On the 24th March, members of the German association ZNM – Zusammen Stark! and Myotubular Trust met at IGBMC in Strasbourg to visit the laboratory of Dr Jocelyn Laporte where Myotubular Trust have been funding projects for a number of years. We had a tour of the IGBMC lab with Dr Laporte, Dr Belinda Cowling and Dr Johann Bohm. Then Dr Cowling presented the latest research findings on their project targeting DNM2 as a cross therapy approach to treating a number of forms of of centronuclear myopathy.

We also met representatives of Dynacure http://www.dynacure.fr/, located on the same research park as IGBMC, and working in collaboration with Dr Laporte’s lab. How patient organisations and Dynacure can work together to provide the patient perspective in development work going forward was discussed.

More background information:

Dr Belinda Cowling, IGBMC, Strasbourg, France (pictured) says:

“In our team we are working on understanding what causes centronuclear myopathies, and trying to develop treatments. We recently discovered that the protein called DNM2, that is mutated in centronuclear myopathy, is highly expressed in myotubular myopathy (also called X-linked centronuclear myopathy), and that reducing DNM2 can dramatically improve the disease in mice.

In addition, we have shown that targeting DNM2 is beneficial to both myotubular and centronuclear myopathies (BIN1-related). BIN1-treated mice are able to walk around the cage, and maintain whole body strength up until at least 18 months of age, nearly a full lifespan for a mouse, indicating targeting DNM2 may be a valid therapy for several forms of centronuclear myopathies. This ongoing work is supported by a grant from the Myotubular Trust” Dr Belinda Cowling.

At the end of October 2016 the creation of a biotech company, Dynacure, was announced. Dynacure is aiming to treat myopathies, with as their first target, myotubular and centronuclear myopathies.

The chosen name of Dynacure stands for targeting Dynamin to cure myopathies.

The team of Jocelyn Laporte and Belinda Cowling based at IGBMC near Strasbourg-France have previously shown that reduction of DNM2 (dynamin 2), a gene implicated in centronuclear myopathy, can effectively rescue myotubular myopathy due to MTM1 loss. Cowling et al., Journal of Clinical Investigation 2014

The first aim of Dynacure is to develop a human drug to normalize DNM2, and revert clinical signs of myotubular myopathy. To reach this goal, Dynacure has teamed up with Ionis Pharmaceuticals (Carlsbad, USA) which will provide its unique expertise in antisense chemistry. Antisense are small pieces of DNA that can specifically fine-tune genes of interest, such as DNM2 in the present application.

“Ionis Pharmaceuticals is a world leading company on antisense chemistry. They have 20 years of expertise in such drug development and several of their compounds are being tested in patients with neuromuscular diseases, as SMA or myotonic dystrophy. Their expertise will thus greatly accelerate the development of a human drug for myotubular myopathy. The fact that such a prominent biotech decided to collaborate with our team even prior to the establishment of the Dynacure start-up is rather unique,” says Jocelyn Laporte.

In parallel, other important investigations, such as testing different methodologies for targeting DNM2 or assessing if such an approach can also rescue other forms of centronuclear myopathies, are ongoing and funded by a grant from Myotubular Trust

Dynacure is based in Strasbourg-France and was launched by the venture capital groups Kurma and Idinvest, Conectus. Based on a tight collaboration with the French technology transfer office Conectus, Ionis Pharmaceuticals, and IGBMC. http://www.conectus.fr/en/dynacure