EditUP, led by researches from University of Trento and Hub Innovazione Trentino (Italy), is one of the supported research teams in the first S3E Start Programme.
Haploinsufficiencies are rare genetic diseases that affect an estimated 15 million patients worldwide, 99% of which remain incurable and manageable only through palliative and symptomatic care. EditUP have devised and patented a genome editing approach capable of precisely modulating the expression of specific genes to compensate for the lack of a disease-related allele and restore the levels of expression within physiological values.
The team are planning to validate their platform with patients affected by Autosomal Dominant Optic Atrophy (ADOA), which affects about 1 in 30,000 people worldwide. ADOA is the most common hereditary optic atrophy disease and is characterized by a bilateral degeneration of the optic nerves. Unlike palliative therapies, RNA and other genome editing approaches, EditUP will provide to patients affected by ADOA a therapy that is safe, personalised and is needed only once for a lifetime.
Learn more about EditUP by watching the pitch done by researcher Alberto Raoss during the Start Open Day on 20th July 2023.
Would you like to get in touch with them? Let us know! Email us at info@south3e.eu