Latest news with #KJMuldoon
Business Wire
5 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
06-06-2025
- Health
- Yahoo
FDA meeting gives window into gene therapy field's angst
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. Anyone looking for evidence of genetic medicine's enormous promise need only read of KJ Muldoon. The 10-month-old infant headed home from a Philadelphia hospital this week, dressed in a celebratory cap and gown, after his life-threatening disease was successfully treated with a bespoke CRISPR therapy. While baby KJ is not cured, the treatment has stabilized his disease, a rare liver condition known as CSP1 deficiency, to such extent he's able to resume eating a normal diet. Doctors, who hurriedly designed and constructed KJ's custom therapy in a matter of months, have backed off supportive medications and hope he'll no longer need a liver transplant. 'Each year, 10 million babies are born with one of about 10,000 known rare genetic diseases, many of which are, in principle, now treatable with genetic medicines,' David Liu, a pioneering CRISPR scientist whose laboratory helped in KJ's treatment, said at a meeting hosted by the Food and Drug Administration Thursday. 'The opportunity created by this perfect storm moment in scientific, medical, regulatory and manufacturing innovation is to provide on-demand genetic treatments like KJ's at scale.' Yet Liu and 22 other gene therapy experts and advocates who attended Thursday's roundtable didn't travel to the regulator's headquarters in White Oak, Maryland to extol the field's advances. By and large, they came to warn of a crisis. There are now dozens of approved cell and gene therapies in the U.S., some of which offer near-curative potential for serious diseases like spinal muscular atrophy, sickle cell disease and acute lymphoblastic leukemia. However, the sector that's produced these therapies is struggling. Investors have soured on genetic medicine as developers struggle to prove they can profitably sell the complex and often hugely expensive treatments. Biotechnology companies are cutting research, laying off staff and, in some cases, shutting down. Large pharmaceutical firms are no longer willing to bet billions of dollars they can surmount the regulatory and reimbursement hurdles that stand in the way of many of these therapies. And academic labs, still bursting with promising new ideas for technologies like CRISPR, now fear their projects will wither on the vine. 'We estimate that over 100 rare disease gene therapy products that had reached clinical stage have been discontinued since 2023 — not because of treatment failure, but because of the risk of market failure,' said Terence Flotte, dean of the University of Massachusetts' T.H. Chan School of Medicine and president of the American Society of Cell and Gene Therapy. 'The scientific advances that we have witnessed are just nothing short of spectacular. It's not hyperbole,' said Crystal Mackall, a professor at Stanford University and founding director of the cancer cell therapy center there. 'Despite this unconditional scientific success, the field is really struggling to deliver these therapies to all patients who can benefit.' Their warnings found a receptive audience in FDA leadership. Commissioner Martin Makary and top official Vinay Prasad, who leads the office that oversees cell and gene therapies, were sympathetic to experts' arguments and pledged to help. 'We are going to continue the successes of the FDA in facilitating the regulatory process for these conditions and these products,' said Makary. 'We're also going to try to improve by creating more efficiencies.' Prasad, who in the past has criticized the FDA's accelerated approval of a gene therapy for Duchenne muscular dystrophy, showed support for flexible trial designs and endpoints when appropriate for the disease or treatment. He also noted the agency accepts that cell and gene therapies don't always comes with transformative potential. 'We understand that progress is not always made in a single leap,' he added. 'We will consider incremental steps forward, because those add up.' The assembled experts came with lists of possible solutions. Carl June, a famed immunologist and cell therapy researcher at the University of Pennsylvania, called for the U.S. to borrow from the two-tier regulatory system used in China, which allows for medical institutions to more rapidly start first-in-human trials under the supervision of local review boards. Don Kohn, a University of California, Los Angeles scientist who has developed gene therapies, asked the FDA to reduce the requirements for 'comparability' testing when companies transition production from academic to commercial settings. Others emphasized the importance of regulatory awards, like the priority review vouchers granted by the FDA to developers of certain therapies, who often sell them for needed capital. And many called for the agency to share more feedback and lessons learned from the applications they receive from industry. Behind all of their suggestions was a consistent concern: If regulators don't help solve the field's problems, the U.S. risks losing its leadership in developing the kinds of treatments that can cure diseases. 'If we don't adapt, the next generation of treatments will emerge abroad,' said June. 'The future of medicine with cell and gene therapy is at stake.' Their message seemed to be heard by Makary and Prasad, who noted that many of the issues raised are on their radar at FDA. Prasad, for instance, noted that they hope to redact and make available more internal documents to aid developers' understanding of what the FDA is looking for. 'This is not a horse and pony show to say we did this,' added Makary. 'This is an honest listening session.' Recommended Reading A bespoke CRISPR therapy suggests a blueprint for treating 'N-of-1' diseases
