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Retinitis Pigmentosa

24
Pipeline Programs
27
Companies
38
Clinical Trials
5 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
4
9
7
2
2
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
467%
RNA Therapeutic
233%
+ 36 programs with unclassified modality

Competitive Landscape

26 companies ranked by most advanced pipeline stage

Ocugen
OcugenPA - Malvern
3 programs
1
1
Sub-Retinal Administration of OCU400-301Phase 31 trial
OCU400 Low DosePhase 1/21 trial
OCU400N/A1 trial
Active Trials
NCT06574997No Longer Available
NCT05203939Active Not Recruiting22Est. Mar 2027
NCT06388200Active Not Recruiting140Est. Feb 2027
Utah Medical
Utah MedicalIreland - Dublin
1 program
1
N-acetylcysteinePhase 31 trial
Active Trials
NCT05537220Active Not Recruiting485Est. May 2030
Biocorp
BiocorpFrance - Issoire
3 programs
1
2
QR-421aPhase 2/31 trial
UltevursenPhase 2/3RNA Therapeutic1 trial
RNA antisense oligonucleotide for intravitreal injectionPhase 21 trial
Active Trials
NCT05085964Terminated21Est. Oct 2022
NCT05176717Terminated5Est. Aug 2022
NCT05158296Terminated7Est. Oct 2022
Nanoscope Therapeutics
4 programs
1
1
Gene Therapy Product-MCO-010Phase 2Gene Therapy1 trial
Gene Therapy product:vMCO-IPhase 1/2Gene Therapy1 trial
Gene Therapy product-MCO-010N/AGene Therapy1 trial
Gene Therapy product:vMCO-IN/AGene Therapy1 trial
Active Trials
NCT06162585Active Not Recruiting18Est. Sep 2027
NCT05921162Enrolling By Invitation11Est. May 2025
NCT04919473Completed11Est. Oct 2020
+1 more trials
Neurotech Pharmaceuticals
3 programs
3
Low Dose NT-501Phase 21 trial
NT-501Phase 21 trial
NT-501Phase 21 trial
Active Trials
NCT00447993Completed67Est. May 2009
NCT00447980Completed73Est. Apr 2010
NCT01530659Completed22Est. Jul 2019
Dompé
DompéItaly - L'Aquila
2 programs
1
1
rhNGF 180 µg/ml eye drops solutionPhase 21 trial
rhNGF 60 µg/ml eye drops solutionPhase 1/21 trial
Active Trials
NCT02110225Completed50Est. Nov 2015
NCT02609165Completed45Est. Feb 2017
Aldeyra Therapeutics
Aldeyra TherapeuticsMA - Lexington
1 program
1
ADX-2191Phase 21 trial
Active Trials
NCT05392179Completed8Est. Jun 2023
SparingVision
SparingVisionFrance - Paris
2 programs
1
SPVN06Phase 1/21 trial
Ophthalmic examinationsN/A1 trial
Active Trials
NCT04285398Active Not Recruiting82Est. Jun 2026
NCT05748873Recruiting33Est. Sep 2030
AbbVie
AbbVieNORTH CHICAGO, IL
1 program
1
400 µg Brimonidine Tartrate ImplantPhase 1/21 trial
Active Trials
NCT00661479Completed21Est. May 2010
Bionic Sight
Bionic SightNY - New York
1 program
1
BS01Phase 1/21 trial
Active Trials
NCT04278131Recruiting20Est. Dec 2029
Sandoz
SandozAustria - Kundl
1 program
1
CPK850Phase 1/21 trial
Active Trials
NCT03374657Active Not Recruiting12Est. May 2026
ProQR Therapeutics
ProQR TherapeuticsMA - Cambridge
1 program
1
QR-421aPhase 1/21 trial
Active Trials
NCT03780257Completed20Est. Oct 2021
ReNeuron
ReNeuronUK - Bridgend
1 program
1
hRPCPhase 1/21 trial
Active Trials
NCT02464436Unknown29Est. Dec 2023
Zambon
ZambonItaly - Bresso
1 program
1
NAC effervescent tabletsPhase 11 trial
Active Trials
NCT03999021Active Not Recruiting30Est. Jun 2027
Ray Therapeutics
Ray TherapeuticsCA - Berkeley
1 program
1
RTx-015Phase 11 trial
Active Trials
NCT06460844Active Not Recruiting10Est. Oct 2030
VeonGen Therapeutics
VeonGen TherapeuticsGermany - Planegg
1 program
1
VG901Phase 11 trial
Active Trials
NCT06291935Recruiting6Est. Apr 2026
Zhongmou Therapeutics
1 program
1
ZM-02-LPhase 11 trial
Active Trials
NCT06292650Recruiting12Est. Dec 2028
Thea Pharma
Thea PharmaMA - Waltham
3 programs
RNA antisense oligonucleotide for intravitreal injectionPHASE_2
QR-421aPHASE_2_3
UltevursenPHASE_2_3RNA Therapeutic
jCyte
jCyteCA - Newport Beach
3 programs
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
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
Mobius Therapeutics
Mobius TherapeuticsMO - St. Louis
1 program
44Ch Bionic Eye DeviceN/A1 trial
Active Trials
NCT03406416Completed4Est. Dec 2020
PYC Therapeutics
PYC TherapeuticsAustralia - Nedlands
1 program
Natural History of PRPF31 Mutation-Associated Retinal DystrophyN/A1 trial
Active Trials
NCT05573984Active Not Recruiting50Est. Nov 2026
Genomics
GenomicsUK - Oxford
1 program
Retina Analysis-mosaicN/A1 trial
Active Trials
NCT03990727Unknown17,000Est. Sep 2025
Intelligent Therapeutics
1 program
Retinal Implant SystemN/A1 trial
Active Trials
NCT00427180Unknown20Est. Dec 2010
Jerusalem Pharmaceuticals
Jerusalem PharmaceuticalsIsrael - Ramallah
1 program
cannabisPHASE_11 trial
Active Trials
NCT03078309Unknown50Est. Dec 2021
Novartis
NovartisBASEL, Switzerland
1 program
CPK850PHASE_1_2

