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.
IC: NHLBINIH Ref. No.: E-165-2014/0TAB No: TAB-2935Advantages: Increased viral titers
Increased transduction efficiency
Large scale vector production
Applications: Gene therapy
Development Status: Early-stage
In vitro data available
In vivo data available (animal)
Provider Technology ID: 2935Updated in TTS: Jul 1, 2015Date Published: Friday, May 15, 2015Provider Classifications: Publications: Patent Application: 62/048,881Patent Authority: USLicensing Contacts: Inventor IC: NHLBINHLBILPM FIrst Name: CristinaLPM Last Name: Thalhammer-ReyeroLPM Address: 6011 Executive Blvd. Suite 325
Room 33LPM City: RockvilleLPM Zip: 20852Inv Is lead: LPM State: MDLPM Phone: [phone]LPM Email: ...

Source: NIH OTT Licensing Opportunities - May 15, 2015 Category: Research Authors: admin Source Type: research