04-06-2025
- Health
Baby saved by gene-editing therapy 'graduates' from hospital, goes home
KJ Muldoon, a 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment, was released from the hospital this week. KJ has spent the majority of his life at Children's Hospital of Philadelphia after being diagnosed with a one-in-a-million, deadly genetic disease shortly after birth. Working quickly, his doctors were able to use new gene-editing technology called CRISPR, designing a bespoke treatment just for him. The treatment, first infused into his body at seven months old, seems to have worked. KJ's body, which was fighting a toxic buildup of ammonia, began to thrive and he quickly gained weight appropriate for a baby of his age. Wearing a cap and gown to symbolize his 'graduation' from the hospital, baby KJ was discharged home to his parents and siblings on Tuesday after spending 307 days at Children's Hospital of Philadelphia. Doctors and nurses gathered for a "clap out" on his way out the hospital doors, and he was escorted home by local law enforcement. KJ's metabolic condition, called carbamoyl-phosphate synthetase 1 deficiency, affects about 1 in 1.3 million people. The disease kills 50% of babies by early infancy. KJ 'had the most severe variant," Dr. Ahrens-Nicklas, one of KJ's doctors at Children's Hospital of Philadelphia, told ABC News. "This meant that we had to expedite the pathway for personalized therapy we were already working on," he said. Gene therapy treatments have already been approved for more common genetic diseases, including the blood disorders sickle cell disease and beta thalassemia, which affect tens of thousands of patients in the U.S. Those treatments are sold by major pharmaceutical and biotechnology companies. KJ's disease is so rare that his doctors were on their own. But thanks to the technology available at Children's Hospital of Philadelphia, his doctors believed they could use a streamlined approach to make their own bespoke therapy, in-house. CRISPR gene-editing technology was the perfect tool for a rare genetic disease like KJ's, and potentially future babies born with slightly different genetic errors. "Think of it like a GPS signal," Dr. Kiran Musunuru, director of the Penn Cardiovascular Institute's Genetic and Epigenetic Origins of Disease Program, told ABC News. "You can change where the GPS is going depending on what specific sequence of genes you want to change." Musunuru says there is still a lot of work to be done on this bespoke treatment to make it feasible, but he is hopeful that more babies with ultra-rare conditions can be treated this way.
Yahoo
04-06-2025
- General
- Yahoo
Baby saved by gene-editing therapy 'graduates' from hospital, goes home
KJ Muldoon, a 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment, was released from the hospital this week. KJ has spent the majority of his life at Children's Hospital of Philadelphia after being diagnosed with a one-in-a-million, deadly genetic disease shortly after birth. Working quickly, his doctors were able to use new gene-editing technology called CRISPR, designing a bespoke treatment just for him. MORE: Doctors save baby's life with first-ever gene fix for deadly rare disease The treatment, first infused into his body at seven months old, seems to have worked. KJ's body, which was fighting a toxic buildup of ammonia, began to thrive and he quickly gained weight appropriate for a baby of his age. Wearing a cap and gown to symbolize his 'graduation' from the hospital, baby KJ was discharged home to his parents and siblings on Tuesday after spending 307 days at Children's Hospital of Philadelphia. Doctors and nurses gathered for a "clap out" on his way out the hospital doors, and he was escorted home by local law enforcement. KJ's metabolic condition, called carbamoyl-phosphate synthetase 1 deficiency, affects about 1 in 1.3 million people. The disease kills 50% of babies by early infancy. KJ 'had the most severe variant," Dr. Ahrens-Nicklas, one of KJ's doctors at Children's Hospital of Philadelphia, told ABC News. "This meant that we had to expedite the pathway for personalized therapy we were already working on," he said. Gene therapy treatments have already been approved for more common genetic diseases, including the blood disorders sickle cell disease and beta thalassemia, which affect tens of thousands of patients in the U.S. Those treatments are sold by major pharmaceutical and biotechnology companies. MORE: FDA approves gene therapies for sickle cell disease, a 'functional cure' for many KJ's disease is so rare that his doctors were on their own. But thanks to the technology available at Children's Hospital of Philadelphia, his doctors believed they could use a streamlined approach to make their own bespoke therapy, in-house. CRISPR gene-editing technology was the perfect tool for a rare genetic disease like KJ's, and potentially future babies born with slightly different genetic errors. "Think of it like a GPS signal," Dr. Kiran Musunuru, director of the Penn Cardiovascular Institute's Genetic and Epigenetic Origins of Disease Program, told ABC News. "You can change where the GPS is going depending on what specific sequence of genes you want to change." Musunuru says there is still a lot of work to be done on this bespoke treatment to make it feasible, but he is hopeful that more babies with ultra-rare conditions can be treated this way. ABC's Dr. Keerthana Pakanati contributed to this report.
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.