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
OcugenSub-Retinal Administration of OCU400-301
Utah MedicalN-acetylcysteine
BiocorpQR-421a
BiocorpUltevursen
jCytehuman retinal progenitor cells
Aldeyra TherapeuticsADX-2191
BiocorpRNA antisense oligonucleotide for intravitreal injection
Nanoscope TherapeuticsGene Therapy Product-MCO-010
jCytehuman retinal progenitor cells
jCytehuman retinal progenitor cells
DompérhNGF 180 µg/ml eye drops solution
Neurotech PharmaceuticalsNT-501
Neurotech PharmaceuticalsNT-501
Neurotech PharmaceuticalsLow Dose NT-501
SparingVisionSPVN06

Showing 15 of 37 trials with date data

Clinical Trials (38)

Total enrollment: 18,611 patients across 38 trials

NCT06388200OcugenSub-Retinal Administration of OCU400-301

A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa

Start: Jun 2024Est. completion: Feb 2027140 patients
Phase 3Active Not Recruiting
NCT05537220Utah MedicalN-acetylcysteine

Oral N-acetylcysteine for Retinitis Pigmentosa

Start: Oct 2023Est. completion: May 2030485 patients
Phase 3Active Not Recruiting

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 20225 patients
Phase 2/3Terminated

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 20227 patients
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

A Study in Subjects With Retinitis Pigmentosa

Start: Jul 2022Est. completion: Jun 20238 patients
Phase 2Completed
NCT05085964BiocorpRNA 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 202221 patients
Phase 2Terminated
NCT04945772Nanoscope TherapeuticsGene Therapy Product-MCO-010

Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]

Start: Jul 2021Est. completion: Jan 202427 patients
Phase 2Completed
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
NCT02609165DompérhNGF 180 µg/ml eye drops solution

Nerve Growth Factor Eye Drops Treatment in Patients With Retinitis Pigmentosa and Cystoid Macular Edema

