A group of hemophilia patients with more than 300 people.
The group leader sent a link to vote on the Longtu app.
"Everyone, hurry up and vote. Whether the children's disease can be cured depends on whether we work hard or not."
"receive"
"+1 received"
"Received +2"
"+3 received"
Xiaomei's mother is also a family member of a child with hemophilia. She has heard of the Longtu app, which is said to be a very popular software recently, but what about this vote? Why is it related to the child's illness?
She couldn't help but be curious: "Brother, group leader, what's going on with this vote? Why does it have anything to do with the children's illness?"
Group leader: "...Are you new here?"
"Yes, I just joined the group today."
"No wonder, it's like this. You should have heard of the genetic medicine of Sanqing Pharmaceutical. The Longtu app is also a software developed by this pharmaceutical company."
Xiaomei's mother timidly replied: "I have heard of it. It is said that this company is very powerful. It has developed many miracle medicines. I even registered this software specially. It is very easy to use."
"As you know, they recently held a voting event on it. Each rare disease patient can vote for his or her disease. In this way, the disease with the largest number of people will be selected, and they will give priority to developing symptomatic genetic drugs. Now you should know How important is this vote?"
Xiaomei's mother was suddenly shocked: "Ah, there is still such a thing, then I have to go 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 were already dozens of options for rare diseases, such as albinism, ALS, osteogenesis imperfecta, pulmonary hypertension, and tuberous sclerosis. syndrome, Fabry disease, etc.
After searching for a while, I found the option of hemophilia. I quickly selected it and then voted.
After the votes were cast, the number of votes for each option was also displayed. The number of hemophilia patients is not small. There are tens of thousands of people across the country. This vote currently has more than 10,000 votes. Not to mention being far ahead, it is still 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.
The one with the most votes is type I diabetes, followed by some common rare diseases, such as ALS and thalassemia. These diseases have a relatively large number of people, and there are already tens of thousands of votes for the options.
The options at the later stage have fewer people, such as Fabry disease, a rare X-chromosome sex-linked lysosomal storage disease, with only about 300 patients nationwide. Although the patients are very united and the number of votes is close to 300, the ranking is still very low because the total number is too small.
The diseases ranked at the bottom received only a pitiful dozen votes. These are simply rare diseases among rare diseases.
The last column of options is 'Others'. After selecting, patients can fill in other rare diseases not listed above.
Xiaomei’s mother took a screenshot of her vote and happily posted it to the group.
"Brother, group leader, I have finished voting. Our votes are still quite high."
The group leader was very happy after seeing this: "Not bad, not bad, we have reached the sixth place, keep it up. Do you know other patient groups? Anyone with hemophilia can canvass for votes. Try to get into the top five, so that the three If Qing Dynasty develops genetic medicine, we can be considered earlier.”
"According to the speed at which Sanqing has developed a genetic drug in the past three months, we will have to wait for a year and a half, and we will probably have a genetic drug 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, then slowly said: "Mother Xiaomei, didn't you pay attention to the voting statement? Only patients and family members can vote. Others can't vote even if they open this page."
"Ah, there is such a thing? Then how does it know whether I am a patient?"
"Don't you need real-name authentication when you register? Then you have to fill in the electronic medical record. Only if you meet the conditions of rare diseases can you vote."
"That's it. It seems that there is no way to canvass votes in the circle of friends. I have several other patient groups, so I will send the link and let them all vote."
Xiaomei's mother was speechless for a moment, so she had no choice but to share the link and continue canvassing for votes among other patient groups.
The same scene also happened among patients with other rare diseases. Each patient's family members were canvassing hard for votes, trying to get the most votes to increase Sanqing's priority for their own disease when developing genetic drugs.
But they don't know that Sanqing's development of genetic medicines is not entirely based on the number of people.
The large number of patients is only one of the three major factors that led Sanqing to choose to conduct research and development.
The other two factors are sponsorship fees and the other decisive factor is the terminal illness index.
