Specialist
Professor at UC Davis School of Medicine
Agenda
- Key trends and developments in the CRISPR technology space, focusing on Mammoth Biosciences
- CRISPR technology competitive landscape and Mammoth Biosciences’ positioning vs competitors
- Mammoth Biosciences’ CRISPR systems and differentiators
- Clinical potential of Mammoth Biosciences’ partnership with Vertex Pharmaceuticals (NASDAQ: VRTX)
- Outlook for Mammoth Bioscences’ DETECTR platform
Questions
1.
Could you highlight any key trends or developments within the broader CRISPR segment? Is anything impacting Mammoth Biosciences?
2.
Where is Mammoth positioned in the competitive landscape, given it’s a small, newer name in biotech? Who does it compete against?
3.
Could you assess Mammoth’s work in creating the CRISPR toolbox while collecting various Cas proteins? I understand the company uses a CRISPR platform across the three areas of diagnostics, genome editing and protein discovery. What makes its approach unique?
4.
Do you have any reservations around the work Mammoth is doing? What challenges could the company face?
5.
How does the specific DNA sequence of PAM [protospacer adjacent motif] with Mammoth’s new Cas family and Cas14 compare to other proteins? We know that SpCas9 [pyogenes Cas9] requires the presence of PAM to target an adjacent sequence for genome editing. Why is it important to have short PAM requirements?
6.
What unlocked potential exists for Mammoth in broadening its toolkit of next-generation CRISPR systems? The company recently received more funding, as you mentioned, and plans to broaden its toolkit with a focus on building its pipeline of in vivo gene-editing therapeutics, specifically using a set of these ultra-small Cas proteins. How exciting might the news of its collaborative deal with Vertex be to this expansion?
7.
What disease areas might have the most promise for Mammoth and Vertex to target? We know they plan on targeting genetic diseases, but have not yet stated which ones. Are there any areas with high unmet need that might benefit from being delivered directly into the body?
8.
What is important to highlight in the differences in Cas proteins needed for diagnostics vs gene editing, given Mammoth is also tackling diagnostics with its CRISPR toolkit? What are the main advantages and disadvantages of its DETECTR platform?
9.
Is there anything else that you’d like to highlight, whether about gene editing, diagnostics or Mammoth’s growth potential?
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