I can extract side effects

"Don't worry, Mr. Wei." Seeing Wei Kang's stunned face, Cao Yang quickly explained, "I didn't want to kidnap morally, and asked Sanqing to develop the Menkes gene medicine for us."

"I mainly want to meet relevant experts, hoping that experts can help answer some questions, and I also want to ask R\u0026D personnel to provide some help for subsequent security tests."

"I have already had some research and ideas about Menkes' gene therapy. It's just that I am not a professional, so I am still lacking in many aspects, and I urgently need the guidance and advice of real experts."

"Oh?" Wei Kang asked curiously, "Then tell me about your thinking first?"

Cao Yang nodded and focused on one by one.

"Since the preparation of the compound, I have been carrying out the gene therapy program mediated by the adeno-associated virus AAV9. After more than a year of hard work, I finally truncated the sequence of the ATP7A gene by analyzing the protein domain and constructed a plasmid. Currently Expression verification and biological activity verification are also performed on 293T cells."

"I have found an AAV packaging company, and I have negotiated a cooperation intention. However, I still don't have a thorough understanding of the immune issues and multiple delivery issues of AAV9 viral vectors, and I need the guidance of vector experts."

"Because the child's mutated gene is expressed throughout the body, AAV9-mediated gene replacement can only solve the genetic problems of some organs with a slow renewal cycle, and the ultimate gene editing in vivo is the most thorough treatment."

"I want to devote myself to the research of CRISPR-CAS9, the latest gene editing tool, after gene replacement therapy, because my child's mutation is a repeat mutation, and an extra A base was inserted at position 1141 of the ATP7A gene sequence, resulting in The stop codon is advanced and protein translation is blocked."

"So I want to use the CRISPR-CAS9 tool to excise the extra A base. I consider using the child's skin fibroblasts as model cells after in vitro culture, design RNA according to the mutation site, and verify the editing efficiency in vitro by constructing plasmids."

"I also hope that there are relevant experts who can help me in this regard."

"If the gene therapy plan like mine is feasible," Cao Yang continued with a sigh of relief, "I still have some patients, all of whom are family members of Menkes patients, and they also want to get the same gene therapy."

He paused for a moment, and said resolutely: "Mr. Wei, I know that research on gene therapy is very expensive, and I have already prepared for it. I invested hundreds of thousands in my self-built laboratory before. I sold my car last month. Yes, plus some savings, there is another half a million yuan, I don’t know how far these funds can go.”

"If it doesn't work, I can go back and sell the house, just to cure my son completely."

"Of course, my patients may not be able to afford such a treatment plan, but there are also some patients who are willing to sell the pot to save their children, and hope that Mr. Wei can give them a chance."

Seeing Cao Yang's earnest face and unusually firm tone, Wei Kang couldn't help but sigh in his heart.

Out of emotion and reason, he wants to help the families of these desperate patients.

At the same time, he was once again shocked by Cao Yang's gene therapy solution.

Is this really a guy with only a high school education?

Why do these plans speak clearly and clearly, and are more detailed than biologists?

It can be seen that the other party has really put in a lot of energy to study and study in order to have such a deep understanding.

However, professional matters should be left to experts.

There has been no symptomatic treatment for Menkes disease, and Wei Kang is not sure whether the solution proposed by the other party is feasible.

You still have to call an expert to come over and discuss it before making a decision.

He smiled, with admiration in his eyes, looked at Cao Yang and said, "It can be seen that you have put in a lot of hard work to come up with such a clear solution, how about this, I will call the relevant experts over, and you can discuss it together, I want How about listening to his opinion first?"

Cao Yang was overjoyed immediately: "Thank you so much, Mr. Wei, this is exactly what I want."

Wei Kang took out his mobile phone, walked to the side, called Tang Que, and told him everything about him.

"Oh my god, is there a family member of a patient with such a strong self-learning ability? He is simply my confidant, don't move! I'll come over right away!"

Tang Que's strange cry came from the phone instantly.

Five minutes later, a whirlwind of white rushed into the meeting room.

"Where's the family? You, you are the family of the patient named Cao Yang, right? It's a pleasure to meet you. Come, tell me about your child's specific situation and your idea of ​​gene therapy."

Tang Que saw Cao Yang as soon as he came in, and sat next to him with a happy face, as enthusiastic as a dog seeing a sausage.

Wei Kang quickly extended his hand and introduced: "This is Tang Que, the director of our biological laboratory. He is responsible for the overall research and development of SMA gene therapy drugs."

Cao Yang immediately stood up in awe, and extended his hand very politely: "I have admired you for a long time, Director Tang, it is a great honor to meet you..."

He wanted to continue the greetings, but Tang Que was a little impatient: "Mr. Cao, don't be so polite, let's talk about business first."

Cao Yang withdrew his hand, nodded in embarrassment, and then took out a thick stack of medical records from the bag under his feet, as well as some prepared materials, and handed them over.

Tang Que flipped through it slowly, while he explained in detail.

The two lowered their heads, watching and discussing.

Wei Kang was listening. Seeing that the two people's discussion became more and more heated, he felt bored like an outsider.

Fortunately, one of these two people is very professional, and the other is quite understanding. There is no such thing as chicken and duck talking, but they both have a tacit understanding.

After some discussion, the two finally reached a consensus.

