Latest news with #CPS1
Business Wire
2 days ago
- Health
- Business Wire
Integrated DNA Technologies Announces Translational CRISPR Portfolio Expansion with Product Innovation Updates
CORALVILLE, Iowa--(BUSINESS WIRE)--Global genomics leader Integrated DNA Technologies (IDT) is revolutionizing the field of translational gene editing with new additions to its end-to-end CRISPR portfolio. These expanded CRISPR translational research solutions are designed to help researchers accelerate more CRISPR-based therapies for patients like KJ Muldoon, an infant who suffered CPS1 deficiency and made history when he received the world's first personalized CRISPR therapy manufactured jointly by IDT and Aldevron. IDT provided the guide RNA (gRNA), off-target analysis services and regulatory support for baby KJ's treatment, which was delivered in a significantly compressed timeline. 'IDT's rich history, deep expertise, and ability to manufacture bespoke products at the highest quality uniquely positions us to consistently innovate on our CRISPR portfolio and support customers at every stage of their gene editing journey' Share Tailored gRNAs to Accelerate CRISPR Discoveries As researchers advance from discovery to clinical applications, the demand for higher purity in CRISPR reagents increases. To meet this need, IDT's chemically synthesized gRNAs are now available for online ordering in a range of modification and purity options. With high purity ideal for translational applications such as gene editing in primary cells and in vivo models, IDT's high-performance liquid chromatography (HPLC)-purified gRNAs are orderable in 2 nmol and 10 nmol yields with larger quantities available by request and ship in as little as 12 business days. Formats include CRISPR-Cas9 gRNA and Custom Alt-R™ CRISPR gRNA. 2' Fluoro, 2' O-Methyl, and other modifications can be added to increase stability and specificity with IDT's custom gRNA tool. Supporting Scientists with Regulatory Filings and Ensuring Safety of Therapeutic Candidates CRISPR-based genome editing allows for targeted editing at specific sites in the genome, but there is potential risk that off-target edits at other locations can occur. To enable scientists to understand where these off-target edits might happen and assess how they might impact safety early in the therapeutic development process, IDT launched UNCOVERseq, off-target nomination services which uses an enhanced GUIDE-seq™ methodology to identify off-target sites for its customers. When paired with IDT's award-winning off-target confirmation services, rhAmpSeq™ CRISPR Analysis System, CRISPR pioneers can confidently accelerate their path to the clinic by obtaining a deeper understanding of editing risks. The custom safety services provided in baby KJ's treatment helped launch UNCOVERseq and represent a major step forward in ensuring the safety of CRISPR-based therapies. Enhancing Safety, Quality and Efficiency: IDT's Growing Pipeline and Future Collaborations Future IDT launches, planned for late 2025, include complementary offerings such as the Alt-R HDR Enhancer Protein, which is designed specifically for therapeutic applications, and manufactured by Aldevron, to meet rigorous quality standards. The HDR Enhancer Protein improves HDR efficiency in difficult-to-edit cells, and maintains safety and cell health. IDT, in collaboration with Aldevron, will also be launching a novel Cas9 mRNA to support early discovery to clinical stage customers. IDT has been a long-time provider of CRISPR gRNA libraries for screening applications and will unveil a new design tool later this year for efficient ordering of custom, configured libraries. 'IDT's rich history, deep expertise, and ability to manufacture bespoke products at the highest quality uniquely positions us to consistently innovate on our CRISPR portfolio and support customers at every stage of their gene editing journey,' said Sandy Ottensmann, VP/GM, Gene Writing & Editing at IDT. 'On the heels of the world's first personalized CRISPR-based therapy, we're honored to bring more CRISPR tools that will enable researchers to make important discoveries, like the one designed for baby KJ, and progress science forward.' Learn more about IDT's CRISPR portfolio here. About IDT Building from a strong foundation of innovation, expertise, and reliability, Integrated DNA Technologies (IDT) has evolved from an oligo manufacturer to a leading genomics provider. We work shoulder-to-shoulder with scientific and global health partners to enable genomics breakthroughs at scale. Our vision of enabling researchers to rapidly move from the lab to life-changing advances reflects our ongoing commitment to a healthier, brighter future for all. IDT is proud to be part of Danaher, a global science and technology leader. Together we combine our capabilities to accelerate the real-life impact of tomorrow's science and technology to improve human health. For more information about IDT, visit and follow the company on LinkedIn, X, Facebook, YouTube, and Instagram. Disclaimer: RUO — For research use only. Not for use in diagnostic procedures. Unless otherwise agreed to in writing, IDT does not intend these products to be used in clinical applications and does not warrant their fitness or suitability for any clinical diagnostic use. Purchaser is solely responsible for all decisions regarding the use of these products and any associated regulatory or legal obligations. Disclaimer: CGMP refers to products manufactured under ICHQ7; IDT engineering runs and CGMP gRNA are for development and investigational use only. The performance characteristics of this product have not been established. This product is not intended to be used as final drug product. The purchaser is solely responsible for all decisions regarding the intended use of the product and any associated legal or regulatory obligations. GUIDE-seq™ is owned by Maxcyte®, Inc.