Sponsorship fees are easy to understand. Some wealthy people or their family members will also suffer from rare diseases. They often spend a lot of money to sponsor 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 whether the disease is fatal, and the other is to see whether there is any medicine available.
At present, less than 5% of rare diseases can be effectively intervened or treated. There are 68 domestically approved drugs for 121 rare disease indications, 48 of which have been included in medical insurance. For these diseases for which drugs are already available, research and development The priority of genetic medicine is obviously not that high.
Just like Menkes syndrome, although there are only 61 people, children with the disease usually only live three to four years, and even if they take relieving drugs such as copper histidine, they cannot live a few more years.
Such a disease has a very high terminal illness index and is undoubtedly a high priority.
In short, these three factors need to be taken into consideration in Sanqing’s list of rare diseases to be allocated 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. Because he is an entrepreneurial veteran, he has already achieved financial freedom.
However, shortly after he turned 40, he discovered that he had become a patient with ALS.
After suffering from this disease, his life took a different path.
He was a powerful business elite in the workplace, but after suffering from ALS, he became a lonely and disappointed terminally ill patient.
In the face of ALS, all his defenses have collapsed.
Amyotrophic lateral sclerosis, also known as amyotrophic lateral sclerosis, is a rare motor neuron disease. Professor Hawking, who is familiar to people, has bravely fought against als for many years.
It has been more than 100 years since the first patient with ALS was discovered, but the cause and treatment have not yet been found clinically. This is a disease more terrifying than cancer. Once diagnosed, it is equivalent to being sentenced to death. received the death penalty.
There are nearly 200,000 ALS patients in our country, and thousands of patients are seeking medical treatment everywhere. Once suffering from ALS, the patient's muscles will gradually atrophy, eventually leading to the inability to walk, difficulty breathing and swallowing, and about 8 Most patients will die within 5 years of diagnosis.
Valley altar
If it could be treated with money, Zhang Lei would not have to worry about his life at all.
Unfortunately, no matter how rich you are, there is no cure for ALS.
Currently, there is only one drug called "Lirutai" in the world, which can delay the patient's life cycle by three months, but it cannot cure ALS.
Doctors often tell patients bluntly that after diagnosis, they will usually die within 5 years.
Zhang Lei was instantly desperate.
He had only been married and had children for less than two years, and he was unwilling to give up just like that no matter how hard he thought about it.
Can’t money buy good health?
Even if he loses all his wealth, he will still find a way to survive for himself.
So, after much thought, he made a crazy decision: to establish a company to overcome the incurable disease of ALS.
Not only that, he also established the 'Healing Mutual Aid House' to provide a platform for patients with ALS to communicate and receive treatment.
In just three years, Zhang Lei invested tens of millions in this platform and established the world's largest single database.
Until Sanqing announced its genetic medicine, this platform had 6 drug lines under development. Although it may not be successfully developed in time, it at least gave people suffering from ALS a glimmer of hope and encouraged them to a large extent. They can make them stronger to face the disease.
Zhang Lei immediately paid attention to Sanqing's research and development of rare disease genetic drugs. Out of caution, he waited for a while. It was not until the second menkes genetic drug was released that he finally made a decision.
Sanqing is indeed powerful and has been continuously developing genetic drugs for rare diseases.
He was immediately excited and quickly contacted Sanqing, willing to spend tens of millions of dollars to sponsor the research and development of genetic drugs for ALS.
After some discussions, a crowdfunding campaign for ALS was officially launched on the Longtu app.
Without saying a word, Zhang Lei immediately donated the first fund, amounting to 10 million.
He also mobilized his contacts to conduct extensive publicity and joined forces with the ALS Charity Foundation to raise funds.
Crowdfunding funds for ALS are growing at a visibly high rate every day.
In just one week, the amount reached tens of millions. Among them, tens of thousands of ALS patients undoubtedly contributed a lot. They contributed with their strength and money. Almost everyone donated hundreds of yuan to put together a An extremely considerable amount.
For Sanqing, this money can already cover part of the research and development costs.
