Oregon Patent of the Month – March 2022

Scientists work tirelessly to develop drugs meant to treat diseases. Unfortunately, even if the drug is effective it may be hindered by delivery. Even just the act of delivering the drug to the proper target in the body requires intensive R&D. This has been one of the many limitations in treating muscle pathologies including Duchenne muscular dystrophy (DMD). Dystrophin is an essential protein but is the largest gene in the body. Because of this, it is unable to fit within the vector meant to deliver it to the muscle.

Sarepta Therapeutics, Inc. is a global biotechnology company focused on developing precision genetic medicine which targets rare diseases. Their current focus is on muscular dystrophy including DMD and limb-girdle muscular dystrophy. They’ve developed a gene therapy engine which provides a framework for creating new therapies. Through this engine, they have developed a compound capable of treating myotonic dystrophy in DMD – and the methods to better target both heart and quadricep muscles.

They’ve developed an antisense compound used to treat both myotonic dystrophy DM1 and DM2. While these two types cause similar symptoms they are each caused by different genetic alterations. DM1 is caused by an alteration in the DMPK gene. DM2 is caused by an alteration in the CNBP gene. Rather than correcting for missing or damaged exons in the dystrophin gene, it aims to deliver a separate, potentially functional version of the gene to take over the job of producing the essential dystrophin protein. Sarepta’s compound takes just a portion of the dystrophin gene, creating a smaller but functional micro-dystrophin gene. The smaller size means it can be better transported.

The antisense compound effectively and selectively blocks the sequestration of muscleblind-like 1 protein (MBNL1) in heat and quad muscles. Designed to penetrate cells, the compound uses a homing peptide which is selective for muscle tissue. This ensures it will target the desired tissues. 

Are you developing new technology for an existing application? Did you know your development work could be eligible for the R&D Tax Credit and you can receive up to 14% back on your expenses? Even if your development isn’t successful your work may still qualify for R&D credits (i.e. you don’t need to have a patent to qualify). To find out more, please contact a Swanson Reed R&D Specialist today or check out our free online eligibility test.

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