Elixirgen Therapeutics Presents Preclinical Data with Bobcat mRNATM Technology in Duchenne Muscular Dystrophy at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

Bobcat mRNA^TM technology is a novel linear mRNA platform able to encode large proteins

Data showed restoration of muscular function in DMD mouse model

BALTIMORE, March 04, 2024 (GLOBE NEWSWIRE) — Elixirgen Therapeutics, Inc., a clinical-stage biotechnology company focused on the discovery, development and commercialization of therapies using its mRNA platforms, today presented preclinical data on its proprietary Bobcat mRNA^TM technology in a poster presentation at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference taking place March 3-6, 2024, in Orlando, Florida. Bobcat mRNA^TM is designed to deliver large protein payloads (over 13 kb), and results show that a Bobcat mRNA^TM encoding a full-length dystrophin protein can restore muscular function in a mouse model for Duchenne muscular dystrophy (DMD).

“We’re excited to be presenting these promising data on our novel Bobcat mRNA^TM technology. Dystrophin, the largest known human gene, is mutated in DMD, which is a rare disease that causes progressive muscle weakness and atrophy,” said Aki Ko, Co-Founder and Chief Executive Officer of Elixirgen Therapeutics. “Our Bobcat mRNA^TM full-length dystrophin approach may be complementary to others currently approved or in development, or may even mitigate some of their issues. We’re pleased to add the Bobcat mRNA^TM technology to our pipeline of RNA technologies and look forward to sharing updates in the months ahead.”

Presentation Title: Development of mRNA Therapeutic Delivering Full-length Dystrophin for Duchenne Muscular Dystrophy
Poster Number: T428

About Elixirgen Therapeutics, Inc.

Elixirgen Therapeutics, Inc. is a clinical-stage biotechnology company focused on the discovery, development and commercialization of therapies using its mRNA platforms and ZSCAN4 technology. The company has one ongoing (NCT04211714) and one completed (NCT04863131) Phase 1/2 clinical trial. For more information, visit ElixirgenTx.com.

Forward-Looking Statements

This press release may contain “forward-looking” statements. Actual results may differ materially from those set forth in this press release due to the risks and uncertainties inherent in pharmaceutical research and development. Any forward-looking statements in this press release speak only as of the date of this press release, and Elixirgen Therapeutics undertakes no obligation to update or revise the statements in the future, even if new information becomes available.

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