Clinical Drug Experience Knowledgebase

Criteria

Inclusion Criteria:

Documented diagnosis of retinitis pigmentosa based on a genetic test confirming the presence of a monogenic mutation that affects a gene involved in the visual signalling process specifically at the level of RPEs, namely RPE65 or LRAT, or MerTK
18 years old ≤ Age ≤ 65 years old

For patient of the first cohort:

Visual acuity ≤ 20/200 in the best eye (legally blind)

- For patient of the second cohort:

20/63 > Visual acuity > 20/200 in the worst eye And
Visible photoreceptor outer nuclear layer (ONL) on a spectral domain optical coherence tomography (OCT) scan

For the two cohorts:

Negative serum pregnancy test in women of childbearing potential (a woman who is two years post-menopausal confirmed by a physician or surgically sterile is not considered to be of childbearing potential)
Female patients of childbearing potential (if sexually active), committed to use two methods of contraception starting from the enrollment, during Mycophenolate Mofetil (MMF) treatment and for 6 weeks after the last dose of MMF
Sexually active men (including vasectomized men) committed to use condoms during from the first day of MMF treatment and for at least 90 days after cessation of treatment
Signed informed consents by the patient or legal guardian(s). For patients unable to give consent, authorization to participate to the study will be collected close to their legally authorized representative
Affiliated to or a beneficiary of a health care system

Exclusion Criteria:

- Patient unable or unwilling to comply with the protocol requirements
History of allergy or sensitivity to one of the products used during the study
Patients with known serious allergies to the fluorescein
Patients with a contraindication to general anesthesia
Prior treatment with a gene or cell therapy product
Patients with chronic hepatitis B or C, i.e. positive hepatitis B surface antigen or hepatitis C RNA viral load positive
Patients infected with Human immunodeficiency virus (HIV)
Anti-HLA antibodies positive at screening
Pregnancy or breastfeeding
Presence of any ocular disease or ocular media opacity which in the opinion of the investigator precludes accurate evaluation
Participation in another drug or device clinical study within last 6 months prior to baseline
Patients known to be affected by pathologies for which the symptoms or associated treatments can alter the visual function and/or affect the retina
Systemic corticosteroid therapy or other immunosuppressive / immunomodulating or anti-retroviral drugs within 2 months prior to baseline
Patients with a contraindication to immunosuppressive/immunomodulating therapy (MMF) such as severe chronic renal impairment, severe digestive system disease, Lesch-Nyhan disease, Kelley-Seegmiller syndrome…
Acute illness or infection within 4 weeks of the anticipated administration of study medication which may interfere with study assessments and immunosuppressive/immunomodulating therapy
Any other condition or history that, in the opinion of the Investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study