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Written | Edited by Wang Cong | Nagashi Typesetting | Shuichengwen A few days ago, Beam Therapeutics announced a collaboration with Apellis Pharmaceuticals to create new gene editing therapies for complement-driven diseases, a type of disease caused by overactive immune system
.
Complement is a group of proteins that are activated in the serum and tissue fluid of normal humans and animals and have enzymatic activity.
Complement helps to replenish antibodies and white blood cells to resist pathogen invasion.
However, when the complement system is over-activated, it will damage itself.
Healthy tissues, leading to inflammation and autoimmune diseases
.
In their five-year cooperation, Beam will receive an advance payment of 50 million U.
S.
dollars and subsequent annual funding of 25 million U.
S.
dollars
.
Both parties will use Beam's base editing technology to discover new therapies for various targets in the complement system
.
This collaboration was reached a few days after Intellia Therapeutics announced the results of the first clinical trial of CRISPR gene editing therapy in individuals
.
On June 26, 2021, NEJM, a top international medical journal, published the results of Intellia Therapeutics' CRISPR gene editing therapy clinical trial
.
This in vivo gene editing therapy uses a lipid nanoparticle (LNP) delivery vector to deliver the sgRNA targeting the disease-causing gene TTR gene and the optimized spCas9 protein mRNA sequence to the liver to treat transthyretin amyloidosis ( ATTR), safe and effective in clinical trials of 6 patients
.
This is the first published clinical trial result of CRISPR gene editing therapy in vivo.
This result has greatly expanded the application range of CRISPR gene editing therapy.
It can be directly injected into the body for efficient gene editing, which opens up new ideas for the treatment of many genetic diseases.
It can be said that this approach has opened up a new era of medicine
.
Affected by this, the stock prices of several listed CRISPR gene therapy companies have risen collectively
.
Unlike companies such as Intellia, Editas, and CRISPR, which focus on the treatment of genetic diseases through gene editing, Beam is moving in a different direction.
It is planning to reset the human immune system through a new single-base editing technology
.
Beam and Apellis will collaborate on six R&D pipelines aimed at targeting complement protein C3 and other targets in the complement pathway to treat eye diseases, liver diseases and brain diseases driven by excessive complement activation
.
Beam Therapeutics was founded in May 2018 by Liu Ruqian, Zhang Feng, and J.
Keith Joung, three gene editing gods.
It was listed on the Nasdaq in February 2020 and has a current market value of more than US$8 billion
.
Zhang Feng (left), Liu Ruqian (middle), and J.
Keith Joung (right) The single-base editing technology that Beam relies on can achieve precise modification of a single base without causing DNA double-strand breaks, so It is considered safer than CRISPR-Cas9
.
But Beam's vision is not limited to the treatment of single-gene genetic diseases, but through the cooperation with Verve Therapeutics and Apellis Pharmaceuticals, which has just reached a cooperation, to expand single-base editing technology to more complex disease treatments
.
Verve Therapeutics is committed to the development of one-time base editing treatments for high cholesterol
.
The therapy aims to reduce cholesterol levels by blocking the PCSK9 gene, thereby preventing and treating cardiovascular diseases
.
Not long ago, the company was listed on Nasdaq, and its current total market value is nearly $2.
8 billion
.
Beam's research and development direction Beam is based on single-base editing technology, which generates precise, predictable and effective genetic modifications on the target DNA sequence by rewriting a single base in the genome
.
At present, base editors can provide a variety of gene editing effects to carry out therapeutic gene intervention, including: correcting disease-causing point mutations (Gene Correction), modifying genes to produce protective mutations (Gene Modification), and activating gene expression ( Gene Activation, Gene Silencing, Multiplex Editing
.
Beam delivery methods Beam is currently developing gene therapy delivery strategies including: electroporation, non-viral vectors (mainly LNP) and viral vectors (mainly AAV)
.
No single delivery technology can deliver drugs to different target organs with the same efficiency
.
Therefore, Beam plans to use electroporation for the delivery of extracorporeal blood cells and immune cells (for example, to treat blood diseases such as thalassemia), use LNP for in vivo delivery of liver and other organs, and use AAV for delivery to the eyes and central nervous system.
.
Beam's R&D pipeline At present, Beam's R&D pipeline can be divided into three categories: electroporation, LNP, and AAV vector according to the delivery technology
.
Among them, electroporation technology is used for tumor treatment of blood diseases, and the treatment of sickle cell disease and β-thalassemia based on electroporation technology has made the fastest progress, and clinical trials have been declared
.
LNP technology is mainly used for the treatment of liver diseases, including α1-antitrypsin deficiency, glycogen storage disease type 1a and the like
.
AAV technology is mainly used in the treatment of ophthalmic diseases, including Stargardt disease (also called fundus yellow spot disease, mostly autosomal recessive inherited disease, common in offspring of close relatives) and so on
.
