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More impressive data from Bluebird’s gene therapy in thalassemia and sickle cell disease

Cell and gene therapy specialist Bluebird going head-to-head with partner Celgene


While the US-based ASCO and ASH congresses tend to get the most important haemato-oncology and blood disorder data readouts, the European Hematology Association (EHA) congress now under way in Stockholm has its share of significant advances this year.

One of the standouts amid all the other blood cancer therapy data released this morning were two updates from Cambridge, Mass-based biotech Bluebird Bio.

The company has revealed more early stage data for its gene therapy platform in two rare blood conditions, promising to transform treatment of both diseases.

New data being presented in Stockholm for its LentiGlobin gene therapy has produced impressive results in both Transfusion-Dependent Beta-thalassemia and severe sickle cell disease – building the promise of a de facto cure for at least some patients.

Bluebird updated its manufacturing process last year, and its LentiGlobin gene therapy is now derived from stem cells harvested from patients’ blood rather than bone marrow. The company says this improved manufacturing process is already producing better results in patients.

However the data is still at an early stage, with the number of patients treated still small, and the question of whether patients in both diseases will sustain a response over time is yet to be proven.

Tomorrow sees a presentation of data from the completed phase 1/2 Northstar (HGB-204) study in adolescents and adults with transfusion-dependent Beta-thalassemia (TDT) and any genotype, and its ongoing, phase III Northstar-2 (HGB-207) study of LentiGlobin in TDT patients and non-β00 genotypes.

The results show that seven out of eight patients no longer need long-term blood transfusions, and all are showing normal levels of haemoglobin.

“The maturing data from HGB-204 and HGB-207 suggest that one-time treatment with LentiGlobin may address the underlying genetic cause of TDT. With our refined manufacturing process, the majority of patients with TDT and non-β00 genotypes are transfusion-free and producing total haemoglobin at normal or near-normal levels,” said David Davidson, M.D., chief medical officer, Bluebird Bio.

Europe’s regulatory the EMA has granted the therapy its special fast-track status for these patients, and unusually, Bluebird plans to file it in Europe before the US.

“We are on track to submit a marketing authorisation application in the European Union later this year, and we continue to work closely with clinical investigators and regulatory authorities to complete our ongoing clinical trials and bring this important treatment option to patients as soon as possible,” added Davidson.

Sickle cell disease

The story is similar in severe sickle cell disease (SCD), where new interim data from the ongoing HGB-206 phase 1 multicentre clinical study of LentiGlobin is also being presented tomorrow.

The first patient treated in the study, now with six months of follow-up, is producing over 60% anti-sickling haemoglobin with a normal total haemoglobin level of 14.2 g/dL –  which means if this persists, this patient is effectively cured of their disease.

Overall results suggest that all of the patients will surpass the original therapeutic target of 30% anti-sickling haemogloblin, freeing them of the disease and its painful complications.

Sickle cell anaemia suffers have to contend with severe anaemia and blocked veins which causes severe pain and lead to organ damage and a shortened life.

“The early data from Group C patients are very exciting and provide increasing confidence that LentiGlobin has the potential to deliver transformative benefit to patients. The longer-term data from patients treated earlier in the study show that levels of anti-sickling HbAT87Q in patients with SCD treated with LentiGlobin remain stable for at least two years,” said Dr. Julie Kanter, a lead investigator of the HGB-206 study.

The ultimate commercial success of the treatments will depend on just how many patients will see a complete remission in their disease, and how many will be spared the damaging symptoms caused by the conditions.

These two major clinical trial programmes are not Bluebird’s only promising candidates – it also has bb2121, its anti-BCMA CAR T Cell therapy for Relapsed/Refractory Multiple Myeloma in development with Celgene, plus Lenti-D, a gene therapy for the rare disease adrenoleukodystrophy.

Bluebird won’t have the Beta-thalassemia market to itself though – Celgene is working with another company, Acceleron, on a rival treatment, luspatercept.

The partners have produced phase II data today at EHA showing strong benefits in transfusion-dependent and non-tranfusion dependent patients.

In the more severe transfusion-dependent cases, 32 patients have been treated with luspatercept, with 41% achieving a reduced transfusion burden of at least 33% in the fixed 12-week intervals including weeks 13 to 24 and weeks 37 to 48 when compared to baseline.

Also set to enter clinical trials in the near future is a collaboration between CRISPR Therapeutics and Vertex for CTX001 in β-thalassemia and sickle cell disease, which will make it one of the first CRISPR-based therapies to reach human studies.

Article by
Andrew McConaghie

15th June 2018

From: Research



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