Retinitis Pigmentosa

8
Pipeline Programs
7
Companies
11
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
1
4
2
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
1100%
+ 10 programs with unclassified modality

Competitive Landscape

6 companies ranked by most advanced pipeline stage

Thea Pharma
Thea PharmaMA - Waltham
3 programs
1
2
QR-421aPhase 2/31 trial
UltevursenPhase 2/3RNA Therapeutic1 trial
RNA antisense oligonucleotide for intravitreal injectionPhase 21 trial
Active Trials
NCT05085964TerminatedEst. Oct 2022
NCT05176717TerminatedEst. Aug 2022
NCT05158296TerminatedEst. Oct 2022
jCyte
jCyteCA - Newport Beach
3 programs
3
human retinal progenitor cellsPhase 21 trial
human retinal progenitor cellsPhase 21 trial
human retinal progenitor cellsPhase 21 trial
Active Trials
NCT04604899Completed30Est. Mar 2022
NCT06912633Recruiting60Est. Sep 2026
NCT03073733Completed84Est. Nov 2020
Novartis
NovartisBASEL, Switzerland
1 program
1
CPK850Phase 1/21 trial
Active Trials
NCT03374657Active Not Recruiting12Est. May 2026
Jerusalem Pharmaceuticals
Jerusalem PharmaceuticalsIsrael - Ramallah
1 program
1
cannabisPhase 11 trial
Active Trials
NCT03078309Unknown50Est. Dec 2021
Astellas
AstellasChina - Shenyang
2 programs
Development of Visual Function Evaluation MethodN/A1 trial
Development of Visual Function Evaluation Method (2)N/A1 trial
Active Trials
NCT03281005Completed18Est. Nov 2019
NCT03626207Completed8Est. Jun 2019
Genomics
GenomicsUK - Oxford
1 program
Retina Analysis-mosaicN/A1 trial
Active Trials
NCT03990727Unknown17,000Est. Sep 2025

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Thea PharmaQR-421a
Thea PharmaUltevursen
jCytehuman retinal progenitor cells
Thea PharmaRNA antisense oligonucleotide for intravitreal injection
jCytehuman retinal progenitor cells
jCytehuman retinal progenitor cells
NovartisCPK850
Jerusalem Pharmaceuticalscannabis
AstellasDevelopment of Visual Function Evaluation Method (2)
AstellasDevelopment of Visual Function Evaluation Method
GenomicsRetina Analysis-mosaic

Clinical Trials (11)

Total enrollment: 17,262 patients across 11 trials

NCT05176717Thea PharmaQR-421a

Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)

Start: Dec 2021Est. completion: Aug 2022
Phase 2/3Terminated
NCT05158296Thea PharmaUltevursen

Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)

Start: Dec 2021Est. completion: Oct 2022
Phase 2/3Terminated
NCT06912633jCytehuman retinal progenitor cells

Safety of a Single, Intravitreal Injection of 6.0M jCell (Famzeretcel) in Retinitis Pigmentosa (RP)

Start: Jun 2025Est. completion: Sep 202660 patients
Phase 2Recruiting
NCT05085964Thea PharmaRNA antisense oligonucleotide for intravitreal injection

An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa

Start: Sep 2021Est. completion: Oct 2022
Phase 2Terminated
NCT04604899jCytehuman retinal progenitor cells

Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa

Start: Dec 2020Est. completion: Mar 202230 patients
Phase 2Completed
NCT03073733jCytehuman retinal progenitor cells

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

Start: Mar 2017Est. completion: Nov 202084 patients
Phase 2Completed
NCT03374657NovartisCPK850

A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa

Start: Aug 2018Est. completion: May 202612 patients
Phase 1/2Active Not Recruiting
NCT03078309Jerusalem Pharmaceuticalscannabis

The Effects of Cannabis on Visual Functions in Healthy and Retinitis Pigmentosa Patients

Start: Sep 2018Est. completion: Dec 202150 patients
Phase 1Unknown
NCT03626207AstellasDevelopment of Visual Function Evaluation Method (2)

Development of Visual Function Evaluation Method (2)

Start: Oct 2018Est. completion: Jun 20198 patients
N/ACompleted
NCT03281005AstellasDevelopment of Visual Function Evaluation Method

Development of Visual Function Evaluation Method

Start: Nov 2017Est. completion: Nov 201918 patients
N/ACompleted
NCT03990727GenomicsRetina Analysis-mosaic

Phenotype Correlates Genotype of Inherited Retina Dystrophies, Retinitis Pigmentosa, Con>Rod Dystrophies.

Start: Aug 2009Est. completion: Sep 202517,000 patients
N/AUnknown

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 actively recruiting trials targeting 17,262 patients
7 companies competing in this space