Severe Sickle Cell Disease - Healing Genes

Severe Sickle Cell Disease

Share This Post

Share on facebook
Share on linkedin
Share on twitter
Share on email

A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in Severe Sickle Cell Disease

A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the LentiGlobin BB305 Lentiviral Vector in Subjects With Severe Sickle Cell Disease

Phase 1 / 2


Several research centers working with bluebird bio seek participants with severe sickle cell disease to receive an experimental gene therapy that modifies the patient’s own blood stem cells (autologous), inserting a functional human beta-globin gene in the lab then returning the cells to the patient along with a drug to boost their growth.

The treatment will require leukapheresis to collect the blood stem cells, chemotherapy before they are returned via a single infusion, and a long-term follow up period of at least 2 years.


  • Be 12 to 50 years of age
  • Have severe SCD. i.e., in the setting of appropriate supportive care measures for SCD (e.g.,pain management plan) have experienced at least 4 severe VOEs in the 24 months prior to informed consent
    • A severe VOE is defined as an event with no medically determined cause other than a vaso-occlusion, requiring a ≥ 24-hour hospital or Emergency Room (ER) observation unit visit or at least 2 visits to a day unit or ER over 72 hours with both visits requiring intravenous treatment
  • Not be a prior receipt of an allogeneic transplant


  1. Screening to confirm eligibility, including imaging procedures, heart and lung tests, and lab tests.
  2. Participant will have leukapheresis: blood will be removed through a needle in the arm while a machine separates the white blood cells. The rest of the blood is returned through a needle in the other arm.
  3. No treatment while the cells are gene-modified in the lab.
  4. Admission to hospital and over several days, chemotherapy drugs and then the gene-modified immune cells are injected. Plerixafor to stimulate the cells’ growth will be given.
  5. Participants will be monitored in the hospital for several days with support medicine, blood and lab tests.
  6. Follow up for ~2 years.

The study centers have not yet been announced. Contact the recruiting center to learn of one near you.

bluebird bio  |  1-339-499-9300  |  [email protected]
bluebird bio
Or go online:

Subscribe To Our Newsletter

Get updates and learn from the best

More To Explore

Vanquishing the Virus: 160+ COVID-19 Drug and Vaccine Candidates in Development

While President Trump continues to promote antimalarial drugs such as hydroxychloroquine sulfate and azithromycin to treat COVID-19—“What do you have to lose?” he exclaimed during the April 4 Coronavirus Task Force Briefing—the global community of drug discovery researchers and biopharmas is developing a much broader