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Posted by: thepinetree on 12/08/2023 01:09 PM
Updated by: thepinetree on 12/08/2023 01:09 PM
Expires: 01/01/2028 12:00 AM
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FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease
Washington, DC...Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy.
Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S. It is most common in African Americans and, while less prevalent, also affects Hispanic Americans. The primary problem in sickle cell disease is a mutation in hemoglobin, a protein found in red blood cells that delivers oxygen to the body’s tissues. This mutation causes red blood cells to develop a crescent or “sickle” shape. These sickled red blood cells restrict the flow in blood vessels and limit oxygen delivery to the body’s tissues, leading to severe pain and organ damage called vaso-occlusive events (VOEs) or vaso-occlusive crises (VOCs). The recurrence of these events or crises can lead to life-threatening disabilities and/or early death.
“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”
Casgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises. Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of genome editing technology. Patients’ hematopoietic (blood) stem cells are modified by genome editing using CRISPR/Cas9 technology.
CRISPR/Cas9 can be directed to cut DNA in targeted areas, enabling the ability to accurately edit (remove, add, or replace) DNA where it was cut. The modified blood stem cells are transplanted back into the patient where they engraft (attach and multiply) within the bone marrow and increase the production of fetal hemoglobin (HbF), a type of hemoglobin that facilitates oxygen delivery. In patients with sickle cell disease, increased levels of HbF prevent the sickling of red blood cells.
Lyfgenia is a cell-based gene therapy. Lyfgenia uses a lentiviral vector (gene delivery vehicle) for genetic modification and is approved for the treatment of patients 12 years of age and older with sickle cell disease and a history of vaso-occlusive events. With Lyfgenia, the patient’s blood stem cells are genetically modified to produce HbAT87Q, a gene-therapy derived hemoglobin that functions similarly to hemoglobin A, which is the normal adult hemoglobin produced in persons not affected by sickle cell disease. Red blood cells containing HbAT87Q have a lower risk of sickling and occluding blood flow. These modified stem cells are then delivered to the patient.
Both products are made from the patients’ own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant. Prior to treatment, a patients’ own stem cells are collected, and then the patient must undergo myeloablative conditioning (high-dose chemotherapy), a process that removes cells from the bone marrow so they can be replaced with the modified cells in Casgevy and Lyfgenia. Patients who received Casgevy or Lyfgenia will be followed in a long-term study to evaluate each product’s safety and effectiveness.
“These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval, reflecting the FDA’s commitment to facilitating development of safe and effective treatments for conditions with severe impacts on human health.”
Data Supporting Casgevy
The safety and effectiveness of Casgevy were evaluated in an ongoing single-arm, multi-center trial in adult and adolescent patients with SCD. Patients had a history of at least two protocol-defined severe VOCs during each of the two years prior to screening. The primary efficacy outcome was freedom from severe VOC episodes for at least 12 consecutive months during the 24-month follow-up period. A total of 44 patients were treated with Casgevy. Of the 31 patients with sufficient follow-up time to be evaluable, 29 (93.5%) achieved this outcome. All treated patients achieved successful engraftment with no patients experiencing graft failure or graft rejection.
The most common side effects were low levels of platelets and white blood cells, mouth sores, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile neutropenia (fever and low white blood cell count), headache and itching.
Data Supporting Lyfgenia
The safety and effectiveness of Lyfgenia is based on the analysis of data from a single-arm, 24-month multicenter study in patients with sickle cell disease and history of VOEs between the ages of 12- and 50- years old. Effectiveness was evaluated based on complete resolution of VOEs (VOE-CR) between 6 and 18 months after infusion with Lyfgenia. Twenty-eight (88%) of 32 patients achieved VOE-CR during this time period.
The most common side effects included stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with chemotherapy and underlying disease.
Hematologic malignancy (blood cancer) has occurred in patients treated with Lyfgenia. A black box warning is included in the label for Lyfgenia with information regarding this risk. Patients receiving this product should have lifelong monitoring for these malignancies.
Both the Casgevy and Lyfgenia applications received Priority Review, Orphan Drug, Fast Track and Regenerative Medicine Advanced Therapy designations.
The FDA granted approval of Casgevy to Vertex Pharmaceuticals Inc. and approval of Lyfgenia to Bluebird Bio Inc.
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The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products.
