Regeneron Pharma and Mammoth Biosciences collaborate on CRISPR based gene editing therapies.
Regeneron Pharmaceuticals, Inc. and Mammoth Biosciences, Inc., announced a collaboration to research, develop and commercialize in vivo CRISPR-based gene editing therapies for multiple tissues and cell types
Regeneron is developing adeno-associated viral vectors (AAVs) using antibody-based targeting to enhance delivery of genetic medicine payloads to specific tissues and cell types. Mammoth is developing novel ultracompact nucleases and associated gene editing systems, with a variety of editing functionalities at a significantly smaller size than other CRISPR-based systems, including first generation Cas9 nucleases. By leveraging Regeneron’s expertise in AAV and antibody engineering and Mammoth’s expertise in ultracompact gene editing systems, the teams will endeavor to create disease-modifying medicines that can be delivered to tissues beyond the liver, to which most gene editing treatments are currently limited.
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