Latest news with #geneediting
Yahoo
7 hours ago
- Business
- Yahoo
Eli Lilly Advances Pipeline and Gene Focus With Verve Acquisition
Eli Lilly and Company (NYSE:LLY) is one of the . Amid carrying on new studies, the company has agreed to acquire Verve Therapeutics. Eli Lilly and Company (NYSE:LLY) is a research-driven pharmaceutical leader headquartered in Indiana. With a 150-year history, the company carries on the business of discovering, developing, manufacturing, and marketing innovative treatments across diseases like diabetes and in fields like oncology, immunology, and neuroscience. Some of the company's notable products, such as Humalog, Trulicity, Mounjaro, and Zepbound, are marketed in over 125 countries. The company is carrying on multiple studies and its latest updates include the Phase 2b clinical study titled 'A Study of Eltrekibart (LY3041658) in Adult Participants With Moderate to Severe Hidradenitis Suppurativa' and Phase 3 clinical study titled 'A Multicentre, Randomized, Double-blind, Placebo-controlled, Parallel Group Phase 3 Efficacy and Safety Study of Lebrikizumab/ LY3650150 in Adults With Chronic Rhinosinusitis With Nasal Polyps on a Background Therapy With Intranasal Corticosteroids.' Along with these studies, the company has a pipeline with potential drugs capable of generating more than $1 billion in annual revenue. Amid these studies, the company has agreed to buy Verve Therapeutics Inc. for up to $1.3 billion. With this acquisition, Eli Lilly and Company (NYSE:LLY) aims to strengthen its positions in gene editing for cardiovascular conditions. With a beta of 0.44 signaling the company's resistance to market conditions, Eli Lilly and Company (NYSE:LLY) has a high anticipated 5-year EPS growth of 41.87%, making the stock a strong contender in our list. While we acknowledge the potential of LLY as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 10 Metal Stocks with Insider Buying in 2025 and 10 Energy Stocks with Insider Buying in 2025 Disclosure. None.
Yahoo
11 hours ago
- Business
- Yahoo
Precision BioSciences Receives FDA Orphan Drug Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
DURHAM, N.C., July 23, 2025--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD). "Receipt of Orphan Drug Designation from the FDA for PBGENE-DMD underscores the tremendous unmet need and urgency to deliver safe treatments that significantly improve muscle function over time for boys living with Duchenne muscular dystrophy," said Cindy Atwell, Chief Development and Business Officer at Precision BioSciences. "This regulatory milestone builds on our recent receipt of Rare Pediatric Disease designation and, together with our preclinical body of evidence, gives us tremendous confidence as we move this program towards the clinic. Looking ahead, we remain in active dialogue with the FDA as we advance PBGENE-DMD toward regulatory milestones, with clinical data anticipated in 2026." The FDA grants Orphan Drug Designation to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides sponsors certain benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity for the drug for the designated orphan indication in the U.S. if the drug is ultimately approved for that use. About PBGENE-DMD PBGENE-DMD is Precision's development program for the treatment of DMD. The approach uses two complementary ARCUS nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene with the aim of restoring near full-length dystrophin protein within the body to improve functional outcomes. PBGENE-DMD is intended to address up to 60% of the DMD patient population. In preclinical studies, PBGENE-DMD demonstrated the ability to target key muscle types involved in the progression of DMD and produced significant, durable functional improvements in a humanized DMD mouse model. PBGENE-DMD restored the body's ability to produce a near full-length functional dystrophin protein across multiple muscles, including cardiac tissue and various key skeletal muscle groups. In addition, PBGENE-DMD edited satellite muscle stem cells, believed to be critical for long-term durability and sustained functional improvement. PBGENE-DMD was recently granted FDA Rare Pediatric Disease (RPD) designation for the treatment of DMD. The Company is advancing the final IND-enabling toxicology studies and is working closely with leading DMD clinicians to design a first in human trial optimized for safety and efficacy. Clinical grade material is in preparation, with initial clinical data expected in 2026. About Precision BioSciences, Inc. Precision's two lead programs, PBGENE-HBV, for chronic Hepatitis B, and PBGENE-DMD, for Duchenne muscular dystrophy, are focused on areas with large patient populations with high unmet need. Precision BioSciences, Inc. is a clinical stage gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company's pipeline prioritizes in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the clinical development and expected safety, efficacy and benefit of our and our partners' and licensees' product candidates and