Eagle Country, Xincun City, a sunny weekend.
Singers opened the medical website he often visited, and a headline suddenly came into view.
"Sanqing, China's leading pharmaceutical company, claims to have developed a genetic drug to treat menkes disease!"
"This drug uses the first-generation gene editing tool zfn to successfully knock out key positions on the mutated gene, thereby achieving the goal of completely curing the patient..."
Singhs was stunned for a moment, and it took him a while to react. After reading the news in one breath, he rushed out of the study with great excitement and found his wife who was taking care of their daughter in the bedroom.
"Dear, guess what I discovered? A Chinese pharmaceutical company has developed a genetic drug that can cure menkes disease. Our daughter can be saved!"
"God, do you know what you're talking about?"
"I swear, this is all true. I saw on the news that this drug has started clinical trials in China, and there are already examples of patients being cured. Yes, they use gene editing treatment methods that can Cure this damn disease once and for all.”
"Ah, great, it's a miracle! Contact this pharmaceutical company quickly, our daughter can't wait."
"Okay, I'll contact the pharmaceutical company right away. Please wait for my good news."
Sings' daughter also suffered from Menkes Syndrome and was notified of death early by the doctor.
However, like most family members of patients, they did not give up easily, but tried their best to save themselves.
Sings is no ordinary person. He is the founder of a famous hedge fund on Wall Street. He manages more than 3 billion US dollars in assets. He was also a partner of Goldman Sachs, and his wife is also a banker.
The two established a charity called the Menkes Foundation a year ago with $10 million of their own investment. The wife serves as the chairman of the foundation and is committed to funding research on this disease.
The foundation's goal is to find a cure for their daughter and 800 other children with menkes as soon as possible before it's too late.
The couple continues to contact powerful biopharmaceutical companies, hoping to provide interested companies with considerable research funds to promote the development of drugs for menkes.
However, even with such strong financial resources and connections, things are still much more difficult than they imagined.
At an annual biology meeting, they invited the CEOs of seven pharmaceutical companies to dinner, but only two came.
Even the two CEOs who came here only did it for the sake of the two famous bankers and had no intention of conducting research and development in this area.
It’s no wonder that these pharmaceutical companies are not interested. The number of patients is too small. Even if tens of millions of charitable funds can be raised to conduct research, the potential sales of this drug are too small to recover the cost.
After some hard work, Singers was almost desperate.
He later found that he could not impress pharmaceutical companies, so he funded some university laboratories and invited experts in related fields to conduct research. However, by this time, he no longer had high hopes, and he simply relied on fate to do his best.
However, they never expected that they would be on the verge of giving up.
I actually saw the news that a Chinese pharmaceutical company was voluntarily researching rare disease drugs, and actually developed a genetic drug that can completely cure menkes.
This was undoubtedly good news from heaven, and the intense emotions between the two broke out instantly.
After a heated discussion, Singhs contacted Sanqing Pharmaceutical in the name of the foundation.
After some detailed inquiries, the existence of the genetic drug was finally confirmed. The two people were so excited that they hugged each other tightly and felt the huge impact of the miracle.
However, it is a pity that Sanqing’s drug is still undergoing clinical trials, and there is currently no clear time for its launch, let alone its overseas launch.
This is not a problem for high-end people with strong social energy like Sings.
He quickly expressed the foundation’s strong interest and expressed its willingness to fully fund overseas clinical trials of this drug.
Originally, according to Weikang's intention, almost all domestic patients with menkes' genetic drugs were cured after completing clinical trials. There was not much motivation to immediately conduct overseas phase III clinical trials. After all, it was expensive and time-consuming, so there was no need to rush. For a while. Gu Yan
But if there is sponsorship from local tycoons, it's a different matter.
So I happily agreed to the request of the wealthy family and conducted the Phase III clinical trials overseas.
With the promotion of Singer's Menkes Foundation, Sanqing's overseas branch's clinical phase III trial application submitted to the FDA was also quite smooth.
In fact, Eagle Country is also very encouraging of rare disease drugs. It provides policy incentives and fast application channels for R&D companies in this area, including a 7-year market exclusivity period, tax exemptions and registration application fee policies.
So not long after, under the foundation's golden bullet offensive, overseas clinical trials of Menkes syndrome began, and dozens of patients became the first batch of lucky ones.
The Singhs' daughter finally got her wish and took life-saving medicine.
This is another touching overseas version of the story of "the old father has no way to find any medicine, so he tries his best to save his daughter and saves himself".
Of course, Weikang did not forget to seize this good opportunity to promote the Longtu app overseas.
Singhs and other family members of children participating in the clinic downloaded and installed the English version from Fruit and Google app stores.
After trying it out for a while, Singhs' eyes widened in surprise.
"Oh my god, this, this is more amazing and more potential than Menkes' genetic medicine. There is no doubt that this is exactly what I imagine the future of medical care to be."
"Hell, why are companies in China taking the lead every step of the way, leaving companies in Eagle Country far behind?"
"Unfortunately, Sanqing does not accept investment from Wall Street. However, I can definitely copy his business model in Yingguo United Pharmaceuticals. This will definitely be the best investment I have ever made in my life."
****
In China, as the clinical trials of menkes are progressing smoothly, 60 patients have received treatment, and their condition has been greatly relieved immediately. As long as the injections are continued every three months, I believe they will be completely cured soon.
Sanqing United Media filmed the entire process into a documentary, starting from when Cao Yang came to visit, then the development process of genetic drugs, until these patients received treatment.
"Selection by Nature: The Story of Gene Editing"
Once the documentary came out, it became a hit all over the Internet, with a rating of 9.8 on major websites, making it comparable to "A Bite of China".
The Longtu app also made a strong appearance in the film, and became popular all over the Internet along with the documentary.
Everyone in the audience noticed the importance of this software for rare disease patients, and out of curiosity, they also downloaded it.
Although they do not need functions such as clinical trial applications and shared laboratories, electronic medical records are still very practical for most people.
Users can record their own health data, take a photo of the examination report and so on after seeing the disease and upload it to form a complete data table, and also make comments and suggestions on the health status.
There is also detailed medical information in the forum, which is also an excellent feature for people who want to seek medical advice.
At least for them, this is a sufficiently authoritative medical forum. They can ask questions about common diseases to the system and get satisfactory answers quickly.
There is no need to worry about the scary results that a random search on Qiandu will reveal the onset of cancer.
This wave of advertising was very successful, and Longtu’s app downloads began to soar instantly.
Sanqing also officially announced the existence of the Longtu app and spared no effort to promote it to patients with rare diseases across the entire network.
Wei Kang has great ambitions, and he has enough strength to support this ambition.
That is, to unite all rare disease patients across the country and completely become a member of medical big data.
