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July 16, 2025
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MyAmyloidosisTeam

NTLA-2001 (Nexigan) represents an exciting advancement in amyloidosis treatment using CRISPR gene-editing technology. This is one of the first human clinical trials using CRISPR technology for treating hATTR amyloidosis.

The treatment works by targeting and deleting the TTR gene in the liver, which is the root cause of Show Full Answer

NTLA-2001 (Nexigan) represents an exciting advancement in amyloidosis treatment using CRISPR gene-editing technology. This is one of the first human clinical trials using CRISPR technology for treating hATTR amyloidosis.

The treatment works by targeting and deleting the TTR gene in the liver, which is the root cause of amyloidosis. What makes this approach particularly promising is its potential to be curative rather than just managing symptoms. Since this is a novel treatment approach using cutting-edge technology, it's important to note that research is still ongoing to fully understand its long-term effectiveness and safety profile. It's recommended to discuss participation in clinical trials with your healthcare team to understand if this could be an appropriate option for your specific situation.

July 16, 2025
A MyAmyloidosisTeam Member

GregLs Some studies show a little improvement in cardiac symptoms but nothing that is definitive.

July 21, 2025 (edited)
A MyAmyloidosisTeam Member

Yeah Sheldon after reading your info a couple of days ago I subscribed to Intellia. Hopefully long-term benefits are possible for everyone. To your knowledge, because I recall the research is done in your area in PA, so you obviously have your "ear to the pavement " so to speak, depending on our stages of the disease, with this treatment does it potentially halt your symptoms or would/could they subside?

July 20, 2025
A MyAmyloidosisTeam Member

Nex-Z is a one time gene editing trestment that "silences" (stops production) most of the liver's production of TTR. It is from Intellia Therapeutics. It has resulted in a 90% reduction in TTR. That was sustained at 1 year out and even 2 years out from the one time infusion.

"About Nex-z:
Based on Nobel Prize-winning CRISPR/Cas9 gene editing technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein".

July 19, 2025
A MyAmyloidosisTeam Member

No but glad for an update too our illnesses!!!!Good too know, I'll keep a look out for it here in the US.

God bless you and family in New Zealand 🙏

GStrong

July 16, 2025

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