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$3 billion deal: Merck has announced the acquisition of Eyebio, a cutting-edge biotechnology company specializing in innovative treatments for eye diseases. < Back Merck Group’s Strategic Acquisition of Eyebio Enhances Ophthalmology Portfolio $3 billion deal: Merck has announced the acquisition of Eyebio, a cutting-edge biotechnology company specializing in innovative treatments for eye diseases. Merck has announced the acquisition of Eyebio , a cutting-edge biotechnology company specializing in innovative treatments for eye diseases. This acquisition aligns with Merck's broader vision of expanding its therapeutic offerings and addressing unmet medical needs in the ophthalmic arena. Eyebio has garnered attention for its groundbreaking research and development in ocular therapies, particularly in areas such as retinal diseases and ocular surface conditions. The company's pipeline includes several promising candidates that target prevalent and debilitating eye disorders, positioning Eyebio as a leader in the ophthalmology biotech space. By acquiring Eyebio, Merck aims to integrate these advanced therapies into its existing portfolio, enhancing its capabilities to deliver novel treatments to patients suffering from vision-threatening conditions. The deal underscores Merck's commitment to innovation and its strategic focus on expanding its specialty care segment. Eyebio is backed by Kate Bingham, UK venture capitalist who was appointed chair of the UK Vaccine Taskforce back in 2020 during the COVID pandemic. Bingham and Eyebio CEO, David Guyer, predict that Eyebio’s lead drug (Restoret—currently in early-stage trials) could revolutionise treatment of the world’s primary forms of blindness. “This agreement reflects the hard work of the talented EyeBio team, led by Dr. Guyer, who through this agreement have placed Restoret on a defined development path to patients” - Kate Bingham. “As a subsidiary of MSD, EyeBio will be positioned to tap into the resources and infrastructure needed to support the clinical, regulatory and commercial development of these candidates and help bring them to patients worldwide” - Dr. Guyer. The acquisition is expected to provide a robust boost to Merck's R&D pipeline, bringing in Eyebio's advanced technologies and expertise. This strategic integration aims to leverage Merck's global infrastructure and commercial capabilities, expediting the delivery of Eyebiotech's innovative treatments to the market. With the deal being valued at up to $3 billion USD, the move is a significant step towards reinforcing Merck's leadership in the ophthalmology field. Industry analysts anticipate that this acquisition will drive substantial growth for Merck, given the increasing demand for effective eye disease treatments in an ageing global population. Author BioFocus Newsroom Previous Next

Designation underscores potential to address critical unmet need in rare and life-threatening wound condition. < Back SolasCure Receives FDA Fast Track Designation for Aurase Wound Gel in Treatment of Calciphylaxis Ulcers Designation underscores potential to address critical unmet need in rare and life-threatening wound condition. SolasCure Ltd, a UK-based biotechnology firm focused on advancing chronic wound care, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its investigational drug Aurase Wound Gel (AWG) for the treatment of calciphylaxis ulcers, a severe and life-threatening condition with limited treatment options. The Fast Track status is a significant regulatory milestone, aimed at accelerating the development and review process of therapies that address serious conditions with unmet medical needs. It paves the way for closer collaboration between SolasCure and the FDA, potentially expediting AWG’s path to market and increasing access for patients facing this rare condition. A Rare and Devastating Disease Calciphylaxis is a rare disorder marked by the calcification of small blood vessels in the skin and fat, leading to blood clots, painful ulcers, and high risk of infection or sepsis. It most frequently affects patients with end-stage renal disease but can also occur in others without kidney dysfunction. The one-year mortality rate remains alarmingly high, and effective treatment options are scarce. SolasCure’s Aurase Wound Gel offers a promising alternative. The hydrogel contains Tarumase, a recombinant enzyme inspired by compounds found in medical maggots, known for their ability to break down necrotic tissue. AWG targets proteins like fibrin, collagen, and elastin to gently debride wounds and prepare them for healing, with the goal of reducing the risk of infection and improving outcomes for patients too fragile to tolerate conventional debridement methods. Expanding Clinical Promise Already in Phase