gene editing approaches; the potential of PBGENE-DMD to safely drive meaningful improvement in functional and durable benefit over time for up to 60% of patients with DMD; the design on PBGENE-DMD to permanently edit a patient's own DNA sequence, resulting in naturally-produced, near full-length dystrophin protein proven known to be functional in humans; the benefits of an Orphan Drug Designation including financial incentives and regulatory exclusivity; the potential value of a Priority Review Voucher(if awarded) including priority review for a different product or sale to another sponsor; the expected timing of regulatory processes and clinical operations (including IND and/or CTA filings, studies, enrollment and clinical data for PBGENE-DMD; and anticipated timing of clinical data. In some cases, you can identify forward-looking statements by terms such as "aim," "anticipate," "appear," "approach," "believe," , "confidence", "contemplate," "could," "design" "designed," "estimate," "expect," "goal," "intend," "look," "may," "mission," "plan," "possible," "potential," "predict," "project," "pursue," "should," "strive," "suggest," "target," "will," "would," or the negative thereof and similar words and expressions. Forward-looking statements are based on management's current expectations, beliefs, and assumptions and on information currently available to us. These statements are neither promises nor guarantees, and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators' or other licensees' ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators or other licensees related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators' or other licensees' development of product candidates; our or our collaborators' or other licensees' ability to advance product candidates into, and successfully design, implement and complete, clinical trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; delays or difficulties in our and our collaborators' and other licensees' ability to enroll patients; changes in interim "top-line" and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; our or our licensees' ability to obtain orphan drug designation or fast track designation for our product candidates or to realize the expected benefits of these designations; our or our collaborators' or other licensees' ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; the rate and degree of market acceptance of any of our product candidates; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate executives and personnel; effects of system failures and security breaches; insurance expenses and exposure to uninsured liabilities; effects of tax rules; effects of any pandemic, epidemic, or outbreak of an infectious disease; the success of our existing collaboration and other license agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of sustained inflation, supply chain disruptions and major central bank policy actions; market and economic conditions; risks related to ownership of our common stock, including fluctuations in our stock price; our ability to meet the requirements of and maintain listing of our common stock on Nasdaq or other public stock exchanges; and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2025, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC's website at and the Investors page of our website under SEC Filings at All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. View source version on Contacts Investor and Media Contact: Naresh TannaVice President of Investor
Globe and Mail
3 days ago
- Business
- Globe and Mail
3 Monster Stocks in the Making to Buy Right Now
Key Points CRISPR Therapeutics is still in its early growth stage. Summit Therapeutics has plenty of upside potential remaining. Viking Therapeutics is targeting two monster markets. 10 stocks we like better than CRISPR Therapeutics › Where can you find the next monster stocks? Check out the biotech space. It's not easy, but investors can sometimes find highly promising biotech stocks that are only in their early innings. Three Motley Fool contributors think they've identified monster stocks in the making. Here's why they picked CRISPR Therapeutics (NASDAQ: CRSP), Summit Therapeutics (NASDAQ: SMMT), and Viking Therapeutics (NASDAQ: VKTX). Image source: Getty Images. A promising company in its early growth stage David Jagielski (CRISPR Therapeutics): Biotech company CRISPR Therapeutics has a market cap of around $5 billion, but it has the potential to be much more valuable in the long run. The gene editing market is still fairly small, but it has a lot of room to grow. Analysts from Grand View Research project that it will expand at a compound annual growth rate of more than 16% between now and 2030, when it will be worth $25 billion. As a leading company in the space, CRISPR Therapeutics is well positioned to benefit from future growth in this area of healthcare. The company, and its key development partner, Vertex Pharmaceuticals (NASDAQ: VRTX), already have an approved gene therapy treatment in Casgevy, which