A Novel Therapeutic Vector for Hemoglobin Disorders

Investigators at the National Heart, Lung, and Blood Institute have designed a novel lentiviral vector as a potential gene therapy for sickle cell anemia and beta-thalassemia. The novel lentiviral vector encodes the beta-globin gene in a forward orientation and can produce 5-10 fold higher viral titer and 4-10 fold higher gene transfer efficiency to hematopoietic stem cells than reverse-oriented lentiviral vectors. In vivo studies conducted in rhesus macaques show beta-globin production after transplantation with this novel lentiviral vector. This technology could provide an alternative therapy for patients suffering from blood disorders associated with beta-globin gene mutations.

Potential Commercial Applications: Competitive Advantages:
  • Gene therapy
  • Increased viral titers
  • Increased transduction efficiency
  • Large scale vector production

Development Stage:
  • Early-stage
  • In vitro data available
  • In vivo data available (animal)


Naoya Uchida (NHLBI)  ➽ more inventions...

John Tisdale (NHLBI)  ➽ more inventions...

Intellectual Property:
U.S. Pat: 10,543,257 issued 2020-01-28
US Application No. 15/510,014
US Application No. 62/048,881
US Application No. 16/717,420

Collaboration Opportunity:

The National Heart, Lung and Blood Institute is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize this technology. For collaboration opportunities, please contact Denise Crooks at crooksd@mail.nih.gov.

Licensing Contact:
Denise Crooks,
Email: crooksd@mail.nih.gov
Phone: 301-435-0103

OTT Reference No: E-165-2014-0
Updated: Sep 21, 2015