More Good News from Bluebird. FDA Grants Breakthrough Therapy Designation.

This is some huge news and bodes well for the future approval of gene therapy for thalassemia.
FDA Grants Breakthrough Therapy Designation to LentiGlobin for Treatment of Beta-Thalassemia Major.

http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2012428

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb. 2, 2015-- bluebird bio, Inc. (Nasdaq:BLUE) a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to LentiGlobin® BB305 Drug Product for the treatment of transfusion-dependent patients with beta-thalassemia major.
LentiGlobin BB305 Drug Product aims to treat beta-thalassemia major and severe sickle cell disease by inserting a functional human beta-globin gene into the patient's own hematopoietic stem cells ex vivo and then returning those modified cells to the patient through an autologous stem cell transplantation.
"The FDA's Breakthrough designation of LentiGlobin highlights that new therapies are needed for the treatment of patients with beta-thalassemia major, especially treatments with the potential to meaningfully reduce or liberate patients from transfusion dependence," said David Davidson, M.D., chief medical officer of bluebird bio. "Our early clinical data investigating the use of LentiGlobin in patients with multiple genotypes of beta-thalassemia major, including beta-0/beta-0, the most severe genotype, are very encouraging, and we remain on track to complete enrollment in the Northstar and HGB-205 studies in 2015. In light of the Breakthrough designation, we look forward to working even more closely with the FDA to expedite the development of LentiGlobin for the treatment of beta-thalassemia major."
The FDA's Breakthrough Therapy designation is intended to expedite the development and review of a drug candidate that is planned for use to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The benefits of Breakthrough Therapy designation include the same benefits as Fast Track designation, plus an organizational commitment involving FDA’s senior managers with more intensive guidance from the FDA. Breakthrough Therapy designation does not however change the standards for approval.
The Breakthrough Therapy designation is supported by data from the ongoing Phase 1/2 Northstar (HGB-204) and HGB-205 studies of LentiGlobin. Findings in eight subjects with beta-thalassemia major were presented at the 56th Annual Meeting of the American Society of Hematology (ASH) in December 2014. In the first four subjects, each of whom had at least three months of follow up, treatment resulted in sufficient hemoglobin production to reduce or eliminate the need for transfusion support among patients with beta-thalassemia major who would otherwise require chronic blood transfusions. These data consisted of the first five subjects treated in bluebird bio's ongoing Northstar Study and the first three subjects from its HGB-205 study. These included the first beta-thalassemia subjects with the beta-0/beta-0 genotype to be treated with LentiGlobin BB305 drug product. The HGB-205 study also included the first subject with sickle cell disease to be treated with gene therapy.
About bluebird bio, Inc.
With its lentiviral-based gene therapy and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and T cell-based immunotherapy. bluebird bio’s clinical programs include Lenti-D™, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of childhood cerebral adrenoleukodystrophy, and LentiGlobin®, currently in three clinical studies: a global Phase 1/2 study, called the Northstar Study, for the treatment of beta-thalassemia major; a single-center Phase 1/2 study in France (HGB-205) for the treatment of beta-thalassemia major or severe sickle cell disease; and a separate U.S. Phase 1 study for the treatment of sickle cell disease (HGB-206). bluebird bio also has a preclinical CAR T cancer immunotherapy program in collaboration with Celgene Corporation, as well as discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies.
bluebird bio has operations in Cambridge, Massachusetts, Seattle, Washington, and Paris, France. For more information, please visit www.bluebirdbio.com.
Forward-Looking Statements
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the potential efficacy and safety of the Company’s LentiGlobin product candidate and the regulatory pathway afforded by Breakthrough Designation by the FDA, in particular statements concerning the reduced or eliminated need for transfusion support in the four initial subjects treated with LentiGlobin drug product, statements concerning the Company’s future plans with respect to LentiGlobin and its other product candidates and statements concerning anticipated enrollment rates and clinical milestones in 2015. It should be noted that the data for LentiGlobin announced from the Northstar and HGB-205 studies at the ASH Annual Meeting are preliminary in nature and the Northstar and HGB-205 studies are not completed. There is limited data concerning long-term safety and efficacy following treatment with LentiGlobin drug product. These data may not continue for these subjects or be repeated or observed in ongoing or future studies involving our LentiGlobin product candidate, including the HGB-205 Study, the Northstar Study or the HGB-206 study in sickle cell disease. It is possible that subjects for whom periodic transfusion support has been reduced or temporarily eliminated may receive transfusion support in the future. It should also be noted that Breakthrough designation does not change the standards for approval and is not a guarantee of success. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the preliminary results from our clinical trials will not continue or be repeated in our ongoing clinical trials, the risk that previously conducted studies involving similar product candidates will not be repeated or observed in ongoing or future studies involving current product candidates, the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates, the risk of a delay in the enrollment of patients in the Company’s clinical studies, the risk that our collaboration with Celgene will not continue or will not be successful, and the risk that any one or more of our product candidates will not be successfully developed and commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.
Availability of other information about bluebird bio
Investors and others should note that we communicate with our investors and the public using our company website (www.bluebirdbio.com), our investor relations website (http://www.bluebirdbio.com/investor-splash.html), including but not limited to investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference calls and webcasts. You can also connect with us on Twitter @bluebirdbio, LinkedIn or our YouTube channel. The information that we post on these channels and websites could be deemed to be material information. As a result, we encourage investors, the media, and others interested in bluebird bio to review the information that we post on these channels, including our investor relations website, on a regular basis. This list of channels may be updated from time to time on our investor relations website and may include other social media channels than the ones described above. The contents of our website or these channels, or any other website that may be accessed from our website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Source: bluebird bio, Inc.
Investor Relations:
bluebird bio, Inc.
Jim DeTore, 339-499-9355
Chief Financial Officer
or
Media:
Pure Communications, Inc.
Dan Budwick, 973-271-6085

