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Verve halts enrollment in lead trial following grade 3 side effects and prioritizes next steps for PCSK9 editing therapy. < Back Verve Therapeutics Halts Gene Editing Trial After Side Effects Verve halts enrollment in lead trial following grade 3 side effects and prioritizes next steps for PCSK9 editing therapy. Verve Therapeutics , a pioneering biotech company focused on developing gene-editing therapies for cardiovascular diseases, recently announced a temporary halt in enrollment for its lead clinical trial. The decision comes in response to the occurrence of Grade 3 elevations in liver enzymes observed in participants receiving its investigational PCSK9 editing therapy. The trial, which aims to evaluate the safety and efficacy of Verve's gene-editing technology targeting PCSK9, a gene linked to high cholesterol and cardiovascular risk, has encountered a setback due to safety concerns. Grade 3 elevations in liver enzymes represent a significant adverse event, prompting the company to prioritize a thorough investigation before proceeding with further enrollment. Verve's decision to pause enrollment aligns with its commitment to patient safety and rigorous clinical evaluation. By taking this precautionary measure, the company demonstrates its dedication to ensuring the integrity and safety of its therapeutic approach. The temporary halt in enrollment follows a series of promising advancements in the field of gene editing, particularly in the context of cardiovascular disease management. Verve's innovative approach leverages cutting-edge technology to target specific genes associated with heightened cardiovascular risk, offering the potential for more precise and effective treatment strategies. Despite this setback, Verve remains steadfast in its mission to advance gene-editing therapies for cardiovascular diseases. The company has outlined plans to thoroughly investigate the observed adverse events and implement appropriate measures to address safety concerns before resuming enrollment in the trial. The decision underscores the inherent challenges and complexities involved in the development of novel therapeutic modalities, particularly in the realm of gene editing. While setbacks are not uncommon in the biopharmaceutical industry, Verve's proactive response highlights its commitment to upholding the highest standards of patient care and scientific rigor. Moving forward, Verve will continue to collaborate with regulatory authorities, healthcare professionals, and patients to navigate the clinical development process effectively. By prioritizing safety and transparency, the company aims to overcome challenges and ultimately deliver innovative therapies that have the potential to transform the landscape of cardiovascular disease treatment. Learn more about Verve Therapeutics here . Author BioFocus Newsroom Previous Next

This study introduces a groundbreaking genetic engineering approach with immense potential for precision medicine. < Back Hit-and-run Epigenome Editing: Gene Therapy Advancements This study introduces a groundbreaking genetic engineering approach with immense potential for precision medicine. Recent research titled "Durable and efficient gene silencing in vivo by hit-and-run epigenome editing" unveils a revolutionary method for controlling gene expression in living organisms. This study introduces a groundbreaking approach called hit-and-run epigenome editing, which promises long-lasting and efficient suppression of targeted genes, holding immense potential for biomedical research and therapeutic applications. Published in Nature , the research marks a significant advancement in the field of gene regulation, addressing a longstanding challenge of achieving durable and effective gene silencing in vivo . Traditional gene editing techniques often face limitations such as transient or off-target effects, resulting in the feed for continuous interventions in order to maintain gene suppression. The novel hit-and-run epigenome editing method circumvents these challenges by precisely modifying the epigenetic marks associated with the target gene. Unlike conventional gene editing approaches, which directly alter the DNA sequence, this technique focuses on modulating the chemical changes that regulate gene expression without permanently changing the genetic code. In the study, Martino Alfredo Cappelluti and team demonstrated the efficacy of hit-and-run epigenome editing in achieving sustained gene silencing in living organisms. By carefully designing and delivering epigenome-editing agents, they successfully suppressed the expression of target genes over extended periods without the need for continuous intervention. This long-term gene silencing was accompanied by minimal off-target effects, ensuring specificity and safety. Moreover, the versatility of hit-and-run epigenome editing offers potential applications across various fields of biomedical research and clinical practice. From investigating the underlying mechanisms of genetic diseases to developing targeted therapies for cancer and other disorders, this innovative approach holds promise for revolutionizing the way we manipulate gene expression in living organisms. The implications of this research are profound, offering new avenues for understanding gene regulation and developing precision medicine strategies. By enabling durable and efficient gene silencing in vivo , hit-and-run epigenome editing opens doors to a wide array of possibilities for advancing both basic science and clinical applications. Author BioFocus Newsroom Previous Next

