Elixirgen Therapeutics’ Bobcat mRNA Therapeutic for Duchenne Muscular Dystrophy: Aki Ko

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"We envision the application of this Bobcat mRNA encoding a full-length dystrophin in a complementary context so that patients that have already undergone clinical trials, for example, would be able to potentially use this complimentary proposed treatment."

Duchenne muscular dystrophy (DMD) is a neuromuscular disease caused by mutations in DMD (encoding dystrophin), which prevent the production of the muscle isoform of dystrophin.¹ In the past decades, disease-modifying therapies have extended the lifespan of patients with DMD yet despite the progress made, many patients with the disorder still face unaddressed issues and seek additional treatment. A suggested and potential option for these patients is RNA therapeutics, which are a group of oligonucleotide-based drugs.

In recent preclinical research, Elixirgen Therapeutics’ Bobcat mRNA therapeutic showed that it enabled successful generation of mRNA encoding a full-length human dystrophin protein. Presented at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-7, in Orlando, Florida, preclinical results using the Bobcat indicated that the produced DMD proteins were stable and remained in the injection site for 3 weeks in skeletal muscles of the mouse models.2

Aki Ko, chief executive officer at Elixirgen Therapeutics, sat down with NeurologyLive® at the conference to discuss the advantages of using mRNA over viral vectors like adeno-associated viral in therapeutics. He also spoke about how Bobcat mRNA potentially addresses historical difficulties in encoding full-length dystrophin. Additionally, Ko talked about the implications of the functional restoration observed in DMD mice model.

REFERENCES
1. Saifullah, Motohashi N, Tsukahara T, Aoki Y. Development of Therapeutic RNA Manipulation for Muscular Dystrophy. Front Genome Ed. 2022;4:863651. Published 2022 May 10. doi:10.3389/fgeed.2022.863651
2. Elixirgen Therapeutics Presents Preclinical Data with Bobcat mRNATM Technology in Duchenne Muscular Dystrophy at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. News Release. Elixirgen Therapeutics. Published March 24, 2024. Accessed March 26, 2024. https://www.biospace.com/article/releases/elixirgen-therapeutics-presents-preclinical-data-with-bobcat-mrnatm-technology-in-duchenne-muscular-dystrophy-at-the-2024-muscular-dystrophy-association-mda-clinical-and-scientific-conference/