Main objectives of Teramet Project are based on the development of new forms of therapy for rare diseases, using cutting edge technologies which may be extrapolated to other diseases with similar causes. So, and in a general way, the objectives of the project can be divided in two big categories:

• To obtain new forms of therapy based on the use of nano-technological platforms (nanoliposomes) functionalized as vehicles for Active Pharmaceutical Ingredients (APIs) from biotechnological origin and also small molecules according to the specific case, to improve efficacy, use of API and to diminish side effects.

• To develop new forms of gene therapy, which may go beyond a chronic treatment for the disease, and advance to a potential curation based on the correction of the genetic inborn errors which cause it.

The project presents a strong innovative profile, as it is based on the use of new generation nano-biotechnological platforms, which posse a high potential for the improvement of the drug based therapies, and, on the other hand, the application of innovative genomic therapies, using vanguard tools which goes beyond conventional gen therapy.

From a socio-economic point of view, Terarmet project will mean not only the entry of new drugs in niche markets which are considered as orphan at the current moment, but it also presents the potential to change the life to hundreds of patients and their families which are already affected by this challenge.