The UK-based biotech company, specialising in synthetic genomics, recently obtained $58 million in Series A financing. < Back Spotlight on Constructive Bio The UK-based biotech company, specialising in synthetic genomics, recently obtained $58 million in Series A financing. Constructive Bio’s recent $58 million Series A financing (bringing the total amount raised by the company to date to $75 million) signals a major milestone in synthetic biology and biomanufacturing. Based in the historic city of Cambridge, Constructive Bio is very much future-focused, operating in the burgeoning realm of creating new biomolecules. By advancing the capacity to "write genomes from scratch," Constructive Bio is pushing synthetic genomics to unprecedented levels, going beyond traditional genetic engineering, with platforms that can design and synthesize entire genomes and precisely tailor protein translation systems. This allows for the creation of entirely novel biomolecules with specific and programmable properties. This holds vast potential for industries reliant on complex molecules, such as pharmaceuticals, consumer goods, and advanced materials. CEO Ola Wlodek had this to say about Constructive Bio’s position, and the impact this financing will provide to them. "Synthetic genomics is redefining how we harness biology. Our suite of proprietary technologies and incomparable team allows us to build novel materials and products that will revolutionise drug manufacturing and discovery. This financing will enable us to accelerate bringing breakthrough products and platforms to market, which will grow our revenue through existing and new paths, ranging from exciting therapeutics to sustainable biomaterials. We see the future of biology, and we're leading the charge." Notably, Constructive Bio’s foundational technology originates from groundbreaking research by founder Professor Jason Chin at the UK’s Medical Research Council’s Laboratory of Molecular Biology. Chin’s landmark achievement—synthesizing the genome of E. coli —laid the groundwork for developing synthetic organisms capable of incorporating non-standard amino acids, an innovation that can create proteins and biopolymers with novel functionalities. This expansion of the genetic code represents a key advancement, enabling Constructive Bio to engineer proteins with enhanced features that are difficult, if not impossible, to produce via natural biological pathways. This funding infusion, led by prominent investors such as Ahren, OMX Ventures, and Paladin Capital Group, reflects strong confidence in Constructive Bio’s ability to commercialize its synthetic biology platforms. The addition of Nobel Laureate Sir Gregory Winter to the board adds extra depth of expertise, as Winter’s background in developing therapeutic monoclonal antibodies complements Constructive Bio’s vision for pioneering therapeutic proteins and enzymes. As Winter noted, "With this investment, we aim to develop Constructive Bio's technology to deliver biomanufacturing at scale. By providing a biological, cost-effective production process, we can help overcome shortfalls in global supplies of therapeutic peptides and proteins such as semaglutide ." Constructive Bio is unique in combining three core technologies—recoded genomes, engineered cell machinery, and expanded molecular chemistry—enabling rapid genome synthesis and versatile biomolecular engineering. This trifecta has allowed Constructive Bio to reduce genome synthesis timelines from years to days, fast-tracking their ability to produce commercial-scale biomaterials. Furthermore, by developing new routes to sustainability in molecule synthesis, Constructive Bio aligns with broader industry goals of sustainable production, reducing dependency on resource-intensive manufacturing processes. The company’s technology represents a remarkable synthesis of computational biology, protein engineering, and molecular chemistry, making it an innovator capable of transforming not just healthcare, but global supply chains for a range of high-demand biological products. The future of synthetic genomics, as Constructive Bio’s rapid growth suggests, is one where the boundaries of natural biology are expanded and applied across industries, making novel therapeutics, sustainable materials, and highly engineered biomolecules a tangible reality. Author BioFocus Newsroom Previous Next

The funding will help complete non-clinical safety and efficacy studies, setting up ABX-01 for a first-in-human clinical trial planned for early 2026. < Back Centauri Therapeutics Secures $5.1M Boost from CARB-X to Advance Novel Antimicrobial into Clinical Trials The funding will help complete non-clinical safety and efficacy studies, setting up ABX-01 for a first-in-human clinical trial planned for early 2026. Centauri Therapeutics, a UK-based biotechnology company specialising in immunotherapy, has received an additional $5.1 million from the Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) to accelerate the development of its lead antimicrobial compound, ABX-01. This latest award brings CARB-X’s total financial commitment to Centauri’s ABX-01 programme to $12.3 million since 2019. The initiative is part of CARB-X’s global mission to tackle the growing threat of antimicrobial resistance (AMR), a crisis projected by the World Health Organization to cause 10 million deaths annually by 2050 if