regulators have approved for both sickle cell disease and transfusion-dependent beta-thalassemia. The companies will share in the profits on the treatment (CRISPR will take 40%). Casgevy is still in its early growth stages. CRISPR is also working on developing treatments for type 1 diabetes and cardiovascular disease. While CRISPR remains unprofitable today, its future does look bright. It's a long road ahead for CRISPR Therapeutics, but with an encouraging pipeline and a lot of growth potential, this could be a monster healthcare stock in the making. The business is well funded with its cash and marketable securities as of the end of March totaling nearly $1.9 billion, putting it in an excellent position to continue investing in its research and development efforts. CRISPR can make for an excellent investment, but it's also one you'll need to be patient with. Still plenty of upside left for this stock Prosper Junior Bakiny (Summit Therapeutics): Few biotech companies have performed better than Summit Therapeutics over the past three years. The company's shares have soared by more than 2,000%, making early investors who held on significantly wealthier in the process. Here's the good news for those who are still considering the stock: There could be a massive upside left for Summit Therapeutics. Though the biotech generates no revenue and is unprofitable, it has one of the more promising pipeline products in the industry. Summit Therapeutics' leading candidate is a cancer medicine called ivonescimab, which it licensed out from Akeso Biopharma (OTC: AKES.F), a China-based drug developer. The medicine has been extensively tested in China, where it has already received approval in certain indications. One of ivonescimab's most promising markets is non-small cell lung cancer (NSCLC). In a phase 3 study in China, it went head-to-head against the market leader and the world's best-selling drug: Merck 's (NYSE: MRK) Keytruda. Ivonescimab emerged victorious, leading to a greater decrease in the risk of recurrence or death among NSCLC patients with a PD-L1 protein overexpression. Summit Therapeutics is conducting clinical studies to support approval in the U.S. and other regions. Furthermore, the biotech is likely to seek a series of approvals and label expansions over the years, based on the number of clinical trials and indications Akeso is targeting in China. Ivonescimab should easily become a blockbuster and generate strong sales for years. That's why it's not too late to invest in Summit Therapeutics. If ivonescimab achieves its full potential, Summit could deliver market-beating returns in the next five to 10 years and establish itself as a prominent player in the biotech industry. Targeting two monster markets Keith Speights (Viking Therapeutics): The big story for Viking Therapeutics is its experimental obesity drug VK2735. The company recently advanced a subcutaneous formulation of this candidate into late-stage clinical testing after announcing highly encouraging results from a phase 2 study last year. Viking is also evaluating an oral version of VK2735 in a separate phase 2 clinical trial, and it expects to report results from that study later in 2025. In addition, the drugmaker plans to initiate a clinical study for a monthly maintenance version of subcutaneous VK2735 in the coming months. Morgan Stanley Research projects that the global obesity drug market could reach $150 billion by 2035. Viking could be in a great position to claim a nice chunk of that market if VK2735 fulfills its potential. Obesity isn't the only big market that the company is targeting, though. In 2023, Viking reported positive results from a phase 2 study of VK2809 in treating metabolic-associated steatohepatitis (MASH), a chronic liver disease also known as nonalcoholic steatohepatitis (NASH). The company is seeking a partner to advance VK2809 into late-stage testing, so it can dedicate its financial resources exclusively to VK2735 for now. Grand View Research predicts the MASH/NASH market could hit $33.8 billion by 2030, so that's a nice opportunity. A clinical trial flop for VK2735 and/or a failure to find a partner for VK2809 could prevent Viking from becoming a monster stock. However, I think the company's chances look pretty good. I also wouldn't be surprised if a larger drugmaker swoops in to acquire Viking. Should you invest $1,000 in CRISPR Therapeutics right now? Before you buy stock in CRISPR Therapeutics, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the 10 best stocks for investors to buy now… and CRISPR Therapeutics wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $652,133!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $1,056,790!* Now, it's worth noting Stock Advisor's total average return is 1,048% — a market-crushing outperformance compared to 180% for the S&P 500. Don't miss out on the latest top 10 list, available when you join Stock Advisor. See the 10 stocks » *Stock Advisor returns as of July 15, 2025
Daily Mail
3 days ago
- Science
- Daily Mail
