Metagenomi, Inc. (MGX), a precision genetic medicines company, on Thursday announced significant progress in its therapeutic development programs, particularly its MGX-001 gene editing candidate for Hemophilia A.
MGX-001, the company's wholly-owned development candidate, has demonstrated sustained Factor VIII activity in nonhuman primates or NHPs for over 16 months.
Metagenomi has engaged with the FDA for initial regulatory discussions and started Good Manufacturing Practices or GxP manufacturing activities for the program.
The company is set to continue the NHP durability study in 2025, with a goal of submitting an Investigational New Drug or IND application in 2026.
In addition to its Hemophilia A program, Metagenomi has made significant strides in its secreted protein deficiency platform, leveraging the MGX-001 gene editing approach. The company plans to demonstrate proof-of-concept in NHPs in 2025.
Metagenomi also remains on track for its Ionis collaboration programs, which target large cardiometabolic indications.
The company expects to nominate one to two development candidates in 2025.
The company also announced the addition of Eric Bjerkholt, CFO of Mirum Pharmaceuticals, to its Board of Directors.
Cash position:
Cash, cash equivalents, and available-for-sale marketable securities were $274.6 million as of September 30, 2024 with a cash runway expected to support operations through 2027.
Metagenomi continues to make advancements in its gene editing technology, including the development of compact SMART nucleases, adenine base editors, and CRISPR-associated transposases (CAST), with upcoming milestones in 2025 and 2026.
MGX shares debuted on the Nasdaq global market on February 9, 2024 under the ticker symbol "MGX.", priced at $15.00 per share.
Currently, MGX is trading at $2.74 down 1.89%.
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