left unchecked. Centauri announced the selection of its first clinical candidate for ABX-01 in March of this year. The broad-spectrum antimicrobial is designed to address multidrug-resistant Gram-negative bacterial infections, particularly in the lungs of severely ill and immunocompromised patients. These infections are among the most difficult to treat and represent a significant cause of hospital-acquired mortality worldwide. ABX-01 is built on Centauri’s proprietary Alphamer ® platform, which merges two distinct antimicrobial strategies into a single molecule. The compound not only directly kills bacteria but also enlists the body’s immune system, using mechanisms such as complement fixation and phagocytosis to clear the infection. This dual-action approach could represent a breakthrough in dealing with drug-resistant pathogens, particularly given the dwindling pipeline of effective new antibiotics. Dr. Jennifer Schneider, Chief Executive Officer of Centauri Therapeutics, underscored the importance of the partnership with CARB-X, commenting that “The unwavering scientific and financial support from CARB-X has provided stability to Centauri as a company, expanded understanding of our Alphamer platform, and enabled us to progress the ABX-01 programme from discovery, through early development, and is now providing a smooth and continuous path towards First in Human clinical studies. We are thankful for CARB-X and their continued engagement and confidence, which has allowed us to move a step closer to delivering a much needed therapeutic for serious, drug-resistant Gram-negative infections, even in the most clinically vulnerable patients.” From an industry perspective, the ABX-01 programme is notable because of its focus on Gram-negative bacteria such as Pseudomonas aeruginosa , Klebsiella pneumoniae , and Acinetobacter baumannii . These pathogens are infamous for their resistance to multiple drug classes, including carbapenems — often considered antibiotics of last resort. Few experimental therapies in development address Gram-negative infections effectively, and those that do often suffer from limitations such as poor safety profiles or limited spectrum of activity. Dr. Erin Duffy, Chief of Research and Development at CARB-X, emphasised the organisation’s long-standing support for Centauri: “We have been proud to support Centauri, beginning with answering key questions on the approach and continuing with the drug discovery that has led to the lead asset of ABX-01 and its progression towards building a dossier to support its advancement into first in human clinical trials.” While pre-clinical results have been promising, the true test will come in human studies, where safety, tolerability, and efficacy in severely ill patients will need to be demonstrated. If ABX-01 meets these milestones, it could become one of the most important additions to the limited arsenal against Gram-negative superbugs in decades. As AMR continues to rise, successes like this will be essential not only for individual patients but also for global health security. Author BioFocus Newsroom Previous Next

Dive into the leading DNA sequencing technologies driving genomic discovery from Oxford Nanopore, Illumina, PacBio, and 10x Genomics. < Back The Four Powerhouses of DNA Sequencing Dive into the leading DNA sequencing technologies driving genomic discovery from Oxford Nanopore, Illumina, PacBio, and 10x Genomics. DNA sequencing has come a long way in the past two decades, revolutionizing how we explore the complexities of life. From understanding disease pathways to advancing personalized medicine, cutting-edge technologies from Oxford Nanopore , Illumina , PacBio , and 10x Genomics are leading the charge. Each of these companies offer unique tools that cater to different scientific and industrial needs. Dive into what makes each one a standout in this dynamic landscape. Oxford Nanopore Technologies: Real-Time Sequencing On the Go Imagine carrying a sequencing device in your pocket and performing genomic analysis anywhere, from a lab bench to a rainforest. That’s the power of Oxford Nanopore Technologies (ONT). Their platforms, including the MinION, GridION, and PromethION, use nanopore-based sequencing—a method where single-stranded DNA passes through a tiny pore, causing electrical disruptions that identify each nucleotide in real time. ONT delivers ultra-long reads, often exceeding hundreds of kilobases. This capability is a game-changer for resolving structural variants and repetitive genomic regions. The portable MinION device enables on-site sequencing for applications like infectious disease tracking and environmental analysis. Real-time sequencing lets researchers make rapid decisions—ideal for clinical and field-based studies. However, while ONT’s technology is versatile, it’s not without challenges. Accuracy, though improving, historically lags behind competitors, and throughput can be inconsistent depending on sample quality. Illumina: The Gold Standard of Short Reads For years, Illumina has set the benchmark for sequencing accuracy and throughput. Their sequencing by synthesis (SBS) technology powers platforms like the NovaSeq and MiSeq, which are staples in laboratories worldwide. With error rates as low as 0.1%, Illumina’s short-read data is trusted for applications like clinical diagnostics and population genomics. The NovaSeq can churn out terabases of data in a single run, perfect for large-scale projects. While equipment costs are high, per-base