Crouzon Syndrome With Acanthosis Nigricans

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Quiver Bioscience (“Quiver”), a discovery technology and therapeutics company advancing programs for the treatment of serious central nervous system (CNS) disorders, announces today a collaboration with the Dup15q Alliance to advance an antisense oligonucleotide (ASO) therapeutic program for chromosome 15q duplication (Dup15q) syndrome using Quiver’s innovative “Genomic Positioning System” (GPS). There are currently no disease-modifying treatments for Dup15q, which remains a substantial unmet medical need. Dup15q is a neurodevelopmental disorder characterized by moderate-to-severe hypotonia, motor and language delays, intellectual disability, epilepsy, and autism spectrum disorder (ASD). Most Dup15q individuals experience seizures and are at an increased risk for sudden unexpected death in epilepsy (SUDEP). Dup15q is associated with genomic copy number variations that result in increased levels of the UBE3A gene product. Dup15q syndrome is clinically related to Angelman syndrome (AS), a distinct neurodevelopmental disorder caused by UBE3A loss of function. ASO therapeutics for AS are currently in clinical trials and have shown early potential benefits in disease modification. The current prevalence estimates for Dup15q syndrome are as high as 1 in 4,000 live births. Genetic medicine approaches, such as ASOs, to reduce UBE3A levels are a viable precision therapeutic path for the treatment of Dup15q and have disease-modifying potential. Quiver has leveraged its unique GPS platform, which integrates unique-in-world, scalable, human single-cell neuronal electrophysiology data with artificial intelligence and machine learning (AI/ML) analytics, and extensive experience in ASO design to robustly model Dup15q syndrome and identify, optimize, and advance effective ASO candidates capable of reducing UBE3A and rescuing associated human Dup15q patient-specific cellular phenotypes. This initial grant formalizes a growing partnership between Quiver and the Dup15q Alliance that has been built over several years. The grant will support requisite preclinical studies to characterize Quiver’s lead therapeutic candidate ASOs and advance towards a transformative treatment for the Dup15q community. “We’re thrilled to continue working shoulder to shoulder with the excellent team at the Dup15q Alliance in advancing this important therapeutic for the community of patients and their families,” says Graham Dempsey, Ph.D., Founder and Chief Scientific Officer at Quiver Bioscience. “This is a devastating disease,” says Mike Porath, Executive Director of the Dup15q Alliance. “We are committed to developing a therapeutic treatment so people with Dup15q syndrome can thrive. We're excited to partner with Quiver to reach our ambitious goals as quickly as possible.” About Quiver Bioscience Quiver Bioscience is a technology-driven company established to create transformational medicines for the brain while simultaneously uncovering new biology and novel, effective drug targets. Using advanced single-cell imaging and multi-omics, we are building the world's most information-rich neuronal insight map via our "Genomic Positioning System". Our approach integrates cutting-edge scalable human models, state-of-the-art technology and proprietary engineering, and learning and surrogate AI/ML models to identify novel therapeutic targets and the best candidate molecules to deliver new and meaningful therapeutics to patients. For information, including additional publications describing the application of Quiver’s GPS to drug discovery, visit www.quiverbioscience.com. About Dup15q Alliance The Dup15q Alliance is a nonprofit organization devoted to bringing awareness to Dup15q syndrome and empowering individuals living with the condition by supporting scientific and clinical research towards effective therapeutics. Media: Noélle Germain +1-617-396-3611 noelle.germain@quiverbioscience.com ‍

Johnson & Johnson and Legend Biotech's additional investment comes just as the so-called Tech Lane facility is set to begin clinical production of Carvykti in a few weeks. On track to meet their long-stated capacity goal for multiple myeloma therapy Carvykti in 2025, Johnson & Johnson and Legend Biotech are already planning the next phase of their cell therapy manufacturing expansion.Based on internal projections for demand, the two companies have approved about $150 million in additional investments to expand the future capacity of a brand-new facility in Ghent, Belgium, Legend CEO Ying Huang, Ph.D., and interim Chief Financial Officer Jessie Yeung said on an investor call Tuesday.J&J and Legend decided on the new investment just as the so-called Tech Lane facility is set to begin clinical production of Carvykti in a few weeks. The site is expected to gain approval to initiate commercial supply by the end of 2025, according to Huang.For the latest expansion, early engineering and design work has begun, and construction is slated to start in the second half of 2025 and expected to be complete in 2028, Yeung said. The addition will primarily