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Real members of MyAmyloidosisTeam have posted questions and answers that support our community guidelines, and should not be taken as medical advice. Looking for the latest medically reviewed content by doctors and experts? Visit our resource section.
A MyAmyloidosisTeam Member asked a question 💭
December 24, 2024
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A MyAmyloidosisTeam Member

Intellia Therapeutics is the pharmaceutical company behind NTLA-2001. It is also known as Nex-z . CRISPR is an acronym: Clustered Regularly Interspaced Short Palindromic Repeats. It is just not FDA approved. Clinical studies and trials have not been completed. I am quite familiar with CRISPR.

July 18, 2025
A MyAmyloidosisTeam Member

Sheldon hope you're having a good day.

Curious if you're familiar with Magnitude NTLA-2001 now from Nexigan CRISPR a gene editing therapy for HTTR-CM? Supposed to be in its infancy stages of being developed

Regards
GStrong

July 17, 2025
A MyAmyloidosisTeam Member

My clinical trial cardiologist's Nurse Coordinator at University of Pennsylvania told me that the Alexion drug may not be available until 3-4 years from now. The drug is in Phase 3 so the complete data will not be submitted for Fast Track Review by the FDA until the trial is completed in 2-3 years.

December 24, 2024

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