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Photo by Chad McNeeley
An investigational gene therapy known as LentiGlobin BB305 has been accepted into the European Medicines Agency’s (EMA’s) Priority Medicines (PRIME) program as a treatment for patients with transfusion-dependent beta-thalassemia (TDT).
LentiGlobin BB305 is created by inserting a functional human beta-globin gene into a patient’s hematopoietic stem cells ex vivo. The cells are then returned to the patient via transplant.
The goal of the EMA’s PRIME program is to accelerate the development of therapies that target unmet medical needs. The program provides enhanced EMA support and increased interaction to developers, in order to optimize development plans and speed regulatory evaluations to potentially bring these therapies to patients more quickly.
To be accepted for PRIME, a therapy must demonstrate potential to benefit patients with unmet medical need through early clinical or nonclinical data.
Bluebird bio, the company developing LentiGlobin BB305, is also participating in the EMA’s Adaptive Pathways pilot program.
Like PRIME, the Adaptive Pathways program aims to expedite patient access to therapies with the potential to treat serious conditions with unmet need. It uses the existing European Union (EU) regulatory framework for medicines, including conditional approval.
“PRIME designation will allow bluebird bio to further improve our communication with European regulators as we continue to refine our evidence generation plan in the context of adaptive biomedical innovation,” said David Davidson, MD, chief medical officer of bluebird bio.
“Overall, we believe this will enable us to accelerate development of LentiGlobin drug product for patients with transfusion-dependent beta-thalassemia, a life-shortening disease with significant unmet medical need.”
“Earlier this year, we completed enrollment in the Northstar (HGB-204) global clinical study of LentiGlobin drug product in patients with TDT, which, along with the supporting HGB-205 study, will form the basis of our eventual application for conditional approval in the EU under the Adaptive Pathways pilot program.” 
Photo by Chad McNeeley
An investigational gene therapy known as LentiGlobin BB305 has been accepted into the European Medicines Agency’s (EMA’s) Priority Medicines (PRIME) program as a treatment for patients with transfusion-dependent beta-thalassemia (TDT).
LentiGlobin BB305 is created by inserting a functional human beta-globin gene into a patient’s hematopoietic stem cells ex vivo. The cells are then returned to the patient via transplant.
The goal of the EMA’s PRIME program is to accelerate the development of therapies that target unmet medical needs. The program provides enhanced EMA support and increased interaction to developers, in order to optimize development plans and speed regulatory evaluations to potentially bring these therapies to patients more quickly.
To be accepted for PRIME, a therapy must demonstrate potential to benefit patients with unmet medical need through early clinical or nonclinical data.
Bluebird bio, the company developing LentiGlobin BB305, is also participating in the EMA’s Adaptive Pathways pilot program.
Like PRIME, the Adaptive Pathways program aims to expedite patient access to therapies with the potential to treat serious conditions with unmet need. It uses the existing European Union (EU) regulatory framework for medicines, including conditional approval.
“PRIME designation will allow bluebird bio to further improve our communication with European regulators as we continue to refine our evidence generation plan in the context of adaptive biomedical innovation,” said David Davidson, MD, chief medical officer of bluebird bio.
“Overall, we believe this will enable us to accelerate development of LentiGlobin drug product for patients with transfusion-dependent beta-thalassemia, a life-shortening disease with significant unmet medical need.”
“Earlier this year, we completed enrollment in the Northstar (HGB-204) global clinical study of LentiGlobin drug product in patients with TDT, which, along with the supporting HGB-205 study, will form the basis of our eventual application for conditional approval in the EU under the Adaptive Pathways pilot program.”
Photo by Chad McNeeley
An investigational gene therapy known as LentiGlobin BB305 has been accepted into the European Medicines Agency’s (EMA’s) Priority Medicines (PRIME) program as a treatment for patients with transfusion-dependent beta-thalassemia (TDT).
LentiGlobin BB305 is created by inserting a functional human beta-globin gene into a patient’s hematopoietic stem cells ex vivo. The cells are then returned to the patient via transplant.
The goal of the EMA’s PRIME program is to accelerate the development of therapies that target unmet medical needs. The program provides enhanced EMA support and increased interaction to developers, in order to optimize development plans and speed regulatory evaluations to potentially bring these therapies to patients more quickly.
To be accepted for PRIME, a therapy must demonstrate potential to benefit patients with unmet medical need through early clinical or nonclinical data.
Bluebird bio, the company developing LentiGlobin BB305, is also participating in the EMA’s Adaptive Pathways pilot program.
Like PRIME, the Adaptive Pathways program aims to expedite patient access to therapies with the potential to treat serious conditions with unmet need. It uses the existing European Union (EU) regulatory framework for medicines, including conditional approval.
“PRIME designation will allow bluebird bio to further improve our communication with European regulators as we continue to refine our evidence generation plan in the context of adaptive biomedical innovation,” said David Davidson, MD, chief medical officer of bluebird bio.
“Overall, we believe this will enable us to accelerate development of LentiGlobin drug product for patients with transfusion-dependent beta-thalassemia, a life-shortening disease with significant unmet medical need.”
“Earlier this year, we completed enrollment in the Northstar (HGB-204) global clinical study of LentiGlobin drug product in patients with TDT, which, along with the supporting HGB-205 study, will form the basis of our eventual application for conditional approval in the EU under the Adaptive Pathways pilot program.”