MGX Remains On Track for Q4:26 IND; Cash Runway Through 2027

研报英文原文证据摘录

MGX Remains On Track for Q4:26 IND; Cash Runway Through 2027

TD Cowen Metagenomi Therapeutics

Global Research May 11, 2026

Investment Thesis

Metagenomi Therapeutics is a biotechnology company focused on the development

of genome editing technologies to potentially cure disease. The company leverages a

novel database of nuclease families uncovered from screening in biodiverse

environments in order to generate a broad toolbox of gene correction approaches.

Using its programmable nucleases, the company can perform more traditional gene

editing without the typical size constraints, complete single nucleotide changes with

base editors, use RNA templates to achieve small edits (prime editing) with their little

RIGS and large edits with their big RIGS, or can enact large gene insertions using

transposases (CASTs).

The company’s lead program is MGX-001 being developed for hemophilia A. Pre-

clinical studies have demonstrated the ability to generate therapeutic levels of FVIII

protein that are well-maintained in early follow-up studies in NHPs. Approved gene

therapies for hemophilia A have shown roll-off of FVIII expression, which has limited

commercial uptake. IND/CTA filings are expected in Q4:26.

Metagenomi also leverages a collaboration with Ionis. The lead indications with IONS

include ATTR, a refractory hypertension program focused on AGT, and a

hypertriglyceridemia program focused on APOC3. Importantly, these collaborations

could be a good source of continued non-dilutive funds as development advances

and some programs offer co-develop optionality. Due to the translatability of the

platform, early de-risking can have important implications across the broad pipeline,

which should enable fast-follow programs over the coming years.