Start: May 2015Est. completion: Feb 201745 patients
Phase 2Completed

Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa

Start: Jan 2012Est. completion: Jul 201922 patients
Phase 2Completed

A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa

Start: Jan 2007Est. completion: Apr 201073 patients
Phase 2Completed

A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa

Start: Jan 2007Est. completion: May 200967 patients
Phase 2Completed

Promising ROd-cone DYstrophy Gene therapY

Start: Apr 2023Est. completion: Sep 203033 patients
Phase 1/2Recruiting
NCT05203939OcugenOCU400 Low Dose

Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis

Start: Jan 2022Est. completion: Mar 202722 patients
Phase 1/2Active Not Recruiting

BS01 in Patients With Retinitis Pigmentosa

Start: Feb 2020Est. completion: Dec 202920 patients
Phase 1/2Recruiting
NCT04919473Nanoscope TherapeuticsGene Therapy product:vMCO-I

Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa

Start: Oct 2019Est. completion: Oct 202011 patients
Phase 1/2Completed

Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene

Start: Mar 2019Est. completion: Oct 202120 patients
Phase 1/2Completed

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

Safety and Tolerability of hRPC in Retinitis Pigmentosa

Start: Dec 2015Est. completion: Dec 202329 patients
Phase 1/2Unknown
NCT02110225DompérhNGF 60 µg/ml eye drops solution

A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP)

Start: Jan 2014Est. completion: Nov 201550 patients
Phase 1/2Completed
NCT00661479AbbVie400 µg Brimonidine Tartrate Implant

An Exploratory Study to Evaluate the Safety of Brimonidine Intravitreal Implant in Patients With Retinitis Pigmentosa

Start: Jul 2008Est. completion: May 201021 patients
Phase 1/2Completed

Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa or Choroideremia Patients (ENVISION)

Start: Aug 2024Est. completion: Oct 203010 patients
Phase 1Active Not Recruiting

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

Start: Feb 2024Est. completion: Dec 202812 patients
Phase 1Recruiting

Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene

Start: Sep 2023Est. completion: Apr 20266 patients
Phase 1Recruiting
NCT03999021ZambonNAC effervescent tablets

FIGHT-RP 1 Extension Study

Start: Jun 2019Est. completion: Jun 202730 patients
Phase 1Active Not Recruiting

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

Start: Sep 2018Est. completion: Dec 202150 patients
Phase 1Unknown

Expanded Access Program for RP in Adults

N/ANo Longer Available
NCT06162585Nanoscope TherapeuticsGene Therapy product-MCO-010

Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study

Start: Dec 2023Est. completion: Sep 202718 patients
N/AActive Not Recruiting
NCT05921162Nanoscope TherapeuticsGene Therapy product:vMCO-I

A Long-Term Follow-Up Study in Subjects Who Received vMCO-I Administered Via Intravitreal Injection

Start: Jul 2023Est. completion: May 202511 patients
N/AEnrolling By Invitation
NCT05573984PYC TherapeuticsNatural History of PRPF31 Mutation-Associated Retinal Dystrophy

Natural History of PRPF31 Mutation-Associated Retinal Dystrophy

Start: Jul 2022Est. completion: Nov 202650 patients
N/AActive Not Recruiting
NCT04285398SparingVisionOphthalmic examinations

Prospective Natural History Study of Retinitis Pigmentosa

Start: Feb 2020Est. completion: Jun 202682 patients
N/AActive Not Recruiting
NCT03626207AstellasDevelopment of Visual Function Evaluation Method (2)

Development of Visual Function Evaluation Method (2)

Start: Oct 2018Est. completion: Jun 20198 patients
N/ACompleted
NCT03406416Mobius Therapeutics44Ch Bionic Eye Device

Study of a Suprachoroidal Retinal Prosthesis

Start: Feb 2018Est. completion: Dec 20204 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

IRIS PILOT - Extended Pilot Study With a Retinal Implant System

Start: Dec 2006Est. completion: Dec 201020 patients
N/AUnknown

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals
5 actively recruiting trials targeting 18,611 patients
27 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.