Highlights from ESGCT 2021: gene therapy clinical data, advances in gene editing & AAV toxicity

admin_vjr_user — Fri, 24 Dec 2021 10:13:24 +0000

Paul Wuh-Liang Hwu, MD, PhD, National Taiwan University Hospital, Taipei, Taiwan, offers some of his highlights from the 2021 European Society of Gene & Cell Therapy (ESGCT) Congress. He discusses his excitement around promising clinical data presented at the meeting evaluating various gene therapy approaches in rare disorders such as mucolipidosis III, as well as advances in gene editing and the promise of base editing. He also considers the importance of talks on the safety issues associated with gene therapy, such as the integration of adeno-associated virus (AAV) vectors into the liver, resulting in toxicity. This interview took place at the ESGCT Virtual Congress 2021.

LYS-SAF302 AAV gene therapy for MPS IIIA

admin_vjr_user — Wed, 22 Dec 2021 18:29:28 +0000

Michael Hocquemiller, PhD, Lysogene, Paris, France, provides an overview of the ongoing Phase II/III AAVance (NCT03612869) study of LYS-SAF302 in patients with Mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo type A. LYS-SAF302 is an adeno-associated virus serotype rh.10 (AAVrh.10) vector carrying functional human N-sulfoglucosamine sulfohydrolase (SGSH) cDNA. This study aims to evaluate the efficacy in improving the neurodevelopmental state of MPS IIIA patients following one-time intracerebral administration of LYS-SAF302. Cerebrospinal fluid biomarker data obtained at one-year follow up supports the biological activity and therapeutic potential of LYS-SAF302. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.

Mucopolysaccharidosis – VJRegenMed

Highlights from ESGCT 2021: gene therapy clinical data, advances in gene editing & AAV toxicity

LYS-SAF302 AAV gene therapy for MPS IIIA