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Pharma Belite Bio Announces Landmark Phase 3 Success for Stargardt Disease Tinlarebant becomes the first therapy to show clinical benefit in a pivotal global trial, reducing retinal lesion growth by 36% and paving the way for a planned FDA filing in 2026. Read More Study Shows TREMFYA® Slows Psoriatic Arthritis Progression at 48 Weeks Long-term APEX results reinforce TREMFYA® as the only IL-23 inhibitor proven to slow structural joint damage in psoriatic arthritis. Read More World-First Preventative Lung Cancer Vaccine Enters Clinical Trial A groundbreaking clinical trial set to begin in summer 2026 will test LungVax, the world’s first vaccine designed to prevent lung cancer in people at high risk, a major milestone in cancer prevention research. Read More Optibrium Launches StarDrop 8, Bringing Real-Time Collaboration to Molecular Design StarDrop 8 integrates real-time collaboration into compound design and optimisation, streamlining decision-making and improving team productivity. Read More Astellas Reports Long-Term Benefits and Strong Safety Profile for IZERVAY™ in Geographic Atrophy GATHER2 extension data show sustained efficacy and no new safety concerns after 3.5 years of treatment. Read More Novo Nordisk Pulls Plug on Cell Therapy in $1.3B Restructuring Push The pharma giant will close its cell therapy unit, cutting 250 jobs and ending work on Type 1 diabetes and Parkinson's programs, as new CEO shifts focus to obesity, diabetes, and liver disease treatments. Read More Load more

New validated protocols on SPT’s firefly platform aim to boost throughput and reproducibility for sequencing labs. < Back SPT Labtech and Twist Bioscience Team-up to Automate NGS Library Prep Workflows New validated protocols on SPT’s firefly platform aim to boost throughput and reproducibility for sequencing labs. SPT Labtech has rolled out new automated workflows for Twist Bioscience’s next-generation sequencing (NGS) library preparation kits on its firefly liquid handling platform, aiming to boost throughput and consistency for genomics laboratories. Developed in collaboration with Twist , the validated methods are accessible via the Firefly Cloud and support Twist’s FlexPrep UHT Library Preparation Kit as well as the company’s Enzymatic Fragmentation Kit 2.0. According to SPT Labtech , the workflows address growing demand from research and core facilities for standardised, hands-free NGS protocols as sequencing volumes continue to scale. The automated FlexPrep workflow integrates on-deck fragmentation and ligation, alongside an auto-normalising protocol that consolidates indexing and final clean-up into four columns of a 96-well plate. The company says this supports higher throughput without requiring access to 384-well thermal cyclers, while also improving reproducibility across samples. In parallel, the integration of Twist’s Enzymatic Fragmentation Kit 2.0 with firefly is intended to offer an efficient sequencing pipeline with reduced error rates and more accurate readouts. SPT Labtech expects ongoing combinations of Twist kits with firefly to expand its catalogue of validated, ready-to-deploy automated workflows. Rob Walton, Chief Executive Officer at SPT Labtech, said: “We are delighted to be working with Twist Bioscience to bring these high-demand methods to our customers. By making these Twist workflows available on Firefly, we are giving users a straightforward way to adopt automation without adding complexity to their existing processes, while access through the Firefly cloud allows laboratories to implement these methods quickly and scale as their needs evolve.” Emily Leproust, Chief Executive Officer and Co-founder of Twist Bioscience, added: “Our customers are increasingly seeking validated, hands-free solutions to improve the efficiency and reproducibility of their NGS workflows. By collaborating with SPT Labtech, we are providing a simple path to automation for our kits, beginning with our new FlexPrep kit, which offers significant benefits in terms of speed and scalability." The workflows are available immediately to Firefly users via the Firefly Cloud. The collaboration reflects a broader trend towards workflow simplification and automation in sequencing, driven by mounting sample volumes and tightening turnaround expectations across research and clinical settings. As libraries become faster and more consistent to prepare, bottlenecks increasingly shift upstream or downstream of sequencing itself. By validating Twist’s kits on firefly and making the methods instantly accessible through the cloud, SPT Labtech is positioning automation as a turnkey choice rather than a complex engineering project. Author BioFocus Newsroom Previous Next

Partnership integrates scalable iPSC workflows with AI-driven 3D organoids to improve drug safety prediction and translational decision-making in rare diseases. < Back iXCells and Rosebud Team Up on Human Organoid Models Partnership integrates scalable iPSC workflows with AI-driven 3D organoids to improve drug safety prediction and translational decision-making in rare diseases. As the life sciences industry intensifies its focus on translational relevance, human-based model systems are increasingly seen as essential for improving decision-making across drug discovery and development. In this context, iXCells Biotechnologies and Rosebud Biosciences have announced a strategic partnership to integrate advanced 3D human organoid technologies into scalable induced pluripotent stem cell (iPSC) platforms. The collaboration brings together iXCells’ established iPSCore platform with Rosebud’s expertise in automated, AI-powered organoid generation and characterization. By combining scalable iPSC differentiation with complex three-dimensional human tissue models, the partners aim to provide drug developers with more predictive systems for assessing tissue-specific toxicity, therapeutic response, and disease mechanisms, areas where traditional preclinical models often lack fidelity. Tackling a key translational