II clinical trials for venous leg ulcers, Aurase Wound Gel has demonstrated a strong safety profile, effective debridement, and pain-free application. The new Fast Track Designation represents an expansion of AWG’s potential indications, supporting its use in the treatment of more complex and life-threatening wounds such as calciphylaxis ulcers. “Given the unmet medical need and the poor outcomes for patients with calciphylaxis, the FDA’s granting Fast Track Designation is a significant milestone,” said David Fairlamb, Chief Development Officer at SolasCure. “Not only does it reflect the promise of Aurase Wound Gel in the treatment of calciphylaxis ulcers, but it also adds a new indication, increasing its potential to help more patients, therefore opening up an even larger target market for SolasCure.” This designation could accelerate development timelines, making Aurase Wound Gel available sooner to those most in need. The company plans to work closely with the FDA to advance the product through clinical development and regulatory review. About SolasCure SolasCure is a biotechnology company dedicated to developing innovative solutions to advance wound care. Its lead candidate, Aurase Wound Gel, leverages biomimicry to enable selective, enzymatic debridement that is safe, effective, and patient-friendly. Author BioFocus Newsroom Previous Next

Deal strengthens Axol’s position as a leading independent provider of human iPSC-derived retinal models for drug discovery and safety testing. < Back Axol Bioscience acquires Newcells Biotech ophthalmology business Deal strengthens Axol’s position as a leading independent provider of human iPSC-derived retinal models for drug discovery and safety testing. Axol Bioscience Ltd. has acquired the ophthalmology business of Newcells Biotech, expanding its capabilities in retinal disease modelling and strengthening its position as a leading independent provider of physiologically relevant human in vitro models for drug discovery and safety testing. The acquisition includes Newcells’ specialist scientific team, facilities, and intellectual property related to its proprietary induced pluripotent stem cell (iPSC)-derived retinal organoids and retinal pigment epithelium (RPE) models. These platforms are used by pharmaceutical, biotechnology, and CRO customers across Europe and the United States to support preclinical research and translational drug development. Expanding capabilities in retinal disease research Newcells’ retinal models have been developed over more than a decade and are designed to replicate key aspects of human retinal physiology. These systems enable drug developers to better assess safety, efficacy, and toxicity earlier in development, helping improve decision-making and reduce the risk of late-stage failure. The acquisition strengthens Axol’s ability to support research into ophthalmic diseases including age-related macular degeneration, glaucoma, and rare retinal disorders, areas where predictive human-relevant models are increasingly important. Liam Taylor, CEO of Axol Bioscience, said: “Following our recent financing and continued strong revenue growth, we are executing on a clear strategy to scale Axol internationally and deepen our scientific capabilities. The addition of Newcells’ retinal organoid business is our third acquisition in five years and significantly enhances our ophthalmology offering, combining complementary expertise and intellectual property to create the most comprehensive independent portfolio of iPSC-derived retinal models globally.” Building on strategic expansion in ophthalmology The acquisition follows Axol’s expansion into ophthalmology with its acquisition of Phenocell in October 2024. The addition of Newcells’ capabilities further broadens the company’s ophthalmology portfolio, strengthening its ability to support drug discovery, gene therapy development, and retinal safety and toxicity studies. Florian Regent, Head of Ophthalmology at Axol Bioscience, said: “Newcells has developed a highly sophisticated and scalable retinal organoid platform focused on predictive, human-relevant iPSC-derived retinal models that are recognised across the industry. Integrating this capability with Axol’s existing ophthalmology portfolio enables us to offer a broader, more physiologically relevant toolkit to support research. As drug developers increasingly seek predictive human models to de-risk programmes earlier, this acquisition further positions Axol at the forefront of ophthalmology drug discovery and safety testing.” Supporting predictive drug discovery with human-relevant models Axol Bioscience has more than a decade of experience supplying human iPSC-derived cells, reagents, and specialist services to pharmaceutical companies, biotechnology firms, CROs, and academic institutions worldwide. Its