Tar­get­AMD 1.0

Age-re­lated Mac­u­lar De­gen­er­a­tion (AMD) is a dis­ease of the light-sen­si­tive part of the eye (reti­na) that is re­spons­ible for vis­ion. AMD is a ma­jor cause of blind­ness in eld­erly people and due to the aging pop­u­la­tion, it has been re­ferred to as a “time bomb” in so­ci­e­ty.

In the wet form of AMD, an im­bal­ance of mainly two factors (vas­cu­lar en­dotheli­al cell growth factor [VEGF] and pig­ment-ep­ithe­lial de­rived factor [PEDF]), leads to the growth of ves­sels in­to the reti­na and cell death. Cur­rently, wet AMD is treated by ap­prox­im­ately monthly in­jec­tions of anti-VE­GF mo­lecules to stop ves­sel growth. How­ever, the the­ra­py im­proves vis­ion in only 30% of treated pa­tients (sta­bil­isa­tion of vis­ion is reached in 60% of pa­tients). To avoid severe side ef­fects, high costs and the over­all con­tinu­ing bur­den on health care as­so­ci­ated with fre­quent drug in­jec­tions, the in­crease of PEDF levels to in­hib­it ves­sel growth and to re­cov­er a healthy cel­lu­lar en­vir­on­ment, would be a vi­able thera­peut­ic al­tern­at­ive.

Tar­get­AMD will ge­net­ic­ally modi­fy (trans­fect) pa­tient-de­rived, Iris Pig­ment Ep­ithe­lial (IPE) cells to in­duce high pro­duc­tion of PEDF in these cells, which will be trans­planted un­der the reti­na of the same pa­tient in one sur­gic­al ses­sion to provide a long-last­ing cure of AMD. IPE cells de­rive from the iris of the eye and small biopsies can be eas­ily har­ves­ted. Stable PEDF gene de­liv­ery will be based on the non-vir­al Sleep­ing Beauty trans­po­son sys­tem, which com­bines the ef­fic­acy of vir­al meth­ods with the safety of non-vir­al tech­no­lo­gies is to be cut off from one loc­a­tion in the gen­ome and to be re­in­ser­ted in­to an­oth­er one. Aca­dem­ic sci­ent­ists and in­dustry part­ners pro­duced in­nov­at­ive gene de­liv­ery tech­no­lo­gies, re­agents, and devices to be trans­lated in­to a simple and safe gene thera­peut­ic treat­ment for wet AMD. Ex­per­i­enced clini­cians and qual­i­fied sci­ent­ists will per­form a clini­cal tri­al, com­pris­ing isol­a­tion and PEDF-trans­fec­tion which con­sists of patient’s iris biopsy and trans­plant­a­tion of trans­fec­ted cells in­to the pa­tient dur­ing a single, 60-minute sur­gic­al ses­sion. The pro­ject brings a sig­ni­fic­ant en­hance­ment on qual­ity of life to AMD pa­tients, high­light the syn­er­gist­ic power of aca­dem­ic, clini­cal and in­dus­tri­al co­operaton to the scien­tific arena, and open new mar­kets for nov­el products for clini­cal ap­plic­a­tions of trans­po­son-based gene the­ra­py to in­dustry.