Kathrin Meyer – VJRegenMed

Neuraminidase could improve AAV9 gene therapy for Batten disease

admin_vjr_user — Thu, 12 Aug 2021 10:07:41 +0000

Kathrin Meyer, PhD, Nationwide Children’s Hospital, Columbus, OH, discusses the administration of neuraminidase prior to or in combination with AAV9 gene therapy in Batten disease, a neurological condition that can lead to macular degeneration. In a mouse model, intravitreal administration of neuraminidase resulted in enhanced delivery of AVV9 into the eye by removing certain sugar residues on entry receptors, improving penetration of the virus into deeper structures of the eye. While additional research is required to assess safety in larger models, neuraminidase pretreatment holds potential in a range of neurological conditions affecting the eye, particularly when targeting the inner nuclear layer. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

Challenges in developing gene therapies for rare diseases & CNS disorders

admin_vjr_user — Thu, 24 Jun 2021 17:15:50 +0000

Kathrin Meyer, PhD, Nationwide Children’s Hospital, Columbus, OH, explains how the small number of patients suffering from ultra rare diseases and challenges in obtaining funding makes it difficult for companies to justify the high costs associated with the development, manufacture and market approval of gene therapies. Furthermore, many rare diseases lack natural history data, rendering clinical trial design difficult, as both patient variability and disease evolution must be considered. The development of therapies for rare diseases affecting the central nervous system (CNS) can be challenging due to the size of the CNS and protection by the blood-brain barrier, making it difficult for homogenous drug action to be achieved across the system. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.

Strategies for advancing gene therapy for CNS disorders

admin_vjr_user — Thu, 24 Jun 2021 17:15:49 +0000

Kathrin Meyer, PhD, Nationwide Children’s Hospital, Columbus, OH, discusses ongoing research into enhancing gene therapies for central nervous system (CNS) disorders, such as through improvement of vector capsid efficiency to reduce the dosage required. Improved methods for therapeutic delivery are also being investigated including ultrasound-guided delivery systems, novel vector formulations and alternative transplantation sites, such as the cisterna magna and intraparenchymal injection. In addition, mathematical modelling and biophysics are being applied to enhance the systemic distribution of cerebrospinal fluid (CSF) delivered therapeutics to prevent hotspot formation and ensure complete coverage of the brain. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.