I can extract side effects.

Chapter 183 The Dawn of Personalized Gene Therapy
A group of more than 300 people with hemophilia.

The group owner sent a voting link on the Longtu APP.

"Everyone hurry up and vote. Whether the children's illness can be cured depends on whether we work hard or not."

"receive"

"+1 received"

"+2 received"

"+3 received"

Xiaomei’s mother is also a family member of a child with hemophilia. She has heard of Longtu APP, which is said to be a very popular software recently, but what happened to this voting?Why is it related to the child's illness?

She couldn't help being a little curious: "Brother, group owner, what's the matter with this vote? Why is it related to the children's illness?"

Group leader: "...Are you new here?"

"Yeah, I just joined the group today."

"No wonder, that's how it is. You should have heard of Sanqing Pharmaceutical's genetic medicine. Longtu APP is also a software developed by this pharmaceutical company."

Xiaomei's mother timidly replied: "I've heard of it. It is said that this company is very powerful. It has developed many magic medicines. I even registered this software specially. It is quite easy to use."

"It's good that you know. Recently, they held a voting activity on it. Every patient with a rare disease can vote for his or her own disease. In this way, the disease with the largest number of people will be selected, and they will give priority to the development of symptomatic gene medicine. Now you should know How important is this vote?"

Xiaomei's mother was startled: "Ah, there is still this matter, then I have to vote quickly."

She clicked on the link posted by the group leader, and it turned out to be a voting event officially released by Longtu. There are already dozens of options for rare diseases, such as albinism, ALS, osteogenesis imperfecta, pulmonary hypertension, and tuberous sclerosis. syndrome, Fabry syndrome, etc.

After searching for a while, I found the option of hemophilia. I quickly selected it and then voted.

After voting, the number of votes for each option is also displayed. The number of patients with hemophilia is not small, and there are tens of thousands of people across the country. There are currently more than 1 votes in this vote. Not to mention far ahead, it is also among the top ten List.

Xiaomei's mother breathed a sigh of relief, feeling a little lucky, and then carefully looked at the number of votes for each option.

Type I diabetes received the most votes, followed by some common and rare diseases, such as ALS and thalassemia. The number of people with these diseases is relatively large, and there are already tens of thousands of votes behind the options.

The later the options, the fewer the number of people, like Fabry disease, which is a rare X-chromosome sex-linked genetic lysosomal storage disease, with only about 300 patients nationwide.Although the patients are very united and the number of votes is close to 300, but because the total number is too small, the ranking is still very low.

For the last few diseases, the number of votes is only a dozen or so. This is simply a rare disease among rare diseases.

The last column of the options is 'Others', and patients can fill in other rare diseases not listed above after selection.

Xiaomei's mother took a screenshot of her vote and happily posted it to the group.

"Brother group owner, I've finished voting, and our votes are still quite high."

The group owner was very happy to see it: "Not bad, it's already No.6, keep it up, do you know other patient groups? Anyone with hemophilia can go to solicit votes. Try to be in the top five, so three If Qing develops genetic medicine, he can consider us earlier."

"According to the speed at which Sanqing has developed a genetic drug in the past three months, we have to wait for a year and a half, and the genetic drug will probably be available."

Xiaomei's mother felt that the group leader was right, and quickly agreed: "Brother, I will post it to Moments immediately and ask everyone I know to vote for me."

The group leader was silent for a while, and then slowly said: "Mother Xiaomei, didn't you pay attention to the voting statement? Only patients and family members can vote, and others can't vote even if they open this page."

"Ah, there is such a thing? Then how does it know if I am a patient?"

"Didn't you need real-name authentication when you registered? Then you have to fill in the electronic medical record. Only if you meet the condition of a rare disease can you vote."

"Well, it seems that there is no way to canvass votes in the circle of friends. I also have a few other patient groups. I will send the link to them and let them vote."

Xiaomei's mother was speechless for a while, so she obediently shared the link and continued to solicit votes in other patient groups.

The same scene also happened to other rare disease patient groups. The family members of each patient were canvassing votes hard, trying to win the most votes and increase the priority of Sanqing's own disease when developing genetic medicine.

But they don't know that Sanqing's research and development of genetic medicine is not all based on the number of people.

The large number of patients is only one of the three factors that made Sanqing choose to conduct research and development.

One of the other two factors is the sponsorship fee, and the other decisive factor is the terminal illness index.

