What is Bobcat mRNA? How Does Bobcat mRNA Work for Duchenne Muscular Dystrophy?

Bobcat mRNA works differently than exon skipping or AAV-microdystrophy mechanisms. This next-generation method could lead to low-cost drugs for Duchenne muscular dystrophy.

Many exon skipping therapies or gene therapies, such as Elevidys, approved by the FDA for the treatment of Duchenne Muscle Disease, worry patients with their exorbitant prices.

According to reports, drugs developed using the Bobcat mRNA method will be more reasonably priced.

What is Bobcat mRNA?

Bobcat mRNA is a proprietary linear mRNA technology that produces high-quality mRNA encoding large proteins (greater than 13 kb) in a highly efficient and cost-effective manner. A lipid nanoparticle (LNP) delivers it. Elixirgen Therapeutics is first looking into how Bobcat mRNA can be used with dystrophin, which is the largest human gene known.

Is Bobcat mRNA Technology Reliable?

The company states that it did not observe any safety issues associated with Bobcat mRNA-DMD injection in preclinical animal studies.

When evaluating the safety of bobcat mRNA in comparison to alternative methods, there are a few crucial aspects to consider. Since mRNA is not a virus, it is exempt from the biosafety standards that an AAV method would need to adhere to in both manufacturing and administration. Notably, mRNA can be redosed, in contrast to AAV. Due to immune response concerns, AAV-based therapies often cannot be dosed more than once.

Can Bobcat mRNA Improve Muscle Strength?

The company conducted a study in mutant mice, a mouse model for DMD, and found functional improvements in muscle strength. The Elixirgen research company says it showed improvements in forearm muscles with weekly doses of mRNA-DMD. It also states that a single injection of mRNA-DMD into the forearm muscles can restore muscle strength in mutant mice.

These studies show that it might be possible to deliver an mRNA that codes for full-length dystrophin for muscle therapy in specific areas and eventually across the whole body. Both local and systemic full-length dystrophin approaches could be useful treatments for DMD patients who can walk and those who can’t.

Advantages of Bobcat mRNA

Current treatments, including exon-skipping drugs and AAV-microdystrophin, have been groundbreaking for patients with DMD. However, there remains a significant need to develop therapeutics that can more effectively prevent or treat the muscle-debilitating symptoms.

Dystrophin is a protein that causes DMD. Therefore, a treatment that restores the entire protein should theoretically restore proper function.

Scientists also know that full-length dystrophin is more stable and has a longer lifespan than the shortened version. Notably, exon skipping would only target a small percentage of the patient population.

A treatment developed with Bobcat mRNA technology and using the full-length version could be used by anyone, regardless of the exact mutation they have.