EXCLUSIVE I went to prison for genetically editing human babies - here's why I'd do it again in a heartbeat
When He Jiankui stood before a court in 2019, he did so accused of a crime no one in history had ever committed. The year prior, Mr He had triumphantly announced the birth of the world's first gene-edited babies - a pair of twins named Lulu and Nana. For that crime, Mr He got three years in jail, a fine of three million Chinese Yuan (£310,000), and instant worldwide infamy. However, out of prison and back in the lab, Mr He has told MailOnline he would do it all again if he had the chance. Speaking from his home in Beijing, where he has remained since Chinese authorities confiscated his passport, he explained his plans to start a new research lab in the US. Totally unapologetic and unrepentant, his ambition seems to have only grown following his years of incarceration. Mr He said: 'When I think about the long term, I would like embryo gene editing to be as popular as the iPhone. 'It's going to be affordable to most families. So we will see the majority of babies born having gene editing, because doing so will make them healthy.' Throughout 2018, Mr He and his colleagues took sperm and eggs from eight couples who had tested positive for HIV and used these to create embryos with IVF. In each of these embryos, he edited a gene to make the children resistant to HIV. He then took those gene-edited embryos and inserted them into women's wombs, where they matured into healthy, living foetuses. In two cases, this resulted in a pregnancy - one leading to the twins Lulu and Nana, who were born in November 2018, and another leading to a third child born later. In doing so, Mr He not only violated China's criminal law prohibiting human genetic engineering, but went well beyond the limits of ethical research. While Mr He had, and still has, his supporters, he was also criticised by researchers around the world who deemed the work premature, irresponsible, and unjustified. Scientists from around the world pointed out that Mr He had made no attempts to ensure that these methods were safe or effective. With our current level of understanding of these techniques, we simply do not know whether what Mr He did will cause lifelong health issues. Who is He Jiankui? He Jiankui is a disgraced Chinese scientist who was jailed for three years in 2019 after announcing he had genetically engineered human babies. Although this was initially met by some as a major scientific breakthrough, criticism followed as the details of his experiments emerged. Mr He had forged ethical review documents, falsified blood tests, hidden from government scrutiny by personally funding the research, and misled doctors into unknowingly implanting gene-edited embryos. Following his release from jail, his application to work in Hong Kong was rejected after it emerged that he lied on his application form. Although Mr He told MailOnline he is in regular contact with the parents of all three children, there is no way to confirm that they really are as healthy as he claims. Likewise, experts in HIV argued that there was 'zero risk' of the children being infected with HIV in the first place, provided the mother took widely available and provenly safe antiretroviral therapy. Dr Kathy Niakan, embryo research group leader at the Francis Crick, said at the time: 'This would be a highly irresponsible, unethical and dangerous use of genome editing technology. 'Given the significant doubts about safety, including the potential for unintended harmful side-effects, it is simply far too premature to attempt this.' Even among researchers who support research into embryo editing, Mr He was slammed for potentially undermining years of work spent convincing the public that these methods could be done ethically. Professor Darren Griffin, a geneticist from the University of Kent, said at the time that Mr He had taken trust in science 'back to the Stone Age'. As the trial took place, it also emerged that Mr He had taken a number of highly unethical and often illegal steps to hide his research from prying eyes. Mr He and his colleagues were accused of forging ethical review documents, misleading doctors into unknowingly implanting gene-edited embryos. How did He Jiankui genetically edit babies? Throughout 2017 and 2018, Mr He recruited eight couples in which the male was HIV positive. He took sperm and eggs from these couples and created fertilised embryos. Using CRISPR gene editing technology, Mr He altered a gene that would give the baby protection against the HIV virus. He then took these embryos and implanted them into their mothers' wombs. Two women then became pregnant and gave birth to three babies, including a pair of twins. Since HIV positive parents are not allowed to receive assisted reproduction, he asked others to take the volunteers' blood tests on their behalf to avoid regulations. It was also revealed that he had used private funds from his two biotech companies to finance the research personally to avoid scrutiny. Despite all that, Mr He, who requested he be introduced as a 'pioneer of gene editing', appeared confused by the criticism. 