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Intellia Therapeutics Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for NTLA-2002 By Investing.com

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Intellia Therapeutics (NTLA) Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for NTLA-2002

Intellia Therapeutics, Inc. (NASDAQ:) today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is an in vivo CRISPR-based investigational therapy designed to inactivate the target gene, kallikrein B1 (KLKB1), to potentially prevent life-threatening swelling attacks in people with HAE.

“The RMAT designation is important recognition for our early clinical data. It indicates that a single dose of NTLA-2002 has the potential to address serious unmet medical need for people living with hereditary angioedema,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We look forward to continuing our productive dialogue with the FDA to accelerate the development of NTLA-2002, an investigational in vivo CRISPR-based therapy, with the goal of bringing forth a potentially transformative treatment to patients more quickly.”

The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising therapeutic candidates, including genetic therapies, which are intended to treat, modify, reverse or cure a serious or life-threatening disease. RMAT designation includes benefits, such as early interactions with the FDA, including discussions on surrogate or intermediate endpoints that could potentially support accelerated approval and satisfy post-approval requirements, and potential priority review of a product’s biologics license application (BLA).

The RMAT is the third special regulatory designation received by Intellia for NTLA-2002. NTLA-2002 was also granted Orphan Drug Designation by the FDA and the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency.

About the NTLA-2002 Clinical ProgramIntellia’s multi-national Phase 1/2 study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II hereditary angioedema (HAE). This includes the measurement of plasma kallikrein protein and activity, as well as HAE attack rate. The Phase 1 portion of the study is an open-label, single-ascending dose design used to identify two dose levels of NTLA-2002 for further evaluation in the Phase 2, randomized, placebo-controlled portion of the study. This…

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