With the eye disease known as achromatopsia, the vivid colors of daylight are overexposed, causing the world to go white. A needle in the eye may sound nightmarish, but for the thousands of people suffering from this form of blindness, injectable viruses repurposed to restore vision offer hope in the form of a cure.

Gene therapy applications and uses range from fighting cancer to curing genetic illnesses that previously meant a lifetime of invasive treatments. The future of gene therapy has never looked more promising.

Current gene therapy uses and developments

As of 2023, patients with leukemia, lymphoma, sickle cell disease, and other rare genetic disorders have shown remarkable improvement during clinical gene therapy trials. Geneticists are focused on curing cancer and neurological, immunological, cardiovascular, and blood diseases first, since these are the most life-threatening and debilitating conditions.

New technology, like ex vivo retroviral and lentiviral gene transfer to T cells, will likely make adaptive cell immunotherapy and other gene therapy uses possible. It’s already leading to breakthroughs in treatments for heritable conditions. The European Union has approved gene therapy for beta-thalassemia, for example.

So far, the U.S. Food and Drug Administration (FDA) has cleared 27 gene therapy and cellular products, with several other gene therapy applications under development—most of them in phase one or two of the clinical trial pipeline.

Having passed the approval process, the current gene therapies on the market still have sky-high costs. Luxturna, for example—a drug that treats an eye disease known as Leber congenital amaurosis—costs a staggering £355,025 ($429,399) per eye. Prices will likely fall as the technology used to create gene therapies becomes more accessible. In the meantime, these breakthrough treatments are limited to people undergoing clinical trials and to the wealthiest patients in the highest-income countries.

Most gene therapy drugs have a short shelf life and must be shipped at very low temperatures. As a result, supply chains and logistics providers have begun expanding their cryostorage capabilities, broadening their network of storage stations. This will make it easier to transfer medications across the globe.

Potential gene therapy applications

The FDA will likely approve over 60 gene therapies within the next seven years. Some gene therapy uses include treating cancer, heart disease, diabetes, cystic fibrosis, AIDS, and diabetes.

Gene-editing technologies, such as CRISPR-based editing, have the potential to precisely modify genomic sequences. Their success in treating beta-thalassemia and sickle cell disease suggests they could also treat similar illnesses. The U.S. National Institutes of Health (NIH), Novartis, and the Bill and Melinda Gates Foundation have teamed up to develop single-injection gene therapy treatments for sickle cell disease and HIV.

Although initial research focused almost solely on rare diseases caused by a single genetic defect, the future of gene therapy will also include research into more common disorders. For example, researchers are using CRISPR-Cas9 to modify wheat gluten, potentially letting people with celiac disease eat the protein without suffering an immune response.

Other gene therapy applications include treating neuromuscular problems. Geneticists are trying to develop new gene therapy and gene-editing techniques to address nervous system disorders that afflict children, such as ataxia telangiectasia and spinal muscular atrophy. Clinical trials that use base editing technology are ongoing.

The debate about the ethics of germline gene editing is still raging. However, somatic gene editing is much less controversial because it only affects the patient, not their potential future children.

The morals of editing embryological genes are also up for discussion—is it OK to treat a child’s severe illness before birth? Few people would argue against that, but what about going a step further and making the child smarter or taller? Governments will need to set policies about the future of gene therapy to address these unique issues.

Ongoing progress

The Global Gene Therapy Initiative plans to launch clinical trials for gene therapies in lower- and middle-income countries within the next few years. This program would help developing nations—which bear the highest disease burden—treat genetic disorders that could otherwise be a death sentence. Reducing medical costs will be paramount to expanding treatment access.

Experts predict gene therapy’s global value will increase to over £16.54 billion ($20bn) by 2027, up from £4.45 billion ($5.38bn) in 2022. As new technologies enter the market, so do new gene therapy companies. The increased competition will broadly benefit the medical field by driving down prices and stimulating innovation.

The future of gene therapy

New gene therapy uses will include treating cancer, diabetes, beta-thalassemia, cystic fibrosis, and other genetic diseases at their source. These gene therapy applications have the potential to radically transform the health care field and improve countless lives.

The treatments currently available to the public are expensive, but as the technology matures, prices will likely fall. The future of gene therapy will involve extraordinary medical breakthroughs. Someday, treating one’s blindness might be as easy as getting a shot.