challenge Rare disease drug development presents distinct challenges, including limited patient populations, biological heterogeneity, and high attrition rates driven by late-stage safety or efficacy failures. As a result, there is growing demand for patient-relevant human models that can help de-risk programmes earlier and guide translational decision-making. Organoids have emerged as a powerful addition to the preclinical toolbox, offering the ability to recapitulate aspects of human tissue architecture and function. However, issues around scalability, reproducibility, and integration into drug development workflows have limited their broader adoption. Through this partnership, Rosebud’s organoid platforms will be embedded within iXCells’ existing iPSC workflows, enabling access to standardized 3D human systems that can be deployed for screening, engineering, multi-omics profiling, and mechanism-of-action studies within a unified platform. Expanding the iPSCore platform For iXCells, the collaboration marks a strategic expansion of its modelling capabilities as the company scales its platform to support more complex and translationally focused programmes. “Predicting human-specific safety and response earlier in development can have a significant impact on timelines and patient outcomes, particularly in rare diseases,” said Steve Smith, Chief Executive Officer of iXCells Biotechnologies. “Integrating Rosebud’s depth in organoid science enhances our end-to-end platform and allows us to deliver models that reflect human biology with greater depth and accuracy.” The integrated offering is designed to deliver the consistency and scalability required by pharmaceutical and biotechnology partners, supporting drug safety assessment, tissue-specific response evaluation, and deeper interrogation of disease biology. Scaling access to organoid technologies For Rosebud Biosciences, the partnership provides a route to broaden access to its industrialised organoid systems. The company has focused on building automation- and AI-first platforms capable of generating large volumes of reproducible, clinically meaningful human tissue data. “Our partnership with iXCells enables us to make advanced organoid systems more accessible to researchers who need models that better represent human biology,” said Kitch Wilson, CEO and Co-Founder of Rosebud Biosciences. “By combining complementary strengths, we can support deeper exploration of disease mechanisms and help drive the next generation of therapeutic discovery.” Rosebud’s platform is designed for high-throughput applications, with the capacity to generate tens of thousands of organoids simultaneously, supporting safety and efficacy testing across multiple human organ systems. Supporting the shift toward human-relevant models The partnership reflects a broader industry shift toward predictive, patient-relevant human models aligned with regulatory and scientific momentum around New Approach Methodologies (NAMs). As developers seek to reduce reliance on animal models while improving translational confidence, integrated platforms that combine iPSCs, organoids, automation, and AI are expected to play an increasingly central role. By uniting scalable stem cell technologies with complex 3D human biology, iXCells and Rosebud aim to help close the gap between in vitro models and clinical outcomes, particularly in rare disease programmes where early insight can be decisive. Author BioFocus Newsroom Previous Next

< Back A Simple Pad Could Transform Cervical Cancer Screening Population-based study shows menstrual blood testing matches clinic-based HPV detection, with far greater convenience. Cervical cancer screening saves lives, but getting screened requires something many women find understandably difficult: a clinic visit, a speculum exam, and a trained healthcare provider. Now, recent research from China highlights that there might be another way, one that requires nothing more than a sanitary pad and a post box. This is of particular importance in UK, where there are high rates of non-attendance at booked screening appointments, driven in large part by anxiety about the procedure itself. Speaking with a friend of BioFocus, who had recently attended her cervical screening, she described the concerning insight her NHS nurse shared: the low attendance rate, which consumes healthcare resources, is prompting discussions about whether the free screening programme can remain sustainable. The nurse went on to say that there is a genuine risk that cost pressures could push these vital services toward a pay-per-use model, a change that might improve attendance among those who book, but would almost certainly discourage many women from seeking screening at all. For a service that should be free, this would be a travesty. In a study published in The BMJ , researchers led by Zheng Hu at Wuhan University tested whether menstrual blood could be used to detect human papillomavirus (HPV), the virus responsible for nearly all cervical cancers. The results were impressive: a small absorbent strip worn during menstruation detected precancerous cervical lesions just as accurately as samples collected by clinicians during pelvic examinations. The study enrolled over 3,000 women aged 20-54 across urban and rural communities in Hubei Province. Each participant provided three samples: menstrual blood collected at home using a prototype "minipad," plus the standard clinic-based cervical sample and Pap smear. Women who tested positive on any method were referred for biopsy to check for cervical abnormalities. The minipad performed remarkably well. For detecting high-grade precancerous lesions (CIN2+), it showed 94.7% sensitivity compared to 92.1% for clinician-collected samples, statistically identical. The negative predictive value was 99.9% for both methods, meaning a negative result was equally reassuring regardless of collection method. Both approaches required similar numbers of follow-up procedures per diagnosis detected (about 10 colposcopies per case of CIN2+). The minipad