Aplastic anaemia: who works on it

One card per institution. Pick a target or a country to narrow the list.

• All targets
• TP53 0
• CD19 3
• JAK 2
• NLRP3 0
• RUNX1 0
• STING 0
• ABL1 0
• CD38 1
• CSF1R 1
• EZH2 0
• FLT3 1
• GPX4 0
• KIT 0
• mTOR 0
• NRF2 0
• PI3K 0
• PPAR 0
• PRMT5 0
• TERT 0
• TLR4 0
• YAP 0

24 of 190 institutions, ordered by recent publications and open trials. This is not a ranking of quality.

Funded research projects that name this disease in their title, terms or summary and have not ended yet: who is funded, where, how much and until when. Organisations only; the funder's page has the details.

• NIH NCI · R01

  Integrating AI and Deep Mutational Scanning to Predict the Cancer-Promoting Effect of Shelterin Mutations

  University of California Berkeley· Berkeley, United States· US$2,197,943 awarded in fiscal year 2026· until Aug 2029

• NIH NHLBI · R01

  Mechanisms of somatic rescue in Fanconi anemia

  Boston Children's Hospital· Boston, United States· US$516,458 awarded in fiscal year 2026· until May 2031

• NIH NHLBI · R01

  Defining the Role of ATR in Hematopoiesis and Clonal Evolution

  Massachusetts General Hospital· Boston, United States· US$649,476 awarded in fiscal year 2026· until May 2031

• NIH NIAID · F31

  A novel genetic variant reveals the molecular and cellular mechanisms of human T cell responses

  Vanderbilt University· Nashville, United States· US$43,714 awarded in fiscal year 2026· until Aug 2029

• NIH NHLBI · K08

  Proteostasis in Hematopoietic Stem Cell Development

  University of California, San Diego· La Jolla, United States· US$168,795 awarded in fiscal year 2026· until Jun 2031

• European Commission (Horizon Europe) · HORIZON-ERC

  Uncovering Genetic Rescue and Functional Rebalancing in Telomeropathies Through Genome Editing and High-Resolution Screening (REBALANCE)

  Genome Research Limited Lbg· Saffron Walden, United Kingdom· EU contribution €2,496,154· until Dec 2031

  The disease is named only in the funder's project summary, not in the title.

• European Commission (Horizon Europe) · HORIZON-EIC

  Exploiting ex vivo expansion and deep multiomics profiling to bring novel, efficient and safer hematopoietic stem cell gene therapies to clinical application (X-PAND)

  Ospedale San Raffaele Srl· Milano, Italy· EU contribution €3,797,563· until Mar 2027· 4 countries

7 of 162 running projects, newest first. Listed amounts together: US$91.0m + €6.3m.

Sources: NIH RePORTER (U.S. National Institutes of Health, public domain) and CORDIS (European Commission, CC BY 4.0). Changes: filtered by disease, organisations matched to ROR.

Terminated, withdrawn and suspended trials with the reason the sponsor gave in the registry, quoted word for word.

• Suspended

  CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia

  NCT06622694· Institute of Hematology & Blood Diseases Hospital, China· Updated 4 Nov 2026

  “Suboptimal treatment response”Registry text, quoted unchanged. Source

• Terminated

  Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic Anemia

  NCT06695741· Institute of Hematology & Blood Diseases Hospital, China· Updated 4 Nov 2026

  “No significant improvement in efficacy was observed.”Registry text, quoted unchanged. Source

• Terminated

  Low-Dose Danazol for the Treatment of Telomere Related Diseases

  NCT03312400· National Heart, Lung, and Blood Institute (NHLBI)· Updated 27 Oct 2026

  “Study was stopped due to poor accrual”Registry text, quoted unchanged. Source

• Terminated

  A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

  NCT05600426· Boston Children's Hospital· Updated 19 Oct 2026

  “Funding terminated”Registry text, quoted unchanged. Source

• Withdrawn

  UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia

  NCT07001254· Anjali Sharathkumar· Updated 16 Oct 2026

  “Withdrawn in HawkIRB”Registry text, quoted unchanged. Source

• Terminated

  REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy

  NCT04409080· Regeneron Pharmaceuticals· Updated 22 Jun 2026

  “Sponsor Decision”Registry text, quoted unchanged. Source

• Withdrawn

  Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia

  NCT05018936· Peking Union Medical College Hospital· Updated 8 Jun 2026

  “The ethical application for the institution was not completed, and the research was not carried out.”Registry text, quoted unchanged. Source

• Withdrawn

  Enarodustat+CsA vs CsA in the Treatment of Newly-diagnosed TD-NSAA

  NCT07522996· Peking Union Medical College Hospital· Updated 4 Jun 2026

  “The registration of this research plan was incorrect. A new registration has been re-applied for.”Registry text, quoted unchanged. Source

• Withdrawn

  Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced AA

  NCT07532408· Peking Union Medical College Hospital· Updated 4 Jun 2026

  “The registration of this research plan was incorrect. A new registration has been done.”Registry text, quoted unchanged. Source

• Withdrawn

  Luspatercept Plus CsA vs CsA for the Treatment of Newly Diagnosed Non-Transfusion-Dependent NSAA

  NCT06424639· Peking Union Medical College Hospital· Updated 4 Jun 2026

  “This is a duplicate registration. The research plan and content have already been updated in another entry.”Registry text, quoted unchanged. Source

Trials whose registry record says individual participant data (IPD) will be shared.

• Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms

  NCT06303193· National Cancer Institute (NCI)· Source

• Low-Dose Danazol for the Treatment of Telomere Related Diseases

  NCT03312400· National Heart, Lung, and Blood Institute (NHLBI)· Source

• Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy

  NCT02979873· National Heart, Lung, and Blood Institute (NHLBI)· Source

• Haploidentical HCT for Severe Aplastic Anemia

  NCT04558736· St. Jude Children's Research Hospital· Source

• A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment

  NCT07786922· Swedish Orphan Biovitrum· Source

• A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia

  NCT03025698· Novartis Pharmaceuticals· Source

• Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia

  NCT05828108· National Heart, Lung, and Blood Institute (NHLBI)· Source

• Phase I Clinical Study of Haplo-HSCT Combined With Hypoxic 3D-Cultured Umbilical Cord MSC for the Treatment of SAA

  NCT07299123· Chinese PLA General Hospital· Source

• REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy

  NCT04409080· Regeneron Pharmaceuticals· Source

• Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia

  NCT05018936· Peking Union Medical College Hospital· Source

Find publications on this disease by a MeSH method term, for example a sequencing or imaging method.

• Single-Cell Analysis 3
• Mendelian Randomization Analysis 4
• CRISPR-Cas Systems 1
• Gene Expression Profiling 6
• Sequence Analysis, RNA 1
• Whole Genome Sequencing 4
• Exome Sequencing 2
• Proteomics 1
• Metabolomics 1
• Flow Cytometry 4
• Immunohistochemistry 1
• Xenograft Model Antitumor Assays 2
• Magnetic Resonance Imaging 2
• Machine Learning 3
• Cohort Studies 1
• Genome-Wide Association Study 3

• PRMT5 inhibition impairs Fanconi Anemia pathway-mediated homologous recombination and enhances the antitumor efficacy of Temozolomide in glioblastoma.

  Cell Death Dis, Apr 2026

• Patient-derived xenograft models of Fanconi anemia-associated head and neck cancer identify personalized therapeutic strategies.

  J Clin Invest, Dec 2025

See which targets of this disease your abstract mentions, and go to the people working on them.