sequencing costs are exceptionally low, making Illumina a cost-efficient option for high-volume projects. But, Illumina’s short-read sequencing (150-300 base pairs) has its limits, especially when it comes to tackling complex genomic regions or assembling new genomes. Hybrid approaches often pair Illumina’s accuracy with long-read technologies to bridge this gap. PacBio: Marrying Length and Accuracy When accuracy and long reads are non-negotiable, Pacific Biosciences (PacBio) delivers. Their single-molecule, real-time (SMRT) sequencing technology is celebrated for generating highly accurate long reads (HiFi reads), which are ideal for tackling complex genomes. Spanning up to 15-20 kilobases with >99.9% accuracy, HiFi reads combine the strengths of long-read sequencing with unparalleled precision. PacBio’s ability to detect DNA modifications, like methylation, adds an epigenetic dimension to sequencing projects. Whether it’s structural variants or phased haplotypes, PacBio’s data excels in delivering comprehensive genomic insights. The trade-off? PacBio’s instrumentation and reagents are costlier compared to ONT or Illumina, and achieving high coverage can increase both expense and time. 10x Genomics: A Multidimensional Perspective While not a sequencing platform itself, 10x Genomics transforms how researchers extract value from sequencing data. Their Chromium platform partitions DNA or RNA into droplets, enabling downstream library preparation for platforms like Illumina. By linking short reads to long genomic fragments, 10x Genomics reconstructs haplotypes and resolves structural variants. From immunology to oncology, Chromium’s single-cell capabilities provide unparalleled insights into cellular diversity and function. The ability to integrate genomic, transcriptomic, and epigenomic data is a boon for systems biology. That said, 10x Genomics is reliant on external sequencing platforms, and its linked-read approach has faced competition from emerging long-read technologies. The Final Word Each sequencing technology has carved out a niche, excelling in different areas of genomics. Oxford Nanopore’s portability and real-time sequencing are unmatched for field applications. Illumina remains the go-to for short-read accuracy and large-scale studies. PacBio’s HiFi reads bridge the gap between accuracy and long-read capabilities, while 10x Genomics unlocks new dimensions in single-cell and multi-omics research. The genomics revolution is far from over, and as these technologies continue to evolve, so too will our ability to decode the complexities of life at unprecedented depth and precision. For researchers and biotech professionals, the challenge lies not in choosing the best technology overall, but the best technology for the question at hand. Author BioFocus Newsroom Previous Next

Revolutionary 3D multi-omics platform reveals new therapeutic targets, promising faster, more successful drug development. < Back Enhanced Genomics and ALBORADA Drug Discovery Institute Accelerate Alzheimer’s Drug Discovery Revolutionary 3D multi-omics platform reveals new therapeutic targets, promising faster, more successful drug development. A pioneering collaboration between biotech innovator Enhanced Genomics and The ALBORADA Drug Discovery Institute at the University of Cambridge has delivered a major breakthrough in Alzheimer’s research. The partnership, supported by Alzheimer’s Research UK, is already generating multiple novel drug targets that were previously undetectable using traditional genomic methods. By harnessing Enhanced Genomics’ proprietary 3D multi-omics platform, scientists have been able to map complex gene-regulatory interactions at an unprecedented scale and resolution. This has revealed critical new insights into the underlying biology of Alzheimer’s disease and accelerated the discovery of high-confidence, genetically validated drug targets. “This collaboration marks a turning point in how we discover drugs for complex diseases like Alzheimer’s,” said Dr. Dan Turner, Chief Scientific Officer at Enhanced Genomics. “Our platform doesn’t just look at the genome—it interprets how different regions of DNA interact in 3D space across specific human cell types. This gives us the power to uncover therapeutic targets hidden in the non-coding genome, something conventional tools simply can’t do.” The ALBORADA Drug Discovery Institute, based at the University of Cambridge, has used these findings to open new research pathways into drug development. “The insights provided by Enhanced’s technology have already led us to previously unknown targets with real therapeutic potential,” said Dr. John Skidmore, CSO at The ALBORADA Drug Discovery Institute. “This could significantly advance our ability to develop effective treatments for Alzheimer’s disease.” Alzheimer’s Research UK, the UK’s leading dementia research charity, sees this partnership as a model for future industry-academic collaborations. “This is the kind of ambitious, high-impact science we need to bring hope to the millions affected by dementia,” said Dr. Julia Dudley, Head of Strategic Programmes. “By combining cutting-edge technology with deep disease expertise, we’re accelerating progress toward the next generation of treatments.” The collaboration not only delivers immediate value in Alzheimer’s research, but also underscores the wider potential of Enhanced’s 3D multi-omics platform. By defining causal biology from disease-associated genetic variants, the technology dramatically improves