Yahoo
24-06-2025
- Business
- Yahoo
CRISPR Therapeutics AG (CRSP): A Bull Case Theory
We came across a bullish thesis on CRISPR Therapeutics AG (CRSP) on Two Natural Cap's Substack. In this article, we will summarize the bulls' thesis on CRSP. CRISPR Therapeutics AG (CRSP)'s share was trading at $41.52 as of 11th June. CRSP's trailing and forward P/E were 22.55 and 23.36 respectively according to Yahoo Finance. A scientist peering into a microscope, researching the next gene therapy breakthrough. CRISPR has re-entered the spotlight thanks to a groundbreaking case where the technology was used to treat Baby KJ, born with a severe genetic liver disorder caused by a CPS1 enzyme deficiency. With only a 50% survival rate in infancy, traditional treatment included heavy medication and a strict low-protein diet. But researchers rapidly developed a personalized CRISPR therapy that KJ has now received three times, allowing him to eat a regular diet and significantly reduce medication use. This marks a major milestone not only for KJ but for the broader gene-editing landscape. At its core, CRISPR-Cas9 was originally a bacterial defense mechanism that evolved to recognize and cut viral DNA. Scientists have since repurposed this system, using modified guide RNAs and engineered Cas9 proteins to target human DNA with remarkable precision. In most therapeutic uses, CRISPR creates double-stranded breaks that are imperfectly repaired by the body—helpful for knocking out malfunctioning genes, as seen with CASGEVY, the FDA-approved therapy for sickle cell disease and beta thalassemia. KJ's case went further, employing 'base editing' to fix a single-letter DNA error using a deaminase-modified CRISPR complex that swapped an adenine for a guanine, correcting the mutation without a DNA break. This was delivered via lipid nanoparticles (LNPs), offering dosing flexibility over traditional viral vectors. The success not only validates the science behind base editing but also uplifts companies like Beam, CRISPR Therapeutics, and Intellia after years of market declines. It also spotlights Danaher's Aldevron and Acuitas Therapeutics, whose platforms were instrumental in the therapy's development, underscoring the growing viability of personalized gene medicine. We previously covered a bullish thesis on CRISPR Therapeutics (CRSP) from wallstreetbets by MADD-Scientis, emphasizing Casgevy's revenue potential, strong cash runway, and optionality in oncology and cardiology. Since the coverage, the stock price has appreciated by roughly 1.3%. Two Natural Cap's thesis adds depth by spotlighting CRSP's role in a groundbreaking base-editing therapy, reinforcing the platform's adaptability. Both cases point to CRSP's leadership in curative gene editing. CRISPR Therapeutics AG (CRSP) is not on our list of the 30 Most Popular Stocks Among Hedge Funds. As per our database, 29 hedge fund portfolios held CRSP at the end of the first quarter which was 27 in the previous quarter. While we acknowledge the risk and potential of CRSP as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an extremely cheap AI stock that is also a major beneficiary of Trump tariffs and onshoring, see our free report on the best short-term AI stock. READ NEXT: 8 Best Wide Moat Stocks to Buy Now and 30 Most Important AI Stocks According to BlackRock. Disclosure: None. This article was 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
Yahoo
04-06-2025
- General
- Yahoo
World's first patient treated with CRISPR gene editing therapy at CHOP returns home
The Brief A ten-month-old baby who has been at the Children's Hospital of Philadelphia (CHOP) since birth finally got to go home with his family. KJ Muldoon made headlines around the globe a few weeks ago for being the first person in the world to receive a breakthrough gene editing therapy that is customized to the patient. The family said after KJ received three infusions in February, March and April. Doctors said the results are very promising so far. CLIFTON HEIGHTS, Pa. - An emotional homecoming occurred in Delaware County for a ten-month-old baby named KJ Muldoon who made headlines around the globe just a few weeks ago. The backstory KJ was born on August 1 and diagnosed with a rare metabolic disease called CPS1 that causes ammonia to build up to a toxic level in the body. In February, doctors treated the infant with a breakthrough and historic CRISPR gene editing therapy, making KJ the very first patient in the world to receive this kind of personalized treatment. KJ received additional infusions of the experimental therapy in March and April. Doctors have told his parents the results so far are very promising. What's New "We went through all of the emotions. You're excited, you're nervous, but we're just glad that he's finally able to be home with us," said Nicole Muldoon, KJ's mother. "We've been operating like five plus one for so long and we're excited to be the six of us moving forward." "We're trying to meet all his developmental milestones and kind of see what he's capable of, but he's already shown us how special he is and I think we're in for a treat." KJ's family and friends hung up welcome home signs and colorful balloons as they waited in anticipation for his homecoming in Clifton Heights. "It's just been a really long fight for him," said Dee Aaron, KJ's grandmother. "Miracles do happen, and it really is a miracle. We didn't think he'd be here." "We're so happy you're home big guy," said Cathy Franklin, KJ's great-grandmother. "He's beautiful, and his parents have been remarkable just remarkable. Stayed so strong and we just prayed for ten months. Here he is!" The staff at CHOP dressed KJ up in a graduation cap and gown for his send-off, and they sent him out the hospital doors in great numbers, cheering on his health and recovery. Once the family made it out of the hospital, police from Upper Darby and Radnor Township escorted the family from CHOP all the way to Clifton Heights. The nonprofit The Delco Group helped arrange the special police escort. "We made one phone call yesterday with Ken Piree from Radnor right to Upper Darby Township. It was just within seconds and that's what you get in Delaware County. Everybody gets each other's back here," said John Port of The Delco Group. What you can do The community has also raised tens of thousands of dollars in a gofundme campaign which will now help support KJ's medical needs moving forward.