'I have three babies born now, and the two families thanked me. They thanked me for what I did for them, for their family. They have a healthy baby and a happy life now,' said Mr He. 'The three babies are normal, healthy, free of HIV. They are living a happy life. That's the proof that what I've done is ethical. 'People don't want to get involved with me because I have a bad name. But actually, nothing happened. There's no bad result, I'm not breaking the law. 'I do everything legally, I do everything ethically. So nothing bad has happened yet.' Although Mr He said he is 'actively' petitioning the Chinese government to legalise embryo gene editing, he doesn't want this to be totally unregulated. In 2018, He Jiankui announced that he had used CRISPR-Cas9 to edit the genes of human babies in an attempt to make them HIV resistant. This was a violation of China's prohibition on human genome editing In fact, he is staunchly opposed to any editing which tries to enhance intelligence, appearance, or create 'super-soldiers' - adding that these should be 'permanently banned'. In Mr He's view, genetically editing embryos is simply the obvious solution to the health problems affecting the world. '10 years ago, my dream was to be a physicist - to be Einstein the second,' said Mr He. 'But then, when I went to the US, my grandfather died of some disease. It would not be a big problem today, but 15 or 20 years ago in China, the medical conditions were not so good. 'I gradually realised that all the problems of physics have mostly been solved, but there are lots of things we could do in human biology and human health.' Mr He describes this as a 'transition moment' in his life, after which he became unflinchingly dedicated to his goal of bringing embryo gene editing to the masses. Gene therapy works by fixing or replacing faulty genes to prevent the body from getting sick or make it able to fight off a disease. This treatment has a lot of promise for tackling conditions such as cancer, Alzheimer's, cystic fibrosis, heart disease, diabetes, haemophilia and AIDS. Why is editing human embryos controversial? He Jiankui's gene editing experiments were roundly criticised by the scientific community, even by those who support embryo research. There is not enough evidence to show that these methods are safe or effective, nor are the potential side effects understood. Mr He made little effort to ensure that the procedure would be safe, and his published results were not peer-reviewed. The procedure was also entirely unnecessary to prevent HIV infection, since a regular course of antiretroviral medication would have the same effect. The drawback is that a single course of gene therapy can be prohibitively expensive, with treatments sometimes costing upwards of £3 million per dose. However, making those same genetic changes on a single-cell embryo before someone is born requires only tiny amounts of medicine. In the future, Mr He said he wants these kinds of preventative treatments to become the norm for the vast majority of children. 'I see this happening very soon, maybe in 10 years,' said Mr He. 'The reason is that it's affordable. The current gene therapy is super expensive. No one can afford it. It's a ridiculous price, and it's not benefiting people at all. 'But embryo gene editing, it only costs a few thousand dollars, so most families can afford it. 'Embryo gene editing is the future. It will get cheap, maybe not as cheap as an iPhone, but maybe as cheap as 10 iPhones.' In service of that goal, Mr He said he is opening a new research lab in Houston, Texas, where he will pursue embryo gene therapies. Mr He said he already has two American researchers lined up to start the lab in early August this year. He insists that there will be 'no pregnancies' and no gene-edited babies born in Austin. However, the same secrecy that plagued his earlier research once again threatens to cast a shadow over this latest project. Mr He refused to disclose any details about the lab, including the names of the researchers or the two Chinese 'colleagues' from his earlier projects that are overseeing the lab on his behalf. Mr He would not even name the disease that the lab is supposed to be investigating, other than to say it was a rare muscular disease. This time around, his sources of funding are also even more obscure. Mr He said he has received private donations from an American family suffering from the unnamed disease, as well as from some 'Southeast Asian families'. He also released a 'meme coin' cryptocurrency called GENE, which supposedly provides direct funding for the research. He claims that these measures are necessary to preserve his employees' safety, fearing attacks on the laboratory if the location were revealed. This comes after a supposed attack by a group of rivals last September, which supposedly led to Mr He needing hospital treatment. However, physical assaults are only the beginning of Mr He's troubles. In addition to the three million Chinese Yuan he was fined, he now says that the local government of Shenzhen is demanding the repayment of a further five million Chinese Yuan (£500,000) lent to Mr He to start a research lab. Mainstream funding bodies have also shunned his research, leading to his application for funding being rejected by the Muscular Dystrophy Association in 2024. Mr He said that researchers have declined to work with him, university professors won't answer his emails, and potential collaborators have refused to be associated with him. Although Mr He said these challenges make him 'angry', he remains characteristically undaunted. 