did show slightly lower specificity (89.1% vs 90.0%), meaning it flagged a few more women for follow-up who ultimately didn't have disease. But this modest difference seems a reasonable trade-off for the potential benefits: privacy, convenience, and dramatically expanded access to screening. What makes menstrual blood work? As blood flows through the cervix and vaginal canal during menstruation, it picks up shed cells from these tissues, including any HPV-infected cells. The researchers designed their sampling strip to adhere to regular sanitary pads, allowing women to use their preferred menstrual products while ensuring standardised collection. When the strip became sufficiently saturated, participants placed it in preservation solution and either mailed it to the lab or handed it to community health workers. The concordance between methods was impressive. When researchers compared specific HPV genotypes detected, the two collection methods agreed 97.7% of the time. Among the most common high-risk types detected were HPV52, HPV16, and HPV58, the same pattern seen with both collection methods. Perhaps most tellingly, 92% of participants in an earlier pilot study preferred self-collection over clinic visits. In a culture where menstruation has traditionally been considered private or even taboo, the acceptance rate suggests the practical advantages, convenience, privacy, and lack of discomfort, can overcome cultural reservations when the health benefits are clear. This matters because cervical cancer remains a major killer, particularly in low- and middle-income countries where 85% of the roughly 661,000 annual cases occur. Screening programmes face persistent challenges such as insufficient healthcare infrastructure in rural areas, cultural barriers around pelvic examinations, lack of trained providers, fear of pain, and the logistical burden of clinic visits. Minipad product and usage Self-collection methods aren't new. Vaginal swabs and cervicovaginal brushes have been studied for years, with relative sensitivities ranging from 77% to 96% compared to clinician sampling. But these still require vaginal insertion, which some women find uncomfortable or culturally unacceptable. In the earlier pilot work for this study, 22.9% of women declined participation specifically because of discomfort with vaginal swabs. Menstrual blood collection sidesteps this entirely. There's no insertion, no manipulation, no medical procedure at all, just a modified sanitary pad worn during a woman's normal menstrual cycle. The researchers even developed a WeChat mini-program called "Early Test" where participants could track their results, ask questions, and access educational resources about HPV and cervical health. The study does have limitations worth noting. Women with negative results on both tests weren't biopsied (which would be unethical given their extremely low risk), so the researchers had to assume these women truly didn't have disease. This could theoretically introduce bias, though previous large studies suggest fewer than 1 in 1,000 such women would have high-grade lesions. There's also an additional biological consideration - because menstrual blood contacts a broader anatomical area than a cervical swab, including the vagina and vulva, it might detect HPV infections from these other sites. This could explain the slightly higher rate of positive results and might mean some women get referred for colposcopy unnecessarily. Future research testing additional biomarkers could help improve specificity. The researchers are careful not to oversell their findings. They're not suggesting menstrual blood testing should immediately replace current screening protocols. But as an alternative for women who can't or won't access clinic-based screening? The evidence is compelling. What happens next matters enormously. The minipad device used in this study is a research prototype, not yet commercially available. Questions about cost-effectiveness, quality control across different laboratories, and integration into existing healthcare systems all need addressing. The researchers acknowledge that implementation research (tracking real-world uptake, retention, and clinical outcomes) will be crucial. But the core finding stands, which is that for detecting the cervical abnormalities that can progress to cancer, menstrual blood seems to work. It's non-invasive, private, convenient, and accurate. In a world where millions of women lack access to screening, that combination could prove genuinely transformative. As the global health community works toward eliminating cervical cancer as a public health problem, innovations like this, ones that meet women where they are rather than demanding they navigate complex healthcare systems, may prove as important as the science itself. Sometimes the biggest breakthroughs aren't about discovering something entirely new, but about finding a simpler way to do what we already know works. Author BioFocus Newsroom Previous Next

New synthetic promoter tech promises sharper targeting for liver gene therapies < Back SynGenSys Launches Synthetic Promoter Library for Liver-Targeted Gene Therapies New synthetic promoter tech promises sharper targeting for liver gene therapies Sheffield-based biotech company SynGenSys has released a new library of synthetic promoters designed to improve precision in liver-directed gene therapies, addressing a persistent challenge in the development of in vivo treatments. The Liver.SET™ library offers researchers a collection of compact, synthetic promoters engineered to drive gene expression specifically in liver tissue while minimising unwanted activity in muscle, a common source of off-target effects in systemically delivered gene therapies. The promoters have been validated in both cell culture and animal models. Addressing manufacturing and delivery constraints One notable feature of the Liver.SET promoters is their low activity in HEK293 cells, the workhorse cell line used to produce adeno-associated virus (AAV) vectors. Premature expression