A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF)

CK0801 (Cord blood-derived T-regulatory cells) consists of ex vivo expanded T-regulatory
cells with a flow cytometry phenotype of ≥ 60% CD4+CD25+ T-regulatory (Treg) cells and < 10%
CD4-CD8+ T-cytotoxic/suppressor cells. The cellular starting material of CK0801 is a single
unit of umbilical cord blood (CBU) from a normal, healthy unrelated donor. Because Treg cells
are present only at low frequency in circulating blood or umbilical cord blood, production of
clinically relevant Treg cell doses requires ex vivo enrichment and expansion of Treg cells
with a CD4+CD25+ phenotype. In their natural state, T-regulatory (Treg) cells play an
important role in maintaining immune homeostasis and limiting autoimmune responses by
modulating both innate and adaptive immunity. Based on literature reports of animal studies
showing induction of immune tolerance by Treg cells in autoimmune diseases, graft-versus-host
disease, and solid organ transplant rejection, the administration of Treg cells in human
clinical trials has become an attractive strategy to induce immune tolerance in patients in a
variety of clinical settings.

Inclusion Criteria:

1. Subjects who fulfill the diagnostic criteria of bone marrow failure syndrome
including: aplastic anemia, myelodysplastic syndrome, or myelofibrosis.

2. HLA matched (≥ 3/6) cord blood unit available for CK0801 generation.

3. Subjects age 18 to 70 years.

4. Bilirubin ≤ 2 x ULN and SGPT (ALT) ≤ 2 x ULN (unless Gilbert's syndrome).

5. Calculated creatinine clearance of > 50mL/min using the Cockcroft-Gault equation.

6. Zubrod performance status ≤ 2.

7. Female subjects of child bearing potential (FPCP) must have a negative urine or serum
pregnancy test. NOTE: FPCP is defined as premenopausal and not surgically sterilized.
FPCP must agree to use maximally effective birth control or to abstain from
heterosexual activity throughout the study. Effective contraceptive methods include
intra-uterine device, oral and/or injectable hormonal; contraception, or 2 adequate
barrier methods (e.g., cervical cap with spermicide, diaphragm with spermicide).

8. Subject has agreed to abide by all protocol required procedures including
study-related assessments, visits and long term follow up.

9. Subject is willing and able to provide written informed consent.

Exclusion Criteria:

1. Subject has received an investigational agent within 4 weeks prior to CK0801 infusion.

2. Subject has received radiation or chemotherapy within 21 days prior to CK0801
infusion.

3. Subject has received prior cord blood-derived T-regulatory therapy.

4. HIV seropositivity.

5. Subject has uncontrolled infection, not responding to appropriate antimicrobial agents
after seven days of therapy. The Protocol PI is the final arbiter of eligibility.

6. Subjects with uncontrolled inter-current illness that in the opinion of the
investigator would place the patient at greater risk of severe toxicity and/or impair
the activity of CK0801

7. Subjects is pregnant or breastfeeding.

8. Bone marrow failure caused by stem cell transplantation.

9. Subjects who are unable to provide consent or who, in the opinion of the Investigator
will be unlikely to fully comply with protocol requirements.