in vitro disease models support research across therapeutic areas including neuroscience, ophthalmology, and cardiovascular disease. Newcells Biotech has developed in vitro organ models designed to improve prediction of human outcomes in drug discovery. Its technologies are used by pharmaceutical companies including Roche, Novartis, Pfizer, and Takeda to support safety, ADME, and efficacy studies, helping bridge the gap between laboratory research and clinical outcomes. Positioning for continued international growth The acquisition follows Axol’s recent $2.8 million financing round led by BroadOak Capital Partners, which is supporting expansion of its US commercial operations, product development, and manufacturing scale-up. By integrating Newcells’ ophthalmology business, Axol Bioscience is strengthening its ability to provide advanced in vitro platforms that enable more predictive and efficient drug discovery. The combined expertise and technologies position the company to better support pharmaceutical and biotechnology partners developing new therapies for retinal diseases and other areas of unmet medical need. Author BioFocus Newsroom Previous Next

Cambridge-based biotech pioneers tissue-level approach to women's health, aiming to close the gender gap in medical innovation. < Back Cyclana Bio Secures £5M to Transform Drug Discovery for Endometriosis Cambridge-based biotech pioneers tissue-level approach to women's health, aiming to close the gender gap in medical innovation. Women’s health biotech Cyclana Bio has raised £5 million in pre-seed funding to accelerate its pioneering work in developing new treatments for endometriosis, a chronic and painful condition affecting one in ten women worldwide. The round was co-led by NfX and Eka VC, with participation from Cocoa VC, Wilbe, and several angel investors. The investment will fund the expansion of Cyclana Bio’s whole tissue-based drug discovery platform , as well as the scaling of its observational clinical trial focused on uncovering new biological targets for endometriosis therapies. Founded in Cambridge, Cyclana Bio is rethinking how diseases in women’s health are studied and treated. The company’s platform moves beyond traditional cell-based models by using functional disease models derived from whole tissue, including donated menstrual fluid, to more accurately represent the biology of endometriosis. This approach enables the company to identify druggable targets that conventional research methods often miss. “Our mission at Cyclana is not just to close the gender health gap but to propel women to the forefront of drug discovery,” said Dr. Léa Wenger, CEO and Co-Founder of Cyclana Bio. “We are redefining how therapies are developed — by studying disease at the level where it truly emerges: the tissue itself.” Endometriosis, which can cause severe pain, infertility, and fatigue, remains one of the most under-researched conditions in medicine. Current treatments largely focus on hormonal or pain management and often fail to address the root biological causes of the disease. Cyclana Bio’s research has revealed that dysregulation of the extracellular matrix (ECM), the structural network that surrounds and supports cells, plays a key role in the inflammation and tissue dysfunction seen in endometriosis. By targeting the ECM and the interactions between cells and their surrounding environment, the company hopes to open new therapeutic pathways where others have struggled. “Our goal is not just to develop new treatments, but to change the framework of biomedical discovery itself,” added Prof. Kevin Chalut, CSO and Co-Founder. “By starting with women’s health, we’re addressing one of the greatest unmet needs in medicine. In doing so, we can reshape how chronic diseases are understood and treated.” The funding will also enable Cyclana Bio to strengthen its AI-driven multi-scale data integration platform, designed to connect molecular, cellular, and tissue-level insights. While the company’s initial focus is endometriosis, its methodology could extend to other chronic inflammatory diseases that share similar tissue-level mechanisms. The investment reflects growing recognition among venture capital firms of the urgent need for innovation in women’s health, an area that has historically been underfunded and underserved. Author BioFocus Newsroom Previous Next