of therapeutic genes during vector manufacturing can reduce yield and quality, so promoters that remain largely silent during production offer a practical advantage. The library's promoters are also designed with compact sequences, which matters when working within the strict packaging limits of AAV vectors - typically around 4.7 kilobases. Every nucleotide counts when fitting a promoter, therapeutic gene, and regulatory elements into such a constrained space. According to SynGenSys, the modular architecture of these promoters allows for rapid customisation, giving developers flexibility to tune expression levels and regulatory behaviour rather than being locked into the fixed characteristics of naturally occurring liver promoters. Building on a platform approach Liver.SET is the second tissue-specific promoter library from SynGenSys, following NK.SET, which targets natural killer cells for cancer immunotherapy applications. The company's proprietary computational platform combines informatics with design algorithms to generate synthetic promoters tailored to specific tissues and therapeutic contexts. The release signals the company's intention to expand into additional tissue targets. SynGenSys has indicated that promoter libraries for muscle, retina, and central nervous system applications are in development, alongside custom design services for bespoke therapeutic programs. Dr. Mike Daniels, Chief Commercial Officer at SynGenSys, commented: "The launch of Liver.SET™ represents another significant milestone for SynGenSys, demonstrating that our platform can deliver synthetic promoter solutions for real gene therapy development needs. We see this as a key enabler for in vivo gene therapies, and a reliable, validated starting point for deeper collaboration with developers seeking to design novel therapeutics with enhanced precision and safety." Why liver specificity is important The liver is a major target for therapeutic transgene expression in gene therapies of inherited diseases such as Haemophilia, Phenylketonuria, and Fabry disease, and in cancer and hepatitis. However, systemic delivery of AAV vectors often results in transduction of multiple tissues, particularly skeletal and cardiac muscle. This can dilute therapeutic effect, raise safety concerns, and complicate dosing strategies. Promoters with strong tissue specificity allow therapeutic genes to be expressed where they're needed while remaining transcriptionally silent elsewhere. This can potentially reduce the vector doses required and improve the therapeutic index, particularly important given ongoing concerns about AAV-related toxicity at high doses. The demonstrated specificity in the Liver.SET library, particularly the active de-targeting of muscle tissue, could prove valuable as developers work to improve the safety profiles of next-generation gene therapies. Whether these synthetic promoters will outperform optimised natural promoters in clinical settings remains to be seen, but the flexibility and customisability they offer represent a meaningful addition to the gene therapy toolkit. Author BioFocus Newsroom Previous Next

The introduction of ISO/IEC 17025-accredited methods for oils and tinctures reflects rising expectations for analytical robustness as cannabinoid formulations diversify and regulatory scrutiny intensifies. < Back Broughton Expands Accredited Cannabinoid Testing The introduction of ISO/IEC 17025-accredited methods for oils and tinctures reflects rising expectations for analytical robustness as cannabinoid formulations diversify and regulatory scrutiny intensifies. An independent partner in regulated life sciences Broughton is an independent UK-based life sciences contract research organisation and scientific consultancy, supporting companies operating in highly regulated markets where confidence in data is critical. From its GMP-compliant, ISO/IEC 17025-accredited laboratories in Lancashire and North Yorkshire, the company provides analytical testing and regulatory support to clients across pharmaceuticals, healthcare, consumer health and emerging life science sectors, working with organisations at every stage of the product lifecycle. Over nearly two decades, Broughton has built its reputation on scientific rigour, regulatory credibility and transparency, combining in-house analytical capability with regulatory and scientific insight. This integrated approach has positioned the company as a trusted partner for organisations navigating complex development pathways and increasing regulatory scrutiny. Expanding accredited cannabinoid analytics Broughton has begun 2026 with the introduction of ISO/IEC 17025-accredited analytical methods for the quantitative determination of cannabinoids in oils and tinctures, marking a significant expansion of its capabilities in one of the life sciences sector’s most technically demanding and closely scrutinised areas. The methods, based on high-performance liquid chromatography with tandem mass spectrometry (HPLC-MS/MS), also apply to selected e-liquids and reflect the growing need for accredited measurement as cannabinoid product formats continue to evolve. Oils and tinctures have diversified rapidly in both concentration and formulation complexity, driven by innovation across medicinal, wellness and adjacent regulated markets. In these oil-based matrices, cannabinoid content can vary depending on raw material source, extraction technique and intended application, making reliable quantification essential for product characterisation, quality assurance and regulatory documentation. Analytical approach and measurement range Broughton’s accredited approach involves dilution of the oil or tincture in solvent, followed by quantitative analysis using calibration curves and internal standard correction to support accuracy. Results can be reported in multiple formats, including milligrams per millilitre, percentage concentration, or milligrams per container, with quantification of Total THC and Total CBD available. This flexibility supports a range of requirements, from