The non-profit organisation CureDuchenne has announced that they have made a $500,000 investment in the company 4D Molecular Therapeutics in order to support the development of gene therapy research. For more detailed information, you can read the original press release here, at Business Wire.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a genetic, life-shortening condition characterised by progressive muscle weakness and degeneration. DMD is caused by a lack of the protein dystrophin, which is crucial for building and maintaining healthy muscle tissues. Children who are affected by DMD will often begin to show symptoms between the ages of three to five. Often, the first muscles to be affected are those around the thighs, hips, and shoulders, before the condition progresses to affect other areas such as the arms, legs, and trunk. Over time, the condition can affect the heart and respiratory muscles.
About CureDuchenne’s Investment
CureDuchenne’s $500,000 investment will go towards 4D Molecular Therapeutics, a company that works on adeno-associated virus (AAV) gene therapy vector discovery and development for severe genetic conditions.
AAV gene therapies use harmless viruses as a vector, or ‘carrier’, to deliver the gene therapy past cell defences and into cells, where they can then deliver the genetic material to restore gene expression and treat the underlying genetic causes of certain conditions.
CureDunchenne is a non-profit organisation that works to prolong and improve people who live with DMD. CureDuchenne is involved in funding research into DMD, training healthcare professionals on standards of care for people with DMD, and provides resources to support families affected by the condition. You can visit their website by clicking here.
For more information about this news, you can view the original press release here.