provide supplies for the EU market, a Legend spokesperson told Fierce Pharma.Over the last several years, J&J and Legend have been busy working to ramp up their cell therapy manufacturing capacity. Since Carvykti’s initial FDA approval in early 2022, supply constraints have limited the CAR-T therapy’s revenue growth. What’s more, an expanded FDA nod in April 2024 in second-line multiple myeloma roughly tripled the med’s eligible patient population.In one of the pair’s recent efforts to boost supply, contract manufacturer Novartis recently won FDA approval to make commercial supply of Carvykti at its site in Morris Plains, New Jersey, Huang said on the call. Also in New Jersey, J&J and Legend said in the fall of 2022 that they would double the investment in their flagship Raritan manufacturing facility to a total of $500 million. Legend now expects regulatory approval of a new section at that site in the second half of this year.The companies have another site in Ghent, called the Obelisc facility, which began commercial production in the third quarter of 2024 and is now operating at full capacity, according to Legend.These recent expansions will help the companies reach a capacity to be able to produce 10,000 doses of Carvykti on an annualized basis this year, a goal that was outlined in 2023.“By the end of this year, we’ll be in a situation where supply is fully meeting the demand where we are, and then we’ll be able to keep pace,” Alan Bash, Legend’s president of Carvykti, said on Tuesday’s call. But as analysts noted on the call, Wall Street’s 2026 consensus sales projection for Carvykti indicates that market watchers were unsure about whether patient demand will start to taper off.As of Tuesday, Carvykti can be administered at 104 certified treatment centers in the U.S. Meanwhile, growing levels of administration in the outpatient setting—and increased use at community practices—offer “significant upside” to the drug's growth trajectory, Bash said.Recipients of CAR-T therapies need to be monitored for the potentially life-threatening side effect of cytokine release syndrome. Because of Carvykti’s delayed onset of te syndrome, community providers are able to utilize outpatient administration, thereby freeing up limited inpatient space.This factor can help increase overall treatment capacity and therefore boost demand. The proportion of outpatient Carvykti use has increased from about 30% a year ago to more than 50% today, according to Bash.J&J and Legend have a three-stage plan to increase adoption in the community setting, where most multiple myeloma patients are treated. Right now, the drugmakers are reaching out to more than 8,000 community oncologists to educate them on Carvykti and to help them refer patients to certified treatment centers.In the second stage, starting later this year, the pair will identify certain regional hospitals and community accounts that are closely affiliated with large hospitals to administer Carvykti on-site, Bash said. For the third stage, which will begin next year, the goal is to have the drug more widely administered in community oncology practices, he added. Looming competition Another key reason analysts have reservations about Carvykti in 2026 is the expected entry of Gilead Sciences and Arcellx’s rival BCMA CAR-T anito-cel. In a pivotal phase 2 trial in late-line myeloma, anito-cel showed what analysts viewed as competitive efficacy and better safety to Carvykti, with the important caveat that cross-trial comparisons carry inherent flaws.On Tuesday’s call, Huang played down the potential threat of anito-cel for Carvykti in 2026. When approved, anito-cel would be entering the late-line setting, whereas the majority of Carvykti’s revenue mix will come from earlier lines, he argued. By the end of 2025, Legend expects about two-thirds to three-quarters of Carvykti revenue will come from second- to fourth-line treatment, he said.In anito-cel's phase 2 trial, one factor that stood out for the drug was the absence of some delayed neurotoxicities, including Parkinsonism, which has recently emerged as a concern for Carvykti. During Tuesday’s call, Legend executives pointed to a recent single-site study by the Colorado Blood Cancer Institute. The small study of 39 patients suggested that unrestrained absolute lymphocyte count (ALC) expansion is predictable of neurotoxicites following Carvykti treatment and that prophylactic treatment with the steroid dexamethasone may be able to mitigate the effect. The study did not separate delayed neurotoxocities from other neurologic complications.Legend is talking to multiple centers to potentially study this phenomenon in an investigator-initiated trial, and it has incorporated the mitigation strategy into ongoing Cartitude trial programs for Carvykti, Chief Medical Officer Mythili Koneru, M.D., Ph.D., said on Tuesday’s call.As to the real world, Koneru noted that an ALC-based monitoring and mitigation strategy is easily applicable, because it’s “a very typical lab result that is checked very frequently and commonly at all centers, including academic centers as well as in a community setting.”

