Intellia to Present First Clinical Data for NTLA-2001, Pioneering Systemic CRISPR Therapy

ATTR amyloidosis is a rare, progressive, and fatal disease affecting approximately 50,000 hereditary cases and 200,000-500,000 wild-type cases globally, causing nerve damage and heart failure.

In the summer of 2021, a biotechnology company named Intellia Therapeutics crossed a threshold that medicine had long approached but never reached: the first clinical evidence that CRISPR gene-editing machinery, delivered through a vein, could rewrite the human genome from within. The therapy, NTLA-2001, targets a rare but fatal disease called ATTR amyloidosis, in which a faulty gene slowly fills the body's nerves and heart with toxic protein. Where previous treatments asked patients to manage their illness for a lifetime, this single injection asked a more ancient question — whether the sourc

Intellia to Present First Clinical Data for NTLA-2001, Pioneering Systemic CRISPR Therapy

Cobertura Relacionada

Receba o Register no seu e-mail