Genetix Pharmaceuticals has launched its clinical program to treat Adrenoleukodystrophy (ALD). It is developing a gene therapy product in collaboration with Professor Patrick Aubourg and Doctor Nathalie Cartier of the National Institute of Health and Medical Research (INSERM).
The company is planning to file an IND to expand the development in the US, and to expand the ongoing trial in France. The current development timeline is expected to make the product commercially available to patients by 2014.
According to Genetix, advantages of gene therapy approach over allogeneic bone marrow transplantation include, applicable to all patients and no limitation to have a matched donor; no risk of death from Graft Versus Host Disease by using patients’ own cells; rapid engraftment/reconstitution of marrow and immune function post transplant due to perfect match between patients’ stem cells and marrow mileu.
Alfred Slanetz, CEO of Genetix, said: “Developing safe and effective treatments for incurable diseases is what our industry is all about. Genetix is now committed to developing this product in the most scientific and ethical way, and to make this product commercially available to all patients afflicted with this devastating disease.”