formulation development and batch verification through to regulatory submissions. The method also accommodates defined analytical ranges aligned to product strength. For formulations containing up to 20 per cent CBD, quantitation is supported from approximately 2.5 mg/g to 245 mg/g, while higher-strength products above 20 per cent CBD can be measured from around 8 mg/g to beyond 800 mg/g. This enables consistent, accredited measurement across both lower- and higher-dose oils and tinctures. Supporting confidence in formulation and compliance For Development Scientist Rowan Baxendale, the significance lies in the confidence such data provides. The availability of validated, accredited cannabinoid measurement in oil-based products strengthens the evidence base that informs formulation decisions, quality control processes and regulatory compliance, particularly in a market where analytical robustness is under increasing scrutiny. Chief Executive Officer Chris Allen places the development within a broader strategic context. As product formats evolve and regulatory expectations continue to sharpen, having established measurement frameworks in place brings clarity and consistency to how cannabinoids are assessed, supporting both innovation and compliance. Integrated science across the product lifecycle This latest capability builds on Broughton’s long-standing position within the life sciences sector, providing integrated analytical, toxicological and regulatory services to clients worldwide. From early development and analytical strategy through to routine quality control, stability studies and post-market support, the company supports products across their full lifecycle. What has consistently set Broughton apart is the way scientific delivery and regulatory understanding are embedded together. In an environment where regulatory scrutiny is intensifying across pharmaceuticals, medical devices and emerging life science technologies, data must not only be scientifically sound but demonstrably reliable, traceable and generated within systems that can withstand sustained inspection. A sustained record of regulatory compliance That expectation is reflected in Broughton’s compliance record. In 2024, the company successfully completed inspections by both the United Kingdom Accreditation Service and the Medicines and Healthcare products Regulatory Agency, with no major or critical observations. These outcomes extended Broughton’s unbroken record of dual ISO 17025 and GMP compliance to 19 years, placing it among a relatively small cohort of UK laboratories to have maintained such consistency over time. The UKAS audit assessed adherence to ISO 17025, the international benchmark for laboratory competence and data integrity, while the MHRA inspection examined GMP compliance across laboratory operations, quality systems and documentation. Together, these frameworks underpin trust in analytical science and regulatory decision-making, particularly where data supports high-value or high-risk development programmes. At Broughton, inspections are approached as part of an ongoing process rather than isolated events. Associate Director of Quality Emmet Tilley describes a culture grounded in openness, where engagement with inspectors is used to refine systems and strengthen practices. Sustained compliance, he notes, reflects an organisational mindset that values rigour and continuous improvement rather than short-term audit success. Consistency under global scrutiny Broughton’s laboratories are routinely inspected not only by UKAS and the MHRA but also by international authorities including the US Food and Drug Administration, reinforcing confidence in data generated for global submissions. For organisations operating across multiple jurisdictions, this consistency can translate into faster decision-making and greater regulatory assurance. As Chris Allen, CEO at Broughton, notes, when development teams trust the integrity of their data, they are able to move forward with greater speed and confidence. That assurance continues to drive Broughton’s investment in technical expertise, digital infrastructure and accredited capability, including its latest expansion in cannabinoid analytics. “Providing accredited analytical methods for oils and tinctures reflects our ongoing commitment to supporting organisations with reliable data,” added Chris Allen, CEO at Broughton. “As product formats evolve, having validated measurement frameworks in place helps ensure consistency and clarity in how cannabinoids are assessed.” In a sector where innovation is advancing rapidly but regulatory tolerance for uncertainty is diminishing, Broughton’s trajectory illustrates how sustained quality management and scientific rigour can act as enablers rather than constraints. By combining accredited analytical science with deep regulatory insight, the company has positioned itself as a long-term partner in development rather than simply a service provider. Author BioFocus Newsroom Previous Next

Collaboration integrates quantum mechanics-based modelling with cutting-edge experimental platforms to accelerate hit discovery for GPCRs and ion channels. < Back Domainex and Pharmacelera Partner to Advance AI-Driven Drug Discovery for Transmembrane Proteins Collaboration integrates quantum mechanics-based modelling with cutting-edge experimental platforms to accelerate hit discovery for GPCRs and ion channels. Drug discovery service provider Domainex and computational chemistry innovator Pharmacelera have announced a strategic partnership to accelerate early-stage drug discovery against transmembrane proteins, including G protein-coupled receptors (GPCRs) and ion channels, two of the most therapeutically important yet technically challenging classes of drug targets. Despite representing more than 60% of all FDA-approved drug targets, transmembrane proteins remain notoriously difficult to study due to their structural complexity, hydrophobicity, and instability outside lipid bilayers. The collaboration integrates Domainex’s proprietary