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Bluebird Bio Reports Third Quarter 2015 Financial Results And Recent Operational Progress

The following excerpt is from the company's SEC filing.

-- Six abstracts accepted for presentation at American Society of Hematology (ASH) Annual Meeting --

-- Increased enrollment target for HGB-206 study of LentiGlobin

in severe sickle cell disease (SCD) from eight patients to 20 patients --

-- Published a paper on megaTAL genome editing platform in

Science Translational Medicine

with collaborators at Seattle Children’s Research Institute --

-- Ended quarter with $901.7 million in cash, cash equivalents, and marketable securities --

-- Company will discuss ASH abstract data and business updates in a conference call tomorrow at 8:30 a.m. ET --

CAMBRIDGE, Mass., November 4, 2015 – bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies, today reported business highlights and financial results for the third quarter ended September 30, 2015.

“2015 continues to be a year of significant advancement for our programs in beta-thalassemia, sickle cell disease and oncology, which is reflected in our strong presence at ASH this year. We are excited about the progress we’ve made in the third quarter in enrolling the HGB-206 study in severe sickle cell disease, and we have made the decision to increase the enrollment target in order to gather more data and provide additional regulatory options,” said Nick Leschly, chief bluebird. “Our balance sheet remains strong, allowing us to continue to execute on our goals heading into 2016. This includes initiating the Phase 1 study for our first clinical oncology program, bb2121, in early 2016 and our plans to present data from the Starbeam study of LentiD in CCALD in the first half of 2016.”

Recent Highlights

DATA FROM HGB-204, HGB-205 AND HGB-206 CLINICAL STUDIES OF LENTIGLOBIN TO BE PRESENTED AT ASH –

Updated data from the HGB-204 study of LentiGlobin in beta-thalassemia major and the HGB-205 study in beta-thalassemia major and severe sickle cell disease (SCD) will be highlighted in separate oral presentations at the ASH annual meeting in December. Initial early data from the HGB-206 study of LentiGlobin in severe SCD will be presented in a poster.

DATA FROM PRE-CLINICAL STUDIES OF BB2121 TO BE PRESENTED AT ASH --

bluebird bio will also present data from its lead oncology program, bb2121 at ASH – the first bluebird bio oncology data to be presented at a major medical meeting. Three poster presentations will highlight preclinical data and manufacturing improvements made to the anti-BCMA CAR-T program.

INCREASED ENROLLMENT TARGET FOR HGB-206 STUDY IN SEVERE SCD

– Announced plan to increase enrollment in the U.S.-based HGB-206 study in severe SCD from eight patients to 20 patients to gain additional data and experience with LentiGlobin in SCD. bluebird bio plans to provide an update on enrollment in all three LentiGlobin clinical studies at the ASH meeting in December.

PUBLISHED GENOME EDITING PAPER IN

SCIENCE TRANSLATIONAL MEDICINE

Along with

at the Center for Immunity and Immunotherapy at Seattle Children’s Research Institute, published

“Efficient modification of

in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template” in

The findings showed that it is possible to edit the

to enable

T cells

both resist and kill HIV. They also showed that the megaTAL

platform allows researchers to place edited genes very precisely in the genome, leading to precise location and control of expression of the edited gene.

Third Quarter 2015 Financial Results and Financial Guidance

Cash...


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