Mammoth Biosciences

We're #hiring a new (Associate) Director/Senior Director, Regulatory Affairs, Therapeutics in Brisbane, California. Apply today or share this post with your network.

We're #hiring a new Director/Senior Director, Regulatory Affairs, Therapeutics in Brisbane, California. Apply today or share this post with your network.

?? In case you missed it, we recently announced a new study, “Single-AAV CRISPR editing of skeletal muscle in non-human primates with NanoCas, a novel ultracompact nuclease” available now on bioRxiv. This research represents a major breakthrough in extending CRISPR’s therapeutic potential to a wide range of diseases that were once beyond the reach of gene editing, addressing many of its current limitations. While current CRISPR systems have offered precision in gene editing, clinical applications have been constrained due to their large size. In our latest study, we identified #NanoCas – an ultracompact nuclease that can be delivered using a single-AAV and capable of highly efficient editing of muscle in non-human primates, a tissue traditionally difficult to target.? This work reflects the dedication of the Mammoth Biosciences team to push the boundaries of what’s possible in gene editing and increase therapeutic accessibility to diseases that have been difficult to treat with existing gene editing technologies! ?? Read the full press release here: https://lnkd.in/g-q5inKZ and access the full study here: https://lnkd.in/g6V6GvZB #CRISPR #GeneEditing #Biotech #PrecisionMedicine

??Our latest #NanoCas research is making waves! At just 1/3 the size of Cas9, #NanoCas maintains exceptional editing efficiency while fitting comfortably within a single AAV delivery system. Our research, featured in @GEN, shows how #NanoCas successfully edits muscle tissue in NHPs, a breakthrough that could enable broader therapeutic applications of in vivo gene editing. We are grateful to Julianna LeMieux and Genetic Engineering & Biotechnology News for their coverage. We're extremely excited about the future of #NanoCas, and what it may mean for patients! #GeneEditing #Biotech #PrecisionMedicine #Innovation

AAV Delivered NanoCas CRISPR System Edits Muscle in Non-Human Primates The research team at Mammoth Biosciences presents new preclinical research on NanoCas—the first ultracompact #CRISPR system capable of efficient extrahepatic editing when delivered systemically using a single AAV vector.

We're #hiring a new Director/Senior Director, Regulatory Affairs, Therapeutics in Brisbane, California. Apply today or share this post with your network.

??CRISPR proteins are getting smaller – but their potential just got bigger. Our latest research on #NanoCas, an ultracompact nuclease, has been featured in Science Magazine! This breakthrough allows #CRISPR to reach tissues that were previously difficult to target, such as muscle, using a single AAV delivery system. Why does this matter? By enabling efficient gene editing in the muscle tissue of NHPs, #NanoCas opens new avenues for treating diseases that were previously out of reach for gene-editing therapies. We’re incredibly proud of this work and grateful to Jennifer Couzin-Frankel and Science Magazine for covering our latest research! #GeneEditing #Biotech #PrecisionMedicine #Innovation

?? ?? ?Excited to share our latest preprint introducing NanoCas - an ultra-compact CRISPR protein 1/3 the size of conventional systems! Despite its small size, NanoCas achieves efficient editing of muscle tissues in NHPs, a CRISPR breakthrough! This work represents a crucial step toward expanding the therapeutic potential of in vivo gene editing beyond the current focus on the liver. Why NanoCas is special: ?? Just 450 amino acids, ~1/3 the size of SpCas9 ? Matches editing efficiency of larger systems ?? Uses only a fraction of an AAV’s ~5kb capacity? ?? Flexible PAM sequence ??? Compatible with single AAV delivery of base editing, RT editing, and epigenetic editing ?? Demonstrated robust editing in skeletal and heart muscle in NHPs! Immensely proud of our team at Mammoth Biosciences who made this possible ??. We dedicate this work to our late colleague Ning Chai, whose contributions were instrumental to this breakthrough. Full preprint here: https://lnkd.in/gcdUfSK3 Looking forward to your thoughts on this work!

?? We are proud to announce our team’s latest breakthrough, “Single-AAV CRISPR editing of skeletal muscle in non-human primates with NanoCas, a novel ultracompact nuclease”. This research is a major step for delivering on the full promise of CRISPR to perform any edit in any cell in vivo, and explores how our ultracompact #CRISPR system, #NanoCas, overcomes the limitations of first-generation gene editors such as Cas9 and achieves efficient muscle editing in non-human primates using a single AAV vector. ??Curious to learn more? Read the press release here: https://lnkd.in/g-q5inKZ and access the full study here: https://lnkd.in/g6V6GvZB #GeneEditing #Biotech #PrecisionMedicine

Mammoth Biosciences aims to bring new genetic medicines to patients in need by leveraging our Ultracompact #CRISPR systems to overcome?gene editing delivery limitations. Our Co-Founder and CEO Trevor Martin will be presenting how #JPM2025