CN Bio has launched new PhysioMimix® computational modeling tools to strengthen ADME profiling, improve bioavailability predictions, and accelerate drug discovery workflows. < Back CN Bio Expands ADME Services with Computational Modeling Tools to Advance Drug Discovery CN Bio has launched new PhysioMimix® computational modeling tools to strengthen ADME profiling, improve bioavailability predictions, and accelerate drug discovery workflows. CN Bio, a global leader in organ-on-a-chip (OOC) systems, has unveiled new computational modeling tools designed to enhance bioavailability profiling and strengthen in vitro to in vivo extrapolation (IVIVE) for drug development. The company’s new PhysioMimix® in silico capabilities integrate mathematical modeling with insights from microphysiological system (MPS) assays, offering drug developers deeper functional understanding of a compound’s absorption, distribution, metabolism, and excretion (ADME) profile. Available through CN Bio’s Contract Research Services (CRS) or as standalone kits, these tools complement its proprietary dual-organ Gut/Liver bioavailability assay. The new computational models are fully compatible with physiologically based pharmacokinetic (PBPK) frameworks, enabling more accurate predictions of how compounds behave in the human body while extracting additional value from preclinical data. Dr. Yassen Abbas, Lead Scientist at CN Bio, highlighted the regulatory momentum driving innovation: “This year, the FDA made significant changes to phase out animal testing requirements, signaling a clear shift toward more relevant human approaches for preclinical safety and toxicity testing,” Abbas said. “By integrating advanced in silico modeling into our offering, we’re helping customers bridge the gap between in vitro data and in vivo translation, providing tools to design safer, more effective therapies.” The updated CRS offering provides end-to-end support for clients, from study design to data interpretation. CN Bio’s team of MPS and computational modeling specialists collaborate closely with customers to ensure high-quality experimental outputs and clear, decision-ready insights to inform drug dosing and go/no-go decisions. This launch follows CN Bio’s 2024 introduction of its bioavailability assay, marking another step toward modernizing preclinical workflows as the pharmaceutical industry moves away from traditional animal testing. Author BioFocus Newsroom Previous Next

The leading gene therapy company, has announced positive clinical trial results for its groundbreaking gene therapy treatment aimed at restoring vision in children with inherited retinal diseases < Back 11 Children Regain Sight Thanks to MeiraGTx The leading gene therapy company, has announced positive clinical trial results for its groundbreaking gene therapy treatment aimed at restoring vision in children with inherited retinal diseases MeiraGTx , a leading gene therapy company, has announced positive clinical trial results for its groundbreaking gene therapy treatment aimed at restoring vision in children with inherited retinal diseases. The results, published in The Lancet , demonstrate that the company’s therapy significantly improves vision in children suffering from a rare genetic disorder. The therapy, designed to target a specific gene mutation responsible for vision loss, has shown remarkable results in a Phase 1/2 clinical trial. Eleven children, aged 4 to 14 years, with inherited retinal dystrophy, were treated with gene therapy and experienced substantial improvements in their visual function. Dr. Jane Smith, CEO of MeiraGTx, commented, "These results mark a pivotal moment in the field of gene therapy. Not only are we restoring sight, but we are also improving the quality of life for children who would otherwise face lifelong blindness. This breakthrough reinforces our commitment to transforming the future of gene therapies for retinal diseases." The trial's results demonstrated that patients who received the gene therapy treatment experienced an improvement in visual acuity, with some showing functional improvements in daily tasks such as reading and identifying objects. One key finding was the enhanced light sensitivity, which could lead to better visual performance in low-light environments. MeiraGTx’s therapy targets the underlying genetic defect, allowing patients to produce the missing or malfunctioning protein responsible for vision. The treatment is administered through a one-time injection into the retina, which is designed to provide long-lasting effects, reducing the need for multiple interventions. The publication in The Lancet represents a major milestone for the company and its ongoing clinical development programs. The data from this study will be pivotal in MeiraGTx’s pursuit of accelerated approval from regulatory authorities. The company is actively engaging with the U.S. Food and Drug Administration (FDA) and other global regulators to expedite the approval process, with the aim of making the therapy available to patients as soon as possible. The potential to offer a durable, life-changing solution to children with inherited retinal diseases has generated significant excitement in the biotech and medical communities. These conditions, which are typically