Leveraging novel CRISPR-Cas enzymes and innovative technologies from our discovery and engineering platform.
The small sizes of our NanoCas and CasPhi nucleases overcome the limitations of viral and non viral delivery that challenge legacy proteins like Cas9.
With alternative and shorter PAM requirements, our novel nucleases can direct edits to an expanded set of DNA sequences with improved specificity.
Covering ultracompact nucleases, novel modalities and beyond.
Our novel editing modalities include base editing, gene writing, epigenetic editing and beyond. Our modalities are uniquely compatible with single AAV delivery, which unlocks the full potential of precision editing.
Our novel Cas nucleases enable genome editing results that challenge legacy Cas nucleases.
Ultracompact CRISPR-Cas systems are ideal scaffolds for in vivo genome editing because they can better leverage existing viral and non-viral delivery methods. Using our protein discovery and engineering capabilities, we continue to build solutions to expand our technology portfolio for diverse therapeutic applications.
Example domain organization of NanoCas and CasPhi compared to commonly used variants of Cas9 and Cas12 with the nuclease domains (RuvC and HNH) indicated. Protein lengths are drawn to scale.
Length distribution of NanoCas and CasPhi systems compared to Cas12a-e and Cas9.
We unlock the full potential of current and future delivery modalities through our ultra compact size and diverse editing techniques.
Novel viral and non-viral techniques including next generation AAV, retargeted LNP, exosomes, eVLP, and beyond
Delivering on unmet patient need through differentiated science.
| Target Tissue | Program | Indication | Editing Approach | Delivery | Partner | Research | Lead Optimization | IND Enabling | Clinical |
|---|---|---|---|---|---|---|---|---|---|
| Liver | MB-111 | FCS + SHTG | Ultracompact Nuclease Knockout | LNP | Internal | ||||
| Undisclosed | Ultracompact Nuclease Multiple Techniques | Multiple | Internal + Partner | ||||||
| Neuromuscular | Undisclosed | Ultracompact Nuclease Multiple Techniques | AAV | Internal + Partner | |||||
| CNS | Undisclosed | Ultracompact Nuclease Multiple Techniques | AAV | Internal + Partner | |||||
Our lead wholly-owned investigational program is designed to be a durable, single-course treatment that permanently inactivates the APOC3 gene in the liver to reduce the production of the apoC-III protein. Disrupting protein production by targeting APOC3 may lead to reductions in plasma triglycerides for patients with elevated levels, including those with life-threatening genetic diseases such as Familial Chylomicronemia Syndrome (FCS) and Severe Hypertriglyceridemia (sHTG). People with severely elevated triglycerides are at high risk for acute and potentially fatal pancreatitis and an increased risk of cardiovascular disease.
FCS is a highly debilitating genetic disorder characterized by plasma triglycerides ranging from 10 to 100 times the normal value, which often results in recurrent episodes of acute pancreatitis and frequent hospitalizations. Because there are no FDA-approved therapies for FCS, patients rely on an extremely restrictive, low-fat diet and face a significant burden on all aspects of their lives.
Partnerships
Mammoth is committed to advancing science internally and through collaborations in the service of its mission to discover, develop and commercialize drugs to improve the lives of patients. Please get in touch with us below if you would like to join us on our journey.