Indian Express
27-05-2025
- Health
- Indian Express
Explained: A first— how a customised gene-editing tool was used to treat 9-month-old boy
A nine-month-old boy, born with a rare genetic disorder, has become the first (known) person to successfully receive a custom gene-editing treatment, a report published on May 15 in the New England Journal of Medicine said. Kyle 'KJ' Muldoon Jr suffers from CPS1 deficiency which causes toxic levels of ammonia to accumulate in his blood. To treat him, scientists and doctors from the University of Pennsylvania and the Children's Hospital of Philadelphia developed a personalised treatment based on 'base editing', a new version of the decade-old CRISPR-Cas9 technology. Scientists say this technology can potentially treat thousands of uncommon genetic diseases. But there remain many roadblocks to its universal adoption. What is CRISPR? Following infection by a virus, humans generate an 'immune memory' in the form of antibodies. When they are infected by the same virus again, these antibodies quickly identify the pathogens and help neutralise them. CRISPR, short for 'clustered regularly interspaced short palindromic repeats', is an immune system found in microbes such as bacteria which fights invading viruses. When a virus infects a bacterial cell, CRISPR too helps establish a memory — but a genetic one, not in the form of antibodies like in humans. When a virus enters a bacterial cell, the bacterium takes a piece of the virus's genome and inserts the DNA into its own genome. CRISPR then produces a new 'guide' RNA with the help of the newly acquired DNA. During a future attack by the same virus, the guide RNA quickly recognises the virus DNA and attaches itself to it. Then, the guide RNA directs an enzyme (a type of protein) called Cas9 to act like 'molecular scissors' to cut and eliminate the virus DNA. In 2012, scientists Jennifer Doudna and Emmanuelle Charpentier replicated this mechanism found in microbes to develop a gene-editing tool, which they called CRISPR-Cas9, a feat which earned them the Nobel Prize for Chemistry eight years later. How does CRISPR-Cas9 gene-editing work? The tool works much like the 'cut-copy-paste', or 'find-replace' functionalities in common computer programmes. Genetic information in DNA is stored as code made up of four chemical bases — adenine (A), guanine (G), cytosine (C), and thymine (T). These bases exist in pairs, which are then stacked one on top of each other, creating the horizontal layers of the double-helix structure of DNA. Note that A always pairs with T, and C always pairs with G. Genetic disorders, like the one KJ suffers from, occur due to the presence of an abnormal DNA sequence, that is, a mispairing (A-G or G-T). The first task for the gene-editing tool is to identify the abnormal DNA sequence behind a patient's ailment. Once the bad DNA is located, scientists create a guide RNA attached to a Cas9 enzyme, which is then introduced to the target cells of the patient. The guide RNA recognises the bad DNA sequence, then the Cas9 enzyme cuts the DNA at the specified location in a process called a 'double-strand break' (since the cut is made on both strands of the DNA). This gets rid of the DNA sequence causing the illness. DNA strands have a natural tendency to reattach and repair themselves, meaning there is a chance that the bad sequence regrows. To tackle this issue, scientists also supply the correct DNA sequence after the 'cutting' process which is meant to attach itself to the broken strands of DNA. Over the years, scientists have made many improvements to the original CRISPR-Cas9 technology, making it safer and more precise. A newer, evolved version of this tool is 'base editing'. How does base editing work? Base editing and CRISPR-Cas9 differ significantly in how they modify DNA. Unlike CRISPR-Cas9, base editing does not make a double-strand break. Rather, it enables targeted single-base conversions with the help of a Cas9 enzyme fused to a base-modifying enzyme. This allows scientists to fix mispairing of the bases by changing one specific base. For instance, mispaired A-C bases can be corrected to A-T by converting C to T. To treat KJ, scientists first determined which mispaired base in his DNA was causing his condition. They then programmed the base editing tool to find and rewrite the target base. This process can be likened to using a pencil and an eraser, rather than scissors and glue, as in CRISPR-Cas9. 'In the older version of CRISPR, scientists were required to provide additional DNA from outside, which would be pasted at the site where the double-strand break takes place. In base editing, however, the system by itself can make a very precise change without the need for a foreign DNA to be inserted,' Debojyoti Chakraborty, principal scientist at CSIR-Institute of Genomics and Integrative Biology, told The Indian Express. 