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Expert Interview

Slingshot members are talking to an expert! The topic is:

A look at the positive results from the Phase 2 Study of NTLA-2002, Intellia's investigational in vivo CRISPR gene editing treatment for HAE

Ticker(s): NTLA

Who's the expert?

Institution: Penn State University

  • Professor, Department of Medicine, Division of Pulmonary, Allergy and Critical Care Medicine at Penn State University.
  • Currently manages over 120 patients with hereditary angioedema and has written guidelines for international management of urticaria, hereditary angioedema and rhinitis with more than 300 publications.
  • Clinical research and clinical care focuses include asthma, COPD, alpha-1-antitrypsin deficiency, urticaria, hereditary angioedema, immunodeficiency, vaccine response and other respiratory, allergic and immunologic diseases.

Interview Questions
Q1.

What are the current treatment options available for hereditary angioedema (HAE), and what is considered the standard of care for these patients?

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Q2.

How effective are the current prophylaxis and on-demand treatments, and what factors influence the choice of treatment for patients?

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Q3.

What are the potential benefits and safety concerns associated with Intellia's CRISPR therapy NTLA-2002, and how does it compare to existing and emerging treatments for HAE?

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Q4.

How do CRISPR gene-editing therapies fit into the current landscape of HAE treatments, and what are the barriers to their widespread adoption in terms of safety, administration, and insurance coverage?

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Q5.

Which patient profiles would be ideal candidates for NTLA-2002, and what level of long-term safety data would be needed to build confidence in its use?

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