CRISPR-Based Therapy for Hereditary Angioedema

Danny M. Cohn; Padmalal Gurugama; Markus Magerl; Constance H. Katelaris; David Launay; Laurence Bouillet; Remy S. Petersen; Karen Lindsay; Emel Aygören‐Pürsün; David Maag; James S. Butler; Mrinal Y. Shah; A. Golden; Yuanxin Xu; Ahmed M. Abdelhady; David Lebwohl; Hilary Longhurst

doi:10.1056/nejmoa2405734

October 24, 2024New England Journal of MedicineOpen Access

CRISPR-Based Therapy for Hereditary Angioedema

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Key Points

To assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of a single dose of the in vivo CRISPR-based therapy NTLA-2002 in adults with hereditary angioedema.
Phase 2 portion of a phase 1–2 randomized, placebo-controlled trial (NCT05120830; EudraCT: 2021-001693-33) enrolling 27 adults with hereditary angioedema.
Participants were randomly assigned in a 2:2:1 ratio to receive a single dose of NTLA-2002 at 25 mg (N=10), NTLA-2002 at 50 mg (N=11), or placebo (N=6).
The primary endpoint was the monthly angioedema attack rate from week 1 through week 16; secondary endpoints evaluated safety, pharmacokinetics, and change from baseline in total plasma kallikrein levels.
From week 1 through week 16, the estimated mean monthly attack rate was 0.70 (95% CI, 0.25 to 1.98) with 25 mg of NTLA-2002, 0.65 (95% CI, 0.24 to 1.76) with 50 mg, and 2.82 (95% CI, 0.80 to 9.89) with placebo, reflecting attack rate reductions versus placebo of -75% and -77%, respectively.
Attack-free status through week 16 was achieved by 40% (4 of 10) in the 25-mg group and 73% (8 of 11) in the 50-mg group, accompanied by mean reductions in plasma kallikrein of -55% and -86%, respectively, while levels remained unchanged with placebo.
The most common adverse events among NTLA-2002 recipients were headache, fatigue, and nasopharyngitis.

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Implication

Randomized trial demonstrates reduced attack rates and plasma kallikrein with NTLA-2002 in hereditary angioedema, indicating potential for durable disease control.

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Cite This Study

Cohn et al. (2024) studied this question.

synapsesocial.com/papers/6a038737630747d66657d89e https://doi.org/10.1056/nejmoa2405734

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CRISPR-Based Therapy for Hereditary Angioedema

Key Points

No abstract yet

Implication

Discussion

Cite This Study

CRISPR-Based Therapy for Hereditary Angioedema

Key Points

No abstract yet

Implication

Discussion

Cite This Study

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