Mammoth Biosciences is a biotechnology company based in Brisbane, California[1] developing diagnostic tests using CRISPR-Cas12a and CRISPR-based therapies using its proprietary ultra-small CRISPR systems. Several CRISPR-Cas systems identified through the company's metagenomics-based protein discovery platform, including members of the Casφ and Cas14 families of CRISPR-associated enzymes, have demonstrated potential for therapeutic genome editing in in vivo settings.[2]
In 2023, the company announced that it will focus on developing CRISPR-based therapies.[8] The company's platform uses "ultra-small" Cas enzymes, such as CasΦ and Cas14, which measure down to a third or less the size of Cas9, and which could allow for easier delivery in vivo through commonly used non-viral and viral delivery mechanisms, including adeno-associated virus vectors.[9][10] Compared to Cas9, CasΦ and Cas14 may also offer less restrictive protospacer adjacent motif requirements, advantageous off-target activity, and allele-specific editing capabilities, which could broaden the range of targetable diseases in the genome.[10][9] Mammoth previously announced partnerships with Vertex Pharmaceuticals (2021) and Bayer (2022) to research ultra-small CRISPR systems for in vivo editing.[11][12]
Related Works
Lucas B. Harrington et al. ,Programmed DNA destruction by miniature CRISPR-Cas14 enzymes. Science362,839-842(2018). doi:10.1126/science.aav4294
Broughton, J.P., Deng, X., Yu, G. et al. CRISPR–Cas12-based detection of SARS-CoV-2. Nat Biotechnol 38, 870–874 (2020). https://doi.org/10.1038/s41587-020-0513-4
Patrick Pausch et al. ,CRISPR-CasΦ from huge phages is a hypercompact genome editor. Science369,333-337(2020). doi:10.1126/science.abb1400
