"Don't worry, Mr. Wei." Seeing Wei Kang's stunned look, Cao Yang quickly explained: "I'm not trying to morally kidnap you. I'm asking Sanqing to develop menkes genetic medicine specifically for us."
"I mainly want to meet relevant experts. I hope the experts can help answer some questions. I also want to ask the R&D personnel to provide some help for subsequent safety tests."
"I already have some research and ideas about gene therapy for menkes. But because I am not a professional, I am still lacking in many aspects and urgently need the guidance and advice of real experts."
"Oh?" Wei Kang said curiously: "Then you can tell me your thoughts first?"
Cao Yang nodded and concentrated on each one.
"While preparing the compounds, I have been carrying out a gene therapy program mediated by 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 the AAV packaging company and have reached an agreement on cooperation. However, I do not have a thorough understanding of the immunity issues and multiple delivery issues of the AAV9 viral vector, 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 slow renewal cycles. The final gene editing in vivo is the most thorough treatment."
"After gene replacement therapy, I want to devote myself to the research of the latest gene editing tool CRISPR-Cas9, because my child's mutation is a repeated mutation, with an extra A base 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 remove the extra A bases. I am considering using children's skin fibroblasts to be cultured in vitro as model cells, designing RNA based on the mutation point, and verifying the editing efficiency in vitro by constructing plasmids."
"I also hope that relevant experts can help me in this regard."
"If a gene therapy program like mine is feasible," Cao Yang took a breath and continued, "I have some patients who 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. After selling the car, plus some savings, I still have about 500,000, but I don’t know how far I can go with these funds.”
"If it really doesn't work, I can go back and sell the house. I just hope that my son can be completely cured."
"Of course, not all of my patients may be able to afford such a treatment plan, but there are also some patients who are willing to sell their medical supplies to save their children, and hope that the General Health Administration can give them a chance."
Seeing Cao Yang's sincere face and extremely firm tone, Wei Kang couldn't help but sigh.
Both emotionally and rationally, he wanted 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 person with only a high school degree?
How come these plans are so clear-cut and more detailed than biological experts?
It can be seen that the other party really put a lot of effort into studying and studying to be able to have such an in-depth understanding.
However, professional matters should still be left to the experts.
There has been no symptomatic treatment for menkes disease, and Wei Kang is not sure whether the plan 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 and showed admiration in his eyes. He looked at Cao Yang and said, "I can see that you have made a lot of efforts to have such a clear solution. Let's do this. I'll call the relevant experts over and you can discuss it together. I want to How about hearing his opinion first?"
Cao Yang was immediately overjoyed: "Thank you so much, Mr. Wei, this is exactly what I want."
Wei Kang took out his cell phone, walked aside, and called Tang Que to tell him everything about this.
"Oh my god, are there family members of patients with such strong self-learning ability? They are simply my confidants. Don't move! I'll be right over!"
Tang Que's strange scream instantly came from the phone.
Five minutes later, a white whirlwind rushed into the conference room.
"Where are the family members? You, you are the family member of the patient named Cao Yang. Nice to meet you. Come, tell me about your child's specific situation and your gene therapy ideas."
As soon as Tang Que came in, he saw Cao Yang and sat next to him with a happy face, as enthusiastic as a dog seeing a sausage.
Wei Kang quickly reached out and introduced: "This is Tang Que, the director of our biological laboratory. He is fully responsible for the development of the SMA gene therapy drug."
Cao Yang was immediately 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, there is no need to be so polite. Let's talk about business first."
Cao Yang nodded. He took out a thick stack of medical cases and some prepared materials from the bag at his feet, and handed them over.
Tang Que slowly looked through it while he explained in detail.
The two lowered their heads and started discussing while watching.
Wei Kang listened on the side and saw the discussion between the two becoming more and more heated, giving him the boring feeling of being an outsider.
Fortunately, one of the two people was very professional and the other was quite knowledgeable. There was no inconsistency between them, but they both had a tacit understanding.
After some discussion, the two finally reached a consensus.
Gusi
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 only read some information before, but never saw a real case, especially the treatment process. In fact, I also benefited a lot."