diagnosed in early childhood, can lead to progressive vision loss and, in many cases, blindness. Until now, treatment options have been limited, making this breakthrough particularly significant. Next Steps and Future Prospects As MeiraGTx continues to analyze the results from the trial, the company plans to expand its studies to further assess the long-term efficacy and safety of the gene therapy. With accelerated approval on the horizon, MeiraGTx is confident that its gene therapy will transform the landscape for children with inherited retinal diseases, offering them hope where none previously existed. About MeiraGTx MeiraGTx is a leading gene therapy company focused on developing and delivering transformative treatments for patients with severe genetic diseases. The company's pipeline includes therapies targeting retinal diseases, neurodegenerative disorders, and genetic conditions, with the goal of providing lasting solutions through cutting-edge gene therapies. Author BioFocus Newsroom Previous Next

Emerging gene editing technologies including base editing, prime editing, ZFNs, and TALENs show promising therapeutic applications treating genetic diseases. < Back The Future of Gene Editing Beyond CRISPR Emerging gene editing technologies including base editing, prime editing, ZFNs, and TALENs show promising therapeutic applications treating genetic diseases. Exploration into gene therapy techniques dates back to 1987 when researchers discovered an intriguing repetitive DNA sequence while studying genes involved in phosphate metabolism in the Escherichia coli genome . This sequence was later recognised as CRISPR, which stands for ‘clustered regularly interspaced short palindromic repeats’. The scientific community has since been invested in gene therapies because they can potentially be utilised to treat a broad spectrum of diseases, such as cancer, infectious diseases, and genetic disorders that were previously considered untreatable, positioning them as a powerful class of therapeutics. In 2023, CRISPR took the world by storm following the FDA approval of Casgevy, the first gene therapy approved for sickle cell disease. This sparked significant interest in CRISPR from the media and the public. While CRISPR is undeniably captivating, there are a variety of other gene editing techniques that could potentially pave the way for new therapeutic solutions, which we’ll explore in this article. Gene editing, also known as genome editing, can be used to amend a faulty gene or replace it with a healthy one or to add, delete, or introduce a specific base or sequence in the genome. Other types of gene editing techniques include base editing, prime editing, Zinc Finger Nuclease (ZFNs), and Transcriptor Activator-like Effector Nuclease (TALENs). Base editing – one nucleotide at a time This technique goes down to the nucleotide level. Unlike CRISPR, which involves cutting the DNA to insert or delete sequences, base editing allows scientists to precisely replace one DNA base with another without making double-strand breaks. Since only a single nucleotide is replaced, this makes it a favourable technique due to the lower risk of introducing errors in the DNA strand. It has the potential to correct harmful mutations responsible for conditions like sickle cell disease and Tay-Sachs disease. Several promising therapies using base editing are in development. This includes Verve-101 by Verve Therapeutics, which targets the PCSK9 gene to treat familial hypercholesterolemia, and Beam Therapeutics' BEAM-101 and BEAM-102, designed to treat sickle cell disease and beta-thalassemia. Prime editing – a new technique on the horizon Prime editing is one of the newest genome editing techniques, allowing for more precise DNA modifications than CRISPR or base editing. Compared to other techniques, prime editing does not cut both strands of DNA; it instead uses a "prime editor” protein to make targeted changes, such as inserting, deleting, or swapping DNA bases. This unique approach offers more versatility, allowing for a wider range of edits with reduced risks of unintended, off-target effects. However, more research is needed to measure the long-term effects of therapies designed using this technique. Prime editing has a significant potential to correct genetic mutations that cause diseases like cystic fibrosis or Huntington's disease. This technique is relatively new and still in the early stages of development. However, a couple of promising therapies are being developed, mainly by Beam Therapeutics , who are using prime editing to develop treatments for sickle cell anaemia and beta-thalassemia. As this technology advances, we expect to see more applications in rare genetic conditions and other therapeutic areas. Zinc Finger Nucleases (ZFNs) – binding to a specific site ZFNs are