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Even the greatest Creator has its work at its own times
.
Embodied in humans, a gene that the creator inadvertently mistakes may bring a person lifelong disease torment
.
The good news is that human beings have always had ambitions to change their lives
.
For a long time, the medical community has been trying to make up for the negligence
of the Creator through acquired efforts.
Based on this, gene editing tools continue to iterate
.
The birth of CRISPR technology has brought gene editing from straight machines to the era of smart machines, and has also brought us closer
and closer to the goal of successfully repairing human genetic defects.
Nowadays, many big names in the medical field are trying to modify and modify specific target genes of the human genome based on CRISPR technology, and from in vitro editing to in vivo editing, the steps are getting bigger
and bigger.
However, from ideal to reality, there is always a gap that needs to be bridged
.
In reality, even Editas, a star company backed by super big coffee, may not always be successful
in the research and development of new technologies.
On November 17, Editas announced that it is suspending clinical studies
of the world's first in vivo gene-editing therapy, EDIT-101.
The reason behind this may be that the efficacy of EBT-101 is less than expected
.
On the same day, affected by this news, the star company's stock price fell by 10.
12%, leaving a market value of only $750 million, nearly 90%
less than the peak market value (6.
8 billion US dollars).
The huge gap also tells us that whether it is big coffee or "black technology", it is difficult to make the research and development of innovative drugs difficult to ascend to the sky
.
/ 01 /
/ 01 /In vivo gene editing therapy with high hopes
In vivo gene editing therapy with high hopesIn theory, CRISPR gene therapy can change genes, repair genes, screen genes, and help humans change DNA and RNA
.
To exaggerate, in the future, with the support of CRISPR gene therapy, humans can become their own creators
.
Of course, it may take a long time
to achieve this goal.
At present, the use scenarios of CRISPR gene therapy are still focused on single-gene genetic diseases
.
According to whether it is edited in vivo, it can be roughly divided into two categories:
One is in vitro gene editing therapy, which extracts the patient's diseased cells for gene editing outside the body, and then infuses the cells back;
The other type is in vivo gene editing therapy, which directly delivers the gene editing system to the patient through the vector, and modifies
the patient's genes in vivo.
In contrast, in vitro gene editing therapy technology is simpler, but more expensive to prepare; In vivo gene editing therapy is more difficult, but it is more cost-effective and imaginative
.
This is because in vitro gene editing therapy is suitable for fewer organs, tissues, or cell types, so most of the indications are focused on blood diseases; In vivo gene editing therapy can be used for a variety of diseases, and the imagination space is larger
.
The pharmaceutical company Editas we are going to talk about today is a pioneer
in in vivo gene editing therapy.
Editas, founded in November 2013, had multiple halos at the beginning of its launch
.
Its founders include Zhang Feng, a biologist who holds a patent for CRISPR gene editing
.
In addition to its bright background, Editas' drug development has not been slow
.
On July 25, 2019, Editas launched a clinical trial of CRISPR/Cas9 in vivo gene editing therapy EDIT-101, which aims to correct genetic defects in retinal photoreceptor cells to treat Leber congenital black mong10
(LCA10), which is also the world's first in vivo gene editing therapy
to enter the clinic.
With leading pipelines and the addition of many big names, investors are looking forward
to the future of Editas.
In January 2021, although no product was yet available, Editas
At one point, the company's market capitalization soared to $6.
8 billion
.
However, even with the blessing of multiple halos, Editas' first in vivo injection CRISPR/Cas9 gene editing therapy has temporarily
overturned.
/ 02 /
/ 02 /EDIT-101 IS PRESSED THE PAUSE BUTTON
EDIT-101 IS PRESSED THE PAUSE BUTTONPreviously, the biggest concern about CRISPR/Cas9 gene editing in vivo was safety
.
The core reason is that CRISPR-Cas is easy to "off-target" and change other normal genes
.
There is no doubt that this mistake will bring new hidden dangers
to the patient's health.
In vitro gene therapy drugs, developers can identify off-target cells and normal cells through specific techniques; And in the body, it is obviously difficult to do this
.
Therefore, the safety of gene editing in vivo is more difficult to control
than gene editing in vivo.
However, in previous clinical trials, EDIT-101 did not appear to have safety issues
.
According to the early clinical results of EDIT-101, patients experienced only mild to moderate treatment-related inflammation during treatment, which can be controlled
with steroids.
However, in addition to the safety of a therapeutic drug, the most fundamental evaluation dimension is the effect
.
Unfortunately, Editas did not perform well in terms of efficacy, and on November 17, it announced the phase I/II clinical trial data
of EDIT-101.
The clinical trial enrolled 14 patients, and only three responded to
EDIT-101 treatment.
Also, 2 of the homozygous patients responded, but only 1 of the 12 heterozygous patients did
.
This means that EDIT-101 is generally effective in heterozygous LCA10 patients and slightly better in homozygous LCA10 patients
.
But know that the number of LCA10 patients is not large, there are about 1500 patients
in the United States.
If this is narrowed down to homozygous LCA10, the number of patients is even smaller, estimated at just 300
.
Let's not talk about whether the effects shown so far by EDIT-101 can generally be clinically successful
.
Taking a step back, even if Editas burns all the money and successfully sends EDIT-101 to market, such a narrow patient population determines that EDIT-101 is unlikely to have much room for imagination
.
It is for this reason that on November 17, Editas announced that it was suspending clinical trials of EDIT-101 and seeking partners to continue developing EDIT-101
.
A pioneer in in vivo gene therapy, EDIT-101 was pressed the pause button
.
/ 03 /
/ 03 /The research and development of innovative drugs is difficult to climb to the sky
The research and development of innovative drugs is difficult to climb to the skyThe failure of EDIT-101 does not mean that in vivo editing therapy will definitely stop advancing, but it can once again tell us that innovative drug research and development is difficult to achieve in one step
.
The development of science and technology has always been a spiral, and new technologies and new products never appear
out of thin air.
In the field of biopharmaceuticals, this conclusion still applies
.
In fact, biopharmaceuticals have always been a field
that requires continuous experience.
Most of these emerging technologies will not grow too easily or too shortly
.
Even technologies such as ADC and bispecific antibodies that seem to be mature today have had a history of arduous struggles when turning a few pages back in their history
.
Failure is the norm for innovative drugs, especially First in class drug development
.
The fall of Editas is not a revelation
for the domestic pharmaceutical market.
In the past, there was no real First in class drugs in China, and most pharmaceutical companies did lower risk me too and fast
Follow drugs, so it's hard to really feel the brutality
of First in class drug development.
Nowadays, with the increasing number of voices against me too, many people have developed First in class worship, and more and more innovative drug players have begun to test the waters of First in class
.
However, before pharmaceutical companies enter this field, they may have to be mentally prepared
in advance.
The real war of nine deaths has just begun
here.
