Gene therapy

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...

Nov 14, 2023

Takeda’s ADZYNMA Approved by FDA as the First and Only Recombinant ADAMTS13 Enzyme Replacement Therapy for the Treatment of Congenital Thrombotic Thrombocytopenic Purpura Takeda has received FDA approval for ADZYNMA (ADAMTS13, recombinant-krhn) for both prophylactic and on-demand treatment in adults and pediatri...

Jul 04, 2023

FDA Approves First Gene Therapy for Severe Hemophilia A BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that the US Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the tr...

Jun 09, 2023

The cell and gene therapy market has seen a revolutionary transition in recent years, with advancements in scientific research and novel methods of treatment driving a rise in development activities. This has resulted in an increase in the number of cell and gene therapy choices available to patients suffering from...

May 16, 2023

Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy Sarepta Therapeutics, Inc., a pioneer in precision genetic medicine for rare diseases, announced that the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 in favor of...