DMD research

DMD Industry roundup (August - October 2022)

As the leading funder of Duchenne muscular dystrophy research in the UK, Duchenne UK is part of a global effort to find effective treatments and improve quality of life for everyone living with DMD.  

We aim to share the most important news in drug development with the DMD community through our quarterly roundup of updates from the DMD biopharmaceutical industry.  

If you are unfamiliar with any of the terminology used, please refer to our Research Glossary.

Pepgen reports positive data on exon skipping trial  

We are pleased to share the latest reported data from the Pepgen phase 1 clinical trial of their exon skipping therapy, PGN-EDO51. The treatment is aimed at people with certain mutations that are amenable (potentially responsive) to skipping exon-51 on the DMD gene (about 13% of boys diagnosed with DMD).  

The results reported are promising, showing that the drug was well tolerated, and achieved high levels of exon 51 skipping following a single administration. In addition, high levels of the drug were found in muscles at 10 days after administration, and sustained at 28 days. This is particularly encouraging, as it suggests the potential for dystrophin accumulation following multiple doses. 

Based on these exciting results, PepGen is planning a multiple ascending dose clinical trial in the first half of 2023.  They are also working to progress other candidates for skipping exons 53, 45 and 44. 

Read PepGen’s letter to the community and press release for more information. 

Sarepta acquires gene therapy delivery technology  

Sarepta, a global biotech company developing gene therapies for DMD and other forms of muscular dystrophy, announced that they are acquiring a new technology to improve gene therapy deliver and reduce side effects from current AAV vectors. This is part of their commitment to developing better, ‘second generation’ gene therapies for DMD.  

The technology, called MyoAAV, can deliver 25-50 times greater mini-dystrophin to skeletal muscles, and 10 to 15 times greater expression is the heart.  

We have welcomed the news of Sarepta acquiring a technology from the Broad Institute, in the US, to improve gene therapy delivery and reduce side effects for current AAV vectors, confirming their commitment to finding better, ‘second generation’, gene therapies for DMD. The technology called MyoAVV can deliver 25–50 times greater mini dystrophin expression to skeletal muscles and 10 to 15 times higher expression in the heart compared to standard AAV vectors.  

The MyoAAV vector is also reported to reduce by 50% accumulation in the liver, suggesting an improved safety profile in patients.  

Please the full story at Muscular Dystrophy news 

Solid Biosciences issues community update  

Solid Biosciences, a company which currently focuses on developing gene therapy for DMD, issued a recent update to the DMD community. They announced their plans to acquire a genetic medicines company and expand into other disease areas. They also announced that they will prioritise their second-generation gene therapy candidate, SGT-003, and pausing activities for the first-generation gene therapy candidate, SGT-001.  

At a time of significant change for the organisation, we welcome Solid’s continued commitment to finding treatments for people living with Duchenne muscular dystrophy.

Vamorolone and timescales for patient access: 

Earlier this month, we shared an update with the DMD community about Santhera’s steroid alternative drug vamorolone. Evidence from clinical trials suggests vamorolone is effective with fewer side effects than steroids currently prescribed for DMD. Santhera are now applying to regulators in different countries for market authorisation.  

Published on 28 October 2022

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