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Regeneron inks $100M deal to develop gene-editing therapies

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Tarrytown-based Regeneron Pharmaceuticals has struck a $100 million deal with Mammoth Biosciences, a California-based biotechnology company, to develop and commercialize gene editing therapies for multiple tissues and cell types, the firms announced Thursday.

Regeneron’s $100 million upfront payment includes a $95 million equity investment, according to the company. Mammoth could receive a total of $370 million per target in development and commercial milestone payments.

 

 

The firms aim to produce CRISPR-based drugs that can be directly infused into patients’ bodies. CRISPR is a technology that scientists use to modify the DNA of living organisms; it has the potential to cure genetic diseases by cutting out targeted parts of DNA sequences.

According to Christos Kyratsous, Regeneron’s senior vice president and co-head of Regeneron Genetic Medicines, the firm chose to partner with Mammoth because the companies are working on aspects of gene editing technology that complement each other.

Regeneron has developed a virus platform that targets tissues and cells outside the liver, Kyratsous told Crain’s, whereas most gene editing therapies are currently limited to the liver. In turn Mammoth has created relatively small CRISPR editing systems that fit Regeneron’s platform, which could help the drugs reach tissues around the body and unlock treatments for diseases. Scientists at Regeneron will lead development and commercialization.

The deal also gives Mammoth the right to opt into co-funding and profiting from collaboration programs with Regeneron, instead of receiving milestone payments. Meanwhile, Regeneron will have access to some of Mammoth’s gene editing technologies for 5.5 years. It can extend access for another two years if it pays an undisclosed research extension fee.

The two firms will pick and research which sequences to target, Kyratsous added, declining to share specifics about which are being considered. The companies seek to begin developing therapies as soon as possible.

Regeneron earned just under $3.5 billion in revenue in the fourth quarter of 2023, a 1% increase over the same quarter of 2022, and is working on drugs to treat genetic diseases including ALS and hemophilia. Its financial results for the first quarter of 2024 will be released on May 2.

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Jacqueline Neber , 2024-04-26 11:33:03

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