First CRISPR therapeutic approved

PacificBlueGator · Nov 19, 2023

For sickle cell disease and beta thalassemia using gene editing. So far very promising results in curing the disease with one application. Conditionally approved in UK, with Europe and US FDA review ongoing. Remarkable how life-changing it is for people who have lived with chronic episodes of debilitating pain their entire lives to suddenly be free from it. Cost, about 2 million, and access will be a big factor with the economics of a single treatment versus a lifetime of non-curing treatments.

UK first to approve CRISPR treatment for diseases: what you need to know

In a world first, the UK medicines regulator has approved a therapy that uses the CRISPR–Cas9 gene-editing tool as a treatment. The decision marks another high point for a biotechnology that has been lauded as revolutionary in the decade since its discovery.

The therapy, called Casgevy, will treat the blood conditions sickle-cell disease and β-thalassaemia. Sickle-cell disease, also known as sickle-cell anaemia, can cause debilitating pain, and people with β-thalassaemia often require regular blood transfusions.

“This is a landmark approval which opens the door for further applications of CRISPR therapies in the future for the potential cure of many genetic diseases,” said Kay Davies, a geneticist at the University of Oxford, UK, in comments to the UK Science Media Centre (SMC).
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l_boy · Nov 19, 2023

A single infusion of a gene-editing medicine may control inherited high LDL cholesterol.

This is an example of a recently tested CRISPR modification that can gene edit to permanently fight high cholesterol.

PacificBlueGator · Nov 19, 2023

l_boy said: ↑

A single infusion of a gene-editing medicine may control inherited high LDL cholesterol.

This is an example of a recently tested CRISPR modification that can gene edit to permanently fight high cholesterol.
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Yes, moving into a large disease indication will test how successful curative treatments will be in crowded space with multiple therapeutic options

G8trGr8t · Nov 20, 2023

Cancer treatments aren't far behind. It is what the covid vaccine people were focused on before covid. Hopefully volums will drop price as it treats many more patients

PacificBlueGator · Nov 20, 2023

G8trGr8t said: ↑

Cancer treatments aren't far behind. It is what the covid vaccine people were focused on before covid. Hopefully volums will drop price as it treats many more patients
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While not gene editing, gene therapy for hematological cancers have already been approved, like Yescarta and Kymriah, and proven to be highly effective as a last option for patients with lymphomas. It's a good question on volume and cost. Most of the programs target rare diseases with few good options for patients with the exception I-boy noted above which would have higher volume.

UFLawyer · Nov 20, 2023

l_boy said: ↑

A single infusion of a gene-editing medicine may control inherited high LDL cholesterol.

This is an example of a recently tested CRISPR modification that can gene edit to permanently fight high cholesterol.
Click to expand...

I am VERY skeptical of these types of treatments and claims. On one hand you have a potential cure and on the other hand you have a potential zombie apocalypse/Planet of Apes scenario.

duggers_dad · Nov 20, 2023

Always skeptical of a need to ‘fight’ high cholesterol.

Gatorhead · Nov 20, 2023

UFLawyer said: ↑

I am VERY skeptical of these types of treatments and claims. On one hand you have a potential cure and on the other hand you have a potential zombie apocalypse/Planet of Apes scenario.
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I am Legend?

UFLawyer · Nov 20, 2023

Gatorhead said: ↑

I am Legend?
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me too

PacificBlueGator · Nov 20, 2023

UFLawyer said: ↑

I am VERY skeptical of these types of treatments and claims. On one hand you have a potential cure and on the other hand you have a potential zombie apocalypse/Planet of Apes scenario.
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The efficacy endpoints are pretty clear, patients no longer have painful episodes caused by the disease. Is your fear of new health technology due to lack of understanding how they work?

