Clinical gene technology in Australia: building on solid foundations

Article Cell & Tissue Engineering

Establishing a robust chimeric antigen receptor T-cell therapy program in Australia: the Royal Prince Alfred Hospital experience

Zlatibor M. Velickovic et al.

Summary: CAR T-cell therapy is a novel cancer treatment that has shown remarkable efficacy in treating patients with relapsed leukemia and lymphoma. Implementing this therapy in a hospital setting requires a multi-disciplinary approach, dedicated funding, and clinical expertise.

CYTOTHERAPY (2022)

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Article Medicine, General & Internal

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

M. C. Ozelo et al.

Summary: The study evaluated the efficacy and safety of valoctocogene roxaparvovec in men with severe hemophilia A, showing an increase in factor VIII activity level, reduced bleeding rates, and factor VIII concentrate use.

NEW ENGLAND JOURNAL OF MEDICINE (2022)

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Review Medicine, General & Internal

20 Years of Legislation-How Australia Has Responded to the Challenge of Regulating Genetically Modified Organisms in the Clinic

Gabrielle M. O'Sullivan et al.

Summary: Since 2001, gene technology in Australia has been regulated under a legislated national Gene Technology Regulatory Scheme, which aims to assess the potential risks of gene technology to human health and the environment. This scheme works alongside the Australian Therapeutic Goods Administration to ensure the safety and efficacy of therapeutic products. Recent reforms to the scheme aim to facilitate the safe development and delivery of gene-based human therapeutics in Australia, serving as a model for other jurisdictions.

FRONTIERS IN MEDICINE (2022)

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Editorial Material Biotechnology & Applied Microbiology

Fourth Boy Dies in Clinical Trial of Astellas' AT132

Alex Philippidis

HUMAN GENE THERAPY (2021)

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Article Medicine, General & Internal

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

Julian D. Gillmore et al.

Summary: The gene-editing therapeutic agent NTLA-2001 effectively reduced serum TTR protein concentrations in patients with ATTR amyloidosis, showing potential safety and pharmacodynamic effects in clinical studies.

NEW ENGLAND JOURNAL OF MEDICINE (2021)

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Review Hematology

Hemophilia Gene Therapy: Approaching the First Licensed Product

Paul Batty et al.

Summary: The clinical potential of hemophilia gene therapy has been pursued for the past 30 years, with expectations of achieving this goal in the near future. While recent late phase clinical trials have shown promising results, there are still issues regarding efficacy and safety that need further attention. Overcoming these challenges is crucial for the widespread application of this treatment paradigm.

HEMASPHERE (2021)

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Article Biotechnology & Applied Microbiology