For the treatment of rare diseases!

Recent popular reports from Yimaike ★NoticeShanghai Minhang Biopharmaceutical Industry Development Conference and 2021 Life and Health Industry Summit Forum is about to open in Shanghai ★Bluebird submitted to FDA the marketing application of β-thalassaemia gene therapy, how to evaluate the safety of viral vectorsmedicine Mai Meng broke the news, click on the picture, and sign up for the conference September 26, 2021/eMedClub News/--Recently, the gene therapy company Taysha Gene Therapies announced that the AAV9-based gene replacement therapy TSHA-102 has been awarded an orphan by the European Commission Drug title, used to treat Rett syndrome
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Rett syndrome is a serious inherited neurodevelopmental disorder caused by mutations in the methylated CpG binding protein 2 (MeCP2) gene on the X chromosome, which is essential for neuronal and synaptic function in the brain
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Rett syndrome mainly occurs in women and is one of the most common genetic causes of severe intellectual disability worldwide, with a prevalence of more than 25,000 in the United States and the European Union
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Early development of Rett patients is normal, and symptoms usually begin to appear when they are 6 to 18 months of age
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Rett syndrome is characterized by rapid developmental regression, leading to intellectual disability, language impairment, inability to use hands purposefully, inconvenience, seizures, heart damage, and breathing problems
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At present, Rett syndrome can be intervened to improve symptoms, but there is no effective cure
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TSHA-102 is a gene therapy delivered in the sheath.
It uses AAV9 capsid to deliver therapeutic genes.
By replacing mutant genes, the expression of silenced genes is enhanced or the expression of genes is reduced to achieve therapeutic effects
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Because of its unique ability to cross the blood-brain barrier, AAV9 has become an ideal carrier for CNS (monogenic central nervous system disease) gene therapy
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In addition, TSHA-102 directly delivers gene therapy drugs to the cerebrospinal fluid through intrathecal administration to promote CNS biodistribution and cell transduction
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This operation is usually carried out in an outpatient clinic, which is very convenient
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And compared with intravenous injection, intrathecal administration allows lower dose treatment
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The MECP2 gene provided by TSHA-102 also includes a novel miRNA response auto-regulatory element (miRARE) to regulate MECP2 expression during transcription
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The miRARE technology is designed to prevent the toxicity associated with overexpression of MECP2
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The positive preclinical data of TSHA-102 in Rett syndrome recently published in the journal “Brain” provides quantitative evidence that miRARE can display MECP2 in wild-type and knockout mouse brain cells.
Gene expression is used to regulate the role model of Rett syndrome
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RA Session II, Taysha’s founder and CEO, said: “Obtaining the European Commission’s orphan drug designation represents an important regulatory milestone.
It has the potential to accelerate the global clinical development of TSHA-102 and is a disease-changing potential.
Sex gene therapy
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"▲Taysha's founder and CEO RA Session II (picture source: Taysha's official website) TSHA-102 has previously been designated by the U.
S.
Food and Drug Administration (FDA) for rare pediatric disease and orphan drug designation
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It is expected that the IND/CTA application for TSHA-102 will be submitted in the second half of 2021, and the Phase 1/2 clinical trial will be launched at the end of 2021
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Taysha Gene Therapies is a patient-centric gene therapy company dedicated to the development and commercialization of AAV (Adeno-Associated Virus)-based gene therapies to treat rare and large patient populations of central nervous system monogenic diseases
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With the AAV9 vector as the core, the company has 18 gene therapy pipelines using intrathecal injection, covering three major categories of neurodegenerative diseases, neurodevelopmental disorders and brain development-related recurrent epilepsy, and a total of 18 central nervous system diseases
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▲Taysha Gene Therapies pipeline (picture source: Taysha official website) Reference materials: 1.
https:// -for-tsha-102-for-the-treatment-of-rett-syndrome/