both the speed and likelihood of success in early drug discovery. Looking ahead, Enhanced Genomics and The ALBORADA Drug Discovery Institute are exploring ways to expand their work into other neurodegenerative diseases. The company is also actively seeking new partnerships with pharmaceutical and biotech organizations interested in incorporating 3D multi-omics into their own drug discovery pipelines. About Enhanced Genomics: Enhanced Genomics is a biotechnology company revolutionizing drug discovery through its proprietary 3D multi-omics platform, capable of identifying high-confidence therapeutic targets from disease-associated variants across the entire genome. The platform enables faster, more efficient, and more successful drug development for common diseases with high unmet need. About The ALBORADA Drug Discovery Institute: Established by Alzheimer’s Research UK, The ALBORADA Drug Discovery Institute focuses on developing treatments that tackle the biological mechanisms driving neurodegenerative diseases, including Alzheimer’s. About Alzheimer’s Research UK: Alzheimer’s Research UK is the UK’s leading dementia research charity, dedicated to accelerating the discovery of new treatments and, ultimately, a cure for dementia. Author BioFocus Newsroom Previous Next

The FDA has approved a new plasma-based biomarker assay, the first blood-based diagnostic for Alzheimer's disease, offering a less invasive and more accessible alternative to traditional methods like PET scans and spinal taps. < Back FDA Clears Blood Test for Diagnosing Alzheimer’s Disease The FDA has approved a new plasma-based biomarker assay, the first blood-based diagnostic for Alzheimer's disease, offering a less invasive and more accessible alternative to traditional methods like PET scans and spinal taps. The U.S. Food and Drug Administration (FDA) has granted 510(k) clearance for the Lumipulse® G β-Amyloid 1-42/pTau217 Plasma Ratio test, marking a significant advancement in the diagnostic landscape for Alzheimer’s disease (AD). Developed by Fujirebio Diagnostics, this in vitro diagnostic is the first FDA-cleared blood test specifically designed to assist in the clinical evaluation of Alzheimer's by detecting key biomarkers linked to the presence of cerebral amyloid plaques. This regulatory milestone reflects a broader shift in the approach to diagnosing neurodegenerative conditions, moving away from highly invasive and resource-intensive methods such as cerebrospinal fluid (CSF) sampling and positron emission tomography (PET) imaging toward more scalable, minimally invasive assays. The Lumipulse G test quantifies the ratio of two critical proteins in blood plasma: β-amyloid 1-42 (Aβ1-42) and phosphorylated tau at threonine 217 (pTau217). These biomarkers are central to Alzheimer’s pathophysiology. Aβ1-42 is involved in the extracellular deposition of amyloid plaques, while hyperphosphorylated tau proteins contribute to the formation of intracellular neurofibrillary tangles, both recognized as defining features of AD. Rather than measuring amyloid deposition directly, the test leverages the pTau217/Aβ1-42 plasma ratio to infer the likelihood of amyloid plaque presence. This method provides clinicians with a biochemical signal that correlates closely with neuropathological status, supporting early-stage clinical decision-making. The FDA's clearance is supported by data from a pivotal clinical study involving 499 individuals with signs of cognitive impairment. Plasma test results were benchmarked against PET imaging and CSF-based assays. The Lumipulse G test demonstrated 91.7% positive predictive agreement and 97.3% negative predictive agreement, indicating a high degree of concordance with established reference standards. Furthermore, fewer than 20% of participants received indeterminate results, suggesting a high rate of diagnostic clarity for the majority of tested individuals. The assay is indicated for adults aged 55 years and older who are already exhibiting symptoms suggestive of cognitive decline, where differential diagnosis between Alzheimer’s and other dementias is clinically relevant. Until now, blood-based assays for Alzheimer’s have been confined to laboratory-developed tests offered by commercial providers such as Quest Diagnostics and Labcorp. These tests, while clinically useful, lacked FDA review and clearance. The Fujirebio assay represents the first step toward standardized, regulated blood biomarker diagnostics that could be implemented across a broader range of healthcare settings. By reducing dependence on CSF analysis and PET imaging, both of which are limited in availability and costly, this blood-based test can improve diagnostic equity and accelerate access to care. It is especially relevant as new disease-modifying therapies, such as monoclonal antibodies targeting amyloid, become available. These treatments are most effective when initiated early, underscoring the need for reliable tools to identify at-risk individuals before substantial neurodegeneration has occurred. Fujirebio’s progress aligns with a larger trend toward innovation in neurodegenerative disease diagnostics. In parallel, the company has expanded its partnership with Japanese pharmaceutical firm Eisai to jointly develop and promote blood-based biomarkers for Alzheimer’s and related disorders. This builds upon earlier collaborations focused on CSF diagnostics and supports the development of companion diagnostics for therapeutics like lecanemab (Leqembi), Eisai’s anti-amyloid