Latest FDA grants Fast Track designation to CK0803 in ALS

Cord blood regulatory T cell therapeutics

Cellenkos is a clinical-stage biotechnology company developing allogeneic, tissue-directed, cord blood-derived T-regulatory cell therapies to resolve inflammation in autoimmune, neurodegenerative and inflammatory diseases.

FDA Fast Track · CK0803 · ALSPhase 2 cleared · CK0801 · Aplastic anemiaIND cleared · CK0802 · Steroid-refractory GVHDOrphan Drug · CK0804 · MyelofibrosisOrphan Drug · CK0803 · ALSOrphan Drug · CK0801 · Aplastic anemiaU.S. Patent US12472182B2
0Patients treated across Cellenkos clinical programs
0Dose-limiting toxicities observed across the Treg platform
0FDA designations: Fast Track and three Orphan Drug
0Product candidates spanning five therapeutic areas

Our science

Restoring the immune system's peacekeepers

Animated diagram of regulatory T cells, effector T cells and tissue Healthy tissue Cellenkos Treg product
01

Tregs protect the body from attack by T cells

Regulatory T cells (Tregs) suppress inflammation and keep T cells and other compartments of the immune system from attacking our own body.

02

Defective Tregs lead to autoimmune disease

Defective Tregs can no longer protect the body from the harmful effect of T cells, leading to autoimmune diseases including lupus, multiple sclerosis and rheumatoid arthritis, inflammatory disorders like Crohn's disease, and bone marrow failure syndromes like aplastic anemia.

03

A first-in-class, cord blood-derived Treg product

Clinically manufactured using Cellenkos' proprietary CRANE® platform from a single qualified umbilical cord blood unit, Cellenkos' Treg product shows superior T cell suppression and anti-inflammatory properties.

04

Infusion overcomes the existing Treg defect

Infusion of Cellenkos' Treg product replaces the defective Tregs and protects the body from T cell attack — releasing IL-10, consuming IL-2 that fuels aggressive T cells, and neutralizing antigen-presenting cells.

05

Restoring immune homeostasis

By preventing T cell attack against one's own body, Cellenkos' Treg product helps restore immune homeostasis — creating the conditions for disease control and tissue recovery.

The CRANE® platform

Cytokine-directed, tissue-targeted Tregs go where inflammation is

CRANE® generates disease-specific Treg products enriched for homing markers, so each candidate travels directly to the tissue where unresolved inflammation drives disease. Select a program to trace its route.

Body map showing where each Treg product homes after IV infusion IV

CK0803

Neurotropic Tregs that cross into the CNS

Engineered to express high levels of CXCR3 and CD11a (LFA-1), CK0803 cells are designed to traffic across the blood-brain and blood-spinal cord barriers and migrate along CXCL10 gradients toward inflamed microglia.

Indication
Amyotrophic lateral sclerosis
Homing
CXCR3 / CXCL10 axis
Status
Phase 1/1b · FDA Fast Track
Cell therapy cleanroom with a scientist handling a cryopreserved cord blood product
  • Off-the-shelfCryopreserved product with a reported shelf life of nearly three years, ready for immediate use.
  • No matchingNo HLA or ABO matching required — derived from qualified U.S. public cord blood units (21 CFR Part 1271).
  • OutpatientPeripheral IV infusion without conditioning chemotherapy or IL-2 support.
  • Protected IPU.S. patent US12472182B2, granted November 18, 2025, covering compositions of regulatory T cells and methods of producing and using them.

Our pipeline

Six Treg products across eight therapeutic areas

Program Indication PreclinicalPhase 1Phase 2Phase 3

All product candidates are investigational and have not been approved by the FDA for any indication.

News center

Latest press releases

All releases on PR Newswire ↗

Publications

Peer-reviewed clinical and translational research

Cellenkos' clinical and preclinical research on cord blood-derived Tregs has been published in NEJM Evidence, Blood, Blood Advances, Annals of Internal Medicine, iScience, Cytotherapy, Cells and Frontiers journals, and presented at the American Society of Hematology Annual Meeting.

