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Bluebird Bio Releases Data on Current Gene Therapy Trials

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Andy Battaglia:
The first patient Bluebird treated has seen a waning of the effects as time has passed. Bluebird is confident that their new vector is much more effective and they have confidence it will continue to work in the long term. Only time will tell.

Bluebird released this update. 


--- Quote ---bluebird bio Announces Presentation at Cooley’s Anemia Foundation Symposium
Professor Marina Cavazzana presents overview on experience with investigational gene therapy in beta-thalassemia
CAMBRIDGE, Mass. – October 19, 2015 – bluebird bio, Inc. (Nasdaq: BLUE) announced that Marina Cavazzana, M.D., Ph.D., professor of hematology and director of the Department of Biotherapy at Hospital Necker, University Paris Descartes and lead investigator for the HGB-205 clinical study, presented a review of the clinical study experience with lentiviral-based gene therapies for beta-thalassemia at the 10th Annual Cooley’s Anemia Foundation Symposium in Chicago today.
Professor Cavazzana summarized results from HGB-205, an ongoing clinical study using LentiGlobin® BB305 for the treatment of beta-thalassemia major and severe sickle cell disease, which were presented earlier this year at the Annual Congress of the European Hematology Association. She also provided an update on subject 1003, a patient with beta-thalassemia major treated by Professor Cavazzana in 2007 in the LG001 study using the first-generation HPV569 lentiviral vector. Following approximately seven years of transfusion independence, subject 1003 has recently required two blood transfusions after experiencing clinical symptoms of anemia. Importantly, both the expression of HbAT87Q and the vector copy number in peripheral blood leukocytes, a measure of the persistence of the gene therapy, have remained largely unchanged. The safety profile for both vectors is consistent with autologous transplantation, with no gene-therapy related serious adverse events.
Development of bluebird bio’s approach to treating hemoglobinopathies was guided by the data collected from the LG001 study, a pioneering clinical study that served to highlight both the potential for gene therapy as an intervention in beta-thalassemia as well as the need for an improved lentiviral vector. To this end, Professor Cavazzana also discussed the advances in the design of lentiviral vectors for hemoglobinopathy gene therapies -- specifically the evolution from the first-generation HPV569 lentiviral vector to bluebird bio’s current lentiviral vector, BB305, which results in substantially improved vector copy number and HbAT87Q expression. “The early clinical experience with HPV569 represented a crucial proof of concept for the potential of gene therapy to bring life-changing treatment to patients in need,” said David Davidson, M.D., chief medical officer, bluebird bio. “The data from the LG001 study were invaluable to our efforts over the last five years to optimize our gene therapy approach with improvements to the potency, robustness and manufacturing for the next-generation lentiviral vector, BB305. LentiGlobin BB305 drug product is being evaluated in three ongoing clinical trials for the treatment of patients with beta-thalassemia major and severe sickle cell disease, and three abstracts related to these ongoing clinical trials have been accepted for presentation at this year’s American Society of Hematology’s Annual Meeting in December.”
--- End quote ---

Andy Battaglia:
A bit more follow up on this first case.


--- Quote ---This patient was treated using an old version of the gene therapy, and tests show that the amount of hemoglobin being produced by the gene-therapy-modified stem cells remained stable. This suggests that the persistence of the gene therapy, even though it’s an older version, did not wear off. However, of course, this is only 8 years after the treatment.
 
No one at the company has spoken with the patient (obviously!), and all treatment decisions are made by the patient and their physician. Regardless of a patient’s hemoglobin level, we recognize that the patient may feel more tired or have other anemia symptoms simply because of other factors, and the decision to transfuse is always be made by the patient and his or her care team.
 
We’ll have more data on the patients being treated with our current version of the gene therapy treatment at ASH later this year. I will email you as soon as any data become publicly available.
--- End quote ---

Nothing has changed. Panic in the stock market is unwarranted.

Canadian_Family:
Andy,

Being in stock markets for the last 15 years, I can say that stocks are speculative in nature (99% of the time). Remember Bluebird was just trading $17 last year, after the announcement of human trials the stock shooted to $181 in few months. The answer is speculation, Bluebird has not made any money from its technology, it is on trails basis but stock is trading $81 these days. It came down quickly because news about competition (SKMCC).

News, speculation, big whales at Bay street and Wall street manipulates money and gets bigger.

Note to others: I do not own Bluebird but watch the stock movement as we need this company to succeed.

Sharmin:
That is very interesting Canadian Family, like you we are invested in bluebird in a far more important way.  They may hold the key to curing our children. 

Sharmin

Canadian_Family:
Agreed. Our hopes and prayers are our investment in Bluebird.

Kindly,

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