agent co-developed with Biogen. Importantly, commercialization of therapies like Leqembi has been hindered by delays in diagnosis and limited access to confirmatory testing. Scalable biomarker tests such as Lumipulse G may help bridge this diagnostic gap, enabling earlier and more confident identification of eligible patients. While this development represents a leap forward, its optimal use still requires careful integration into clinical workflows. Blood biomarker results must be interpreted in the context of comprehensive assessments, including neurological exams, cognitive testing, imaging, and family history. Clinicians must also consider individual variability in biomarker expression, comorbidities, and genetic factors such as APOE genotype when interpreting results. To support this evolving paradigm, the Alzheimer’s Association is preparing to release clinical practice guidelines focused on the use of blood biomarkers in specialty care. These will provide evidence-based recommendations for appropriate patient selection and test interpretation and are expected to debut at the 2025 Alzheimer’s Association International Conference (AAIC). With more than 6.9 million Americans currently living with Alzheimer’s, a number projected to nearly double by 2050, the need for scalable, cost-effective diagnostic strategies is pressing. Blood-based biomarker testing represents a crucial step in addressing this gap. Not only does it enable earlier intervention, but it also supports a more efficient clinical workflow that could ultimately reduce the economic and human burden of dementia. The FDA’s clearance of the Lumipulse G blood test signals the beginning of a new chapter in Alzheimer’s diagnostics, one that is rooted in molecular science, guided by robust clinical data, and oriented toward accessibility and earlier disease detection. As the field continues to mature, further innovations in blood-based biomarker platforms may pave the way for even broader regulatory approvals and integration into routine cognitive health assessments. Author BioFocus Newsroom Previous Next

Collaboration with SRI demonstrates Nuvec®’s ability to selectively deliver RNA to cancer cells, advancing prospects for targeted, orally delivered RNA therapeutics. < Back N4 Pharma and SRI Advance Targeted RNA Delivery with Nuvec® Platform for Cancer Therapies Collaboration with SRI demonstrates Nuvec®’s ability to selectively deliver RNA to cancer cells, advancing prospects for targeted, orally delivered RNA therapeutics. N4 Pharma plc (AIM: N4P) has released new data from its collaboration with SRI International demonstrating the potential of its proprietary Nuvec® delivery platform to precisely target RNA payloads to cancer cells, a breakthrough that could reshape how RNA therapeutics are developed and delivered. The findings point to a future where common cancers such as lung, breast, and pancreatic could be treated with targeted RNA therapies, potentially in oral tablet form and with fewer side effects than conventional chemotherapy. The collaboration combined SRI’s targeting molecules with Nuvec® particles to deliver small interfering RNA (siRNA) payloads to non-small cell lung cancer cells. Results showed that Nuvec® functionalised with a targeting molecule binding to the αvβ6 adhesion protein, a marker overexpressed in many epithelial cancers, achieved precision delivery and selective uptake. siRNA payloads were active only when carried by targeted Nuvec® particles, confirming their ability to direct therapeutic action to diseased cells while sparing healthy tissue. “These recent data from our collaboration with SRI are particularly exciting because they represent the first example of the use of Nuvec® for the potential treatment of some of the most common and life-threatening cancers,” said Nigel Theobold, CEO of N4 Pharma. The results further validate Nuvec® as a differentiated RNA delivery system, supporting its broader application in oncology and beyond. Alongside its potential for targeted delivery, the platform offers multiple advantages including the ability to carry more than one RNA therapy in a single particle, stability suitable for oral delivery, low immunogenicity, and scalability in manufacturing. “Targeting RNA therapies to particular cell types is highly sought after by companies developing RNA therapeutics,” Theobold added. “We have now demonstrated Nuvec®'s ability to do this in multiple systems, which we believe sets it apart from other RNA delivery methods.” The global RNA therapeutics market, valued at $13.7 billion in 2023 and forecast to reach $18 billion by 2028, is growing rapidly but faces persistent challenges in manufacturing and delivery. Targeting specific tissues and reducing systemic toxicity remain among the biggest hurdles. Nuvec®’s latest data suggest it could help overcome these barriers and support deal-making with RNA therapy developers looking for next-generation delivery solutions. N4 Pharma raised capital earlier this year to generate data supporting Nuvec®’s key claims, including multi-RNA delivery, targeted uptake, oral dosing, and stability, with the aim of advancing both licensing opportunities and its own RNA therapeutics pipeline. Its lead program, N4 101, is an oral anti-inflammatory therapy for inflammatory bowel disease (IBD) designed to showcase the platform’s capabilities. Author BioFocus Newsroom Previous Next

