A Donegal man who took part in an Amyloidosis treatment trial has welcomed positive developments announced this week.
James Green from Letterkenny is one of just three patients in the world to take part in Phase 1 of a genome editing therapy trial.
James, who works as an accountant, was diagnosed with Hereditary Amyloidosis four years ago. It is estimated that 1% of the Donegal population carries a strand of this condition known as ‘Donegal Amy’.
Soon after his diagnosis, James decided to enrol in a clinical-stage trial in the UK targeting the treatment of the rare and progressive disease. (Featured photo: James Green in London during his second Crispr infusion in May 2023)
Leading medical company Intellia Therapeutics has this week announced the successful results of the dose-escalation therapy conducted on three patients administered with two doses of NTLA-2001 over two years.
The NTLA-2001 treatment is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein and causes serious complications in the heart, nerves, digestive system, and other tissue of ATTR amyloidosis patients.
First-ever clinical data demonstrates redosing with a CRISPR-based medicine isn’t always necessary, but it is possible. According to Intellia CEO John Leonard, M.D, this represents an exciting advancement in the pursuit of treatment of other diseases where patients might need more than one dose to reach the desired therapeutic effect.
These data from the Phase 1 study of NTLA-2001 were presented at the Peripheral Nerve Society Annual Meeting this week in Montreal, Canada.
James’ trial results have shown a 95% median reduction in serum TTR in his body. Delighted with the outcome, he said: “It’s fantastic news for patients with hereditary and wild type amyloidosis as both conditions are caused by the breakdown of the transthyretin protein, TTR, produced in liver.
“The trial drug NTLA 2001 edits the DNA code to achieve a deep reduction in the level of transthyretin circulating in the bloodstream. The phase 1 clinical trial has already been expanded in to a Phase 3 magnitude trial with patients now being enrolled in the United States.
“The NTLA 2001 trial drug alongside other new therapies in development for amyloid removal have the potential to be a cure for transthyretin amyloidosis. I am clinically well and don’t require any ongoing treatment. The freedom that gives is immeasurable.”
James on a trek to Everest Base Camp last October
While James himself does not need re-dosing, Intellia says the ability to re-dose is a key advantage of its non-viral, lipid nanoparticle (LNP)-based delivery platform.
Development and commercialisation of NTLA-2001 is led by Intellia as part of a multi-target collaboration with Regeneron.