“A phase 3 trial of Intellia Therapeutics’ in vivo gene-editing therapy lonvoguran ziclumeran (lonvo-z) has hit its primary endpoint, leading the biotech to start a rolling submission for FDA approval. 

The study randomized 80 people with hereditary angioedema (HAE) to receive lonvo-z or placebo. HAE is a rare genetic condition characterized by recurrent and potentially life-threatening swelling. Noting that overproduction of bradykinin drives the disease, Intellia bet it could improve outcomes by using CRISPR to inactivate a gene needed to produce the peptide. 

Intellia’s phase 3 Haelo data validate the hypothesis, while leaving the question of whether the bet will pay off commercially unanswered. One infusion of lonvo-z reduced swelling attacks by 87% compared to placebo during the six-month efficacy evaluation period, hitting the trial’s primary endpoint.”

From Fierce Biotech.