Cao Yang said with a happy face: "Thank you so much, Director Tang, for your explanation. It really enlightened me and enlightened me."

Tang Que waved his hand: "Your child's case is also very typical. I have only read some materials before, and have never seen a real case, especially the treatment process. In fact, I have also benefited a lot."

"In general, your thinking is correct. The success rate of research and development in this direction is quite high. Because rare diseases are diseases caused by single gene mutations, from the perspective of research and development, the biological signaling pathway is clear and it is easier to succeed. "

Cao Yang was overjoyed immediately: "Great, you are an expert, so I am more confident."

He turned to look at Wei Kang: "I don't know what Mr. Wei said? Are you willing to develop related genetic medicine?"

"If Sanqing is unwilling, I can understand. I just hope that I can keep in touch with Director Tang, and I can always ask for advice if I have any questions in the future."

"I will continue to build the AAV9 virus vector by myself, find companies willing to cooperate to sign contracts, conduct animal experiments, and then find doctors who are willing to assist to promote clinical treatment research. In short, everything will go through the legal gene drug declaration process and strive to promote this launch of the drug."

He obviously came prepared, with a plan in mind, and his thinking was very clear, and he came up with the follow-up work plan one by one.

Wei Kang and Tang Que couldn't help admiring him for his composure and orderliness, and looked at him with admiration.

Tang Que kept winking at Wei Kang.

Wei Kang didn't bother to talk to him, and looked directly at Cao Yang in front of him.

He has made a decision in his heart.

"Cao Yang, I can tell you that Sanqing is willing to help you as much as it can, and is also willing to develop a gene therapy drug for Menkes disease."

"In fact, we are already heading in this direction. SMA genetic medicine is only the first step. We will continue to conduct genetic research on other rare diseases in the future, striving to mature the technology as soon as possible and help as many patients as possible. "

Cao Yang was overjoyed immediately, and stood up abruptly: "Is this true? Ah, Mr. Wei, no, I'm not doubting you, I'm so happy, I've been working on this matter for a long time, although some companies are willing to help , but we are all limited in strength and have not made much progress.”

He sat down and rubbed his hands excitedly: "Now that I have your promise, I feel relieved."

Wei Kang smiled: "Don't get excited, this is what we should do. Sanqing has always supported the research and treatment of rare diseases."

"By the way, how many patients do you have in total?"

"We have 61 patients, all in one group, which is all the Menkes patients in the country. We are all very united. These patients have helped me a lot, and everyone is working wholeheartedly to promote this matter."

"61 people, the number of people is indeed too small. Unless the price is sky-high, such a drug will not be profitable at all. It is simply a loss of money for charity. No wonder many companies are unwilling to do related drug research and development." Wei Kang thought in his heart. Sighing, he felt more sympathy for the situation of this group of people.

He glanced at Tang Que who was eager to try beside him, and said with a smile.

"I guess Director Tang will start the research and development project when he goes back. You can help contact these patients and provide relevant cases together. We also need to do genetic testing for them to determine each person's condition, so that we can do in-depth research on this disease. Research."

"It's up to Director Tang to decide what happens next. I can't intervene."

"The number of you is very small. When the research results are available, everyone should be able to participate in clinical trials, so that even patients with family difficulties don't have to worry about medical expenses."

Cao Yang immediately burst into tears of gratitude: "Thank you Mr. Wei, you really have a heart of great love, and you are so considerate of everyone, I thank you for them."

"I'll go back and tell them the good news, and then the contact person will come over to assist with the follow-up research."

He stood up, bowed deeply to the two, and went out.

Seeing Cao Yang's leaving figure, Wei Kang couldn't help being filled with emotion, he sighed to Tang Que beside him, "Poor parents in the world."

Tang Kuang nodded: "So we can help a little."

Wei Kang looked at the big man in front of him with great interest: "Tsk tsk, you are such a mighty big man, but your heart is so soft, isn't it a kind of cute contrast?"

Tang Que was taken aback: "Ah? Isn't my appearance cute?"

"Ahem." Wei Kang almost choked on his saliva, and said speechlessly, "Okay, different people have different opinions."

"I'm just a little surprised. I just found out today that what you love the most is not cockroaches, but human beings."

Tang Que looked at him in surprise: "It's not human beings I love, but children."

"Most patients with genetic diseases start to develop the disease from infancy. These children are the most pitiful. I can't bear to see such a lovely child suffer from childhood and die early in the torment of the disease."

Wei Kang was stunned, and asked incredulously: "So you study rare diseases because you like children? It's purely for your own preference? Not for the dream and moral sense of saving all mankind?"

Tang Que took it for granted and said, "Of course, scientists have their own preferences when doing research. Besides, newborn children are the most pure and lovely, and have not been polluted by this evil world. Looking at those innocent eyes , It breaks my heart to think that they might not survive."

"What about adults?" Wei Kang felt that something was wrong.

Tang Que showed a disgusted expression: "Eh, boss, adults are not cute."

"Although I don't want to admit it, I do prefer animals, plants, microbes, and human babies... That's why I choose molecular biology, just need to study various cells."

Wei Kang: "...you are happy."

He couldn't help but be speechless. It turns out that in this guy's heart, human cubs are in the same position as cockroaches, potted plants and bacteria.

As for adult humans, their status is not as good as that of cockroaches.