Open to reprint this article open to reprint: just leave a message in this article to inform
.
Complement is a group of proteins that are activated in the serum and tissue fluid of normal humans and animals and have enzymatic activity.
Complement helps to replenish antibodies and white blood cells to resist pathogen invasion.
However, when the complement system is over-activated, it will damage itself.
Healthy tissues, leading to inflammation and autoimmune diseases
.
In their five-year cooperation, Beam will receive an advance payment of 50 million U.
S.
dollars and subsequent annual funding of 25 million U.
S.
dollars
.
Both parties will use Beam's base editing technology to discover new therapies for various targets in the complement system
.
This collaboration was reached a few days after Intellia Therapeutics announced the results of the first clinical trial of CRISPR gene editing therapy in individuals
.
On June 26, 2021, NEJM, a top international medical journal, published the results of Intellia Therapeutics' CRISPR gene editing therapy clinical trial
.
This in vivo gene editing therapy uses a lipid nanoparticle (LNP) delivery vector to deliver the sgRNA targeting the disease-causing gene TTR gene and the optimized spCas9 protein mRNA sequence to the liver to treat transthyretin amyloidosis ( ATTR), safe and effective in clinical trials of 6 patients
.
This is the first published clinical trial result of CRISPR gene editing therapy in vivo.
This result has greatly expanded the application range of CRISPR gene editing therapy.
It can be directly injected into the body for efficient gene editing, which opens up new ideas for the treatment of many genetic diseases.
It can be said that this approach has opened up a new era of medicine
.
Affected by this, the stock prices of several listed CRISPR gene therapy companies have risen collectively
.
Unlike companies such as Intellia, Editas, and CRISPR, which focus on the treatment of genetic diseases through gene editing, Beam is moving in a different direction.
It is planning to reset the human immune system through a new single-base editing technology
.
Beam and Apellis will collaborate on six R&D pipelines aimed at targeting complement protein C3 and other targets in the complement pathway to treat eye diseases, liver diseases and brain diseases driven by excessive complement activation
.
Beam Therapeutics was founded in May 2018 by Liu Ruqian, Zhang Feng, and J.
Keith Joung, three gene editing gods.
It was listed on the Nasdaq in February 2020 and has a current market value of more than US$8 billion
.
Zhang Feng (left), Liu Ruqian (middle), and J.
Keith Joung (right) The single-base editing technology that Beam relies on can achieve precise modification of a single base without causing DNA double-strand breaks, so It is considered safer than CRISPR-Cas9
.
But Beam's vision is not limited to the treatment of single-gene genetic diseases, but through the cooperation with Verve Therapeutics and Apellis Pharmaceuticals, which has just reached a cooperation, to expand single-base editing technology to more complex disease treatments
.
Verve Therapeutics is committed to the development of one-time base editing treatments for high cholesterol
.
The therapy aims to reduce cholesterol levels by blocking the PCSK9 gene, thereby preventing and treating cardiovascular diseases
.
Not long ago, the company was listed on Nasdaq, and its current total market value is nearly $2.
8 billion
.
Beam's research and development direction Beam is based on single-base editing technology, which generates precise, predictable and effective genetic modifications on the target DNA sequence by rewriting a single base in the genome
.
At present, base editors can provide a variety of gene editing effects to carry out therapeutic gene intervention, including: correcting disease-causing point mutations (Gene Correction), modifying genes to produce protective mutations (Gene Modification), and activating gene expression ( Gene Activation, Gene Silencing, Multiplex Editing
.
Beam delivery methods Beam is currently developing gene therapy delivery strategies including: electroporation, non-viral vectors (mainly LNP) and viral vectors (mainly AAV)
.
No single delivery technology can deliver drugs to different target organs with the same efficiency
.
Therefore, Beam plans to use electroporation for the delivery of extracorporeal blood cells and immune cells (for example, to treat blood diseases such as thalassemia), use LNP for in vivo delivery of liver and other organs, and use AAV for delivery to the eyes and central nervous system.
.
Beam's R&D pipeline At present, Beam's R&D pipeline can be divided into three categories: electroporation, LNP, and AAV vector according to the delivery technology
.
Among them, electroporation technology is used for tumor treatment of blood diseases, and the treatment of sickle cell disease and β-thalassemia based on electroporation technology has made the fastest progress, and clinical trials have been declared
.
LNP technology is mainly used for the treatment of liver diseases, including α1-antitrypsin deficiency, glycogen storage disease type 1a and the like
.
AAV technology is mainly used in the treatment of ophthalmic diseases, including Stargardt disease (also called fundus yellow spot disease, mostly autosomal recessive inherited disease, common in offspring of close relatives) and so on
.
Open to reprint this article open to reprint: just leave a message in this article to inform