Qureight’s AI-powered analysis of Insilico Medicine’s Phase IIa trial data for the novel IPF drug rentosertib supports its preliminary efficacy and underpins plans for expanded global clinical trials. < Back Qureight Analysis Supports Efficacy of Insilico Medicine’s AI-Discovered IPF Drug, Rentosertib, in Phase IIa Trial Qureight’s AI-powered analysis of Insilico Medicine’s Phase IIa trial data for the novel IPF drug rentosertib supports its preliminary efficacy and underpins plans for expanded global clinical trials. Qureight, a UK-based techbio company specializing in AI-powered analytics for respiratory diseases, today announced pivotal findings from its collaboration with Insilico Medicine, supporting the preliminary efficacy results of rentosertib (ISM001-055), a novel TNIK inhibitor under development for idiopathic pulmonary fibrosis (IPF). The analysis, powered by Qureight’s deep-learning image biomarkers and advanced clinical data analytics platform, confirmed that baseline disease severity was statistically comparable between cohorts in Insilico’s Phase IIa trial. This reinforces the validity of the trial’s positive outcomes and bolsters plans to expand the study into larger global patient populations. Rentosertib, discovered using Insilico’s generative AI technology, is a first-in-class small molecule targeting the TNIK pathway, implicated in the fibrotic processes that drive IPF. In findings published recently in Nature Medicine , the drug demonstrated preliminary clinical efficacy, with improvement in forced vital capacity (FVC) at 12 weeks, and validated TNIK as a novel therapeutic target for IPF for the first time. Using what is believed to be the world’s largest IPF biorepository, Qureight’s proprietary HRCT (high-resolution computed tomography) biomarkers showed that trial participants were representative of the broader global IPF patient population. This provides further support for Insilico’s plan to scale rentosertib trials internationally. Dr. Muhunthan Thillai, Co-founder and CEO of Qureight, emphasized the impact of the partnership: “The results from this initial project demonstrate the impact of our expertise, AI-powered analytics platform, and specialised patient datasets to support the progression of promising new therapies. We hope to extend the application of our technologies to future development stages to accurately quantify the impact of TNIK as a novel mechanism for the treatment of IPF.” Dr. Alex Zhavoronkov, Founder and CEO of Insilico Medicine, highlighted the broader implications: “Our collaboration with Qureight illustrates the transformative potential of AI in both drug discovery and development, paving the way for faster and more innovative therapeutic and clinical trial advancements.” The findings from this first analysis will be presented at the upcoming European Respiratory Society (ERS) Congress in Amsterdam from 5–9 September 2025. About IPF and Rentosertib Idiopathic Pulmonary Fibrosis is a chronic and progressive lung disease marked by irreversible scarring, affecting an estimated five million people worldwide. With limited treatment options and poor prognosis, the need for disease-modifying therapies is urgent. Rentosertib, a novel TNIK inhibitor discovered via AI, aims to halt or reverse lung fibrosis, offering a potentially groundbreaking therapeutic alternative. Early results indicate that it may offer meaningful clinical benefit for IPF patients globally. Author BioFocus Newsroom Previous Next

Kadimastem has secured shareholder approval for a merger with NLS Pharmaceutics, aiming to achieve a Nasdaq listing and advance its cell therapy treatments for amyotrophic lateral sclerosis (ALS). < Back Kadimastem Renews Push for Nasdaq Listing Kadimastem has secured shareholder approval for a merger with NLS Pharmaceutics, aiming to achieve a Nasdaq listing and advance its cell therapy treatments for amyotrophic lateral sclerosis (ALS). Kadimastem is getting a second chance for Nasdaq, after failing last year. The shareholders of cell therapy developer Kadimastem have approved the merger with NLS Pharmaceutics , a biotech that went public in 2021, potentially paving the path for its much-awaited listing in the exchange. Israel-based Kadimastem develops off-the-shelf cell therapy treatments, with its lead candidate targeting amyotrophic lateral sclerosis, also known as ALS. Kadimastem almost had a non-binding agreement to merge with IM Cannabis almost a year ago, securing it on Nasdaq. That deal was aborted after the planned legalization of cannabis in Germany meant that IM Cannabis backed out of the deal. Come July, Kadimastem had another path forward when it inked a binding term sheet with NLS. In 2023, NLS slashed it's headcount by 50% and began looking for strategic alternatives. Kadimastem saw NLS as an opportunity to get its stock listed on Nasdaq. The scheme was approved by Kadimastem's shareholders this week, and NLS is expected to convene in the coming weeks to seek the final approval of its own investors. Under the deal, Kadimastem would reach U.S. public markets and the much-needed cash to support its pipeline of cell therapies. The financial hurdle is not very high; the completion needs only $600,000 from NLS and $3.5 million from Kadimastem. Once the deal is closed, the Israeli biotech intends to start an ALS candidate Phase 2a U.S. trial. So far, Kadimastem's cell therapy has given promising results in early testing. It treated 10 ALS patients before COVID-19 forced a halt in a Phase 1/2a trial. The treatments seemed to slow disease progression at three months using the ALS Functional Rating Scale-Revised (ALSFRS-R). By the six- and twelve-month follow-ups, the rates of patients' deterioration were back to the pre-treatment levels. Author BioFocus Newsroom Previous Next