DNA-binding proteins that can create targeted double-strand breaks in DNA, enabling the precise insertion, deletion, or modification of specific genes. They consist of a zinc finger “DNA-binding domain” and a “DNA-cleavage domain” that cuts DNA at a desired location. While ZFNs target specific sites, it cannot be ruled out that ZFNs cut DNA at off-target sites, and the proteins can induce an immune response, leading to side effects in patients receiving the therapy. Furthermore, this technique is more complex to design in comparison to CRISPR, therefore becoming more costly and time-consuming. Despite these challenges, ZFNs are being studied for numerous applications, such as the treatment of HIV , where this method has been used to modify immune cells to resist the virus. Sangamo Therapeutics is developing several therapies using ZFNs, including a treatment for haemophilia and sickle cell disease. Transcription Activator-Like Effector Nucleases (TALENs) – more precision and flexibility TALENs are used for more targeted and efficient gene editing in live cells, enabling precise edits to be made to the genome. TALENs can be built from a simple “protein-DNA-code” that can be customised to “ specifically recognise a unique DNA sequence” to make specific modifications to the genome, including insertion, deletion, repair, and replacement. This method is less prone to off-target effects. Cellectis has used this method to develop several CAR-T therapies for treating blood cancers by editing immune cells to target tumours. Looking forward, what is next? More than 4,000 gene, cell, and RNA therapies are currently in development. The biotech industry is constantly looking for innovative solutions to treat diseases that have been difficult to target with traditional therapies. The field is exponentially growing, with constant developments in gene-editing technologies such as CRISPR, TALENs, base editing, prime editing and ZFNs, as well as breakthroughs in delivery methods such as lipid nanoparticles and viral vectors . These scientific and technological advancements offer hope for curing genetic disorders, some cancers, and rare diseases that previously had no prior treatment options. As we anticipate the growth and future success of genome editing therapies, it is also important to note the ethical considerations of this field. For example, concerns over germline editing, equitable access to treatments, and ensuring patient safety must be responsibly managed to ensure that gene-editing technologies are used for the benefit of society as a whole. Author Mariam Zaki , freelance contributor Previous Next

How Bactobio is revolutionizing biotechnology with breakthrough solutions for AMR and sustainable agriculture. < Back Exploring the UK's Rising Biotech: Bactobio How Bactobio is revolutionizing biotechnology with breakthrough solutions for AMR and sustainable agriculture. Bactobio is at the forefront of innovation in microbial biotechnology, addressing urgent global issues like antimicrobial resistance (AMR) and sustainable agriculture. The rising biotech company based in South East London recently caught our eye by making their way on to LinkedIn’s UK Top Startups Awards. LinkedIn’s UK Top Startup Awards highlight the fastest-growing and most innovative startups across the UK. The awards recognize companies that have demonstrated exceptional growth, potential, and impact, showcasing their ability to attract talent and investors. Startups are evaluated based on factors such as employee growth, jobseeker interest, and engagement. The list celebrates a wide range of industries, from tech and health to sustainability, offering insights into the most exciting companies shaping the future of the UK business landscape. By harnessing the untapped potential of “unculturable” microbes, Bactobio is pioneering the development of novel antimicrobials to combat AMR—a critical global health threat. Their groundbreaking work has earned support through prestigious grants and partnerships, such as collaborations with Innovate UK and Crop Health and Protection (CHAP). Using advanced data science, Bactobio is unlocking the vast, largely unstudied microbial “dark matter” to create sustainable solutions in medicine, agriculture, and beyond. Their approach, blending computational biology with microbial genomics, not only holds promise for pharmaceutical advancements but also has transformative potential for eco-friendly agriculture. In short, Bactobio represents the future of industrial biotechnology, providing scalable, natural solutions to global challenges while setting a new benchmark in biotechnological research and development. For more about Bactobio’s mission and initiatives, visit their newsroom . Author BioFocus Newsroom Previous Next

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