'I think it's totally fair. It's fair, because every pioneer, every prophet, has to serve those difficulties and until one day they have been fully recognised by society,' said Mr He. 'People only believe what they want to believe. So, only when those old people die, the young people will accept it. It's natural.' As for why he is so willing to endure these hardships, Mr He appears to be caught between two conflicting motivations. At times, he seems genuinely concerned for those he sees as needlessly suffering from curable conditions, and outraged that gene therapy is only affordable for the super-rich. But at others, his underlying drive seems to be nothing more than a simple desire for fame. Mr He is obsessed with social media, in particular X, where he has amassed 135,000 followers. He claimed to be the 'most influential biologist on X' and boasted that he had 'more followers than Nobel Prize winners' while bitterly complaining that he had been banned from social media in China. He candidly told MailOnline: 'One side, I am always looking for fame. I want my name to be put into history. 'Second, I want to bring glory to my country, China. I want to make it so the Chinese people feel proud of me.' Although he claims that he only wants fame 'on the condition that it's better for the patient', his focus always seems to be on proving to the world that he was right. Asked whether going to jail, facing millions in fines, and becoming an outcast from the world of science was worth it, Mr He replied without hesitation: 'Of course. That's worth a Nobel Prize.' And despite his criminal record and lax attitude towards medical ethics, Mr He doesn't see this as an unreasonable goal at all. Comparing himself to the inventor of IVF, Sir Robert Geoffrey Edwards, Mr He said: 'When Edwards was given the Nobel Prize in 2010, there were already five million IVF babies born all over the world. 'So when we have five million babies born with gene editing, they will give me the Nobel Prize.' 'One day, when China has changed its law, I will have a Nobel Prize.' WHAT IS CRISPR-CAS9? Crispr-Cas9 is a tool for making precise edits in DNA, discovered in bacteria. The acronym stands for 'Clustered Regularly Inter-Spaced Palindromic Repeats'. The technique involves a DNA cutting enzyme and a small tag which tells the enzyme where to cut. The CRISPR/Cas9 technique uses tags which identify the location of the mutation, and an enzyme, which acts as tiny scissors, to cut DNA in a precise place, allowing small portions of a gene to be removed By editing this tag, scientists are able to target the enzyme to specific regions of DNA and make precise cuts, wherever they like. It has been used to 'silence' genes - effectively switching them off. When cellular machinery repairs the DNA break, it removes a small snip of DNA. In this way, researchers can precisely turn off specific genes in the genome. The approach has been used previously to edit the HBB gene responsible for a condition called β-thalassaemia.
Yahoo
4 days ago
- Business
- Yahoo
BMO Capital Maintains a Buy on Prime Medicine (PRME), Sets a $10 PT
Prime Medicine Inc. (NASDAQ:PRME) is one of the . On July 16, BMO Capital analyst Kostas Biliouris maintained a Buy rating on Prime Medicine Inc. (NASDAQ:PRME) and set a price target of $10.00. The analyst based the rating on the company's innovative gene editing technology. A scientist examining a microchip and circuit board in a hi-tech lab. Prime Medicine Inc. (NASDAQ:PRME) recently secured additional funding totaling $39 million from the Cystic Fibrosis Foundation, which, according to the analyst, highlights the potential of its prime editing platform and preclinical data in cystic fibrosis. Biliouris reasoned that the company's notable 90% editing efficiency attained in the second patient treated ex vivo, when coupled with this financial backlog, reduces the risk of Prime Medicine Inc.'s (NASDAQ:PRME) platform and increases its credibility. Headquartered in Cambridge, MA, Prime Medicine Inc. (NASDAQ:PRME) is a biotechnology company that develops one-time curative genetic therapies. While we acknowledge the potential of PRME as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 30 Stocks That Should Double in 3 Years and 11 Hidden AI Stocks to Buy Right Now. Disclosure: None. This article is originally published at Insider Monkey. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
