CRISPR candidate may offer “functional cure” for HAE

NZCR Principal Investigator, Dr Hilary Longhurst has presented promised results of the Phase1/11 NTLA- 2002 clinical trial (UNIVERSE) conducted at NZCR at the American College of Allergy, Asthma and Immunology (ACAAI) meeting this week in Louisville, Kentucky.

The sponsoring company Intellia Therapeutics reported new Phase I/II data for NTLA-2002 suggesting the in vivo CRISPR gene-editing therapy, which is being developed for patients with hereditary angioedema (HAE), led to “robust reductions” in plasma kallikrein levels and HAE attack rates at all doses tested. Commenting on the findings presented this weekend at the American College of Allergy, Asthma & Immunology (ACAAI) conference, CEO John Leonard said “we see early evidence that our one-time CRISPR-based investigational therapy may offer patients suffering from HAE a functional cure for their disease.”

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