What are some notable successes of gene therapy in treating diseases?

Gene therapy has shown remarkable progress in treating various genetic diseases over the past few years.  Its applications in treatment of the genetic disorders are extended beyond traditional therapies. These techniques are being explored as treatment of many genetic disorders including Duchenne muscular dystrophy, cystic fibrosis, Alzheimer’s disease, Parkinson’s disease, and Huntington disease.

Here are some notable successes that offer hope and innovative treatments for patients:

Blood Disorders

Sickle Cell Disease and Beta Thalassemia

Recent breakthroughs have been made in treating these inherited blood disorders:

• The FDA approved Casgevy and Lyfgenia in 2023 for sickle cell disease patients 12 years and older
• Casgevy uses CRISPR/Cas9 gene editing to increase fetal hemoglobin production, preventing red blood cell sickling
• Lyfgenia uses a lentiviral vector to produce a modified hemoglobin that reduces sickling risk
• Early clinical trials of CRISPR-based therapies have shown promising results, with patients experiencing improved hemoglobin levels and reduced symptoms

Cancer Immunotherapy

Chimeric Antigen Receptor (CAR) T-cell therapy has emerged as a groundbreaking treatment for certain cancers:

• In 2017, Kymriah (tisagenlecleucel) became the first FDA-approved CAR-T cell therapy for treating B-cell acute lymphoblastic leukemia (B-ALL)
• Clinical trials showed an 82% overall remission rate in pediatric and young adult patients with relapsed or refractory B-ALL
• Several CAR-T cell therapies targeting CD19 or BCMA have since been approved for various B-cell leukemias, lymphomas, and multiple myeloma

Inherited Eye Disorders

Luxturna became the first FDA-approved in vivo gene therapy for a rare inherited eye disorder.  It has shown success in improving vision in patients with certain genetic mutations causing retinal dystrophy.

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Spinal Muscular Atrophy (SMA)

While not a traditional gene therapy, Spinraza, an antisense oligonucleotide therapy, has shown significant benefits for SMA patients:

• It has improved motor function in many patients, allowing those with severe cases to breathe, swallow, and sit upright independently
• Over 10,000 people worldwide have been treated with Spinraza since 2016

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Other Successes

Gene therapy has also shown promise in treating:

• Other inherited immune deficiencies like Wiskott-Aldrich syndrome and chronic granulomatous disease
• Metabolic disorders such as metachromatic leukodystrophy and mucopolysaccharidosis

These successes demonstrate the growing potential of gene therapy to provide long-lasting or even curative treatments for a range of genetic and acquired disorders.