Back to discover
Disease

Retinitis pigmentosa and erythrocytic microcytosis

Late-stage therapeutic development

Also known as RPEM, retinitis pigmentosa and erythrocytic microcytosis; RPEM.

24
Clinical trials
5
Associated genes
1
Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 3 clinical trials expected to report results, the earliest in Q2 2029.
Upcoming Milestones
Clinical Milestones10View all 10
Results posted 1
Results expected 3
Recently completed 3
Other updates 3
+2 more in the activity timeline below
Activity timeline10

Clinical trials

16 sponsors · 3 new · 2 completed in the last 12 months (net +2)

The current development programme across all trial phases.

Clinical programme
24
All trials
6
Active
18
Late-stage
6
Completed
Late-stage studies

A Phase 3, Double-Blind, Randomized Trial Evaluating the Efficacy and Safety of Elritercept (TAK-226) Compared to Placebo in Participants With Myelofibrosis and Anemia on Concurrent Ruxolitinib Therapy

Phase 3 · Recruiting · Takeda

A Phase 3, Multicenter, Open-Label, Randomized Trial to Compare the Efficacy and Safety of Elritercept Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in ESA-naïve Adult Participants Who Require Red Blood Cell Transfusions

Phase 3 · Recruiting · Takeda

A Phase 3, Double-blind, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK Inhibitor Therapy and Who Require Red Blood Cell Transfusions

Phase 3 · Active, not recruiting · Celgene

A Randomized, Controlled, Masked, Multi-center Study Evaluating the Efficacy, Safety, and Tolerability of Two Doses of AGTC-501 Compared to an Untreated Control Group in Male Participants With X-linked Retinitis Pigmentosa

Phase 2/3 · Active, not recruiting · Beacon Therapeutics

NAC Attack, A Phase III, Multicenter, Randomized, Parallel, Double Masked, Placebo-Controlled Study Evaluating the Efficacy and Safety of Oral N-Acetylcysteine in Patients With Retinitis Pigmentosa

Phase 3 · Active, not recruiting · Johns Hopkins University

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period

Phase 3 · Active, not recruiting · Agios Pharmaceuticals, Inc.
Recently completed

Optimizing Dosing Strategies in Oral Iron Supplementation

Phase 4 · Completed · Luzerner Kantonsspital

A Multicenter, Randomized, Double-blind, Parallel Controlled Clinical Study on the Efficacy and Safety of Compound E Jiao Jiang(cEJJ）in the Treatment of Postpartum Anemia

Phase 4 · Completed · DongE E Jiao Coporation Limited

Comparative Analysis of Oral Iron With Injectable Ferric Carboxymaltose for Treatment of Post Partum Iron Deficiency Anaemia

Phase 2/3 · Completed · ESI-PGIMSR, Basaidarapur

Hydroxyurea Therapy for Neurological and Cognitive Protection in Pediatric Sickle Cell Anemia in Uganda: A Single Arm Open Label Trial, "BRAIN SAFE II"

Phase 3 · Completed · Global Health Uganda LTD

Prevention of Iron Deficiency Anemia Post-delivery (PRIORITY Trial): A Randomized Controlled Trial of the Global Network for Women's and Children's Health Research

Phase 3 · Completed · NICHD Global Network for Women's and Children's Health

The Effect of Preoperative Ferric Carboxymaltose Administration on Mortality in Geriatric Hip Fractures

Phase 4 · Completed · Istanbul University

Associated genes

5 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

TRNT10.76CRBN0.51RUSC10.04CD300LD0.04NTAQ10.03

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Protein cereblonvia CRBN0.51

Reference

Authoritative identity, definition & identifiers.

Synonyms

RPEM, retinitis pigmentosa and erythrocytic microcytosis; RPEM

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.