UFLawyer · Nov 20, 2023

PacificBlueGator said: ↑

The efficacy endpoints are pretty clear, patients no longer have painful episodes caused by the disease. Is your fear of new health technology due to lack of understanding how they work?
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L0L. No. I fear the people pushing it don't understand how they work...20 years from now.

l_boy · Nov 20, 2023

UFLawyer said: ↑

I am VERY skeptical of these types of treatments and claims. On one hand you have a potential cure and on the other hand you have a potential zombie apocalypse/Planet of Apes scenario.
Click to expand...

Seems pretty straightforward. A person has a genetic propensity to high cholesterol. For a rare variant it can be deadly. There is also a rare variant where people have hardly any LDL and those people don’t get heart disease and they are otherwise healthy.

There are drugs that can correct for this, and there are some in testing in pill form that are more effective than statins and have no known side effects. The genetic approach accomplishes the same thing, except permanently.

If I had the hypercholestolia or whatever it’s called, I’d gladly take the genetic alteration vs likely die early due to clogged arteries. Down the road it could also be a treatment for normal people with high cholesterol, but I suspect that will be many years in the future.

UFLawyer · Nov 20, 2023

l_boy said: ↑

Seems pretty straightforward. A person has a genetic propensity to high cholesterol. For a rare variant it can be deadly. There is also a rare variant where people have hardly any LDL and those people don’t get heart disease and they are otherwise healthy.

There are drugs that can correct for this, and there are some in testing in pill form that are more effective than statins and have no known side effects. The genetic approach accomplishes the same thing, except permanently.

If I had the hypercholestolia or whatever it’s called, I’d gladly take the genetic alteration vs likely die early due to clogged arteries. Down the road it could also be a treatment for normal people with high cholesterol, but I suspect that will be many years in the future.
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F’in with genetics to combat cholesterol seems a tad bit risky. My 90 year old mother, who was a RN, has had high cholesterol for the last 50 years. No meds. She has zero blockages and can still kick my butt. I am a hard no on the risk for reward here. Now, sickle cell, Parkinson’s or other bad diseases, I support the risk for reward.

duggers_dad · Nov 21, 2023

Healthcare devising potentially risky treatments for made-up diseases.

PacificBlueGator · Nov 21, 2023

UFLawyer said: ↑

L0L. No. I fear the people pushing it don't understand how they work...20 years from now.
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In actuality, it's the patients, parents and advocacy groups pushing for treatments, especially for rare diseases where there are few options. For people living with SCD, this is a life-changer. Here's an article on a patient in the trial, to give you that perspective: https://www.washingtonpost.com/science/2023/04/28/sickle-cell-disease-treatment-gene-therapy/

There are known and unknown risks with any novel treatment. Unfortunately, no one has a magic crystal ball to predict every outcome and there's always a risk to benefit to consider based on the testing before a clinical trial, and patients are well-informed before taking part in trials. For gene therapy trials in particular there is a mandatory 15-yr follow up for patients, due to potential for cancer, although the risk is very low. However, to just generically say something bad may happen some day, is quite the self-defeat and taking that perspective we would never have treatments for any disease.

UFLawyer · Nov 21, 2023

PacificBlueGator said: ↑

In actuality, it's the patients, parents and advocacy groups pushing for treatments, especially for rare diseases where there are few options. For people living with SCD, this is a life-changer. Here's an article on a patient in the trial, to give you that perspective: https://www.washingtonpost.com/science/2023/04/28/sickle-cell-disease-treatment-gene-therapy/

There are known and unknown risks with any novel treatment. Unfortunately, no one has a magic crystal ball to predict every outcome and there's always a risk to benefit to consider based on the testing before a clinical trial, and patients are well-informed before taking part in trials. For gene therapy trials in particular there is a mandatory 15-yr follow up for patients, due to potential for cancer, although the risk is very low. However, to just generically say something bad may happen some day, is quite the self-defeat and taking that perspective we would never have treatments for any disease.
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that is not what I am saying at all. I just have trepidation about messing with Genes/DNA. For terminal diseases and life debilitating illnesses I support, always with caution.

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