- Bluebird Bio, a Cambridge, Massachusetts-based biotech, on Thursday opened enrollment of a Phase 3 trial of its LentiGlobin gene therapy, adding a key improvement to the manufacturing process for producing the hematopoietic stem cells used in its therapy.
- Importantly, the manufacturing changes will be covered by the same Investigational New Drug (IND) application used for prior studies of LentiGlobin, which is being tested for treatment of a certain type of beta-thalassemia.
- Bluebird stock moved higher by a little over 7% in early Thursday trading on the news.
BlueBird's Lentiglobin gene therapy involves the extraction of patient hematopoietic stem cells (HSC), which are then inserted with targeted gene sequences and re-infused back into the patient.
In the case of patients with beta-thalassemia, a rare blood disorder, Bluebird hopes to reduce the number of transfusions needed. The newly initiated study will aim for transfusion independence, or a 12-month transfusion free period after a transplant with modified HSC.
Bluebird said the study would feature an improved manufacturing process which the company hopes will increase hemoglobin levels in patients. More specifically, two unnamed additives will be incorporated in the transduction step of the process, hopefully increasing vector copy number and the percentage of successfully transduced cells.
This change won't require any new filing with the FDA, either. "Notably, no new IND was required for the incorporation of new manufacturing process," Jefferies analyst Gena Wang wrote in a note to investors.
This will allow Bluebird to proceed with its study without delay, although Wang doesn't expect any clinical data from the trial this year.
The new manufacturing process will also be used in HGB-206, another study currently studying LentiGlobin in sickle cell disease.