https://www.youtube.com/watch?v=-O_vxNKEoYY?si=JzFn3xehhj511KQj

U.S. Food and Drug Administration

FDA NEWS RELEASE

FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

For Immediate Release:December 08, 2023

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.”

https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease

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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.

https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease


Cc OAM4J Nlfpmod Dominique Mynd44 Puskin FOD Mukina

More photos.

Me thinks this is a welcomed development, but I'm always skeptical about anything that has to do with gene alteration

Let me restate that my views are my personal opinion, you're entitled to hold any view opposite/parallel to mine

I thought Nigeria was the first to record breakthrough in sickle cell treatment.

So what happen to all the report submitted to WHO seeking approval.

Oh I forgot, everything must come from them first.

https://www.google.com/url?sa=t&source=web&rct=j&opi=89978449&url=https://cvcnigeria.org/nigeria-records-breakthrough-in-sickle-cell-treatment/&ved=2ahUKEwiSyumGjoSDAxV2UkEAHVtICY0QFnoECCMQAQ&usg=AOvVaw07YAGVNqrrsepSh8cH8Q7b

K

See people maximising their brain potentials. 👍

Plenty of adverse conditions in this part of the world will not allow us do same.

Nice One

Very expensive process. This is a step forward and it's not as easy as it seems.

People should still just avoid marrying if not compatible.

Y'd be surprised to find out that this research was funded by a Private Business individual/Entity.
In Nigeria, the only thing our Business people know is to Import. Even if other parts of the World pause development for 200 years, ain't sure we will Catch up.

Oyibo always looking for ways to develop themselves. Nigerians always looking for PhD (pull her/him down). Our lawmakers will be the first to turn it down so that they will continue to enjoy their medical tourism and loot monies.

While Nigeria pastors are here busy casting imaginary demons away and accusing husband family of being witches as if wife no get sister- inlaw

Great breakthrough!

Good development . Probably it might take years before it reaches Africa.And please house what are the cons and pros of marrying someone with SC genotype?

You forgot to add that it costs $2.2M per treatment.

Supremos:

https://www.youtube.com/watch?v=-O_vxNKEoYY?si=JzFn3xehhj511KQj

U.S. Food and Drug Administration

FDA NEWS RELEASE

FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

For Immediate Release:December 08, 2023

https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease

chai! Which kind brain these people get.
It's amazing. From one innovation to another. God please give me that kind brain 🙏 creative brain.

This is good news!! We are 100million light years away from these progressive countries and it keeps getting worse.

Tonytonex:
chai! Which kind brain these people get.
It's amazing. From one innovation to another. God please give me that kind brain 🙏 creative brain.

Actually, there brain is not special, they just have a better enabling environment to thrive. That's why Nigerians who go there in educational pursuit also thrive even though they mostly go at adulthood and don't have a good head start..

If we had good leaders in Nigerian who allowed our educational system blossom instead of continuously stifling it, this people will not see our back because we Nigerians are hardworking and determined and that's why you will see even secondary school students studying all night.

Some people fill their heads with advanced biology
Some people fill their heads with mohammed's quran
Some people fill their heads with holy ghost fire
Some people fill their heads with ba-la-blue

If you need your head filled up or your head fills empty, DM me,

We move!!!

If you're AS or AC, don't ever marry your type!

Marry AA like us!

That's the best cure without side effects and lifetime monitoring!

I remember when I was on SIWES in one lab like that close to the former Moshood Abiola Polytechnic Abeokuta. A family were advised not to get married but the pastor told them to go on that there's nothing God can't do!

⏩ They got married and had two kids both SS. The first died after wasting a lot of time and funds around 12 years.

They were still hoping the second would survive back then!

Great news.

Thank God. Men no body can say smack about USA.

I know say God will see us through.

Good bless America! Religion will not allow us Blacks to be innovative.

Tonytonex:
chai! Which kind brain these people get.
It's amazing. From one innovation to another. God please give me that kind brain 🙏 creative brain.

Brother, these guys are seriously blessed with Brains. Just imagine if Yankee wasn't there? They are truly a global superpower

Gh

See a country with vision ,no be to dey buy cars and share money all the time..

KingAzubuike:
You forgot to add that it costs $2.2M per treatment.

no matter the cost my dear, life is priceless you know! People are buying material things worth far more than that.
And to come out with that innovation and/or treatment is not a month or year job. It took years of research.
Kudos to them.