experimental platforms with Pharmacelera’s advanced AI and quantum mechanics (QM) driven modelling technologies to address these longstanding challenges. Domainex will deploy its Polymer Lipid Particle (PoLiPa) technology to stabilise membrane proteins within polymer nanodiscs, enabling their purification and interrogation in a near-native state. This capability will be coupled with its Direct-to-Biology (D2B) platform for rapid parallel synthesis and biological screening, streamlining the path from chemistry to assay-ready material. Pharmacelera brings its exaScreen® and PharmScreen® platforms, which apply quantum mechanics-based molecular descriptors and AI-driven virtual screening to explore chemical space beyond the reach of traditional docking algorithms. This integration of physics-informed modelling with high-quality biochemical data is expected to generate hit series with improved novelty, diversity, and developability. “This collaboration between Domainex and Pharmacelera combines innovative technologies and represents optimal approaches for drug discovery programmes and is part of our commitment to achieve faster and successful approvals for our customers,” said Dr. Hayley French, CEO of Domainex. “By pairing our cutting-edge AI models with Domainex’s experimental data and technology platforms, we can accelerate the path from concept to candidate,” added Dr. Enric Gibert, CEO of Pharmacelera. “Our joint efforts will make traditionally intractable targets more accessible to rational drug design.” Domainex operates state-of-the-art facilities in Cambridge, UK, and recently expanded to Cambridge, MA, to better serve North American biotech partners. Pharmacelera, headquartered in Barcelona, develops quantum mechanics, AI, and high-performance computing (HPC) powered software solutions for drug discovery and collaborates with leading pharmaceutical and biotech firms across Europe and the US. By combining advanced computational chemistry with cutting-edge experimental biology, this alliance seeks to overcome key bottlenecks in membrane protein drug discovery, enabling faster hit identification and lead optimisation for diseases driven by these complex targets. Author BioFocus Newsroom Previous Next

Scientists are proposing a lunar biobank to preserve the world's endangered species. < Back A Modern Day Noah's Ark? Scientists are proposing a lunar biobank to preserve the world's endangered species. As many of us are aware, the effects of climate change are becoming more and more apparent. From the melting of the polar ice caps to powerful life-threatening hurricanes to blazing hot summers, all have a huge impact on people, wildlife and the environment. Scientists are now gathering plans to send plant and animal cells to the moon , in attempts to safeguard Earth’s biodiversity! Within the moon’s south pole there is a permanently shaded area which typically remains around -196 degrees Celsius, which would allow these endangered cells to be protected long term through a method known as cryopreservation. Usually when cells are being collected, they are stored within biorepositories , essentially a huge library keeping the biospecimens safe. The idea is to have a biorepository within this shadowy region of the moon. This idea brings hope that soon to be extinct creatures can continue to exist through the protection of their cells. What species would go on the moon first? Mary Hagedorn and her colleagues of the Smithsonian National Zoo and Conservation Biology Institute in Washing DC wrote about this lunar Noah’s Ark proposition in the summer of 2024 in a published article for BioScience. She explained how the species to be stored on the moon would be prioritised, starting with endangered species, species with a large impact on habitats, also known as ecosystem engineers, and species with the potential to help humans with space exploration. Why on the moon? So, why does this biorepository need to be on the moon. Ironically, as well as protecting these species from climate change, the biorepository also needs to be protected from the impacts of changing climates on Earth. Something that the Svalbard Global Seed Vault in Norway know all too well about. The Svalbard Global Seed Vault utilises the freezing temperatures of the Arctic Circle to give home to hundreds of thousands of seeds in a “doomsday” style storage facility. The collection is the largest in the world and “contains 13,000 years of agricultural history” claims Brian Lainoff, who is the lead partnership coordinator of the company (Crop Trust) who manage the vault. In 2017, melting permafrost due to climate change resulting in unseasonably warm temperatures led to the vault being flooded putting the seed collection at risk. Thankfully, the global seed vault is still operating today, working hard to collect and preserve the world’s crops. Researchers explain how this event highlights the need for an alternative plan to protecting our biorepositories. What issues are involved with this plan? As exciting as this plan for a lunar Noah’s Ark sounds, there are plenty of questions still left unanswered such as, how would the different levels of radiation and gravity on the moon affect the samples? What about the dust on the moon, could this contaminate the samples and get into storage containers? Not to mention the fluctuating temperatures which still occur despite the region of the moon’s south pole being consistently dark. Moreover, another big challenge would be coordinating and getting stakeholders and nations invested into the project. To conclude, this idea is still in its early stages and would require a lot of hard work and collaboration for it to come to fruition. However, this is still an incredibly cool concept to think about as well as its potential to protect Earth’s precious biodiversity. Author Mia Butterworth-Brooks Previous Next

