India, April 27 -- Intellia Therapeutics Inc. (NTLA) reported positive Phase 3 results from its global HAELO trial, showing that a single dose of its in vivo CRISPR gene-editing therapy, lonvoguran ziclumeran (lonvo-z), met the primary and all key secondary endpoints in hereditary angioedema.
Intellia mentioned that the data mark a major milestone as the first Phase 3 results ever reported for an in vivo gene-editing treatment.
Hereditary angioedema (HAE) is a rare genetic disorder that causes unpredictable and potentially life-threatening swelling attacks. Many patients rely on chronic prophylactic therapies- often weekly injections or daily oral medications- to prevent attacks, yet breakthrough episodes remain common.
Strong efficacy w...