The Chief Executive Officer of Genethon, Frederic Revah, made the announcement on the YouTube channel of AFM-Telethon that the GNT0004 gene therapy, which is presently undergoing clinical trials, will produce outstanding outcomes for patients suffering from Duchenne muscular dystrophy (DMD).
Transcript of Genethon CEO Frederic Revah Speech
Hello I’m Frederic Revah. I’m the CEO of Genethon.
Genethon is a leading pioneer in gene therapy. We are an R&D organization
dedicated to delivering Gene therapies to patients affected with rare diseases. We were created in 1990 by a patient organization, the French Muscular Dystrophy Association, the AFM-Telethon. We’ve been involved in gene therapy since 1997 and we’re one of the very first actors in the field.
Over the time we have developed a compelling pipeline of products in the clinic. There are 13 products today in clinical trial stemming from our research.
Products for neuromuscular diseases for liver disorders, for eye disorders, for immune deficiencies. All these therapies we develop represent a strong hope for families, and they dramatically improve the lives of patients.
One of the most spectacular examples is the first ever gene therapy for neuromuscular disease, a gene therapy for spinal muscular atrophy, which is today marketed worldwide and which is based on science and patents from Genethon.
Today I want to tell you about the unique therapy we are developing for Duchenne Muscular Dystrophy. This product, GNT0004, has the potential of overturning the disease in Duchenne patients.
Duchenne is a devastating disease which causes irreversible muscle degenerations. It affects boys who will progressively lose their ability to walk and then to breathe. These kids will stop walking beyond the age of 12 and then they will get weaker and weaker. Most of them will die by the age of 20.
Duchenne is the most complex disease we’ve ever tackled. Our objective is to replace the missing Dystrophin Gene in patients.
This gene is too large to fit into a gene therapy vector. We thus had to engineer a reduced but functional form of the gene microdystrophin, which retains the functional and biological features of the parent gene and which can fit into a vector.
This we’ve done with one of the world leading experts in dystrophin biology, Professor George Dickson, in London.
We have combined this microdystrophin gene with a very safe AAV Vector.
In initial preclinical studies, this product has shown spectacular results with a very strong efficiency. Moreover, this effect is long lasting and we’ve seen protection from the disease in dogs up to 10 years today after single injection.
Now where are we with GNT0004 Gene Therapy?
The product is now ready to enter phase three after initial clinical phase. The results we’ve seen in the first patients treated are spectacular.
Patients treated display a much higher clinical score than untreated patients from our natural history study. And this clinical score it really means their ability to walk, to stand, to climb up the stairs.
Patients that are treated show preservation of their score, while untreated patients show a steep drop in this score.
I want to stress the fact that we use here a low dose. We know how much dose is important in terms of safety for gene therapy.
Well these clinical results are correlated with a high expression of the therapeutic transgene in the muscles of patients.
We’ve also looked at a blood marker which is sky high in, patients affected with muscular disease. Because this marker is really due to muscle degeneration. Well this specific marker shows a steep drop in the patient treated with GNT0004.
We see that we have with those elements expression of the transgene decrease in the degeneration blood marker very strong and converging elements.
The message is clear.
GNT0004 has the potential of becoming Best in Class for Gene therapies for Duchenne Muscular Dystrophy.
We are committed to bringing it to diseased kids and their families.
