Gene therapy PBGM01 was approved by EMA orphan drug for the treatment of GM1 gangliosidosis
基因疗法
z-bio
2021-02-10
339
Focusing on the development of single gene transformation therapy for rare central nervous system diseases, the European Commission (EC) has led the treatment of GM1 gangliosidosis in the company on the 26th. (GM1) The title of the gene therapy candidate PBGM01 orphan drug. PBGM01 is an adeno-associated virus vector (AAV) gene delivery therapy for the treatment of infant GM1. PBGM01 delivers the functional GLB1 gene encoding β-gal to the brain and surrounding tissues, thereby reducing the accumulation of GM1 gangliosides and reversing neurotoxicity, thereby protecting and developing the neurological function of GM1 patients, thereby increasing their potential and patient survival time . The drug has previously been awarded the title of "orphan drug" and "rare pediatric disease" by the US FDA for the treatment of GM1. GM1 is an inherited lysosomal disease caused by β-galactosidase deficiency, and its inheritance method is autosomal recessive inheritance. It is caused by a mutation in the GLB1 gene encoding lysosomal acid β-galactosidase (β-gal). By accumulating the toxic level of GM1 gangliosides in neurons throughout the brain, the reduced β-gal activity can lead to the rapid development of neurodegenerative diseases.
There is no age limit for the onset of GM1, but it is the most serious in the infant form. Baby GM1 accounts for about 62.5%, and is characterized by hypotonic and progressive central nervous system attacks within 6 months. Dysfunction and rapid degeneration. The life expectancy of GM1 babies is 2-4 years, and about 0.5-1 years of age is affected for every 100,000 live births. Currently, there are no approved therapies that can change the progression of the disease.
PassageBio plans to conduct a phase I/II clinical open-dose clinical study of PBGM01 for pediatric patients with GM1 from the end of 2020 to the beginning of 2021. The initial safety is 30 days, and I plan to protect it. Gender and biomarker data for the first half of 2021. The European Union's designation of orphan drugs is an important milestone for Passage Bio PBGM01. This will provide waiting patients with this long-awaited treatment, promote early drug development, and encourage researchers to make further efforts to alleviate patient suffering.
