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NCT04819841RECRUITINGanonymous

A Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease

Sponsor

Source record

Kamau Therapeutics

Phase

Source record

Phase 1/2

Modality

AI-normalized

small molecule

Target

AI-normalized

CRISPR-Cas9 edited and sickle mutation-corrected hematopoietic stem cells (HSPCs)

Indication / condition

AI-normalized

Sickle Cell Disease

Intervention

Source record

nula-cel Drug Product

Source & freshness

Source record

NCT ID

NCT04819841

Original source

ClinicalTrials.gov

Source last updated

Mar 11, 2026

Ingested at

Jun 12, 2026

Internal sync

Jun 12, 2026

Model version

trialsignal-ai-v1

Normalized confidence

96%

Validation status

validated

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View original source fields

NCT ID

NCT04819841

Title

A Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease

Sponsor

Kamau Therapeutics

Status

RECRUITING

Phase

Phase 1/2

Condition raw

Sickle Cell Disease

Condition normalized

Sickle Cell Disease

Modality raw

small molecule

Modality normalized

small molecule

Target raw

CRISPR-Cas9 edited and sickle mutation-corrected hematopoietic stem cells (HSPCs)

Target normalized

CRISPR-Cas9 edited and sickle mutation-corrected hematopoietic stem cells (HSPCs)

Interventions

nula-cel Drug Product

Public preview

Source record

Kamau Therapeutics is advancing nula-cel, a gene therapy product aimed at treating severe Sickle Cell Disease (SCD) through the correction of the HbS mutation. The market for SCD therapies is expanding, with increasing demand for innovative treatments that address the underlying genetic causes of the disease. As a first-in-human study, the results could position Kamau Therapeutics favorably against competitors in the gene therapy space, particularly those focusing on hematological disorders. The estimated completion dates suggest a timeline that could allow for early market entry if successful, potentially leading to significant revenue opportunities given the high unmet need in this patient population. However, the presence of established players in the gene therapy market necessitates careful monitoring of competitive developments and regulatory pathways.

AI-generated analysis supports research triage only. Verify source records, publications, sponsor disclosures and IP databases before making diligence decisions. Model: trialsignal-ai-v1.

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