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Re: De: FDA Approves *SECOND* Cell-Based Gene Therapy to Treat Adult and Pediatric Patients  

By: Decomposed in 6TH POPE | Recommend this post (1)
Thu, 18 Aug 22 5:47 PM | 32 view(s)
Boardmark this board | 6th Edition Pope Board 		Msg. 34674 of 60008
(This msg. is a reply to 34669 by Fiz)
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fizzy:

Re: “Regardless, I thought this was very noteworthy and was hoping De, in particular, might give his opinion?”I'm a big fan of gene therapy - for those who have a serious enough problem. Like you, I consider gene therapy as a preventative to be downright evil. Physicians shouldn't be messing with healthy people's biology.

That said, I've never heard of beta-thalassemia. I see, though, that it's a rare disease - about 3,500 Americans have it. Some of those have beta thalassemia minor and aren't even aware that they've got it. Therefore, I don't see this as being a big money maker for Bluebird Bio, Inc. or a stock that would interst me. Take note:

"This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives."

BLUE's stock is up 55% since August 1st but is down 14% today. IMO, today's drop is appropriate as this is a big nothing burger investment-wise. The drug isn't even new, having been approved in the Netherlands three years ago though I'm not sure it continues to be approved. I'm seeing something about its approval having been withdrawn.







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Gold is $1,581/oz today. When it hits $2,000, it will be up 26.5%. Let's see how long that takes. - De 3/11/2013 - ANSWER: 7 Years, 5 Months

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The above is a reply to the following message:
De: FDA Approves *SECOND* Cell-Based Gene Therapy to Treat Adult and Pediatric Patients
By: Fiz in 6TH POPE
Thu, 18 Aug 22 4:34 PM 		Msg. 34669 of 60008

http://www.fda.gov/news-events/press-announcements/fda-approves-first-cell-based-gene-therapy-treat-adult-and-pediatric-patients-beta-thalassemia-who

"Today, the U.S. Food and Drug Administration approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

“Today’s approval is an important advance in the treatment of beta-thalassemia, particularly in individuals who require ongoing red blood cell transfusions,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “Given the potential health complications associated with this serious disease, this action highlights the FDA’s continued commitment to supporting development of innovative therapies for patients who have limited treatment options.”

Beta-thalassemia is a type of inherited blood disorder that causes a reduction of normal hemoglobin and red blood cells in the blood, through mutations in the beta-globin subunit, leading to insufficient delivery of oxygen in the body. The reduced levels of red blood cells can lead to a number of health issues including dizziness, weakness, fatigue, bone abnormalities and more serious complications."

I wonder if this would have gotten approval without without the mRNA Covid vaccines? I believe it is nearly the same tech, a LOT more appropriately applied?

Regardless, I thought this was very noteworthy and was hoping De, in particular, might give his opinion?

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