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No Subject
Posted on: 2023-12-08 13:16:29
By: Anonymous
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Damn it
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Re:
Posted on: 2023-12-08 15:20:51
By: Anonymous
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This vaccine costs 2 million dollars plus other treatments. There is no way that Americans with this disease can afford these treatments unless you are an elite DEMORAT!
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Lies, Lies, Lies, they're all LIES BY GOD!!!
Posted on: 2023-12-08 15:57:54
By: Anonymous
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December 6, 2023 6:28pm
President Joe Biden on Wednesday slapped down a question on his alleged role in the Biden family's business dealings and his reported meetings with the associates of his brother James and son Hunter.
New York Post reporter Steven Nelson had pressed the president on why he interacted with "so many of your son and brother's foreign business associates?" to which Biden angrily retorted "I'm not going to comment. I did not
and it's just a bunch of lies."
"They're lies. I did not. They're lies," he repeated.
YEAH JOE, LIES LIKE IN
" MY SON HAS DONE NOTHING WRONG"
" I HAVE NEVER DISCUSSED MY SONS OVERSEAS BUSINESS" TO
"I HAVE NEVER BEEN IN BUSINESS WITH MY SON" TO
" I HAVE NEVER TALKED TO ANY OF MY SONS BUSINESS ASSOCIATES" TO
" I MAY HAVE CALLED SOME OF MY SON'S BUSINESS PARTNERS ABOUT THE WEATHER" TO
I HAVE NOT NOR HAD ANYONE IN MY FAMILY TAKEN MONEY FROM COMMUNIST CHINA"
EVERY SINGLE ONE HAS BEEN PROVEN TO BE A LIE, SO JOES RIGHT!!! JOE HAS TOLD NOTHING BUT LIES
AND THE IGNORANT LIBERAL LEFT STILL CAN'T SEE THEIR OWN IGNORANCE! MAYBE IT'S BECAUSE OF THE EMBARRASSMENT OF BEING SO IGNORANT TO OF VOTED FOR A SENILE, OLD, WEAK, FEEBLE, CAREER POLITICIAN WHO CANT TALK WITHOUT READING WHAT HE'S SUPPOSE TO SAY. HE CAN'T TAKE QUESTIONS FROM ANY REPORTER WITHOUT HAVING THE QUESTION BEFOREHAND. WHO'S WEAKNESSES AND FOREIGN POLICY IGNORANCE ON ALL TOPICS HAS SO FAR STARTED TWO WARS.
👏 GREAT JOB TO THE IGNORANCE OF THE DEMOCRAT VOTERS FOR ELECTING THE WORST PRESIDENT IN OUR HISTORY
THAT'S WHEN HE REMEMBERS HE IS THE PRESIDENT, ON ANY GIVEN DAY HE CAN'T REMEMBER IF HE IS LET ALONE WHAT DAY OF THE WEEK IT IS.
PS: SHOCKING NEWS JUST SHOCKING!!! GRAND JURY INDICTS HUNTER BIDEN ON NUMEROUS MISDEMEANORS AND FELONY CHARGES RANGING UP TO 17 YEARS PLUS IN PRISON....PRESIDENT BIDEN SPEAKS OUT, " MY SON HAS DONE NOTHING WRONG EXCEPT HAVING THE LAST NAME OF BIDEN"
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Re: Lies, Lies, Lies, they're all LIES BY GOD!!!
Posted on: 2023-12-08 16:29:07
By: Anonymous
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HAHAHAHAHAHA 🤣 LMAO HAHAHAHAHAHA
LISTEN TO HUNTER BIDEN CRYING AND WHINING SAYING ITS ONLY HAPPENING BECAUSE REPUBLICANS ARE AFTER HIS DAD.
SO IT MUST BE OKAY HUNTER TO WRITE OFF $800,000 TO CLAIM STRIPPERS, ESCORTS, AND SEX CLUBS AS A BUSINESS WRITE OFF?
HERE'S THE HEADLINE ON JUST ONE FOX NEWS ARTICLES "Defiant Hunter Biden says Republican 'motherf---ers' are 'trying to kill me'
Hunter Biden dismissed the idea of the 'Biden Crime Family,' arguing his dad released decades-worth of his tax returns
MY SON HAS DONE NOTHING WRONG!!! OKAY JOE, WE BELIEVE YOU.
