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Recent popular reports from Yimaike ★ Nanjing · Big coffee gathered in May2021 nucleic acid drug development forum announced schedule ★ May Nanjing2021 nucleic acid drug development forum is about to open April 26, 2021 / Yimaike news eMedClub News/--Recently, Apic Bio announced that the US FDA has approved the new drug research application (IND) for its main gene therapy candidate product APB-102 for the development of SOD1 type amyotrophic lateral sclerosis (ALS).
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by muscle weakness and ultimately paralysis caused by the loss of motor neurons.
Most patients died of respiratory failure within 5 years of onset.
The disease can be caused by a variety of gene mutations, sporadic or familial.
About 10% of patients with ALS have known driver genes, and about 20% of these patients are related to mutations in the SOD1 gene that encodes superoxide dismutase.
The currently approved ALS therapy can only delay the progression of the disease, but cannot solve the underlying genetic factors of the disease.
APB-102 is a gene therapy that uses AAV as a carrier to deliver anti-SOD1 microRNA to target cells.
The microRNA can bind to SOD1 mRNA, thereby reducing the production of pathogenic proteins, and is designed to slow or reverse the progress of SOD1 ALS.
Preclinical conceptual studies have proved that APB-102 can inhibit the activity of mutant SOD1 gene.
The 2021 nucleic acid drug development forum will kick off in Nanjing from May 7th to 8th, 2021, to welcome the "new era of super drugs".
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by muscle weakness and ultimately paralysis caused by the loss of motor neurons.
Most patients died of respiratory failure within 5 years of onset.
The disease can be caused by a variety of gene mutations, sporadic or familial.
About 10% of patients with ALS have known driver genes, and about 20% of these patients are related to mutations in the SOD1 gene that encodes superoxide dismutase.
The currently approved ALS therapy can only delay the progression of the disease, but cannot solve the underlying genetic factors of the disease.
APB-102 is a gene therapy that uses AAV as a carrier to deliver anti-SOD1 microRNA to target cells.
The microRNA can bind to SOD1 mRNA, thereby reducing the production of pathogenic proteins, and is designed to slow or reverse the progress of SOD1 ALS.
Preclinical conceptual studies have proved that APB-102 can inhibit the activity of mutant SOD1 gene.
The 2021 nucleic acid drug development forum will kick off in Nanjing from May 7th to 8th, 2021, to welcome the "new era of super drugs".
