Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients

An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment.

A serious adverse event (SAE) is any untoward medical occurrence at any dose that leading to the following: Results in death; Life-threatening, refers to an event in which the patient is at risk of death at the time of the event; it does not refer to an event which hypothetically might have caused death if it were more severe; Significant or permanent disability/incapacity, where disability refers to a serious disruption and damage of a person's ability to perform normal life functions; Requires inpatient hospitalization or prolongation of existing hospitalization; Congenital anomaly or birth defect; Other medically important events.

IOP refers to the fluid pressure inside the eye. Monitoring IOP ensures that interventions do not inadvertently increase IOP to dangerous levels. IOP will be measured using a clinical tonometry device.

Multi-Luminance Mobility Test (MLMT) is used to evaluate how well individuals with visual impairments can navigate and perform tasks in different lighting conditions. This test is particularly relevant for conditions that affect night vision or light adaptation, such as retinitis pigmentosa or other forms of inherited retinal dystrophies.

Quality of Life will be measured using Visual Function Questionnaire (VFQ-25) or other similar questionnaires before and after treatment

BCVA of both eyes will be assessed using the early treatment of diabetic retinopathy study (ETDRS) chart or tumbling "E" chart or other avaliable measurement. This approach was chosen to facilitate visual acuity testing in subject who cannot recognize letters.

Color discrimination testing measures an individual's ability to perceive and differentiate hues across the visible spectrum under standard luminance level. Instead of relying on fine spatial detail, these tests challenge the visual system to detect subtle differences in wavelength-an ability that is often impaired in advanced retinal disease.

Visual field will be assessed by Humphrey perimetry, changes in VFI, MD, PSD will be analyzed.

FAF is a non-invasive imaging technique that provides critical information about the health of the retina, which is vital for the functioning of the retina.

Central Foveal Thickness refers to the thickness of the retina at the fovea, the central part of the macula. Changes in CFT can indicate various retinal conditions, including macular edema, macular degeneration, and central serous retinopathy.

Central Macular Thickness is the measurement of the thickness of the macula, which includes the fovea and the small surrounding area. The macula is responsible for central vision and visual acuity.

Fundus photography is used to document and monitor changes in the eye over time. It's vital for assessing the conditions of retina and monitoring the effects of treatments.

Multi-luminance chess board test (MLCBT) is a functional vision assessment specifically designed for individuals with severe visual impairment. During the test, participants are asked to identify the orientation of light target and avoid chessboard pattern obstacles presented under multiple illumination levels that simulate real-world environments, from very dim to bright light conditions. By evaluating performance across luminance tiers, MLCBT provides a sensitive measure of residual spatial vision and contrast-dependent functional gains, enabling the detection of clinically meaningful improvements that are not captured by conventional visual acuity testing.

The Multi-Luminance Shape Discrimination Test (MLSDT) evaluates functional vision by measuring a participant's ability to recognize geometric shapes presented at high contrast under different luminance levels. Shapes such as circles, squares, or triangles are shown one at a time, and the participant must correctly identify each target. By testing performance across a range of light intensities that mimic everyday environments, MLSDT sensitively captures changes in spatial perception and object recognition. This makes it particularly valuable for assessing treatment-related improvements in individuals with profound vision loss, where traditional acuity metrics may not reflect meaningful functional gains.

The Full Stimulus Test (FST) is a test to assess the functional integrity of the entire visual pathway, from the retina to the visual cortex. It often used in studies involving retinal dystrophies or certain neuro-ophthalmological conditions, FST might be used to assess the efficacy of a treatment or intervention.