Cambridge biotech’s Activace™ platform offers a scalable, DNA-preserving method to unlock cancer biomarkers from liquid biopsies < Back Tagomics Breakthrough Study Showcases New Epigenomic Profiling Technology in Cell Reports Methods Cambridge biotech’s Activace™ platform offers a scalable, DNA-preserving method to unlock cancer biomarkers from liquid biopsies Cambridge-based biomarker discovery company Tagomics Ltd. has unveiled a groundbreaking study that could reshape the way scientists study the human epigenome and advance liquid biopsy diagnostics. Published this week in Cell Reports Methods , the peer-reviewed paper highlights the company’s Active-Seq technology, the foundation of its proprietary Activace™ platform, which enables genome-wide profiling of unmethylated DNA with unprecedented precision. The research, titled “Genome-wide profiling of unmodified DNA using methyltransferase-directed tagging and enrichment” , builds on work from the University of Birmingham. It demonstrates how Tagomics’ enzymatic approach to epigenomic profiling can identify biomarkers linked to cancer and other diseases by targeting unmethylated DNA regions, critical signals often missed by current technologies. Traditional methods for measuring DNA methylation, the chemical modifications that regulate gene activity, have long been a challenge in liquid biopsy applications, where only trace amounts of cell-free DNA (cfDNA) are available. Active-Seq sidesteps these limitations with a conversion-free workflow that preserves the DNA sequence and works with inputs as low as one nanogram. Integrated into the streamlined Activace platform, this approach allows for scalable, high-resolution analysis across large patient cohorts. In their colorectal cancer study, Tagomics scientists used Active-Seq to detect thousands of abnormally methylated genomic regions, both hyper- and hypomethylated, in tumour-derived samples. These signals, strongly associated with cancer biology, could enable earlier detection and improved characterisation of disease through non-invasive blood testing. The study also points to the potential of this technology to trace the tissue of origin of cfDNA, a key hurdle in liquid biopsy diagnostics. Dr. Robert Neely, Chief Scientific Officer and co-founder of Tagomics, called the publication a “major milestone” for the company: “We show that our platform enables the sensitive detection of unmethylated genomic regions, which are key markers for DNA tissue of origin,” Neely said. “This paper highlights the insights our approach can deliver into the biology of cell-free DNA, and we’re excited about the opportunities it opens up for cancer diagnostics and patient safety monitoring.” With Activace positioned as a scalable solution for biomarker discovery, Tagomics is aiming to push the boundaries of epigenomic research and accelerate the development of liquid biopsy diagnostics for oncology and beyond. Author BioFocus Newsroom Previous Next

The pharmaceutical giant invests in AviadoBio, giving them an option to acquire the London-based biotech's gene therapy aimed at frontotemporal dementia (FTD). < Back Astellas Pharma Invests $50 million in AviadoBio to Bolster Gene Therapy Offering The pharmaceutical giant invests in AviadoBio, giving them an option to acquire the London-based biotech's gene therapy aimed at frontotemporal dementia (FTD). Astellas Pharma has invested $50 million in AviadoBio, giving the pharmaceutical giant an option to acquire the London-based biotech's gene therapy aimed at frontotemporal dementia (FTD). AviadoBio's innovative approach targets a specific protein deficiency associated with the disease, aiming to deliver long-term therapeutic benefits. Specifically, Astellas is interested in AviadoBio's single dose AAV-based gene therapy, AVB-101, currently in phase 1/2 clinical trials. The partnership also includes potential milestone payments if development progresses successfully, showcasing Astellas' commitment to gene therapy as a major focus for treating neurodegenerative diseases. The deal is part of Astellas’ broader strategy to expand its gene therapy pipeline. AviadoBio's CEO, Lisa Deschamps , commented “As we complete dosing of the first cohort of patients in our phase 1/2 ASPIRE-FTD trial of AVB-101, we are excited about the potential of this collaboration to help address the unmet need that exists today in frontotemporal dementia". Of the $50 million total sum being paid by Astellas Pharma, $30 million constitutes an upfront payment while the remaining $20 million comes in the form of an equity investment. In return, Astellas will be granted the option of being the therapy's exclusive global license holder. Astellas Pharma is a Japanese multinational pharmaceutical company focused on developing innovative therapies in various areas, including oncology, immunology, and gene therapy. It is heavily investing in cutting-edge medical technologies, such as gene therapies, to address complex diseases. AviadoBio , a London-based biotech, specializes in gene therapies for neurodegenerative diseases. The company is working on treatments that target specific genetic causes of these conditions, with a particular focus on frontotemporal dementia (FTD), aiming to deliver durable therapeutic benefits. Author BioFocus Newsroom Previous Next