DMD is a rare genetic condition that causes gradual muscle damage and weakness. Credit: StudioMolekuul/Shutterstock.com.

Avidity Biosciences has obtained orphan drug designation from the US Food and Drug Administration (FDA) for its AOC 1044 to treat Duchenne muscular dystrophy (DMD) in people with mutations amenable to exon 44 skipping (DMD44).

DMD is a rare, early-onset genetic condition that causes gradual muscle damage and weakness due to the loss of the dystrophin protein.

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

AOC 1044 provides phosphorodiamidate morpholino oligomers to skeletal muscles and cardiac tissue.

Its purpose is to selectively exclude exon 44 of the dystrophin gene, thereby facilitating the production of dystrophin.

The Phase 1/2 EXPLORE44 clinical trial is currently evaluating AOC 1044 as a treatment for individuals with DMD44. It marks the initial step in assessing a series of AOCs being developed by the company to address DMD.

Avidity intends to disclose findings from the EXPLORE44 trial’s segment involving healthy volunteers in the fourth quarter of 2023. It is currently enrolling participants living with DMD44 in the trial.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

In April 2023, the company secured fast-track designation from the FDA for AOC 1044 to treat DMD44.

Avidity chief medical officer Steve Hughes stated: “There are currently no treatment options that target the underlying cause of DMD44. AOC 1044 is designed to specifically skip exon 44 of the dystrophin gene to enable the production of functional dystrophin protein.

“We look forward to advancing AOC 1044 in clinical development and bringing this very important treatment to patients as quickly and safely as possible.”