Taysha Gene Therapies Announces First Pediatric Patient Dosed with TSHA-102

Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercialising AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that the first paediatric patient has been dosed with TSHA-102 in the REVEAL Phase 1/2 paediatric trial…

Australia led the world charge of the Anavex2-73 Rett study

As most would know, Australia led the world charge of the Anavex2-73 Rett study here are some accounts of lived experiences from Patient stories currently on compassionate use of drug in Australia. Patient stories now on compassionate use in Australia   Annelise's Story      Click Here   Jess's Story   …

Gene therapy explained.

  Introduction. The worldwide Rett community is excited as two biotechnology companies have recently either started- or announced plans to start- gene therapy clinical trials.  However, gene therapy has different meanings including gene editing (by editing DNA or RNA by CRISPR technology), gene addition (by delivery of genes that encode,…

FDA approval for the Neurogene gene therapy strategy.

            Preclinical studies leading to FDA approval for the Neurogene gene therapy strategy. Introduction and background. Members of the Rett community were excited to learn that Neurogene received approval in January 2023 from the US Food and Drug Administration (FDA) to perform a Phase 1/2…

Letter to the Rett Syndrome Community: Neurogene’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology

Dear Rett Syndrome Community, Earlier this year Neurogene shared that the FDA cleared our Investigational New Drug (IND) application for NGN-401 gene therapy for the treatment of female children with Rett syndrome. NGN-401 is an investigational adenoassociated virus (AAV) gene therapy, using Neurogene’s Expression Attenuation via Construct Tuning (EXACT) gene…

Progress towards a cure for Rett Syndrome by RNA editing.

Introduction. I have previously written articles which appear on the RSAA website that describe strategies designed to cure Rett Syndrome; these strategies, listed below, are designed to result in the expression of functional MeCP2 protein in brain cells of individuals with Rett Syndrome. Gene therapy DNA editing RNA modification and…

Purified MeCP2: An Alternative Potential Cure For Rett Syndrome.

Recognised strategies to cure Rett Syndrome include gene therapy using adeno-associated virus (AAV) to deliver a functional MeCP2 gene, gene correction by diverse strategies which correct the mutation in the gene and X-chromosome reactivation, possible because cells in females with Rett Syndrome contain a functional copy of the MeCP2 gene…

Gene Therapy for Rett syndrome : ASGCT abstract reports

The annual meeting of the American Society for Gene and Cell Therapy (ASGCT) was held recently.  I read the abstracts related to gene therapy for Rett Syndrome as such abstracts often act as pointers to the direction in which current research is heading and have presented a summary in this…
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