Biotech Strategy Blog

Commentary on Science, Innovation & New Products with a focus on Oncology, Hematology & Cancer Immunotherapy

Posts tagged ‘lentiglobin’

ASH 2015 Hematology Orlando

ASH 2015 — taken before 7am!

Orlando – a presentation in the plenary session at #ASH15, the 2015 annual meeting of the American Society of Hematology, is the pinnacle of any doctor or researcher working in the hematology field.

Yesterday, we had the privilege to interview Dr Richard Stone (Dana-Farber Cancer Institute) ahead of his plenary presentation at ASH:

Abstract 6: The Multi-Kinase Inhibitor Midostaurin (M) Prolongs Survival Compared with Placebo (P) in Combination with Daunorubicin (D)/Cytarabine (C) Induction (ind), High-Dose C Consolidation (consol), and As Maintenance (maint) Therapy in Newly Diagnosed Acute Myeloid Leukemia (AML) Patients (pts) Age 18-60 with FLT3 Mutations (muts): An International Prospective Randomized (rand) P-Controlled Double-Blind Trial (CALGB 10603/RATIFY [Alliance])

Anyone who has been to an ASH education session on AML knows how hard a nut it is to crack, so it’s wonderful to see some positive data, in what is commonly considered to be a “graveyard” disease.

The trial has taken a long time to come to fruition, so all credit to Dr Stone and colleagues. We’ll be writing up more about the data in our post meeting coverage.  For additional background, you can also check out our FLT3 preview in AML, which details some of the history and context for this study. The data from the phase 3 study is likely to form the backbone of a registration filing for Novartis with this compound in the near future based on successfully meeting the trial endpoints.

We also kick off today’s highlights with quick reflections on some of the hot topics that emerged yesterday including Bluebird Bio’s lentiglobin, Bellicum’s pipeline and .

During the day, as the opportunity presents, we’ll also be providing commentary on sessions we attend.

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Beta thalassemia isn’t something you read much about in the medical lay press, at least until recently.  Part of the problem is the lack of approved therapies, as well as the dearth of new products being evaluated in this condition.  It’s also more common in the Mediterranean, Middle East and Asia compared to the US, where it’s medical cousin, sickle cell anemia, dominates.

Things are changing on the horizon though with the advent of new approaches in gene therapy and gene editing, which have enabled new compounds to be developed that strike right at the source of the problem – mutated genes – rather than tackle the symptoms associated with the complications that can arise.

As such, this new approach is potentially transformative and therefore of great medical interest.

bluebird bioIn the first of our two part series, we take a look at what the clinical impact of treating thalassemia patients really means and what’s happening next in sickle cell disease in a interview with Dr Alexis Thompson, the PI for the Bluebird Bio Northstar trial with lentiglobin that was presented at ASH last month.

Her perspectives offer a fascinating insight into this novel therapy now that the first 4 patients have been successfully treated.

In the second part of the series later this week, we will also take a look at Bluebird Bio the company, and their approach to gene therapy with lentiglobin and CAR T cell therapy with an interview with their CEO, Nick Leschly.

Interested readers can log-in or sign up below to learn more about lentiglobin in beta thalassemia.

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