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Intellia and ReCode join forces on cystic fibrosis gene therapy

EditorEmilio Ghigini
Published 15/02/2024, 12:48
© Reuters.

CAMBRIDGE, Mass. & MENLO PARK, Calif. - Intellia Therapeutics, Inc. (NASDAQ:NTLA), known for its CRISPR-based gene editing technologies, and ReCode Therapeutics, a genetic medicines company, have announced a partnership to develop genomic medicines for cystic fibrosis (CF). The collaboration aims to harness Intellia's gene editing platform and ReCode's proprietary lipid nanoparticle delivery system to target CFTR gene mutations responsible for CF.

The strategic alliance is set to focus on creating treatments for CF patients with limited or no existing therapeutic options. Intellia will lead the design of the gene editing components, while ReCode will oversee preclinical and clinical development, along with global commercialization for certain programs. Intellia is eligible for development and commercial milestone payments and royalties on sales, with an option to take charge of U.S. commercialization for some programs.

John Leonard, M.D., CEO of Intellia, expressed enthusiasm for the potential impact on CF patients' lives, emphasizing the importance of extending the reach of their CRISPR platform beyond liver targets. ReCode's CEO, Shehnaaz Suliman, M.D., echoed this sentiment, highlighting the collaboration's potential to accelerate the delivery of next-generation gene editing therapeutics to patients.

CF is a life-threatening genetic disorder marked by the buildup of thick mucus in the lungs and other organs, which can cause severe respiratory and digestive issues. Intellia's in vivo programs focus on editing genes within the body, while their ex vivo efforts involve engineering cells outside the body for cancer and autoimmune disease treatments.

ReCode's SORT lipid nanoparticle platform is designed for precise delivery of genetic medicines to specific organs and tissues. The company is advancing its pipeline, which includes mRNA-based therapies for primary ciliary dyskinesia and a subset of CF patients unresponsive to current treatments.

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This collaboration represents a significant step in the development of gene therapies for CF, a condition with a critical need for more effective treatments. The partnership's success will depend on the integration of Intellia's gene editing technology with ReCode's delivery system, aiming to bring transformative solutions to the CF community.

The information in this article is based on a press release statement.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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