Sponsorship fees are easy to understand. Some rich people or their family members will also suffer from rare diseases. They often spend a lot of money sponsoring pharmaceutical companies or biotechnology companies to develop new drugs and do their best to save themselves.

The terminal illness index has two points, one is to see if the disease is fatal, and the other is to see if there is any medicine available.

At present, less than 5% of rare diseases can be effectively intervened or treated. There are 121 domestically approved drugs for 68 rare disease indications, of which 48 have been included in the medical insurance. The priority of genetic medicine is obviously not that high.

Just like Menkes syndrome, although the number of patients is only 61, the children usually only live for three or four years. Even if they take relievers such as copper histidine, they cannot live for a few more years.

Such a disease has a very high index of terminal illness, so it undoubtedly has a high priority.

In short, on Sanqing’s list of rare diseases to overcome, these three factors need to be taken into consideration in order to allocate limited R&D resources and strive to save more patients.

******
Zhang Lei, 42 years old, is an executive of a major Internet company and the vice president of Penguin. As a veteran of entrepreneurship, he has already achieved financial freedom.

However, shortly after his [-]s, he found out that he was suffering from ALS.

After suffering from this disease, his life took a different path.

He is an all-powerful business elite in the workplace, but after suffering from frostbite, he has become a lonely and disappointed terminally ill patient.

In the face of ALS, his defenses have all collapsed.

ALS, also known as amyotrophic lateral sclerosis, is a rare motor neuron disease. The well-known Professor Hawking has fought bravely with ALS for a long time.

It has been more than 100 years since the first patient with ALS was discovered, but the etiology and treatment of the disease have not yet been found clinically. This is a disease that is more terrible than cancer. Once diagnosed, it is equivalent to being sentenced. got the death penalty.

There are nearly 20 patients with ALS in my country, and tens of thousands of patients seek medical treatment everywhere. Once suffering from ALS, the muscles of the patients will gradually atrophy, eventually leading to inability to walk, difficulty breathing and swallowing, and about 8 Successful patients die within 5 years of diagnosis.

If he could be treated with money, Zhang Lei would not have to worry about his life at all.

It is a pity that there is no cure for ALS no matter how rich you are.

At present, there is only one drug called "Lirutai" in the world, which can delay the life cycle of patients by three months, but it cannot cure ALS.

And doctors often tell patients bluntly that after diagnosis, they usually die within 5 years.

Zhang Lei was instantly desperate.

He had only been married and had children for less than two years, so no matter how he thought about it, he was unwilling to give up like this.

Can money not buy good health?

Even if he loses all his wealth, he still has to fight for a way out for himself.

So, after thinking about it, he made a crazy decision: to establish a company to overcome the terminal disease ALS.

Not only that, he also established the "Gradually Healing Mutual Aid House", a platform for ALS patients to communicate and treat.

In just three years, Zhang Lei invested tens of millions of dollars in this platform to build the world's largest single database.

Until Sanqing announced the gene drug, there were already 6 drug lines under development on this platform. Although it may not be successful in time, it at least gave people with ALS a glimmer of hope and encouraged them to a large extent. They can make them face the disease stronger.

Zhang Lei immediately paid attention to the research and development of Sanqing's rare disease gene drug. Out of caution, he waited for a while until the second Menkes gene drug came out, and he finally made a decision.

Sanqing is indeed capable, and has been continuously developing genetic medicines for rare diseases.

He was immediately excited, and quickly contacted Sanqing, willing to contribute tens of millions of dollars to sponsor the research and development of genetic medicine for ALS.

After some discussions, the crowdfunding campaign for ALS was officially launched on the Longtu APP.

Without further ado, Zhang Lei immediately donated the first fund, which was 1000 million.

He also mobilized his contacts for extensive publicity, and joined hands with the ALS Charity Fund to raise funds.

The crowdfunding funds for ALS are growing rapidly every day with visible figures.

In just one week, the amount has reached tens of millions. Among them, tens of thousands of patients with ALS undoubtedly contributed a lot. They have the strength to contribute, and they have money to contribute. Almost everyone donated hundreds of yuan to make up a A very considerable amount.

For Sanqing, this amount of money can already cover part of the research and development costs.

So, not long after, the crowdfunding campaign ended successfully, raising a total of nearly 6000 million funds, and Sanqing also announced that it would set up a dedicated research and development team for ALS, striving to develop genetic drugs as soon as possible.

