Ralf Schmid – VJRegenMed

Challenges with translating gene therapies for neurodevelopmental disorders

admin_vjr_user — Fri, 31 Dec 2021 10:38:55 +0000

Ralf Schmid, PhD, MSCR, University of Pennsylvania, Philadelphia, PA, discusses the major challenges associated with the translation of gene therapies for neurodevelopmental disorders (NDDs) to the clinic. He explains the difficulty in translating therapies for NDDs from mouse models into non-human primate models and humans, given the increased brain size and complexity which support higher-level brain function. In addition, when attempting to use gene therapy approaches to restore lost gene expression seen in NDDs such as CDKL5-deficiency disorder (CDD), it can be difficult to model and predict which neurons need to be targeted in order to achieve lasting benefit in patients. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.

Non-viral gene delivery & gene editing: highlights from ESGCT

admin_vjr_user — Fri, 31 Dec 2021 10:38:53 +0000

Ralf Schmid, PhD, MSCR, University of Pennsylvania, Philadelphia, PA, describes some of his highlights from the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021, which included important basic and translational research in cell and gene therapies. Dr Schmid discusses advances seen in non-viral delivery strategies for gene therapies following lessons learned from the development of the COVID-19 vaccine. He also explains that much progress has been made in gene editing during the pandemic including the development of new strategies for improving safety and efficacy of gene editing, which hold promise for the treatment of brain disorders in the future. This interview took place at the ESGCT Virtual Congress 2021.

The gene-replacement therapy landscape for CDKL5-deficiency disorder

admin_vjr_user — Wed, 22 Dec 2021 18:29:34 +0000

Ralf Schmid, PhD, MSCR, University of Pennsylvania, Philadelphia, PA, discusses the current therapeutic landscape for CDKL5 deficiency disorder (CDD), a rare neurodevelopmental disorder that affects young children and is caused by loss-of-function mutations in the cyclin-dependent kinase-like 5 (CDKL5) gene. CDD is characterised by seizures and intellectual disability and current therapies are only able to ameliorate symptoms. Dr Schmid describes the rationale behind the investigation of potentially curative gene-replacement therapy approaches in order to restore CDKL5 protein expression and provides an overview of ongoing research in this area. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.

AAV9-mediated gene therapy for CDKL5-deficiency disorder

admin_vjr_user — Wed, 22 Dec 2021 18:29:31 +0000

Ralf Schmid, PhD, MSCR, University of Pennsylvania, Philadelphia, PA, describes ongoing research into the development of an adeno-associated virus (AAV) serotype 9-mediated gene replacement therapy to restore lost cyclin-dependent kinase-like 5 (CDKL5) protein expression seen in children with CDKL5 deficiency disorder (CDD). Administration through the cerebrospinal fluid (CSF) appears to be safer than system intravenous administration which requires higher dosages and therefore results in toxicity. He provides an overview of preclinical research in a CDD mouse model, as well as translational studies in non-human primates evaluating vector distribution and the gene expression profile of CDKL5 in the brain. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.