6 specific solutions to the challenges of gene therapies… including cost reduction and local manufacturing

The Dubai Future Foundation revealed that recent years have witnessed a boom in the field of gene therapy, with the approval of more than 20 gene therapies, in addition to hundreds of clinical trials to treat rare genetic diseases, such as monogenic diseases, cancers, blood diseases, immune and inflammatory disorders, and even some neurological and cardiac disorders.

She confirmed in a recently issued report entitled “Global Solutions for a Shared Future” in a special edition of her knowledge report “Future Opportunities: 50 Global Opportunities,” which was reviewed by Emirates Today, that gene therapy and gene editing are not just medical techniques, but rather represent a scientific revolution that redraws the features of modern medicine, which represents a ray of hope for thousands of families burdened by genetic diseases with the health and psychological challenges they create, pointing out that the use of gene therapies to treat more diseases is expected to expand in the future. Noting that some gene therapies cost more than $2.1 million per dose, making them inaccessible to a large number of families and health systems.

Ways of confrontation

The Foundation has presented six solutions that are considered among the most prominent ways to confront the challenges of gene therapies and ensure that the aspirations of millions of families around the world are met and give them real hope for recovery. They include: “Making technological progress to reduce costs, innovating new financial tools that include flexible pricing policies, subscription options, and innovative financing tools that reduce the burden on families and health systems, and broad global cooperation that ensures everyone benefits from these treatments, supporting and diversifying their distribution methods, ensuring that they are equitably available to everyone, building local manufacturing capabilities, and simplifying procedures for approving genetic treatments.”

In detail, the Dubai Independent Foundation proposed a future scenario in which it is possible to reach an international agreement on human DNA, and the world agrees to a charter for genetic editing, entitled “What if the world agrees to a charter for genetic editing?” And the possibility of reaching an international agreement on human DNA, stressing that gene therapy represents a ray of hope for thousands of families burdened by genetic diseases with the health and psychological challenges they cause, and that most of these diseases still have no definitive treatment, and even the available treatments require strict adherence to medical or nutritional systems based on the results of genetic tests, as is the case with phenylketonuria, which is “a rare genetic disorder that causes brain damage, delayed growth, and behavioral problems,” or citrullinemia. It is a “genetic disorder that affects the ability of the liver,” and patients remain vulnerable to serious complications that may threaten their lives once treatment plans are violated.

She emphasized that gene therapy is an exceptional opportunity, as it not only alleviates symptoms, but also targets the roots of the problem by repairing or replacing the defective gene, noting that recent years have witnessed a boom in this field, with the approval of more than 20 gene therapies, in addition to hundreds of clinical trials to treat rare genetic diseases, such as monogenic diseases, cancers, blood diseases, immune and inflammatory disorders, and even some neurological and cardiac disorders.

The most prominent treatments

She explained that among the most prominent gene therapies are the drug “Spinraza” and the drug “Zolgensma” to treat spinal muscular atrophy, which is a very serious disease that weakens muscle strength. Thanks to them, many patients have regained motor abilities that were previously thought to be irretrievable, in addition to the drug “Zynteglo” intended for patients with beta thalassemia, which is a disease resulting from a genetic defect that affects the composition of blood hemoglobin, and in which the patient depends on blood transfusions. It allowed patients to do without periodic blood transfusions.

The treatments also include the drug “Lenmeldy”, approved by the US Food and Drug Administration to treat one of the most prominent rare genetic disorders, which is known scientifically as “metachromatic leukodystrophy,” which causes the accumulation of fatty substances in the nervous system, which leads to gradual damage to the brain and spinal cord and may end in early death. “T-cell therapies” (CAR-T), which reprogram the immune system, have proven unprecedented effectiveness in confronting types of cancer that have eluded other treatments.

She added that despite this progress, gene therapies face major obstacles, and one of the biggest of these global challenges is the high cost that makes them inaccessible to many families and health care systems. Other challenges are related to unequal access, the complexity of supply chains, and the need for shared safety data.

Blood disorders

Dubai Future stated that it is expected that the use of genetic therapies to treat more diseases will expand in the future, especially hereditary blood disorders and metabolic diseases, and in order for these treatments to reach more and more patients, their cost must be reduced, their distribution methods must be supported and diversified, and their availability to everyone is ensured in a fair manner, noting that this will only be achieved through close cooperation between governments, researchers, doctors, the industrial sector and patient associations, in order to build local manufacturing capabilities, simplify procedures for approving treatments, and strengthen partnerships between… Public and private sectors.

She emphasized that the high cost is one of the most prominent of these challenges, as some treatments, such as “Zolgensma,” cost more than $2.1 million per dose, which makes them unavailable to a large number of families and health systems, in addition to technical obstacles, as researchers seek to make gene therapies safer and more precise in targeting cells, pointing out that early clinical trials conducted between 2010 and 2020 showed challenges related to the immune response to these treatments, and in order to overcome these. Challenges: Technological progress must be made to reduce costs, and new financial tools must be invented, including flexible pricing policies, subscription options, and innovative financing tools that reduce the burden on families and health systems, with the need for broad global cooperation to ensure that everyone benefits from these treatments.

She added that gene therapy and gene editing are not just medical techniques, but rather represent a scientific revolution that is reshaping the features of modern medicine, and with continued scientific progress and global commitment to the principle of justice, this field may be able to meet the aspirations of millions of families around the world, and give them real hope for recovery.

Regarding the expected time frame for achieving this opportunity, Dubai Future explained that establishing a global genetic charter and developing a common framework for the use of gene therapies requires efforts that extend across multiple levels, starting from individuals and research institutions, to regional and international frameworks, all the way to the international level. Given the complexity of this task and the depth of its ethical and regulatory implications, this work is likely to extend over decades, with gradual progress accompanied by increasing international consensus on standards and governing frameworks.

“Dubai for the Future”:

• Some treatments cost $2.1 million per dose, threatening equitable access.