Cerevance has announced topline results from its Phase 2 ASCEND trial of solengepras, showing a small but non-significant improvement in motor symptoms and potential benefits for non-motor symptoms, with a strong safety profile. < Back Cerevance Reports Phase 2 Results for Solengepras in Early-Stage Parkinson’s Disease Cerevance has announced topline results from its Phase 2 ASCEND trial of solengepras, showing a small but non-significant improvement in motor symptoms and potential benefits for non-motor symptoms, with a strong safety profile. Cerevance , a clinical-stage biopharmaceutical company, has announced topline results from its Phase 2 ASCEND trial evaluating solengepras as a monotherapy for early-stage Parkinson's disease. The findings were presented at the AD/PD™ 2025 International Conference in Vienna. Solengepras is an oral, non-dopaminergic therapy designed to modulate brain circuits responsible for motor and non-motor functions without directly affecting dopaminergic pathways. This approach aims to reduce motor complications such as dyskinesia and minimize "OFF" periods. In the ASCEND trial, solengepras demonstrated a small, non-statistically significant improvement in motor symptoms from baseline to week 12 compared to placebo, as measured by the Movement Disorder Society – Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Parts II and III. While the primary endpoint was not met, trends toward improvement were observed in several patient-reported measures, including: MDS-UPDRS Part I (-1.38, p=0.12) MDS-UPDRS Part II (-0.4, p=0.62) Non-Motor Symptoms Scale (-1.7, p=0.59) Epworth Sleepiness Scale (-0.3, p=0.62) These results suggest potential benefits in functional and non-motor aspects of Parkinson's disease. Solengepras was generally well tolerated, with all patients in the treatment arm completing the 12-week trial and no discontinuations due to treatment-related adverse events. No serious adverse events related to solengepras were reported, and most were mild and transient. Notably, fewer non-motor symptom-related adverse events occurred in the treatment arm compared to placebo. Dr. Aaron L. Ellenbogen, primary investigator of the ASCEND trial, emphasized the need for therapies addressing both motor and non-motor symptoms in Parkinson's disease. He noted that solengepras' novel mechanism offers promise in this area. Cerevance is continuing its pivotal Phase 3 ARISE trial to evaluate solengepras as an adjunctive therapy in Parkinson's disease, with topline data expected in the first half of 2026. Author BioFocus Newsroom Previous Next

OXGENE, a WuXi Advanced Therapies company, hopes to pioneer a new frontier in Gene Therapy GMP manufacturing. < Back OXGENE Seeks to Overcome Challenges in Gene Therapy Production OXGENE, a WuXi Advanced Therapies company, hopes to pioneer a new frontier in Gene Therapy GMP manufacturing. The rapid advancement of gene therapies has broadened the scope of how we can treat challenging diseases. There are now over 2,000 gene therapies in the global pipeline and more than 20 currently on the market. However, these therapies are hindered by challenges in large-scale manufacturing, particularly in the production of plasmids - which house the therapeutic gene of interest, and which are contained within the viral vectors that actually deliver the gene. As the demand for gene therapies grows, companies are in a competitive race to develop manufacturing technologies that can streamline production, reduce costs, and accelerate turnaround times. Despite significant investments in the gene therapy field, the development of large-scale manufacturing technologies has not kept pace with the therapeutic advancements. The complexity of gene therapy development, involving viral particles and cell lines, is a significant challenge in achieving consistent production. Unlike conventional drug manufacturing processes, the smallest change in production conditions can lead to significant variations in the gene therapy end product. One of the primary obstacles is the use of plasmids in viral vector production. Plasmids are small, circular pieces of DNA. In nature, plasmids are found in microbes, such as bacteria. They are relatively easy to manipulate and can be constructed to encode genes of interest. As such, they are a critical raw material for many therapeutic modalities. Producing plasmids at a Good Manufacturing Practice (GMP) level, as required by regulatory authorities, is complex and expensive. The limited global availability of GMP-grade plasmids further exacerbates the challenge. Furthermore, plasmids exhibit genetic instability, making them susceptible to unwanted genetic changes during the scaling process. In response to these challenges, OXGENE , a WuXi Advanced Therapies (WuXi ATU) company, have developed TESSA™ and XLenti™. These technologies offer innovative alternatives to traditional plasmid-based approaches. TESSA™ leverages a modified adenoviral vector for scalable adeno-associated viral vector manufacturing, eliminating the need for plasmids. XLenti™ integrates DNA into cells to produce lentiviral vectors without introducing new plasmids. WuXi ATU is adapting its facilities to manufacture TESSA™ vectors and recombinant adeno-associated viral vectors at a GMP level. This innovation represents an important step forward in the goal of optimizing large-scale gene therapy production in order to make it more accessible and cost-effective. By overcoming the limitations associated with plasmids and introducing scalable, cost-effective technologies, the industry is poised to make gene therapies available to a broader patient population, offering hope for those with difficult-to-treat diseases. Author BioFocus Newsroom Previous Next

