Transgene-triggered safety concerns in DMD gene therapies

MDA aims to enhance understanding and formulate mitigation strategies for transgene-triggered adverse events. Examples of research that might be supported by this RFA include, but are not limited to, the following:

• Identification of ‘at risk’ patient genotypes for transgene-triggered responses as it relates to DMD mutation types
• Further investigation into the nature of immunoreactive epitopes in the dystrophin gene
• Re-design of dystrophin transgene therapies to minimize transgene-triggered responses
• Development of immunosuppression strategies to minimize transgene-triggered responses
• In vitro assays and/or models predictive of transgene-triggered immune responses

 

 

 

 

Funding: Research- $100,000 per year

Duration: 2 years

 

 

 

Research Authority due dates: 4.7.22

LOI (required) due date:  11.7.22

Full application due date: 29.8.22