Intellia’s NTLA-2002 Receives Orphan Drug Designation
Intellia Therapeutics received Orphan Drug designation from the FDA for its investigational candidate NTLA-2002 in the treatment of patients with hereditary angioedema, a disorder characterized by recurrent episodes of severe swelling.
The drug candidate features in vivo CRISPR/Cas9 genome editing capabilities. The company said NTLA-2002 is systemically administered to knock out the target gene kallikrein B1 to reduce plasma kallikrein activity and prevent hereditary angioedema attacks.
NTLA-2002 is currently being evaluated in a phase 1/2 study in adults with type I and type II forms of hereditary angioedema, the company said.
The Orphan Drug designation is used to support the development and evaluation of new treatments for rare diseases, qualifying sponsors for incentives like tax credits and possible exemption from user fees.
Intellia Therapeutics
Intellia Therapeutics is a biotechnology company developing biopharmaceuticals using a CRISPR gene editing system invented by Jennifer Doudna (with colleagues at University of California, Berkeley) and Virginijus Šikšnys (with colleagues at Vilnius University). The company has partnerships with Novartis and Regeneron.
September 12, 2022
