Intellia Therapeutics Receives European Union Orphan Drug Designation for NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema
CAMBRIDGE, Mass., Nov. 14, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative single-dose therapeutics leveraging CRISPR-based technologies, today announced that the European Commission (EC) has granted orphan drug designation to NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is an in vivo CRISPR-based investigational therapy designed to prevent potentially life-threatening swelling attacks in people with HAE.
“The European Union orphan drug designation for NTLA-2002 represents another important milestone for Intellia as we continue to make rapid progress in the development of a novel, potential one-time treatment for people with hereditary angioedema,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We are on track to complete enrollment of the Phase 2 portion of the study in the coming weeks, which will bring us one step closer to our goal of delivering a potentially life-changing treatment for people who suffer from this serious and debilitating disease.”
Orphan drug designation in the European Union (EU) is granted by the EC based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. To qualify for orphan drug designation, a candidate therapy must be intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating disease that occurs in not more than five in 10,000 people in the EU. The designation provides regulatory, financial and commercial incentives to develop therapies for rare diseases where there are either no satisfactory treatment options or significant benefit to those affected by the disease.
Intellia has received five special regulatory designations for NTLA-2002. NTLA-2002 was also granted Orphan Drug Designation and RMAT Designation by the U.S. Food and Drug Administration (FDA), the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), as well as PRIME designation by the EMA.
About the NTLA-2002 Clinical Program
About Hereditary Angioedema
About Intellia Therapeutics
Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates, including NTLA-2002; the risk that NTLA-2002 will not be successfully developed and commercialized; and the risk that the results of preclinical studies or clinical studies, such as the Phase 1/2 clinical study of NTLA-2002, will not be predictive of future results in connection with future studies for the same product candidate or Intellia’s other product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K and quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.
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