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Gene Therapy

Stem Cell Gene Therapy for Sickle Cell Disease

Phase 1 & 2
Waitlist Available
Led By Gary Schiller, MD
Research Sponsored by Donald B. Kohn, M.D.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Inadequate clinical response to hydroxyurea (HU), defined as any one of the following outcomes, while on HU for at least 3 months:
- 2 or more acute sickle pain crises requiring hospitalization
Must not have
Patient must not have any known cancer or other malignant disease or active infection by CT or MRI of head, chest or ultrasound of abdomen
Poorly controlled hypertension as determined by BP with systolic >135 or diastolic >95 mmHg despite treatment.
Screening 3 weeks
Treatment Varies
Follow Up up to 24 months
Awards & highlights


This trial will test a new treatment for sickle cell disease that uses the patient's own blood cells.

Who is the study for?
Adults with severe sickle cell disease who have had complications like pain crises, stroke, or acute chest syndrome despite treatment can join this trial. They must not be pregnant, have HIV/HCV/HTLV-1/hepatitis B/Cancer, and should not have a suitable sibling donor for transplant.Check my eligibility
What is being tested?
The trial is testing a gene therapy where patients' own blood stem cells are modified with the βAS3-FB vector to potentially treat sickle cell disease. It's in Phase I to check safety and initial effectiveness.See study design
What are the potential side effects?
Potential side effects may include reactions at the infusion site, immune response against the modified cells, or other unforeseen issues due to genetic modification of blood cells.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
My condition did not improve after taking hydroxyurea for 3 months.
I have been hospitalized for sickle cell pain crises at least twice.
I can care for myself but may need occasional help.
I am 18 years old or older.
I've had a severe lung issue not caused by asthma in the last 2 years despite treatment.
I have had a stroke or a lasting brain problem for more than a day.
I had episodes of swollen fingers or toes in my childhood.
I have severe bone tissue death.
I have been receiving regular blood transfusions for over a year to manage my sickle cell disease.
My hemoglobin levels have not increased by more than 1.5 gm/dl from the baseline, or I need transfusions to keep my hemoglobin above 6.0 gm/dL.
My sickle cell disease is confirmed by genetic testing.
I was hospitalized for a severe chest problem in the last 2 years.
I have been hospitalized for sickle cell pain crises at least twice in the last 2 years.
I have had priapism at least twice.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
I don't have any other cancer or active infections.
My blood pressure is high (>135/95) despite taking medication.
I do not have active infections like HIV, Hepatitis B/C, HTLV-1, CMV, or parvovirus B19.
My lung function is significantly reduced.
My liver tests are much higher than normal.
My kidney function tests are not within the normal range.


Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 24 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Evaluation of Safety

Trial Design

1Treatment groups
Experimental Treatment
Group I: βAS3-FB vector transduced peripheral blood CD34+ cellsExperimental Treatment1 Intervention
This is a single arm study without randomization. All subjects will receive the intervention of BetaAS3 lentiviral vector-modified autologous peripheral blood stem cell transplant.

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments for Sickle Cell Disease (SCD) include hydroxyurea, red blood cell transfusions, and investigational gene therapies. Hydroxyurea increases fetal hemoglobin (HbF) production, reducing red blood cell sickling and pain episodes. Red blood cell transfusions increase the number of normal red blood cells, improving oxygen delivery and reducing sickled cells. Gene therapy, such as autologous transplant of lentiviral vector modified peripheral blood, aims to correct the hemoglobin defect at the genetic level, potentially offering a long-term solution by enabling the production of normal hemoglobin. These treatments are crucial for SCD patients as they can reduce complications, improve quality of life, and increase survival rates.
Gene therapy for sickle cell disease.Gene therapy for sickle cell disease.Gene therapy for sickle cell disease.

Find a Location

Who is running the clinical trial?

Donald B. Kohn, M.D.Lead Sponsor
1 Previous Clinical Trials
10 Total Patients Enrolled
California Institute for Regenerative Medicine (CIRM)OTHER
65 Previous Clinical Trials
3,171 Total Patients Enrolled
Giulia Parisi, PhDStudy DirectorUniversity of California, Los Angeles

Media Library

βAS3-FB vector transduced peripheral blood CD34+ cells (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02247843 — Phase 1 & 2
Sickle Cell Disease Clinical Trial 2023: βAS3-FB vector transduced peripheral blood CD34+ cells Highlights & Side Effects. Trial Name: NCT02247843 — Phase 1 & 2
Sickle Cell Disease Research Study Groups: βAS3-FB vector transduced peripheral blood CD34+ cells
βAS3-FB vector transduced peripheral blood CD34+ cells (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02247843 — Phase 1 & 2
~0 spots leftby Dec 2025