to make proteins that are essentially the building blocks of the human body. They contain regulatory instructions, so our bodies know when to use those particular genes – clever, right?!

Yet sometimes, there can be a glitch in the system – either one inherited from our parents or one that arises over time after our genes become damaged through mutation, which, unfortunately, can lead to disease.

Traditional treatment methods tend to manage diseases, usually requiring a lifetime of injections, infusions, monitoring, adjustments, and ongoing doctor’s appointments. To combat the burden of ongoing treatments and daily disease management, gene therapy aims to prevent and cure genetic disorders by targeting the root cause.

Well, how does it work exactly?

By examining the source of genetic disorders, scientists have begun looking at our genes to treat or even prevent diseases – who would’ve thought?

Generally, gene therapy takes on a few different forms, such as replacing a damaged gene with a healthy copy of it, inactivating a gene that is malfunctioning, or introducing a new gene to the body that might be needed for regular function.

For example, to correct a disease in which a specific enzyme is missing, adding the necessary gene component to produce the absent enzyme would fix the disease’s underlying problem.

In other scenarios, harmless viruses serve as ‘packets’ that carry a new gene to wherever it is needed in our bodies.

What kinds of illnesses can gene therapy treat?

To be honest, in the grand scientific scheme of things, gene therapies are pretty new on the scene. The very first gene therapy – Kymriah, or CTL019, developed by Novartis, that targets acute lymphoblastic leukemia – was approved just a few years ago, in 2017. Since then, the FDA has authorised other kinds of gene therapies to treat diseases including multiple myeloma, melanoma, CALD (cerebral adrenoleukodystrophy), lymphoma, beta-thalassemia, spinal muscular atrophy, and leber congenital amaurosis.

Of course, due to the nature of the treatments and the fact they bear a significant amount of risk, the time it takes for them to be approved and come to market is not as instantaneous as other treatments and therapies within Life Sciences. In fact, in 2021, the FDA approved over 50 drugs, but only two of them were gene therapies. Still, that small number meant that approved gene therapies grew by 10%.

Looking at gene therapies in 2022, Bluebird, who developed Zynteglo to remedy beta-thalassemia and Skysona to treat CALD, currently have another kind of gene therapy under review by the FDA for a rare brain disorder. Similarly, BioMarin, Pfizer, Sarepta, Solid Bio, and UniQure all have potential gene therapies that could seek approval in the next year.

So, as trials continue and scientists uncover more breakthroughs, experts predict that gene therapy holds promise to treat a range of diseases including cancer, cystic fibrosis, heart disease, diabetes, hemophilia, and AIDS.

So, what does investment look like for gene therapy today?

As more gene therapies secure FDA approval and investors note the vast potential of this type of treatment, capital flowing into this research area is growing at a remarkable rate. Cell & Gene’s 2022 Investment Outlook notes the exceptional levels of investment in the space, from $362 million in 2020 to nearly $68 billion in 2021 – roughly a third of all private investment made into Life Sciences!

But that’s not all. According to Evaluate Pharma, between 2021 and 2026, cell and gene therapies are forecast to grow at a CAGR of 63%. For comparison, conventional drug sales are expected to grow at a CAGR of 6%, and biologics sales have an estimated CAGR of 5% for that same time period.


Of course, like with all areas of research, gene therapy is no stranger to stumbling blocks and setbacks as specialists in the field continue to venture into uncharted territory. Still, impressive prospects, an increasing number of clinical catalysts, and improved regulatory approvals are what continue to drive investment and innovation in the space.

There’s no doubt in our minds that cell and gene therapy will grow to become its own defined sector within the Life Sciences ecosystem, and we couldn’t be more excited to see how the space transforms in the next couple of decades – similarly to how biotech went on to dominate!

If you’re hiring or looking for a new role within cell and gene therapy, contact one of our experts today to see how we can support you – after all, that’s what we’re known for!