Hematologic Diseases
Recent clinical, regulatory, research and industry developments relating to this disease.
Artificial Intelligence-Assisted Diagnostic Cytology and Genomic Testing for Hematologic Disorders.
Idecabtagene Vicleucel in Relapsed and Refractory Multiple Myeloma.
CRISPR-Cas9 technology and its application in haematological disorders.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 2 clinical trials expected to report results, the earliest in Q1 2027.
- Q1 2027Phase 1/1b Study of T-allo10 Infusion After HLA-Partially Matched Related or Unrelated TCR αβ+ T-cell/ CD19+ B-cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation (αβ Depleted-HSCT) in Children and Young Adults Affected by Hematologic Malignancies
- Q4 2028The Safety and Efficay Investigation of IL-5 CAR-T Cell Therapy for Patients With Refractory/Relapsed Eosinophilic Leukemia
Clinical MilestonesViewHide
- 2026-01-08Phase 1/1b Study of T-allo10 Infusion After HLA-Partially Matched Related or Unrelated TCR αβ+ T-cell/ CD19+ B-cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation (αβ Depleted-HSCT) in Children and Young Adults Affected by Hematologic MalignanciesResults expected Q1 2027
- 2025-12-10The Safety and Efficay Investigation of IL-5 CAR-T Cell Therapy for Patients With Refractory/Relapsed Eosinophilic LeukemiaResults expected Q4 2028
- 2030-03-01A Phase II Trial of CD34+ Enriched Transplants From HLA-Compatible Related or Unrelated Donors for Treatment of Patients With Hematologic MalignanciesWithdrawn
- 2026-05-01An Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of PUL-042 Inhalation Solution in Subjects With Hematologic Malignancies and Recipients of Stem Cell TransplantationWithdrawn
- 2025-09-22A Randomized, Placebo-controlled, Double-Blind (Sponsor Unblind), Parallel Group, Single Dose, Dose Escalation Phase I Study in Sickle Cell Disease Participants, to Evaluate the Safety, Tolerability, and Pharmacokinetics of GSK4172239DTerminated
- 2030-03-01ClinicalA Phase II Trial of CD34+ Enriched Transplants From HLA-Compatible Related or Unrelated Donors for Treatment of Patients With Hematologic MalignanciesWithdrawn
- 2026-05-01ClinicalAn Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of PUL-042 Inhalation Solution in Subjects With Hematologic Malignancies and Recipients of Stem Cell TransplantationWithdrawn
- 2026-04-25ClinicalA Study to Evaluate the Efficacy and Safety of CD7CAR-T Bridging to Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Refractory or Relapsed CD7 Positive Malignant Hematologic DiseasesPrimary completion
- 2026-01-08ClinicalPhase 1/1b Study of T-allo10 Infusion After HLA-Partially Matched Related or Unrelated TCR αβ+ T-cell/ CD19+ B-cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation (αβ Depleted-HSCT) in Children and Young Adults Affected by Hematologic MalignanciesResults expected Q1 2027
- 2025-12-10ClinicalThe Safety and Efficay Investigation of IL-5 CAR-T Cell Therapy for Patients With Refractory/Relapsed Eosinophilic LeukemiaResults expected Q4 2028
- 2025-09-22ClinicalA Randomized, Placebo-controlled, Double-Blind (Sponsor Unblind), Parallel Group, Single Dose, Dose Escalation Phase I Study in Sickle Cell Disease Participants, to Evaluate the Safety, Tolerability, and Pharmacokinetics of GSK4172239DTerminated
Clinical trials
The current development programme across all trial phases.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes8
- Hematologic Diseases2
- Artificial Intelligence1
- CRISPR-Cas Systems1
- Hematopoietic Stem Cell Transplantation1
- Immune System Diseases1
- Immunotherapy, Adoptive1
- Neoplasms1
- Receptors, Antigen, T-Cell1
Leading journals6
- Blood1
- Bone marrow transplantation1
- British journal of haematology1
- Cells1
- Journal of clinical oncology : official journal of the American Society of Clinical Oncology1
- The New England journal of medicine1
Leading researchers8
- Yakoub-Agha I2
- Ackermann J1
- Anderson LD Jr1
- Ayuk F1
- Bachy E1
- Basak GW1
- Berdeja J1
- Berger C1
Affiliations (unnormalised)6
- and German Cancer Research Center1
- Erasmus Medical Center1
- Fondazione IRCCS Policlinico San Matteo and University of Pavia1
- From the Jerome Lipper Multiple Myeloma Center1
- Hospital de la Princesa1
- Hospital Universitario Puerta de Hierro Majadahonda-Universidad Autónoma de Madrid1
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Disease profile
A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.
Hematologic diseases are disorders of the blood and blood-forming tissues. The literature grounding here emphasizes their diagnosis, genetics, and therapy, reflecting a broad group of inherited and acquired conditions affecting hematology.
The supplied grounding does not support a single unified cause for hematologic diseases because it refers to a broad disease category rather than one disorder. It does note that inherited hematological disorders can arise from disease-causing mutations, and that hematological malignancies involve molecular mechanisms that can be studied at the genetic level.
The grounding supports that hematologic diseases can involve abnormalities in blood and blood-forming tissues, including mutation-driven disorders and hematological malignancies. It also indicates that molecular mechanisms are relevant to these conditions, but does not provide a single shared biological pathway across the category.
The supplied grounding does not identify general risk factors for hematologic diseases as a category. It only indicates that inherited hematological disorders are linked to underlying mutations, which is a causal feature rather than a population risk factor.
The grounding supports diagnosis-focused approaches including cytology, cytogenetics, immunophenotyping, sequencing, and AI-assisted analysis to improve diagnostic accuracy and efficiency. For therapy, it supports gene therapy and genome editing approaches such as CRISPR-Cas9 for inherited hematological disorders, and broader molecularly informed treatment development for hematologic malignancies.
AI-generated summary grounded in MeSH and 2 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.
Reference
Authoritative identity, definition & identifiers.
Disorders of the blood and blood forming tissues.
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.