WOULD YOU LIKE TO TALK ABOUT THOSE 20 LLC'S UNDER THE BIDEN TRADEMARK NAME AND THE MILLIONS OF DOLLARS THAT WERE DEPOSITED INTO THEM THEN DISPERSED INTO YOUR FAMILY? THEN IF YOU WOULDNT MIND MR PRESIDENT, HOW WERE YOU ABLE TO AFFORD TWO MULTIMILLION DOLLAR HOMES ON A SENATOR'S SALARY? YOU ALWAYS SOUNDED SO HUMBLE SAYING HOW YOU WERE THE POOREST SENATOR IN CONGRESS. WERE YOU INCLUDING YOU MILLIONS NOT REPORTED TO THE IRS , MR PRESIDENT?
PS: THANK YOU MR PRESIDENT FOR GOING AFTER TAX CHEATS, BIT PLEASE DON'T PARDON THIS ONE, WE KNOW HE'S NEVER HAD A GOOD FATHER TO TEACH HIM THE BASICS, AFTER ALL HIS SISTER IS A RECOVERING ALCOHOLIC AND DRUG ADDICT TOO, SHE BLAMED HER DAD FROM WHEN SHE WAS A LITTLE GIRL AND MOLESTING HERE, MAKING HER TAKING SHOWERS WITH HIM.
THOSE KIDS SURE HAD A TERRIBLE FAMILY HOUSEHOLD.
JUST REMEMBER.....MY SON HAS DONE NOTHING WRONG!!!
SEASONS GREETINGS TO EVERYONE MERRY CHRISTMAS EVERYONE AND HAPPY NEW YEARS TO ALL THE TAX CHEATERS PRESIDENT BIDEN VOWS TO PAY THEIR SHARE!!!
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Re: Lies, Lies, Lies, they're all LIES BY GOD!!!
Posted on: 2023-12-08 16:45:26
By: Anonymous
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2016 debate against Hillary Clinton.
How come you don't pay any taxes Donald?
I do pay taxes.
$1500 a year?
Yes.
That doesn't sound fair.
It sounds like you're a criminal.
No, it makes me smart.
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No Subject
Posted on: 2023-12-09 06:32:05
By: Anonymous
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First of all. Anyone who still believes trump is qualified to be a president. Is clearly not smart. We would call that repeating the same mistakes over and over.
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Re:
Posted on: 2023-12-09 06:35:23
By: Anonymous
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He claimed his taxes were under audit. And he would release them. He never did. Tried to hide the fact he paid vertically zero taxes. And paid more taxes to China then in the United States.
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Re:
Posted on: 2023-12-09 06:38:26
By: Anonymous
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The fraud in New York. He has already been found guilty. This part of that trial will determine how much he must pay back. And if he can continue to do business in New York.
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Re:
Posted on: 2023-12-09 06:45:42
By: Anonymous
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He has sued countless people in his time. Trying to used his money and the courts to punish his enemies So when he looks like his about to cry. What does 70% of the country say. Fu— off lying criminal insurrectionist.
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Re:
Posted on: 2023-12-09 07:41:08
By: Anonymous
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Black Lives Don't Matter
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Re: bldm
Posted on: 2023-12-09 07:51:20
By: Anonymous
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Brought to you by a racist Calabama dotard.
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Re: bldm
Posted on: 2023-12-09 12:38:14
By: Anonymous
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I'm Anti doofus thank you sniveler...,,,-)
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Re: bldm
Posted on: 2023-12-09 13:14:33
By: Anonymous
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I'm a doofus but look who's on all fours this won't hurt Sniveler it's meat injection time
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Re: Lies, Lies, Lies, they're all LIES BY GOD!!!
Posted on: 2023-12-09 07:55:18
By: Anonymous
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HEY MR. ALL CAPS!!!! MIGHT WANT TO UPDATE YOUR MEDS!!! YOUR GONNA BLOW A VALVE MORON!!!
HAHAHAHAHAHAHAHAHAHAHAHAHAHAHAHAHAHAHAHA
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Re: Lies, Lies, Lies, they're all LIES BY GOD!!!
Posted on: 2023-12-09 08:11:10
By: Anonymous
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I BLEW MY NUT ALL OVER SNIVELER'S FACE...,,,
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Re: Lies, Lies, Lies, they're all LIES BY GOD!!!...my nut
Posted on: 2023-12-09 08:55:41
By: Anonymous
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The delusional pervert can't seem to stay in his hole.
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Re: Lies, Lies, Lies, they're all LIES BY GOD!!!...my nut
Posted on: 2023-12-09 13:48:05
By: Anonymous
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Because I'm in yours
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Re: Lies, Lies, Lies, they're all LIES BY GOD!!!
