Near future
No Result
View All Result
27 September 2023
  • Home
  • Tech
  • Health
  • Environment
  • Energy
  • Transports
  • Spazio
  • AI
  • concepts
  • H+
Understand, anticipate, improve the future.
CES2023 / Coronavirus / Russia-Ukraine
Near future
  • Home
  • Tech
  • Health
  • Environment
  • Energy
  • Transports
  • Spazio
  • AI
  • concepts
  • H+

Understand, anticipate, improve the future.

No Result
View All Result
Medicine

The first CRISPR therapy arrives in 2023: it will hit thalassemia and rare blood diseases

The first CRISPR gene editing application against thalassemia and other blood diseases will change the future of drug development.

July 2 2022
Gianluca RiccioGianluca Riccio
⚪ 4 minutes
Share6Pin2Tweet4SendShare1ShareShare1
stop thalassemia

READ IN:

You all know CRISPR, by now: the real, great novelty of genetics in recent years (which resulted in scientists Jennifer Doudna and Emmanuelle Charpentier il 2020 Nobel Prize in Chemistry). Until a few years ago, a technique to "cut and sew" parts of DNA seemed like science fiction, rather than medicine.

And now here we are: the very first treatment that uses this gene editing technique to combat thalassemia and other rare inherited blood disorders, affecting millions of people around the planet, could arrive in the coming months.

Exa-Cel: thalassemia, sickle cell anemia and more in focus

The therapy, called Exa-cel (short for exagamglogene autotemcel), was created as part of a $ 900 million collaboration between Vertex, specialist in rare diseases, e CRISPR Therapeutics. It has already shown promise in assisting patients with beta thalassemia and sickle cell anemia, two rare genetic blood disorders.

La beta thalassemia, in particular, it is characterized by damaged or missing genes that reduce the production of hemoglobin (an essential oxygen-carrying protein). This can lead to enlargement of the liver, spleen or heart and bone malformations. One in every 100.000 people in the world is afflicted with it, and regular blood transfusions are needed to keep it from becoming fatal.

Sickle cell anemia is instead attributed to a defective gene that changes the consistency of hemoglobin, preventing healthy blood cells from carrying oxygen around the body.

thalassemia
Thalassemia forces patients to take constant transfusions to avoid the worst.

2023, the year of contact

Exa-cel has greatly reduced the blood transfusions needed by thalassemia sufferers and the worst conditions associated with this disorder. He did this for months, years following the therapy. An impressive amount of results presented last month paved the way for this first gene-editing treatment, which is now reaching the general public.

Exa-cel is expected to go through regulatory approval in the US, UK and Europe by the end of this year, allowing it to receive marketing authorization in 2023. There we are.

The article continues after the related links

Spider silk from modified silkworms: stronger than Kevlar and 100% natural

How CRISPR can feed the planet

How does the CRISPR Exa-cel therapy against thalassemia and sickle cell disease work?

In the case of Vertex and CRISPR Therapeutics, CRISPR gene editing (performed outside the body) is used "ex vivo" (on a sample taken from the patient). Stem cells are extracted, cellular DNA is modified by exa-cel to stimulate the formation of a type of hemoglobin that the body produces only in childhood, and the modified cells are reinserted into the patient. The production of healthy hemoglobin and red blood cells increases, the damage is reduced or eliminated.

The clinical data presented at the 2022 European Hematology Association Congress in Switzerland are overwhelming. All patients with beta thalassemia or sickle cell anemia undergoing Exa-cel had a near-zero need for transfusions. Of the 44 patients with thalassemia, 42 did not have a single one during all 37 months of post-therapy observation. 2 reduced the blood needed for transfusion by 75% and 89%.

What about the 31 patients with severe sickle cell anemia? None experienced vaso-occlusive crises in the 32-month post-therapy observation. On average, they had 4 seizures a year before receiving Exa-cel.

thalassemia
CRISPR and other gene therapies will revolutionize the production of drugs and treatments over the next decade

Huge potential

CRISPR is not the only type of gene therapy currently in development. There are several, and according to MIT's NEWDIGS drug development program, more than 60 gene and cell therapies could be on the market by 2030.

This will forever change the way we think about diseases that are now considered "incurable". We could gradually develop gene and cellular therapies to treat many ailments, from rare diseases to HIV through to heart ailments.

We start with thalassemia, but the road has just been opened.

Tags: CRISPRthalassemiagene therapy

Latest news

  • On Mars in 26 days with the new airbrush solar sail
  • Amazon invests in Anthropic: the AI ​​war rages on
  • Smile: Drug that regrows teeth reaches clinical trials
  • Without a brake from the institutions, will AI lead to private neo-feudalism?
  • When will we become extinct? Here comes a (nice) prediction from the supercomputer
  • DNA and longevity: how 'jumping genes' could extend our lives
  • TeddyGPT arrives, the first AI teddy bear that talks and learns with children
  • Minimum wage: a step forward or a leap in the dark for Italy?
  • Solar cars could halve the need for charging
  • The crazy food of the future: from edible clouds to mussel ceramics


GPT Chat Megaeasy!

Concrete guide for those approaching this artificial intelligence tool, also designed for the school world: many examples of applications, usage indications and ready-to-use instructions for training and interrogating Chat GPT.

To submit articles, disclose the results of a research or scientific discoveries write to the editorial staff

Enter the Telegram channel of Futuroprossimo, click here. Or follow us on Instagram, Facebook, Twitter, Mastodon e LinkedIn.

FacebookTwitterInstagramTelegramLinkedInMastodonPinterestTikTok

The daily tomorrow.


Futuroprossimo.it provides news on the future of technology, science and innovation: if there is something that is about to arrive, here it has already arrived. FuturoProssimo is part of the network ForwardTo, studies and skills for future scenarios.

  • Environment
  • Architecture
  • Artificial intelligence
  • Gadgets
  • concepts
  • Design
  • Medicine
  • Spazio
  • Robotica
  • Work
  • Transports
  • Energy
  • Edition Francaise
  • Deutsche Ausgabe
  • Japanese version
  • English Edition
  • Portuguese Edition
  • Read more
  • Spanish edition

Subscribe to our newsletter

  • The Editor
  • Advertising on FP
  • Privacy Policy

© 2023 Near future - Creative Commons License
This work is distributed under license Creative Commons Attribution 4.0 International.

No Result
View All Result
Understand, anticipate, improve the future.
  • Home
  • Tech
  • Health
  • Environment
  • Energy
  • Transports
  • Spazio
  • AI
  • concepts
  • H+