Title: NICE Recommends Upstaza for Treating Rare Genetic Disorder
Introduction:
The National Institute for Health and Care Excellence (NICE), the UK’s healthcare treatment advisory body, has recommended the use of Upstaza (CRISPR/Cas9 gene editing therapy) for treating hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). This genetic disorder causes misfolding and deposition of a protein called transthyretin in various organs, leading to organ damage and, in some cases, organ failure. In this blog, we will explore the key points surrounding NICE’s recommendation of Upstaza and how it may benefit patients with hATTR amyloidosis.
Key Points:
- Understanding Hereditary Transthyretin-Mediated Amyloidosis:
Hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) is a rare genetic disorder that affects approximately 10,000 – 30,000 people worldwide. It is caused by a hereditary mutation in the transthyretin gene, leading to the production of abnormal transthyretin protein. The misfolded protein accumulates as amyloid fibrils in various organs such as the liver, heart, and nervous system, eventually causing organ damage and, in some cases, organ failure.
- Overview of Upstaza (CRISPR/Cas9 Gene Editing Therapy)
Upstaza (Patisiran) is a CRISPR/Cas9 gene editing therapy that involves using small molecules to alter the genetic information responsible for the abnormal production of the transthyretin protein. It is infused intravenously every three weeks and aims to slow or even halt disease progression or adverse outcomes from hATTR amyloidosis.
- NICE’s Recommendation of Upstaza:
The NICE has recommended Upstaza (Patisiran) for treating hATTR amyloidosis. The recommendation follows the success of clinical trials of the therapy, which demonstrated significant health benefits, including a reduction in the burden of the disease and the risk of potentially life-threatening complications. Despite the high cost of treatment, NICE believes that the benefits justify the cost, and the approval will enable patients across the UK to access this innovative therapy.
- The Potential Benefits for Patients:
NICE’s recommendation of Upstaza provides hope to individuals living with hATTR amyloidosis who previously had limited treatment options. Upstaza targets the underlying genetic mutation of hATTR amyloidosis and may slow or stop the progression of the disease, offering a promising treatment option that may improve patient outcomes and quality of life.
- Future Developments in Gene Therapy:
The recommendation of Upstaza by NICE highlights the potential of gene therapy and CRISPR/Cas9 technology in developing targeted treatments for rare genetic disorders. The growing body of research in gene editing therapies offers hope for treating inherited genetic diseases in a more precise and targeted way than current treatments.
Conclusion:
NICE’s recommendation of Upstaza (CRISPR/Cas9 gene editing therapy) for treating hereditary transthyretin-mediated amyloidosis raises hope for individuals living with this rare genetic disorder. The recommendation highlights the potential of gene editing therapies, which offer a more targeted and precise way of treating genetic diseases like hATTR amyloidosis. While the cost of treatment is high, NICE believes that the benefits of Upstaza justify its cost, enabling patients across the UK to access this innovative therapy, potentially leading to improved outcomes and quality of life. The future of gene editing therapies looks promising, and it may offer hope for individuals living with other rare genetic disorders.