Andy, Thank you for posting this.....This is the best news ever.....Praying for BlueBird bio's continued success.........

This is amazing! Thank you Andy for posting...getting closer and closer

This is such a wonderful news of this year. Also look at the test results they have quoted below in that article:

The Breakthrough Therapy designation is supported by data from the ongoing Phase 1/2 Northstar (HGB-204) and HGB-205 studies of LentiGlobin. Findings in eight subjects with beta-thalassemia major were presented at the 56th Annual Meeting of the American Society of Hematology (ASH) in December 2014. In the first four subjects, each of whom had at least three months of follow up, treatment resulted in sufficient hemoglobin production to reduce or eliminate the need for transfusion support among patients with beta-thalassemia major who would otherwise require chronic blood transfusions. These data consisted of the first five subjects treated in bluebird bio's ongoing Northstar Study and the first three subjects from its HGB-205 study. These included the first beta-thalassemia subjects with the beta-0/beta-0 genotype to be treated with LentiGlobin BB305 drug product. The HGB-205 study also included the first subject with sickle cell disease to be treated with gene therapy.

There is another option emerging which will preclude the need BMT for a permanent cure. This technology focuses on accelerated fetal hemoglobin production that will alleviate disease symptoms and transfusion need.

http://www.prnewswire.com/news-releases/sangamo-biosciences-announces-fda-acceptance-of-ind-to-initiate-clinical-trial-of-its-novel-zfp-therapeutic-for-beta-thalassemia-300030614.html

RICHMOND, Calif., Feb. 4, 2015 /PRNewswire/ -- Sangamo BioSciences, Inc. (NASDAQ: SGMO) announced today that an Investigational New Drug (IND) application for the company's SB-BCLmR-HSPC genome editing approach, which is designed to provide a  one-time lasting therapy for beta-thalassemia, has been accepted by the U.S. Food and Drug Administration (FDA) and is now active. This enables Sangamo to initiate a Phase 1/2 clinical trial of the ZFP Therapeutic in transfusion-dependent patients with beta-thalassemia major.  The trial, which is expected to begin in 2015, is designed to assess the safety and tolerability, and measures of efficacy of this novel approach. Sangamo is developing the therapy in collaboration with Biogen Idec.

"We believe that a single treatment with SB-BCLmR-HSPC has the potential to provide a lasting therapeutic solution  for transfusion-dependent beta-thalassemia with significant safety advantages over existing transplant therapies that involve hematopoietic stem progenitor cells (HSPCs) from a matched related donor," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "We are using our genome editing technology to target a key genetic switch in a patient's own HSPCs to enable continued production of fetal hemoglobin in the red blood cells of adults.  We know that elevated production of fetal globin can ameliorate disease symptoms of hemoglobinopathies such as beta-thalassemia.  We are also developing this strategy for sickle cell disease."

In May 2013, Sangamo was awarded a $6.4 million Strategic Partnership Award from CIRM, California's stem cell agency, to develop a ZFP Therapeutic for beta-thalassemia. The four-year grant provided matching funds for preclinical work to support the IND application and for a Phase 1/2 clinical trial, which will be carried out at multiple centers, including UCSF Benioff Children's Hospital Oakland.

"Our research and development team has worked hard to rapidly advance SB-BCLmR-HSPC through preclinical development and the IND application process," said Edward Lanphier, Sangamo's president and CEO. "We are very pleased to be in a position to move this therapy into the clinic with the aim of providing transfusion-dependent beta-thalassemia patients with a one-time treatment for this devastating disease."

