Understanding gene therapies

Gene therapy is a category of biological products approved by the Food and Drug Administration (FDA) that seeks to modify or manipulate the expression of a gene or to genetically alter the biological properties of living cells to treat or potentially cure a disease or condition.

For example, one of the first gene modification therapies on the market was Luxturna®, which treats Leber congenital amaurosis (LCA). LCA is a rare, inherited eye disease that can lead to severe vision loss or blindness. LCA is caused by a mutation of a retinal gene that shares responsibility for providing instructions to form various proteins involved in healthy vision. Luxturna, which is administered through a medical procedure one eye at a time, modifies the defective gene so the body can begin producing the necessary proteins. This prevents or drastically slows the rate of vision loss.

Gene therapy can be used to treat conditions within a wide range of therapy classes. There are a growing number of FDA-approved gene therapies on the market today, including:

Zolgensma® improves life expectancy for spinal muscular atrophy (SMA) in infants, the leading genetic cause of infant mortality.

Roctavian® was the first gene therapy for the treatment of hemophilia A, a rare genetic bleeding disorder that primarily affects males.

Lenmeldy®, one of the most expensive gene therapies on the market, treats children with the rare genetic disease metachromatic leukodystrophy (MLD).

Casegevy® and Lyfgenia® were developed to treat sickle cell disease, a group of inherited blood disorders that affects approximately 100,000 people in the U.S

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The average cost of developing a successful drug is $2.6 billion. For gene therapies, the average cost of research and development soars to $5 billion, which largely gets passed on to patients and their insurance providers.

Gene therapy differs from other targeted medicine as its treatment is intended to provide long-term benefit by altering the specific genetic defect. To justify clinical cost and risk, conditions appropriate for gene therapy usually have high rates of mortality or progressive co-morbidities, such as permanent organ damage and severe disability.

These conditions may also require complex and expensive medical treatment and other interventions. While gene therapies cannot reverse existing organ damage or disabilities, they can prevent the need for long-term adherence to complex maintenance medications, improving the patient’s quality of life.

With such high risks and rewards associated with these conditions, gene therapies can carry prices from $850,000 to $4.25 million or more per treatment.

Orphan status gene therapy drugs are used to treat extremely rare diseases. For example, AADC deficiency affects how a person’s cells send signals to each other. People born with this condition do not make enough aromatic l-amino acid decarboxylase (AADC), an enzyme that is critical to brain and nervous system function. AADC affects just 1 in 11 million.

Another example is X-linked adrenoleukodystrophy, which affects the nervous system and adrenal glands, leading to progressive loss of the protective fatty tissue that surrounds nerves in the brain and spinal cord. The incidence is 1 in 318,000 male births.