Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa

There is no effective treatment for RP; once photoreceptors are lost, they do not regenerate.
The rate of deterioration of vision varies from person to person, with most people with RP
legally blind by age 40. Preclinical studies demonstrated that transplantation of retinal
progenitor cells into the eye can result in both photoreceptor replacement and significant
slowing of host photoreceptor loss. Thus, the primary goal of this therapy is to preserve,
and potentially improve, vision by intervening in the disease at a time when dystrophic host
photoreceptors can be protected and reactivated. Based on the demonstration of acceptable
safety and tolerability in a phase 1/2a study, this phase 2b study is designed as a
controlled comparison of the changes in visual function and functional vision in subjects who
receive a single jCell injection in comparison to a comparable sham-treated control group of
subjects with RP.

Inclusion Criteria:

Clinical diagnosis of RP confirmed by ERG and willing to consent to mutation typing, if not
already done Best corrected visual acuity (BCVA) 20/80 or worse and no worse than 20/800
Adequate organ function and negative infectious disease screen Female of childbearing
potential must have negative pregnancy test and be willing to use medically accepted
methods of contraception throughout the study

Exclusion Criteria:

Eye disease other than RP that impairs visual function Pseudo-RP, cancer-associated
retinopathies History of malignancy or other end-stage organ disease, or any chronic
disease requiring continuous treatment with system steroids, anticoagulants or
immunosuppressive agents Known allergy to penicillin or streptomycin Treatment with
corticosteroids or any investigational or neuroprotectant therapy within 90 days of
enrollment Cataract surgery within 3 months prior to enrollment