Sangamo's ZFP Therapeutic® for Hemoglobinopathies
Sangamo's proprietary ZFN genome editing technology enables multiple approaches to the correction of beta-thalassemia and sickle cell disease (SCD). Both diseases manifest after birth, when patients switch from producing functional fetal gamma-globin to a mutant form of adult beta-globin, which results in their condition. Naturally occurring increased levels of fetal hemoglobin have been shown to reduce the severity of both beta-thalassemia and SCD disorders in adulthood. Sangamo's genome editing can be used to precisely disrupt key transcriptional regulators in HSPCs to reverse the switch from expression of the mutant adult beta-globin back to the production of functional fetal gamma-globin.

A bone marrow transplant (BMT), of HSPCs from a "matched" related donor (allogeneic BMT) is curative for both diseases. However, this therapy is limited due to the scarcity of matched donors and the significant risk of Graft versus Host Disease (GvHD) after transplantation of the foreign cells. By performing genome editing in HSPCs that are isolated from and subsequently returned to the same patient, an autologous HSPC transplant, Sangamo's approach eliminates both the need for a matched donor and the risk of acute and chronic GvHD. The ultimate goal of this approach is to develop a one-time treatment for beta-thalassemia.

About Hemoglobinopathies
Mutations in the genes encoding beta-globin, a subunit of the oxygen-carrying protein of red blood cells, lead to the hemoglobinopathies beta-thalassemia and SCD. There are several forms of beta-thalassemia caused by mutations in the beta-globin gene; broadly the disorder results in defective production of red blood cells leading to life-threatening anemia, enlarged spleen, liver and heart, and bone abnormalities. Severe beta-thalassemia major requires regular, often monthly, blood transfusions and subsequent iron-chelation therapy to treat iron overload. The CDC estimates that 2,000 people have beta-thalassemia in the United States, and an unknown number carry the genetic trait and can pass it on to their children.  Thalassemia is most common among people of Mediterranean descent and is also found among people from the Arabian Peninsula, Iran, Africa, Southeast Asia and Southern China.

About Sangamo
Sangamo BioSciences, Inc. is focused on Engineering Genetic CuresTM for monogenic and infectious diseases by deploying its novel DNA-binding protein technology platform in therapeutic gene regulation and genome editing. The Company has ongoing Phase 2 clinical trials to evaluate the safety and efficacy of a novel ZFP Therapeutic® for the treatment of HIV/AIDS (SB-728-T) and NGF-AAV for Alzheimer's disease (CERE-110). Sangamo's other therapeutic programs are focused on monogenic and rare diseases. The Company has formed a strategic collaboration with Shire International GmbH to develop therapeutics for hemophilia, Huntington's disease and other monogenic diseases, and with Biogen Idec for hemoglobinopathies, such as sickle cell disease and beta-thalassemia. It has also established strategic partnerships with companies in non-therapeutic applications of its technology, including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the Company's website at www.sangamo.com.
ZFP Therapeutic® is a registered trademark of Sangamo BioSciences, Inc.

This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, references relating to research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo's ZFP technology platform, the potential of Sangamo's ZFP technology to treat beta-thalassemia and sickle cell disease, the safety of the approach of using ZFN-mediated genome editing, and Sangamo's collaboration with Biogen for the treatment of hemoglobinopathies, beta-thalassemia and sickle cell disease. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the safety, tolerability and efficacy of ZFNs and ZFP TFs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's public filings with the Securities and Exchange Commission, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo assumes no obligation to update the forward-looking information contained in this press release.

Thanks for sharing Himanshu.

"We are using our genome editing technology to target a key genetic switch in a patient's own HSPCs to enable continued production of fetal hemoglobin in the red blood cells of adults.  We know that elevated production of fetal globin can ameliorate disease symptoms of hemoglobinopathies such as beta-thalassemia.  We are also developing this strategy for sickle cell disease."

I am always a big fan of this technique as these gamma chains capable to producing fetal hemoglobin goes silent after six month of birth. This techinique excludes the need of chemotherapy (if I am not mistaken) releiving the patient from side effects of Chemo. This is much natural cure than anything else in progress.

This is amazing! Thank you both Andy and Himanshu for posting.

Regards,
Ashutosh T

Wow,

2015 is a holy grain for Thalpals!

Himanshu, thanks so much for posting that!

Canadian Family, yes, it would be much appreciated as a patient to have therapy without having to undergo Chemo.

Best,

-P

Thank you for sharing Andy and Himanshu,

I agree Canadian Family.  I believe that over time the use of chemotherapy will be tapered and reduced.  As the process is fine tuned and refined.

My greatest concern with gene therapy has always been chemotherapy - if it can be avoided or reduced it would be best. 

Such great things happening for thalassemia and sickle cells patients.  Praying for a cure for a soon.

Sharmin

This is really amazing new for all the thals, thanks Andy and Himanshu for sharing!!

I truly hope these treatments soon become success and be available to larger community....