Every Cure will leverage Google Cloud's infrastructure and AI technologies, including Gemini 2.0, to accelerate discoveries and improve patient outcomes PHILADELPHIA and SUNNYVALE, Calif., Jan. 13, 2025 /PRNewswire/ -- Every Cure, a leading nonprofit dedicated to identifying and validating new uses for existing drugs, today announced an expanded collaboration with Google Cloud to advance AI-driven drug repurposing. By leveraging Google Cloud's AI tools, including Gemini 2.0 large language models to power Every Cure's MATRIX platform, Every Cure aims to accelerate the discovery, validation, and global dissemination of life-saving treatments for diseases lacking effective therapies. More than 300 million people globally suffer from diseases without available treatments, while many face barriers to accessing existing therapies. Drug repurposing utilizes existing medicines, which are often affordable, widely accessible generics with established safety profiles and extensive data, to address this unmet need. By combining Every Cure's mission-driven expertise with Google Cloud's advanced AI capabilities, the collaboration seeks to transform healthcare delivery and patient outcomes worldwide. Every Cure researchers use Google Cloud's infrastructure to stay at the cutting edge of drug repurposing, an approach that can bypass the prolonged timelines and high costs associated with traditional drug development. The Every Cure Leadership Team has already advanced over a dozen repurposed treatments for diseases they weren't initially intended for, including Castleman disease, DADA2 syndrome, POEMS syndrome, and angiosarcoma. By leveraging iterative learning cycles, Every Cure will not only advance computational biology but also deliver immediate benefits to patients with Google Cloud. "We are so excited about the potential for this collaboration with Google Cloud to rapidly scale the impact that Every Cure can make on patients' lives. We created Every Cure to treat patients with existing drugs as quickly as possible and this collaboration supercharges our ability to do this," said Every Cure Co-Founder & President David Fajgenbaum, MD, MBA, MSc. This collaboration focuses on three key use cases: Improving accuracy of AI-driven drug repurposing predictions Every Cure's MATRIX platform will use Google Cloud's AI technology, including Gemini 2.0, to analyze complex biomedical knowledge and integrate diverse data sources, including omics datasets, clinical data, real-world evidence and biomedical knowledge graphs. This synergy will enhance prediction accuracy, identifying overlooked therapeutic opportunities, and accelerating hypothesis generation. Validating predictions through accelerated preclinical testing and optimized clinical trials By leveraging Gemini 2.0-driven trial design and predictive analytics, Every Cure will streamline in vitro and in vivo preclinical testing and clinical trial workflows to confirm efficacy and safety of repurposed drugs. Every Cure will also use Google Cloud's AI-powered tools to select optimal trial designs, reducing resource use and ensuring more efficient evaluation of treatments. Ensuring global adoption of validated treatments to save lives With the help of Google Cloud's scale and infrastructure, Every Cure will ensure that validated repurposed drugs are rapidly adopted into clinical practice, particularly in underserved regions. A few examples of Google Cloud and Workspace tools that are used as part of this collaboration are Google Kubernetes Engine (including GPUs), Ray on VertexAI, BigQuery and Cloud Storage to facilitate the secure dissemination of clinical guidelines. Meanwhile Data Studio provides visual dashboards for global stakeholders. "Every Cure is doing incredible work with the power of cloud and AI technologies by repurposing medicine to save lives," said Scott Penberthy, Senior Director of Applied AI, Office of the CTO, Google Cloud. "We're delighted customers are tapping into the power of GPUs, TPUs, search and AI models that have been purpose built for Google systems, now available on Google Cloud. We are proud to collaborate with them on this journey." This collaboration builds on a successful collaboration between Every Cure and Google Cloud. Google Cloud has supported Every Cure with infrastructure and services to manage biomedical datasets and execute complex computations. With the help of Gemini 2.0's advanced capabilities, Every Cure aims to transform healthcare by accelerating the pace of innovation while ensuring that breakthroughs reach patients worldwide. Every Cure has already identified a number of promising drug repurposing opportunities through its AI-powered pipeline for diseases with major unmet need that are undergoing further investigation, including a neurodevelopmental disorder involving folate receptor auto-antibodies which can be helped with a generic drug that repletes the folate deficiency and can address neurodevelopmental challenges in these patients. About Every Cure Every Cure is a nonprofit biotech organization committed to unlocking the potential of existing medicines to treat as many diseases as possible. By harnessing AI-driven platforms, rigorous preclinical and clinical testing, and global dissemination strategies, Every Cure accelerates the delivery of life-saving treatments to patients worldwide. Every Cure has been profiled on Good Morning America, USA Today, and the Wall Street Journal and selected as both an Audacious Project grantee from TED and a Founder's Prize recipient from Elevate Prize Foundation. Learn more at . Media Contact: [email protected] SOURCE Every Cure WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED