Gene therapy

Aug 22, 2025

Vertex Pharmaceuticals and CRISPR Therapeutics have become the first companies to secure FDA approval for a CRISPR-based gene-editing therapy. Vertex Pharmaceuticals, in partnership with CRISPR Therapeutics, has achieved a historic milestone with the FDA approval of exagamglogene autotemcel (exa-cel, branded as ...

Feb 07, 2025

Acute Myeloid Leukemia (AML) is a fast-moving and aggressive cancer that demands timely and effective treatment. With the current acute myeloid leukemia therapies showing limited success for many patients, the focus has shifted to breakthrough AML drugs that are redefining the treatment landscape. From innovative s...

Dec 27, 2024

Sickle cell disease is a hereditary blood disorder marked by abnormal hemoglobin, which leads to the deformation of red blood cells into a sickle shape. It is a major global health issue, especially common in individuals of African, Middle Eastern, Mediterranean, and South Asian descent. Based on DelveInsight's ...

Nov 18, 2024

Parkinson’s disease is the second most common neurodegenerative disorder after Alzheimer’s disease. It affects about 10 million people worldwide. The average age of onset for Parkinson’s disease is around 60; however, approximately 10-15% of patients experience early-onset PD before the age of 50. In 2023, there...

Oct 10, 2024

Over the past decades, there has been an eruption in understanding treatment strategies of gliomas with the development of clinical immunotherapy, providing exceptional success in employing checkpoint inhibitors and cancer vaccines as treatment options that have given significant momentum to the growth of the GBM m...

Aug 23, 2024

Neuromuscular disorders (NMD) encompass a broad spectrum of conditions impacting the peripheral nervous system, which comprises all motor and sensory nerves linking the brain and spinal cord to the body. These disorders comprise various classes of diseases, such as muscular dystrophies, myopathies, motor neuron dis...

Aug 23, 2024

Duchenne Muscular Dystrophy (DMD) is a rare disorder, but it is one of the most common genetic conditions, affecting roughly 1 in every 3,500 male births worldwide. Gene therapy for Duchenne Muscular Dystrophy (DMD) is poised to revolutionize the treatment landscape by addressing the underlying genetic cause of the...

Jul 26, 2024

Gene therapy is becoming a promising solution for retinal degenerative diseases, as the retina offers an excellent setting for studying and treating eye conditions. Importantly, it was the first tissue to receive approved gene therapy for genetic disorders in the United States. To date, only one retinitis pigmentos...

Mar 04, 2024

Gene therapy is becoming a promising solution for retinal degenerative illnesses, particularly because the retina offers an excellent avenue for studying and treating eye-related issues. Importantly, it is the first tissue in the United States to receive approval for gene therapy in cases of inherited disorders. ...

Dec 15, 2023

History has been created as the world captures the significance of the FDA’s approval of Casgevy (exa-cel), a collaboration between Vertex Pharmaceuticals and CRISPR Therapeutics, for the treatment of sickle cell disease (SCD). This groundbreaking therapy represents a long-awaited potential cure for the debilitatin...