Bluebird Bio Releases Data on Current Gene Therapy Trials

Thank you Andy, that is wonderful to hear.  I hope that the patients are all doing well. 

Hiya Andy,

Please rest, rest, rest.
May God shower you with His special blessings.
Thank you for everything and I hope you get well soon.

Emby

  wondering when my cutiepie would be cured of this curse. there is querry can anyone help? do thal major have body temp bit higher than normal kids. My cutiepie is having fever for last 8 days and after giving course of 8 days antibiotics and negative blood and urine culture and -ve widal for typhoid and malaria antigen her temp is varying from 98.7f to 99.5f. what should i do

nanuaru73,

How is your child's temperature now? 

Gene therapy is in the trial phases at this time.  Presently adults and possibly some teens are being treated.  Once safety is better understood the trials will open to younger patients.  Focus on keeping a good hemoglobin level, low iron levels and nutritious food for now.  Gene therapy is showing great promise - it will hopefully cure all our children one day.

thanks for ur reply. she is much better now. at once stage she was investigated for t.b. also but by god grace every thing came out normal. no dignose was made and it was thought to be long case of viral fever. Is it benfi ial for her to go for swi.ming

Bluebird Bio Releases Data on Current Gene Therapy Trials - Jefferies 2015 Global Healthcare Conference

http://investor.bluebirdbio.com/phoenix.zhtml?p=irol-eventDetails&c=251820&eventID=5195088

Thank you Parin for sharing...very exciting

http://www.thestreet.com/story/13181244/1/bluebird-ceo-gene-therapy-study-plans-on-track-despite-request-to-go-slow-with-kids.html

Andy, what are your thoughts on this?

JV,

I will comment after my next call with Bluebird. I could speculate, but would rather be sure.

I think this is great news for anyone concerned about fertility following bmt or gene therapy:

http://www.iflscience.com/health-and-medicine/first-child-born-woman-who-had-ovary-tissue-removed-and-frozen-during-childhood

Sharmin...you bring up an excellent point...

I have received further explanation about the RAC recommendation.

"The RAC brought up challenges intrinsic to developing novel therapeutics in the pediatric population across therapeutic areas and modalities - not unique to gene therapy trials. Ultimately, evaluating the risks and benefits for this population are decisions that are really made on an individual basis, by individual families, investigators and sites.
 
And, as our CEO told Adam Feuerstein from The Street, “We respect RAC and the process. RAC took a reasonable position when it comes to dealing with kids. We disagree and believe the decision [to proceed with a gene therapy pediatric study] should be placed in the hands of the institutional review boards, the physicians of patients and their families.”
 
(My Question to Bluebird). Is Bluebird bound by RAC or can they proceed on their own?

"The outcome of the RAC review is a series of recommendations and advice, and it does not constitute a formal approval of a proposed protocol. The RAC review is specific to the U.S. and their comments are provided to the IRBs at U.S. trial sites, who individually decide on whether to follow the recommendations.
 
We do appreciate the recommendations from the RAC members and will take the RAC feedback on the timing of initiating HGB-208 under advisement. Of course, we will continue to work closely with the regulatory authorities and our clinical study sites to pursue appropriate accelerated regulatory approval pathways in the U.S. and the EU."

Thank you for the follow up Andy...doesn't sound like it will be a problem but further evaluation in children cant hurt either.

Bluebird feels that it is their decision to make and that their scientists are in the best position to know if trials with children should commence. The RAC has made a rather general recommendation covering all research using children in trials. I feel that specific therapies must stand on their own merits.

Good News.

Andy,

Thanks for sharing Blue thoughts.