According to a story from Newswise, the genome editing company Intellia Therapeutics has recently announced that the first patient has been dosed in its phase 1 trial, which will test its investigational therapy NTLA-2001 as a treatment for hereditary transthyretin amyloidosis (hATTR) in patients with polyneuropathy. The company has further plans to investigate the treatment in patients with cardiomyopathy as well. Intellia is focused on developing potentially curative treatments utilizing CRISPR/Cas9 technology.
About HATTR Amyloidosis
HATTR Amyloidosis is a disease which is characterized by the build up of amyloid fibrils in body tissue. Amyloid fibrils are a type of abnormal protein. The disease is caused by a heritable genetic mutation. Symptoms of the illness include swelling, heart failure, irregular heartbeat, shortness of breath, fatigue, weight loss, easy bruising and bleeding, stroke, lung problems, enlarged liver, and changes in skin color. Treatment approaches for hATTR amyloidosis includes liver transplant; This can actually cure the disease, but the procedure carries substantial risks and side effects. This form of amyloidosis has a better prognosis than some of the more common types with some patients surviving for over a decade. It is estimated to affect around 50,000 people worldwide. To learn more about amyloidosis, click here.
With limited treatment options available for this illness aside from a risky liver transplant, there is an clear and present need for more effective therapies in hATTR amyloidosis. NTLA-2001 utilizes innovative CRISPR/Cas9 gene editing technology, which has the potential to herald a major breakthrough in the treatment of diseases with a known genetic component. It is the first therapy using CRISPR that is delivered in a systemic fashion. The goal of this treatment is to alter the RNA of the causative mutation to reduce the production of transthyretin.
About The Trial
The phase 1 trial consists of two parts and will enroll a total of 38 participants between age 18 and 80. This study is currently enrolling patients in the UK. The study will begin with a single escalating dose and then an expansion cohort once the ideal dosage level has been identified. Intellia hopes that NTLA-2001 could be a potentially curative treatment for hATTR amyloidosis.