dm+d

Unassigned

New Medicines

Retinitis pigmentosa

Information

New molecular entity
GenSight Biologics
GenSight Biologics

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase II Clinical Trials
Yes
Yes
Jan 17Has orphan drug status in EU & US [3].

Category

Gene therapy containing a halorhodopsin gene in a specific AAV (adeno associated virus) variant, which delivers the gene into cone photo-receptors only, thereby restoring their sensitivity to light
Prevalence in all ages is approximately 1 in 4,000. Three types have been found determined by age of onset. The premature type occurs at a mean age of 7.5 years; there is a second type occuring at 17 years of age, and a senile type with onset in the fifties. There are no geographical or sex predilections. However, because of X-linked varieties, men may be affected slightly more than women [1].
Retinitis pigmentosa
Intravitreal

Trial or other data

Jul 20PI/II PIONEER trial continues to recruit. Collection of primary outcome data due to complete Dec 21 and the study should finish Dec 25 [4].
Aug 19PI/II PIONEER trial is recruiting [2].
May 19The company announces that an independent Data Safety Monitoring Board (DSMB) reviewed safety data of all treated patients in all four cohorts and made recommendations before increasing the doses. The DSMB confirmed that there were no safety issues for the first cohort of three subjects who received a single intravitreal injection of 5e10 vg combined with a wearable optronic visual stimulation device. The DSMB recommended moving forward as planned without any modification in the protocol and recruiting the second cohort of three subjects receiving an escalating dose of 1.5e11 vg [3].
Sep 18PI/II PIONEER trial to evaluate the safety and tolerability of GS030 in adults with retinitis pigmentosa starts (GS030_CLIN_001; NCT03326336). 18 patients will be recruited in the UK (Moorfields), France and the US. Primary outcome is safety. The study is due to complete Jan 25 [2].