"In general, your idea is right. The success rate of research and development in this direction is quite high. Because rare diseases are caused by single gene mutations, from the perspective of research and development, the biological signaling pathways are clear, making it easier to succeed. "
Cao Yang was immediately overjoyed: "That's great. You are an expert. Now that you say this, I feel more confident."
He turned to look at Wei Kang: "I wonder what President Wei said? Are you willing to develop related genetic drugs?"
"I can understand if Sanqing is unwilling. I just hope that I can keep in touch with Director Tang and ask for advice from time to time if I have any questions in the future."
"I will continue to build the aav9 virus vector myself, look for companies willing to cooperate to sign contracts, conduct animal experiments, and then find doctors willing to assist to promote clinical treatment research. In short, everything will follow the legal genetic drug application process, and strive to promote this The drug is now on the market.”
He obviously came prepared and had a plan in mind, and his thoughts were very clear, and he laid out the follow-up work plan one by one.
Wei Kang and Tang Que couldn't help but admire his calmness and clear organization, and looked at him with admiration.
Tang Que kept winking at Wei Kang.
Wei Kang was too lazy to pay attention to him and looked directly at Cao Yang in front of him.
He had already made a decision in his mind.
"Cao Yang, I can tell you that Sanqing is willing to provide you with the help it can and is also willing to develop gene therapy drugs for menkes disease."
"In fact, we are already moving in this direction. SMA gene drugs are 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 and stood up suddenly: "Are you saying this in front of me? Ah, Mr. Wei, no, I am not doubting you, I am too happy. I have been working on this matter for a long time, although some companies are willing to Help is provided, but our capabilities are limited and there has been little 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 the same group. This is all the menkes patients in the country. We are all very united. These patients have helped me a lot, and everyone is working hard to promote this matter."
"There are 61 people. There are indeed relatively few people. No wonder many companies are unwilling to do related drug research and development." Wei Kang sighed inwardly, feeling a little more sympathetic to the situation of this group of people.
He glanced at Tang Que who was beside him and was eager to try, and said with a smile.
"I estimate that Director Tang will start establishing research and development projects when he returns home. You can help contact these patients and provide relevant cases. We also need to do genetic testing for them to determine each person's situation so that we can conduct in-depth research on this disease. Research."
"What kind of treatment route will be adopted in the future depends on Director Tang. I can't intervene."
"There are only a small number of you. When there are research results, everyone should be able to participate in clinical trials, so even patients with family difficulties don't have to worry."
Cao Yang burst into tears of gratitude: "Thank you, Mr. Wei, you really have a great heart and think about everyone so much."
"I will go back right away and tell them the good news, and then contact someone to come over and assist with follow-up research."
Looking at Cao Yang's leaving figure, Wei Kang couldn't help but sigh with emotion. He sighed to Tang Que beside him: "What a pity the hearts of parents in the world."
Tang Duo nodded: "So what we can do is a little bit."
Wei Kang looked at the big black man in front of him with great interest: "Tsk, tsk, you are such a powerful guy, but your heart is so soft. Isn't it a kind of cute contrast?"
Tang Que was stunned: "Huh? Don't I look cute?"
"Ahem." Wei Kang almost choked on his saliva and said speechlessly, "Okay, this is a matter of opinion."
"I'm just a little surprised. I just discovered today that your favorite thing is not cockroaches, but humans."
Tang Que looked at him in surprise: "The one I love is not a human being, but a child."
"Most patients with hereditary diseases start to suffer from the disease as babies. These children are the most pitiful. How can I bear to watch such a cute child suffer from the disease since childhood."
Wei Kang was stunned and asked in disbelief: "So you study rare diseases to save children?"
Tang Que said matter-of-factly: "Of course, newly born children are the purest and cutest, and have not been polluted by this evil world. Looking at those innocent eyes, thinking that they may not survive, my My heart is broken.”
"What about the adults?" Wei Kang felt that something was wrong.
Tang Que showed a slightly disgusted expression: "Oh, boss, adults are not cute."
"Although I don't want to admit it, I do prefer animals, plants, microorganisms, and human babies... This is why I chose molecular biology. I just need to study various cells."
Wei Kang: "... As long as you are happy."
He couldn't help but hold his forehead and was speechless. It turned out that in this guy's heart, human cubs had the same status as cockroaches, potted plants and bacteria.