'As a result, base editing has fewer components and is compact, making it easier to package in delivery vehicles, which can take it to target cells,' he said. In 2023, Chakraborty and his team tried to develop a similar tool to treat a patient with a rare neurodegenerative disease. But she passed away before the experiment could be carried out. Will base editing become commonplace soon? Chakraborty said the successful use of base editing for treating KJ has given hope to doctors treating people with rare genetic disorders for whom no medical treatments were currently available. However, it is unlikely that such technologies will become commonplace any time soon, first and foremost due to the prohibitive costs of such treatments. Even if it were to become widely available, base editing would not be accessible to most people. (KJ's treatment was funded by research institutes and biotechnology. While they did not make any official disclosure regarding its cost, it is likely to be in the range of hundreds of thousand dollars, maybe more). Also, the base editing tool created to help KJ was a one-off treatment, meaning it was designed specifically for his unique genetic disorder and cannot be used to treat other individuals with different disorders. This poses a unique challenge with regards to scaling up such technologies for mass consumption, something that disincentives pharmaceutical companies to invest in their development. Getting regulatory approvals is another issue. 'To do such a thing in India is very difficult because it also means that you will have to get rid of red tapism,' Chakraborty said. It remains to be seen how researchers make such personalised treatments more accessible. Till then, only a few fortunate people like KJ will benefit from base editing therapies.
Gulf Today
21-05-2025
- Health
- Gulf Today
US baby with rare illness treated with tailor-made gene edit
A US infant with a rare condition has become history's first patient to be treated with a personalized gene-editing technique that raises hopes for other people with obscure illnesses, doctors said Thursday. The wee pioneer is KJ Muldoon, now a 9-and-a-half-month-old boy with chubby cheeks and big blue eyes. Shortly after birth, he was diagnosed with a rare and serious condition called CPS1 deficiency. It is caused by a mutation in a gene that produces an enzyme key to liver function, and prevents people with it from eliminating certain kinds of toxic waste produced by their metabolism. "You Google 'CPS1 deficiency' and it's either fatality rate or liver transplant," the baby's mother, Nicole Muldoon, says in a video released by Children's Hospital of Philadelphia, where the baby was treated. KJ Muldoon (centre) sits with his siblings. AP With the prognosis grim, doctors suggested something that had never been done before: a personalized treatment to fix the baby's genome using what amounts to a pair of molecular scissors -- the technique called Crispr-Cas9, which earned its creators the Nobel prize for chemistry in 2020. The boy's father said he and his wife faced an impossible decision. "Our child is sick. We either have to get a liver transplant or give him this medicine that's never been given to anybody before, right?" said Kyle Muldoon. In the end, they agreed to have the child treated with an infusion created just for him to fix his genetic mutation -- incorrect DNA letters in the several billion that make up the human genome. "The drug is really designed only for KJ, so the genetic variants that he has are specific to him. It's personalized medicine," said Rebecca Ahrens-Nicklas, a member of the medical team who specializes in pediatric genetics. KJ Muldoon sits with his parents, Kyle and Nicole Muldoon, and his siblings. AP Once the tailor-made infusion reaches the liver, the molecular scissors contained in it penetrates cells and goes to work editing the boy's flawed gene. The results were promising for other people with genetic conditions, said the medical team, which published their study Thursday in the New England Journal of Medicine. KJ can now follow a diet richer in proteins -- his condition prohibited such before -- and does not need as much medicine as he used to. But he will need to follow-up long term to monitor the safety and efficacy of the treatment, the team said. Ahrens-Nicklas said she hoped this achievement will allow the boy to get by with little or no medication some day. "We hope he is the first of many to benefit from a methodology that can be scaled to fit an individual patient's needs," the doctor said. Agence France-Presse
