VivoSeq
Ophthalmology

Retinitis Pigmentosa

53

Pipeline Programs↗

27

Active Trials↗

28

Companies Involved↗

Retinitis Pigmentosa is a moderately active landscape with 53 programs across 28 companies. The most active players are Zhongmou Therapeutics, VeonGen Therapeutics GmbH, TO Pharmaceuticals. 5 therapies are approved with 6 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

63/100

Pipeline Velocity

25/100

Mech. Concentration

11/100

Completion Rate

84/100

Attrition Rate

9/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

Z

Zhongmou Therapeutics

2 programs

Phase 1V

VeonGen Therapeutics GmbH

1 program

Phase 1T

TO Pharmaceuticals

1 program

Phase 1AbbVie logo

AbbVie

4 programs

Phase 2B

Beacon Therapeutics

3 programs

Phase 2N

Neurotech Pharmaceuticals

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

73%

Small molecule

44 of 60 classified programs — the dominant modality here.

27%ALL OTHERSGene therapy813%Cell therapy610%Protein / enzyme12%RNA therapy12%

Small molecule leads; the ring shows all 60 classified programs, each share of the total. Modality classified for 99% of 74 pipeline programs.

Analysis

Trial Status

80

clinical trials

Completed36 · 45%
Recruiting12 · 15%
Unknown status10 · 13%
Active, not recruiting10 · 13%
Terminated7 · 9%
Not yet recruiting4 · 5%
Other1 · 1%

Clinical Trials

Trial Activity

80 trials

A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
Recruiting·OOctant, Inc.·Phase 1/2·NCT07408232·50 enrolled
Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients
Recruiting·ZZhongmou Therapeutics·Phase 1·NCT06292650·12 enrolled
A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa
Recruiting·KKiora Pharmaceuticals, Inc.·Phase 2·NCT06628947·36 enrolled
Safety and Tolerability Study of Gene Editing Drug ZVS203e in Participants With Retinitis Pigmentosa
Recruiting·PPeking University Third Hospital·Phase 1·NCT05805007·9 enrolled
An Open-label, Dose-ascending Study of IGT001 for Retinitis Pigmentosa
Recruiting·SShanghai General Hospital, Shanghai Jiao Tong University School of Medicine·Phase 1·NCT06936787·21 enrolled
Safety of a Single, Intravitreal Injection of 6.0M jCell (Famzeretcel) in Retinitis Pigmentosa (RP)
Recruiting·JjCyte, Inc·Phase 2·NCT06912633·60 enrolled
Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
Recruiting·VVeonGen Therapeutics GmbH·Phase 1·NCT06291935·6 enrolled
A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)
Recruiting·KKiora Pharmaceuticals, Inc.·Phase 1/2·NCT05282953·48 enrolled
Safety and Efficacy of Stem Cell Small Extracellular Vesicles in Patients With Retinitis Pigmentosa
Recruiting·MMahidol University·Phase 1/2·NCT06242379·15 enrolled
Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa
Recruiting·AAAVantgarde Bio Srl·Phase 1/2·NCT06591793·15 enrolled
A Study to Investigate the Safety of DSP-3077 After a Unilateral Eye Injection in Male and Female Participants 18 Years of Age or Older With Retinitis Pigmentosa
Recruiting·Sumitomo Pharma America, Inc. logoSumitomo Pharma America, Inc.·Phase 1/2·NCT06891885·12 enrolled
BS01 in Patients With Retinitis Pigmentosa
Recruiting·BBionic Sight LLC·Phase 1/2·NCT04278131·20 enrolled
Oral N-acetylcysteine for Retinitis Pigmentosa
Active, not recruiting·JJohns Hopkins University·Phase 3·NCT05537220·485 enrolled
A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa
Active, not recruiting·Novartis logoNovartis·Phase 1/2·NCT03374657·12 enrolled
Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
Active, not recruiting·BBeacon Therapeutics·Phase 1/2·NCT03316560·29 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)