Intellia, ReCode partner on genetic medicines for cystic fibrosis

2024-02-15

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基因疗法引进/卖出

Intellia Therapeutics will work with a private biotechnology company to develop genetic medicines for cystic fibrosis, with an initial focus on people who have limited or no available treatment options.

The collaboration pairs Intellia’s gene editing platform, which is based on Nobel Prize-winning CRISPR technology, with ReCode Therapeutics’ method for delivering genetic medicines to specific organs.

With cystic fibrosis, mutations in a gene named CFTR causes thick mucus to accumulate in the lungs and other organs. This can lead to serious health issues, including respiratory failure and life-threatening infections.

“By combining our highly synergistic technologies and capabilities, we are excited about the potential to enable a faster path for next-generation gene editing therapeutics to CF patients,” said Shehnaaz Suliman, ReCode’s CEO, in a Thursday statement announcing the deal.

In that statement, Intellia noted how its platform includes “DNA writing” technology, a tool that has attracted considerable interest and investment in recent years. Tessera Therapeutics, for example, was created by Flagship Pioneering — the same biotech incubator that founded Moderna — and is built around “gene writing” technology that it claims can alter DNA in various ways, from changing single units of genetic material to adding in entire genes.

Those qualities may make gene writing especially useful in diseases like cystic fibrosis where the genetic causes are well understood. Last year, the Cystic Fibrosis Foundation, a patient advocacy group, partnered with Flagship to create potentially curative treatments using technology from some the latter’s portfolio companies. The deal, like that of Intellia and ReCode, is prioritizing patients for whom there aren’t drugs available.

In the U.S., medicines developed by Vertex Pharmaceuticals are, by the company’s estimates, able to treat about 90% of cystic fibrosis patients. The remaining 10% have different mutations that make them ineligible for Vertex’s therapies. Experts believe genetic therapies offer the best shot at treating these patients.

Intellia, per its new agreement, will be responsible for the “editing strategy” and components of the experimental therapies. ReCode, meanwhile, will lead the subsequent development of these therapies through preclinical and human testing. ReCode will also lead global commercialization efforts for certain programs that emerge from the collaboration.

Financial terms of the deal weren’t disclosed. But the companies did say Intellia will be eligible to receive development and commercial milestone payments, as well as sales royalties from products stemming from the collaboration. Additionally, it can exercise an option to lead commercialization in the U.S. for certain programs.

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