Disease Treatments

Advancements in Gene & Cell Therapies

Researchers have developed new technologies, assessed advances in different animal models, and tested new gene and cell therapies in many human clinical trials of different diseases. Some exciting improvements have been observed in several areas. Each trial has advanced our understanding of the complex interactions between different tissues and highlighted challenges for further research. Some of the most notable advancements in gene and cell therapies are discussed below.

Treatments by Disease

Inherited Retinal Diseases

On December 19, 2017, the FDA approved Voretigene Neparvovec (Luxturna, Spark Therapeutics), an innovative one-time gene therapy treatment used to improve vision in patients with established genetic vision loss due to Leber congenital amaurosis or retinitis pigmentosa, both inherited retinal disease

Cancer and Immunotherapies

In 1891, the American surgeon William Coley undertook the first systematic study of immunotherapy for cancer, using first live bacteria, then heat-killed bacterial toxins to stimulate the immune system to attack and resolve bone cancers.

Cardiovascular Diseases

Cardiovascular diseases (malfunctioning of the heart and the blood vessels) are on the rise in the developing world, mainly due to sedentary behaviors, diet choices, longer lifespans and the unique stresses that arise from modern lifestyles.

Infectious Diseases

The impact of infectious disease on human health is immense. Historically, antimicrobial drugs and vaccines have transformed this medicine in this field and continue to do so. 

Lysosomal Storage Diseases

Lysosomal storage diseases (LSDs) are a family of more than 40 distinct diseases resulting from enzymatic deficiencies leading to accumulation of unwanted substances in lysosomes of various cell types.

Inherited Immunodeficiencies

Immunodeficiency is a defect in the immune system that prevents immune resistance to infectious disease and some forms of cancer.

Musculoskeletal Diseases

There are dozens of monogenic disorders of skeletal muscle which cause progressive disability and frequently premature death.

Neurodegenerative and Movement Disorders

Neurodegenerative diseases encompass a spectrum of disorders, which are characterized by progressive degeneration of neurons - electrically excitable cells that transmit information. 

Neurosensory Disorders

Neurosensory disorders such as vision and hearing loss are debilitating disorders that can dramatically affect quality of life, often with few treatment options available for patients.

Red Blood Cell Disorders

Red blood cells (RBCs, also called erythrocytes) are the most common cell type in the blood, with the primary duty of carrying oxygen from the lungs to the body’s tissues using the iron-containing molecule hemoglobin, which also gives blood its red color.

Respiratory Diseases

Alpha1 anti-trypsin deficiency is genetic disease associated with mutations within the SERPINA1 gene which presents with symptoms of emphysema, chronic obstructive pulmonary disease (COPD), and occasionally chronic liver disease. 

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