Gene-editing Therapy NTLA-2001 Given Orphan Drug Status by FDA
fapnewstoday.com The U.S. Food and Drug Administration (FDA) has given orphan drug designation to NTLA- 2001, an investigational gene-editing therapy for familial amyloid polyneuropathy (FAP) and other forms of transthyretin (ATTR) amyloidosis. … The aim of NTLA-2001 is to disrupt the TTR gene, thus decreasing transthyretin production and preventing the buildup of these harmful protein […]
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
Julian D. Gillmore et al. Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein in tissues, predominantly the nerves and heart. NTLA-2001 is an in vivo gene-editing therapeutic agent that is designed to treat ATTR amyloidosis by reducing the concentration of TTR in serum. It […]
Let’s Tag Team This Until Everybody Understands: The Modified Spike Protein is Dangerous and For Very Specific Reasons
Jessica Rose There’s an article that was published yesterday… [that] confirms that the structures inducing arterial embolisms in post-injected individuals are not blood clots … I think we are dealing with a devilishly insipid hand-crafted (modified) RNA whose protein by-products (that appear to be multi-dimensional), following either translation, or digestion for MHC presentation, are amyloidogenic. Clinical presentation […]
