Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials

Still waiting for the update news too!!

tired of hearing from so many years, treatment is coming, trials are coming and finally nothing happens ? It is very sad to hear all this. When will the real picture come in front of us.

Don't lose hope Sagar.. maybe the time is not coming yet, maybe God put it in delay just to give us the very best later..

Have faith..

any update ?

bluebird bio to Present at the Leerink Swann 2012 Global Healthcare Conference
February 09, 2012 08:03 AM Eastern Time
CAMBRIDGE, Mass.--(EON: Enhanced Online News)--bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that Nick Leschly, chief executive officer, is scheduled to present at the Leerink Swann 2012 Global Healthcare Conference on Wednesday, February 15, 2012 at 2:15PM EST at the Waldorf Astoria Hotel in New York City.

Mr. Leschly is expected to discuss the company’s vision for developing gene therapies for genetic diseases with high unmet medical needs, including beta-thalassemia, sickle cell disease and childhood cerebral adrenoleukodystrophy (CCALD). The presentation will include updates on the launch of the company’s pivotal CCALD clinical trial, as well as patient data updates and enrollment of the ongoing beta-thalassemia/sickle cell disease trial.

there may be an update for errant gene next month as well. 

This is a latest reply from Mr. Pat Girondi

Dear Family Member

We just sent the IND to the FDA.
 
We have answered all of there queries and hope that we can begin quickly.
 
My best to you and your family,
 
Pat girondi

Thanks Pat, you are a hero.

Thank you Canadian Family.  Praying for us all...

Sharmin

Dear All,

is it possible to make it happen world wide, because I came from Indonesia.

Thank you.

Dear All,

Any updates from Pat?? or FDA???

There was supposed to be a response from the FDA this past week but still no word. Hopefully, we will hear something this week.

Hoping and praying for a positive response so that the trials can commence...

Hi,

Can anyone shed some light on what exactly this is and how would it be beneficiary to us, Majors? Thanks.

In gene therapy, normal beta globin genes are introduced into the body, so that normal beta globin can be produced. The goal is an Hb of at least 10, and it is believed that Dr Sadelain's process can do this. This would free the patient from transfusions.

Brilliant. Please let us know the updates, whenever you have them, Andy.

Pratik.