Metagenomi, Inc. will showcase new pre‑clinical data on its APOC3 gene‑editing program at the Nature Conference “Cracking the Code: Nucleic Acid Medicines Coming of Age,” scheduled for December 8‑10 in Boston, MA. The presentation, led by Senior Vice President of Research Alan Brooks, will detail a CRISPR/Cas‑mediated APOC3 knockout that could serve as a one‑time treatment for severe hypertriglyceridemia.
The APOC3 program is the third target in Metagenomi’s collaboration with Ionis Pharmaceuticals, expanding the partnership beyond the company’s lead hemophilia A program (MGX‑001). The data build on Ionis’s experience with APOC3 therapeutics, such as AKCEA‑APOCIII‑LRx, and could open a large market for a condition linked to cardiovascular risk and pancreatitis. The presentation follows Metagenomi’s Q3 2025 earnings, in which the company reported a net loss and a decline in collaboration revenue, and a 25% workforce reduction that was part of a broader restructuring aimed at extending the cash runway into Q4 2027.
Analysts have adjusted price targets downward—Jefferies cut its target from $21 to $9 and Chardan lowered its target from $11 to $7—while maintaining buy ratings. The adjustments reflect concerns over the company’s recent restructuring and cash burn, but the presentation may temper investor enthusiasm by highlighting the potential upside of a successful APOC3 program.
CEO and senior research leadership emphasize disciplined spending and a focus on high‑value programs. The company’s strategy is to leverage its metagenomics‑derived gene‑editing platform to advance multiple indications, with the APOC3 data serving as a key milestone that could reshape the pipeline and investor perception.
The presentation is a significant operational milestone that could broaden Metagenomi’s therapeutic portfolio and influence future development priorities. Its impact will depend on the robustness of the pre‑clinical data and the company’s ability to translate these findings into clinical progress.
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