Expert review on novel gene therapies of haemophilia A
ASC Therapeutics has announced the peer-reviewed, open-access publication of “Hemophilia A Gene Therapy: Current and Next-Generation Approaches” in the journal Expert Opinion in Biological Therapy.
This publication provides an update of scientific and clinical advances in gene replacement therapies for haemophilia A with focus on:
- Current and next-generation approaches to achieve a safe, durable, and stable transgene expression while avoiding the challenges of factor VIII replacement therapies
- A thorough review of current and past clinical studies with gene therapies conducted in patients with haemophilia A
- Addressing the critical role of the viral construct towards decreasing the therapeutic dose and minimising cellular stress, induction of the unfolded protein response, and the resulting loss of protein production in liver cells
- Second-generation gene therapies incorporating chimaeric DNA sequences in the transgene can increase clotting factor synthesis and secretion, and advance the efficacy, safety and durability of gene replacement therapy for haemophilia A as well as other blood clotting disorders.
Professor Steven W Pipe said “This comprehensive review summarises several decades of work of my team and others demonstrating the critical role of cellular stress and the unfolded protein response in the impaired production of clotting factors in the liver. The development of a novel bioengineered construct that has been proven in pre-clinical studies to improve biosynthesis, protein folding, and secretion of factor VIII leads me to predict that in a clinical setting it will reduce therapeutic dosing and increase durability.”
Dr Oscar G Segurado, Chief Medical Officer at ASC Therapeutics, added “We are thrilled that the journal Expert Opinion in Biological Therapy has published this seminal review to inform the medical and scientific community of these exciting developments with gene therapies in haemophilia A. This review describes step-by-step the critical elements required for a durable, safe and effective biosynthesis and production of a clotting factor in liver cells. We strongly believe that learning about the underlying molecular, cellular and protein biology of clotting factor production will advance our competitive edge in the field of liver-targeted gene therapies.”
Click here to read the article.