  • 2025
    NEJM Evidence · 4(5)

    Clinical safety and preliminary efficacy of regulatory T cells for ALS

    Shneider NA, et al. · DOI 10.1056/EVIDoa2400249 · Editorial: Regulatory T cell-based therapies — a new piece of the ALS therapeutic puzzle?

  • 2025
    Blood · 146(Suppl 1):5602 · ASH 2025

    Dose intensive regimen of CK0804 Tregs in myelofibrosis

    Masarova L, et al. · DOI 10.1182/blood-2025-5602

  • 2025
    Frontiers in Immunology · 16:1449693

    Ruxolitinib synergizes with regulatory T cells to improve inflammation but has no added benefits in decreasing albuminuria in SLE

    Lyu M-A, et al. · DOI 10.3389/fimmu.2025.1449693

  • 2024
    iScience · 27:110830

    CXCR4-enriched T regulatory cells preferentially home to bone marrow and resolve inflammation

    Huang M, et al. · DOI 10.1016/j.isci.2024.110830

  • 2024
    Frontiers in Transplantation · 3:1448650

    Cord blood T regulatory cells synergize with ruxolitinib to improve GVHD outcomes

    Zeng K, et al. · DOI 10.3389/frtra.2024.1448650

  • 2024
    NEJM Evidence · 3(6)

    Phase 1 study of CK0801 in treatment of bone marrow failure syndromes

    Kadia TM, Huang M, et al. · DOI 10.1056/EVIDoa2300362 · Editorial: Adoptive T regulatory cell therapy takes an important step

  • 2024
    Blood · 144(Suppl 1):999 · ASH 2024 oral presentation

    A phase Ib, open-label study of add-on therapy with CK0804 in participants with myelofibrosis and suboptimal response to ruxolitinib

    Masarova L, et al. · View abstract in Blood

  • 2023
    Blood Advances · 7(13):3075

    Randomized, double-blinded, placebo-controlled trial of allogeneic cord blood T-regulatory cells for treatment of COVID-19 ARDS

    Gladstone DE, et al. · DOI 10.1182/bloodadvances.2022009619

  • 2023
    Frontiers in Immunology · 14:1217121

    Allogeneic cord blood regulatory T cells decrease dsDNA antibody and improve albuminuria in systemic lupus erythematosus

    Lyu M-A, et al. · DOI 10.3389/fimmu.2023.1217121

  • 2023
    Cells · 12:1880

    Adjunct therapy with T regulatory cells decreases inflammation and preserves the anti-tumor activity of CAR T cells

    Zeng K, et al. · DOI 10.3390/cells12141880

  • 2023
    Cytotherapy · 25:245–253

    Allogeneic cord blood regulatory T cells can resolve lung inflammation

    Lyu M-A, et al. · DOI 10.1016/j.jcyt.2022.10.009

  • 2020
    Annals of Internal Medicine · 173(5):OC1 · Annals On Call

    Regulatory T cells: treatment for COVID-19?

    Centor RM, D’Alessio FR · DOI 10.7326/A19-0037

  • 2020
    Annals of Internal Medicine · 173(10):852–853

    Regulatory T cells for treating patients with COVID-19 and acute respiratory distress syndrome: two case reports

    Gladstone DE, et al. · DOI 10.7326/L20-0681 · Full text (PMC)

  • 2015
    Blood · 125(9):1502–1506

    Ex vivo fucosylation of third-party human regulatory T cells enhances anti–graft-versus-host disease potency in vivo

    Parmar S, et al. · DOI 10.1182/blood-2014-10-603449

  • 2014
    Cytotherapy · 16(1):90–100

    Third-party umbilical cord blood-derived regulatory T cells prevent xenogenic graft-versus-host disease

    Parmar S, et al. · DOI 10.1016/j.jcyt.2013.07.009

  • 2006
    Cytotherapy · 8(2):149–157

    Ex vivo expanded umbilical cord blood T cells maintain naive phenotype and TCR diversity

    Parmar S, et al. · DOI 10.1080/14653240600620812

Fluorescent regulatory T cells under a microscope

Join our team

Build the next class of living medicines in Houston

We are growing our footprint in the Houston life sciences ecosystem. If you work in cell therapy manufacturing, clinical operations, translational science or quality, we would like to hear from you.

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