Antisense Oligonucleotide Therapy for Retinal Dystrophy

Age: Any Age

Sex: Female

Trial Phase: Phase 1 & 2

Sponsor: n-Lorem Foundation

Must not be taking: Steroids, Amiodarone, Plaquenil, others

Disqualifiers: Ocular infection, Macular edema, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 24 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Retinal Dystrophy due to PRPH2 mutation

Are You a Good Fit for This Trial?

This trial is for individuals with Retinal Dystrophy due to a specific PRPH2 mutation. Participants must have certain vision capabilities, agree to contraception if applicable, and be able to travel for study visits. Excluded are those with conditions that may interfere with the study or pose risks, such as high eye pressure, recent eye treatments, or other genetic causes of retinal disease.

Inclusion Criteria

Have a BCVA in the worse eye of at least Count Fingers (CF) or better

Have written informed consent and able to comply with all study requirements

For women of childbearing potential for whom the Investigator considers that the potential benefit outweighs any risk to the unborn fetus, a highly effective method of contraception must be used

Exclusion Criteria

Any condition that in the opinion of the Investigator would ultimately prevent the participant from completion of the study procedures

Intraocular pressure in the eye(s) to be treated is greater than 25 mmHg

Presence of any intravitreal device

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a personalized antisense oligonucleotide (ASO) treatment for Retinal Dystrophy

24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• nL-PRPH2-001

Trial Overview The trial involves a personalized antisense oligonucleotide (ASO) drug called nL-PRPH2-001 designed specifically for one participant with Retinal Dystrophy caused by the PRPH2 mutation. The focus is on assessing the safety and effectiveness of this tailored treatment approach.

How Is the Trial Designed?

Group I: Open Label Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

n-Lorem Foundation

Lead Sponsor

Trials

5

Recruited

5+

University of California, San Diego

Collaborator

Trials

1,215

Recruited

1,593,000+