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    Home > Medical News > Medical World News > Gene therapy upstart plans to raise 125 million US dollars through IPO

    Gene therapy upstart plans to raise 125 million US dollars through IPO

    • Last Update: 2020-02-04
    • Source: Internet
    • Author: User
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    Editor's note: in the past few years, adeno-associated virus (AAV) has made a lot of progress in the treatment of nervous system diseases (CNS) With the further study of the pathogenesis of single gene genetic CNS, the treatment of this kind of disease with AAV vector also appears Today, passage bio, a gene therapy company, announced plans to raise $125 million in an IPO Last year, the company completed two rounds of financing of $225 million to develop its treatment drugs for rare single gene central nervous system diseases It is worth mentioning that Professor James Wilson, co-founder and chief scientific adviser of passage bio, is one of the global leaders of gene therapy and director of the gene therapy program at the University of Pennsylvania In gene therapy for CNS, most companies use adeno-associated virus 9 (AAV9) as virus vector According to Dr Wilson's test results in non-human primates, passage bio selects the most suitable AAV vector and route of Administration for its candidate drugs, so as to reduce the cytotoxicity, improve the efficiency of transduction, and reduce the neutralizing antibody rate Photo source: passage bio official website Among the candidate drug combinations of passage bio, there are three leading gene therapies for GM1 ganglioside disease (GM1), frontotemporal dementia (FTD), and Krabbe disease GM1 is an autosomal recessive genetic disease, which is caused by the inactivation mutation of lysosomal enzyme β - galactosidase (glb1), which is necessary for the degradation of GM1 ganglioside and keratin sulfate Infantile GM1 is the most common and serious form of the disease, which usually has 4-month-old gait abnormality and 6-month-old developmental degeneration Currently, there are no approved treatments to alleviate the disease FTD refers to the dementia syndrome of middle-aged and old patients with personality change, speech disorder and behavior abnormality These symptoms are related to the characteristic patterns of neurodegeneration that affect the frontal and temporal cortex In 5% - 10% of FTD patients, the pathogenic loss of function mutation can be identified in the gene encoding the precursor of granular protein, a ubiquitous lysosomal protein The patients generally showed progressive course of disease and survived for 8 years on average Krabbe disease is a rare and life-threatening lysosomal storage disease, which occurs in the early stage of patients' life and is caused by the mutation of GalC gene Early childhood Krabbe disease usually occurs before six months of age The symptoms of these patients include apnea, peripheral neuropathy, severe weakness, unresponsiveness to stimulation, epilepsy, etc At present, the company has three other preclinical development plans to treat patients with heterochromic leukodystrophy (MLD), amyotrophic lateral sclerosis (ALS) and Charcot Marie Tooth disease 1A subtype (cmt2a) with mutations in the ARSA gene, respectively In addition, passage bio can also exercise the option of six other rare single gene CNS indications to the University of Pennsylvania before the expiration of the 2022 cooperation agreement reference material: [1] Early stage biotech Passage Bio files for a $125 million IPO,Retrieved February 03,2020,from https:// [2] Passage Bio official webside,Retrieved February 03,2020,from https://
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