This is not the first crowdfunding of scientific research projects, but it is the first crowdfunding of gene medicine.

The success of crowdfunding undoubtedly stimulated other patients with rare diseases, and they began to follow suit and launched crowdfunding on Longtu one after another.

If the crowdfunding is successful, the funds will become Sanqing’s research and development sponsorship fee. If it fails, the funds will be returned to the user’s account.

Therefore, the patients are also very relieved. They just want to promote the research and development of Sanqing's genetic medicine for their own diseases as soon as possible.

After all, who doesn't want to get rid of pain completely with a few injections like SMA and Menkes patients.

However, not all crowdfunding projects can be successful immediately. Sanqing undoubtedly has its own standards for choosing the next research and development target. If it does not meet the requirements for the time being, it can only wait slowly.

With the advancement of crowdfunding and voting activities, the problem of identifying and finding patients with rare diseases is gradually being resolved.

Compared with the disease management of common diseases and chronic diseases, patients with rare diseases are more sticky to the service. Some patients will spontaneously build a platform to help each other, exchange information, popularize medicines, and share good doctors with patients. .

As the first platform in China that fully serves patients, Longtu not only improves patients' awareness of the disease, allows patients to be diagnosed and treated early, but also allows patients to unite and make their voices heard. Drug development has had some impact.

Never had such a moment, the patients discovered that the strength displayed by their unity was so powerful.

And the legendary personalized gene therapy is so close to me.

Just when Sanqing broke into the field of rare diseases, opened up new territories, and made a lot of money.

Global pharmaceutical companies are also staring at this piece of uncultivated virgin land, making arrangements in advance and speeding up their staking.

We must know that the more difficult it is to treat rare diseases, the more opportunities there are for pharmaceutical companies on this track.

From the perspective of market size, compared with the oncology field that has become a red sea, rare diseases are a huge blue ocean with amazing market potential.

Major multinational pharmaceutical companies are developing orphan drugs successively, such as Novartis, Roche, Bayer, Pfizer, Takeda, etc.

Compared with other diseases, orphan drug research and development is relatively high investment, low risk, and high output, which has been repeatedly verified in overseas markets.

The number of patients with many rare diseases is higher than that of some high-profile tumor types, such as leukemia and melanoma.Moreover, most rare diseases are chronic diseases, and most patients need to take medicine for life, and the average medication cycle is far longer than that of tumor drugs.

Industry reports show that the domestic rare disease track is on the eve of take-off, and it is estimated that the market size will exceed 5 billion US dollars within 10-100 years, and this only accounts for 3%-5% of the global rare disease market, or only equivalent to the orphans of Eagle Country 2020% of the scale of the drug market in 10.

Last month, Pfizer announced a new organizational structure adjustment in China, and rare diseases were included as one of the six independent business units.

It is reported that in terms of rare disease business, Pfizer has launched a variety of rare disease drugs in China, mainly covering the three fields of hemophilia, polyneuropathy, and transthyretin amyloid cardiomyopathy.

Recently, AstraZeneca has also made frequent moves in the field of rare disease business. It not only announced the official establishment of Huaxia Rare Disease Business Department in September last year, but also completed a $9 billion acquisition of rare disease giant Alexion in July. M&A cases.

Roche spent $8 billion last August to reach a multi-target strategic cooperation and licensing agreement with Shape Therapeutics to jointly develop gene therapy drugs targeting specific targets in the field of Parkinson's disease and rare diseases.

In fact, as early as 2 years ago, Roche Pharmaceuticals acquired gene therapy pioneer Spark Therapeutics for $43 billion. This company has a very rich pipeline, covering genetic eye diseases, hemophilia, lysosomal storage diseases, and neurodegenerative diseases. disease.

Three years ago, Takeda spent $3 billion to acquire rare disease giant Shire Pharmaceuticals.

According to incomplete statistics, in the past five years, the acquisitions and mergers and acquisitions in the field of rare diseases have accumulated more than 5 billion US dollars.

The major pharmaceutical giants are gearing up for the field of rare diseases, preparing to flex their muscles and kill you to the death.

But now, another monster has rushed into this vibrant field with unlimited potential.

As soon as he appeared on the stage, he blazed a trail of blood with an unrivaled momentum, standing on the top of the peak and looking down on the world.

(End of this chapter)