In a recent study, researchers from the University of Auckland, Amsterdam University Medical Center, and Cambridge University Hospitals revealed a revolutionary gene-editing therapy that is changing the lives of hereditary angioedema patients in New Zealand, the Netherlands, and the UK (via Medical Xpress).
Here's How Gene-Editing Treatment Changed the Lives of Hereditary Disorder Patients Across 3 Countries
The Groundbreaking Treatment
Led by principal investigator Dr. Hilary Longhurst, the team introduced a cutting-edge gene-editing therapy, NTLA-2002, to over ten patients suffering from hereditary angioedema.
This rare genetic disorder causes severe, unpredictable swelling attacks, often proving fatal and severely impacting daily life.
What sets NTLA-2002 apart is its use of CRISPR/Cas9 technology to target the KLKB1 gene, which is responsible for producing plasma prekallikrein.
The therapy reduces total plasma kallikrein levels by editing this gene, effectively preventing angioedema attacks. Promisingly, interim results from the Phase I study indicate a potential permanent cure for patients.
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The Treatment's Safety and Effectiveness
One of the most encouraging aspects of the study is the lack of serious or long-term side effects from the single-dose infusion given over two to four hours.
All patients showed reductions of up to 95% in total plasma kallikrein protein and angioedema attacks, indicating that the therapy was effective.
Dr. Danny Cohn from the Amsterdam University Medical Center expressed optimism, stating, "We've never been closer to the ultimate treatment goal of normalizing hereditary angioedema patients' lives and offering total control of the disease."
We learn from the report that patients from the initial study will be followed up for 15 years to assess long-term safety and efficacy. A larger Phase II trial is already underway, with a Phase III trial slated to commence in the second half of 2024. The progress is a beacon of hope for the estimated one in 50,000 individuals globally living with hereditary angioedema.
What's Next?
Beyond the profound impact on hereditary angioedema, the therapy's potential is significant. Dr. Padmalal Gurugama from Cambridge University Hospitals highlighted its potential to significantly improve patients' lives, emphasizing that the disorder often leads to misdiagnosis and unnecessary treatments.
NTLA-2002 stands out among CRISPR therapies. While the only approved therapy, CASGEVY, is an ex vivo treatment, NTLA-2002 operates in vivo, editing the targeted gene directly within the body.
The implications extend beyond hereditary angioedema, with CRISPR technologies showing promise in treating various genetic diseases, cardiovascular issues, cancer, and autoimmune diseases.
Importantly, NTLA-2002 only affects the patient and does not pass on to their children. This ensures that the disorder is not inherited, offering families affected by hereditary angioedema a new ray of hope.
With the potential for a single treatment to provide lifelong relief, researchers are ushering in a new era in healthcare, with CRISPR technologies leading the way in rewriting the stories of people affected by genetic disorders.
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