DMD research

DMD Research roundup – Dec 2021 – Feb 2022

Duchenne UK is integral in the global efforts to develop effective treatments for Duchenne muscular dystrophy (DMD) and improve the standards of care. We aim to empower the DMD community by informing them about the research landscape in the UK and beyond, and the scientific progress in the fight against DMD.  

This quarter’s  roundup focuses on the latest developments in DMD from the pharmaceutical industry.

Phase 3 trial of CAP-1002 therapy launching in US 

Capricor Therapeutics is launching a Phase 3 trial of their experimental cell therapy, called CAP-1002. The drug is thought to regulate the activity of the immune system to help restore muscle cells.  

Findings from an earlier trial, called HOPE-Duchenne, indicated that treatment with CAP-1002 improved muscle and heart function in people with DMD. 

It is hoped that the treatment will be effective for patients in the later stages of the disease, making it one of the few treatments suitable for older patients. It is expected to be equally effective in all DMD patients regardless of the specific disease-causing mutation. 

Read the full press release 

Sarepta’s gene therapy trial shows improved motor abilities  

The latest data from Sarepta’s trial of their gene therapy treatment, SRP-9001, indicated that children showed a marked improvement in motor abilities after being treated with the therapy. Results showed that these patients scored higher on the North Star Ambulatory Assessment (NSAA) – a measure of functional motor abilities – than a group of patients given a placebo.  

Read the full press release 

SOLID gene therapy pipeline community update  

SOLID Biosciences provided an update on their pipeline of DMD treatments to the DMD community in January 2022, including an update on the latest data from their gene therapy clinical trial and data from their pre-clinical research.  

Read the letter here 

Dystrogen Therapeutics announces first patient dosing of Chimeric Cell Therapy for DMD and reports on 6-weeks outcome 

Dystrogen Therapeutics announced that they have dosed the first patient in a trial of chimeric cell therapy for DMD. ‘Chimeric’ cells are made by fusing normal human muscle cells with a muscle cell from a person with DMD. It is hoped that this method could improve muscle function, without causing an immune response (one of the major challenges of gene therapy).  

Read the press release here 

Antisense Therapeutics received positive PIP decision from UK MHRA  

Antisense Therapeutics announced that they have received a positive decision from the Medicines and Healthcare products Regulatory Agency (MHRA) on their submission for a paediatric clinical plan of the antisense inhibitor treatment ATL1102, they are developing for DMD. This means that Antisense will be able to conduct a Phase II/III trial of the therapy in the UK. ATL1102 has showed to have potent anti-inflammatory effects by blocking key cells that drive inflammation in DMD, both in animals and in humans. 

Read the full press release here 

RegenXBio receives FDA approval for gene therapy trial  

RegenXBio announced that they have received approval from the US Food and Drug Administration (FDA) to run a clinical trial of their gene therapy treatment for DMD. They expect to initiate the trial in the US in the first half of 2022.  

Read the full press release 

Myogene Bio award to accelerate novel gene-editing therapy to the clinic 

Earlier this year we announced that US company Myogene Bio secured a $3.4M grant from CIRM to accelerate a novel gene-editing treatment to the clinic. Duchenne UK committed to co-fundMyoGene’s grant application alongside the National Institute of Arthritis and Musculoskeletal and Skin Diseases and Parent Project Muscular Dystrophy, helping to advance this essential pre-clinical research.   

Read the full press release 

An overview from Italian Parent Project Conference 

Our Director of Research, Dr Alessandra Gaeta, attended the Italian Parent Project annual conference on 17-20 February. The four-day event was rich with academic and industry talks, highlighting the latest developments in basic and translational research and in the clinic. The full programme and the recording of the talks will be available shortly here. 

Here are some additional updates from the conference that we would like to highlight to you. 

Published on 22 February 2022

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