Posted on: 2023-12-09 08:56:03
By: Anonymous
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John John
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Re: Lies, Lies, Lies, they're all LIES BY GOD!!!
Posted on: 2023-12-09 12:05:21
By: Anonymous
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Biden makes the news again and it's not about falling down, or unable to complete a sentence, or his son facing multiple felony charges, how weak and feeble he is, or his failures on foreign policies and causing two wars, or his failures on protecting our borders, or I better stop or I'll be here for hours on all his failures. No, nope, Biden broke another record, his approval ratings fell AHAIN TO AMOTHER ALL TIME LOW!!! Yup, he's officially the worst president in history, and probably the most loved president in the History of our enemies!!!
"President Biden's approval rating has hit an all-time low, and he trails former President Donald Trump in a hypothetical head-to-head 2024 matchup."
On the bright side, Joe doesn't know it, he doesn't even know he's the President. 🤣
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No Subject
Posted on: 2023-12-09 09:05:33
By: Anonymous
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Spence Ranch Feed & Supply could you please trim Trump's claws he's tearing up my back while we're having sex, Thank You Sniveler
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Re:
Posted on: 2023-12-09 09:06:35
By: Anonymous
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Thay Goe Ping Pow
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Re:
Posted on: 2023-12-09 09:20:30
By: Anonymous
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Spence feed, please also mop the floors and get better lighting. Your "store" is disgusting. Seems like the last time it was cleaned was for those pinetree.nuts advertisements 20 years ago.
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Re:
Posted on: 2023-12-09 09:53:05
By: Anonymous
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Sounds like you been bent over a bag of seed there with hard feelings.
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Re:
Posted on: 2023-12-09 11:51:47
By: Anonymous
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That's not my dog so don't thank me anti. Maybe you should stop having sex with dogs? Just a thought, and it is against the law.
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Re:
Posted on: 2023-12-09 12:22:02
By: Anonymous
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You're a dog and I haven't been arrested yet! :-)
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Re:
Posted on: 2023-12-09 12:23:14
By: Anonymous
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Ruff Ruff
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Re:
Posted on: 2023-12-09 12:28:09
By: Anonymous
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Sniveler you're going to have to do better than that, GO SIT DOWN or better yet I have something you can sit down on.
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Re:
Posted on: 2023-12-09 12:39:47
By: Anonymous
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I wish I could right bettur, Thank You Sniveler.
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Re:
Posted on: 2023-12-09 12:37:24
By: Anonymous
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Anti is one lame duck thank you sniveler...,,,-)
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Re:
Posted on: 2023-12-09 13:16:41
By: Anonymous
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Quack Quack
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No Subject
Posted on: 2023-12-09 13:18:43
By: Anonymous
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Sniveler how's things at the Shi*hole on Live Oak Dr.?
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Re:
Posted on: 2023-12-09 13:46:06
By: Anonymous
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I'm surprise you still have a internet connection that the natives haven't stolen the cable for the copper in the hood.
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Re:
Posted on: 2023-12-09 15:23:51
By: Anonymous
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Any tree lighting this weekend so I can wear my boxers on backwards, Trailer Trash
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Re:
Posted on: 2023-12-10 07:36:24
By: Anonymous
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STFU Snivleler thank you anti...,,,
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Re: I'm surprise...
Posted on: 2023-12-09 16:13:00
By: Anonymous
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Boy, you're fukn stupid.
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Re: I'm surprise...
Posted on: 2023-12-09 20:23:25
By: Anonymous
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Piss Poor sentence composition
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Re: I'm surprise...
Posted on: 2023-12-09 23:56:54
By: Anonymous
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Ahhhh, the success of the educational system in California is alive and well as reflected in the above comments.
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Re: I'm surprise...
Posted on: 2023-12-10 07:45:44
By: Anonymous
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Well thank you volunteer librarian
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Re: I'm surprise...
Posted on: 2023-12-10 20:26:49
By: Anonymous
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I know it’s a lot to ask a liberal democrat. Roll over to RUMBLE. New room Junkies Cartoons. And with common sense watch how embarrassing the Democrats have become. It’s a disgrace. They have forgotten they work for us not personal profit.
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Republicans
Posted on: 2023-12-11 06:22:46
By: Anonymous
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Republicans stupidly matters.
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