So, not long after, the crowdfunding campaign came to a successful conclusion, with a total of nearly 60 million raised. Sanqing also announced that it would set up a dedicated ALS research and development team to strive to develop genetic drugs as soon as possible.
This is not the first crowdfunding for a scientific research project, but it is the first crowdfunding for genetic drugs.
The success of crowdfunding has undoubtedly stimulated other patients with rare diseases, and they have also begun to follow suit and launched crowdfunding on Longtu.
If the crowdfunding is successful, the funds will become Sanqing's R&D sponsorship fees. 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 Sanqing's research and development of genetic drugs for their diseases as soon as possible.
After all, who doesn’t want to be like patients with SMA and Menkes, who can completely relieve their pain with just a few injections?
However, not all crowdfunding can be successful immediately. Sanqing undoubtedly has its own standards when choosing its next research and development target. If it does not meet the standards for the time being, it can only wait slowly.
With the advancement of crowdfunding and voting activities, the problem of screening and finding rare disease patients is gradually being solved.
Compared with the disease management of common diseases and chronic diseases, patients with rare diseases have greater stickiness to services. Some patients will also spontaneously set up platforms to help each other, exchange information, popularize medicines, and share good doctors with their patients. .
As the first platform in China that fully serves patients, Longtu not only improves patients' understanding of the disease, allowing them to make early diagnosis and treatment, but also allows patients to unite and make their voices heard, and express their opinions on the pharmaceutical companies. Drug research and development has had a certain impact.
There has never been a moment like this when the patients discovered that the power displayed by their unity was so powerful.
And the legendary personalized gene therapy is actually so close to me.
Just when Sanqing broke into the field of rare diseases, broke new ground and gained a lot.
Global pharmaceutical companies are also eyeing this uncultivated virgin land, making plans in advance and accelerating their efforts to enclose the land.
You know, 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, which has become a red ocean, rare diseases are a huge blue ocean with amazing market potential.
Major multinational pharmaceutical companies are successively developing orphan drugs, such as Novartis, Roche, Bayer, Pfizer, Takeda, etc.
Compared with other diseases, orphan drug research and development is relatively high-investment, low-risk, and has high output. This has been repeatedly verified in overseas markets.
The number of patients with many rare diseases is higher than that of some cancer types that have attracted much attention, such as leukemia and melanoma. Moreover, most rare diseases are chronic diseases, and most patients require lifelong medication. The average medication cycle is far longer than that of oncology drugs.
Industry reports show that the domestic rare disease track is on the eve of taking off, and the market size is estimated to exceed 10 billion US dollars in 5-10 years. This only accounts for 3%-5% of the global rare disease market, or is only equivalent to the orphans of Eagle Country. 10% of the size of the pharmaceutical market in 2020.
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 Pfizer’s rare disease business has launched a number of rare disease drugs in China, mainly covering the three fields of hemophilia, polyneuropathy, and transthyretin amyloidosis cardiomyopathy.
In recent times, AstraZeneca has also frequently made moves in the rare disease business. Not only did it announce the formal establishment of the China Rare Disease Business Unit in September last year, it also completed a US$39 billion acquisition of the rare disease giant Alexion in July. M&A cases.
Roche Pharmaceuticals spent US$3 billion in August last year 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 US$4.3 billion. This company has a very rich pipeline, covering genetic eye diseases, hemophilia, lysosomal storage diseases, and neurodegenerative diseases. disease.
Three years ago, Takeda Pharmaceutical spent a huge sum of US$65 billion to acquire rare disease giant Shire Pharmaceuticals.
According to incomplete statistics, in the past five years, the cumulative value of acquisitions and mergers and acquisitions in the field of rare diseases has exceeded 100 billion US dollars.
The major pharmaceutical giants are gearing up to enter the field of rare diseases, preparing to flex their muscles and kill you to the death.
But now, another giant beast has entered this vibrant field with unlimited potential.
As soon as he appeared on the stage, he carved a bloody path with unrivaled power, standing on the top and looking down on the world.
