Elixirgen Therapeutics’ Bobcat mRNA Therapeutic for Duchenne Muscular Dystrophy: Aki Ko
The chief executive officer at Elixirgen Therapeutics discussed the company’s mRNA as a promising therapeutic avenue for Duchenne muscular dystrophy. [WATCH TIME: 6 minutes]
WATCH TIME: 6 minutes
"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.
