Major advances in treatment through gene therapy

Scientists had already proposed the concept of gene therapy in 1972, 31 years before the release of the complete sequence of the human genome at the end of the "Human Genome Project". The reason for this was that researchers saw a clear possibility that genetic diseases could be treated by altering DNA, but due to the limitations of the technology at that time, they could not make any further progress. 50 years later, today gene therapy has not only become a reality but also a rapidly developing field of medical science. Readers interested in the topic of gene therapy may find the following points interesting to know.

Gene therapy statistics

By mid-2022, more than 2,000 gene therapies were in the works worldwide, ranging from early-stage research to late-stage clinical testing. This number is double compared to three years ago. These scientific advances are coming in the form of economic benefits. The global gene therapy market is expected to grow from $5.33 billion in 2022 to $19.88 billion in 2027.

Creating new possibilities for new technologies

Sequencing the first human genome took 13 years and cost three billion dollars. Today, sequencing a human genome would cost just a few days and a little more than a hundred dollars. The ability to read DNA in less time and at less cost has made it possible to discover new ways to modify DNA. Over the past 10 years, the advent of next-generation genetic engineering tools (CRISPR, CAS9 hisse editing, Prime editing) along with the ability to target cells around the body has allowed gene therapy to Made safe, efficient and accessible.

New companies are coming into existence as a result of the invention of new devices. In 2021, 1,308 developers working on gene therapy and related technologies raised $22.7 billion in venture capital, a 57 percent increase from two years earlier.

Many diseases can be cured

New therapies are becoming possible with the invention of new gene therapy devices. These include cancer, neurological, blood, immunological and cardiovascular diseases, which are already changing people's lives. Significant improvement has been observed against many types of cancer during clinical trials.

Compared to conventional drugs, which require a person to take for life and provide temporary relief, the therapy will be a one-time step with long-term benefits and potentially disease-fighting benefits. Complete recovery will be achieved.

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Carefully review regulatory bodies

It is expected that more than 60 gene therapies will be approved in the US by 2030, and the US FDA is working rapidly to verify their safety and effectiveness.

Record prices for gene therapies

Leber predicts that the cost of gene therapies will skyrocket once they receive regulatory approval. Gene therapy for Congenital Amaurosis, a rare eye disease that causes blindness, will cost around eight and a half million dollars, while gene therapy for thalassemia (a rare blood disorder) will cost $2.8 million. The trend of high prices for gene therapies is not expected to break anytime soon. These high prices are due to manufacturing costs, limited number of relevant patients, investment in further research and development and other factors in the healthcare market.

This is why individuals and companies providing gene therapy services are coming up with unique payment methods, such as payment in installments over a period of time or payment based on treatment results.

High-income countries benefit

On the one hand, where the health system of the United States and the European Union is working on providing therapy facilities to the patients, the rest of the world is watching all this with curiosity. Low- and middle-income countries in particular lack access to gene therapy, even though these countries bear 90 percent of the world's disease burden.

As of August 2022, there were approximately 1,000 ongoing clinical trials of gene therapies, but less than five percent of these trials were in middle- and low-income countries (not including China) and only four were in Africa.

The need to narrow the gap

The US National Institutes of Health and the Bill and Melinda Gates Foundation, along with several private pharmaceutical companies, have singled out a cure for HIV and "speckle cell disease". Working on developing a shot therapy. In this regard, clinicians, scientists, advocates and community members have formed the Global Gene Therapy Initiative (GGTI), which plans to initiate clinical trials of gene therapy in low- and middle-income countries over the next few years.