Exclusive distribution deal will bring Droplex digital PCR diagnostic kits to more laboratories across Europe, supporting biomarker-driven treatment decisions in major cancers. < Back Bio-Rad and Gencurix Expand European Access to Precision Cancer Testing Exclusive distribution deal will bring Droplex digital PCR diagnostic kits to more laboratories across Europe, supporting biomarker-driven treatment decisions in major cancers. Bio-Rad Laboratories has extended its partnership with Korean diagnostics company Gencurix through a new strategic agreement that will make Droplex digital PCR oncology testing kits more widely available across Europe. The deal establishes Bio-Rad as the exclusive distributor of Gencurix’s CE-IVD marked Droplex assays, which run on Bio-Rad’s QXDx ddPCR systems. The kits enable highly sensitive mutation detection from formalin-fixed paraffin-embedded samples and liquid biopsies in several major cancers, including melanoma, colorectal cancer and non-small cell lung cancer. The agreement builds on the companies’ existing collaboration and includes provisions for joint marketing as well as collaborative product development. It aims to accelerate access to advanced diagnostic tools that support precision medicine in oncology. “We are proud to partner with Gencurix to expand our ddPCR offering and deliver CE-IVD marked ddPCR oncology diagnostics kits to laboratories across Europe,” said Steve Kulisch, Vice President Product Management, Digital Biology Group, Bio-Rad Laboratories. “This partnership will provide clinical laboratories with access to highly sensitive and reliable molecular tools, supporting compliance while ensuring the highest standards of diagnostic accuracy. Most importantly, Gencurix’ Droplex testing kits, combined with our QXDx ddPCR platform, will enable physicians to make more informed treatment decisions based on biomarker testing, thereby contributing to improved patient outcomes.” “This newly expanded strategic partnership with Bio-Rad represents a significant milestone for Gencurix, validating the clinical value of our Droplex technology,” said Sang Rae Cho, CEO of Gencurix. “Bio-Rad's extensive European distribution capabilities and deep expertise in digital PCR technology make them the ideal partner to bring our oncology testing solutions to a broader market. This partnership will accelerate access to our innovative diagnostic tools for oncology applications throughout Europe, supporting improved patient outcomes through precision medicine.” As the demand for biomarker-driven treatment selection continues to grow, the expanded partnership positions both companies to play a larger role in delivering reliable molecular diagnostics to European oncology laboratories Author BioFocus Newsroom Previous Next

New investments and next-generation inhaler technology aim to reduce the climate impact of respiratory care while maintaining treatment access and safety. < Back Every Breath Counts: Toward Sustainable Respiratory Care New investments and next-generation inhaler technology aim to reduce the climate impact of respiratory care while maintaining treatment access and safety. Today, rising temperatures, worsening air quality, and increasingly frequent extreme weather events are no longer distant threats : they are daily realities, especially for people living with respiratory conditions like asthma or chronic obstructive pulmonary disease (COPD). The climate crisis is a health crisis In 2024 , the world surpassed the 1.5°C temperature increase once projected for 2050. Even small shifts in temperature or humidity can alter mortality rates for vulnerable populations, and outdoor air pollution alone causes 4.2 million premature deaths each year . So, the question is no longer if we need to act — but how fast we can It’s time to rethink how we deliver care. Clinical innovation is essential, but sustainability is also critical for patient well-being and long-term health system resilience. After all, healthcare systems are responsible for up to 5% of global greenhouse gas emissions . That’s why healthcare professionals, policymakers, and the pharmaceutical industry must work together to deliver concrete solutions to benefit both patients and the planet. Consider this: what if the medicine that helps you breathe may also contribute to rising temperatures that make it harder to breathe? Respiratory inhalers are a case in point: they’re essential for millions of patients, yet their climate impact is frequently overlooked. In fact, today, pressurized metered dose inhalers (pMDIs) used currently contain propellants with high global warming potential and while these products can be lifesaving, they also contribute to healthcare carbon emissions. This raises a critical question: how do we ensure patients get the treatments they need without confronting them with the choice between their health and the planet’s? The answer lies in innovation that’s both patient-centered and planet-conscious Developing carbon minimal pMDIs is one such step. With the usage of next-generation propellants, these inhalers will ensure treatment continuity providing products with the same active ingredients, efficacy, safety and patient usability, but with a lower carbon footprint . But innovation alone isn’t enough. To reduce the climate impact of inhalers, we need a broader shift: achieving accurate and early diagnosis of asthma and COPD, improving patient education and inhaler technique, and fostering sustainable treatment habits. This includes reducing over-reliance on short-acting beta-agonist (SABA) inhalers—which can contribute to both higher greenhouse gas emissions and poorer disease control—and encouraging adherence to maintenance therapies that improve long-term outcomes. This is how we move from intention to impact: by embedding sustainability into every layer of respiratory care As the link between climate and health becomes clearer, governments and international bodies are stepping in to accelerate change and implement regulations, essential tools for safeguarding public health. The Kigali Amendment, signed in 2016, aims to phase down hydrofluorocarbons (HFCs) (including hydrofluoroalkanes (HFAs))—potent greenhouse gases—prompting scrutiny of their use in pMDIs. The EU's 2024 Fluorinated Greenhouse Gases Regulation includes pharmaceutical HFCs under a quota system, with safeguards to ensure supply and allow time for transition. In the United States, the American Innovation and Manufacturing Act (2020) gave the Environmental Protection Agency (EPA) the authority to reduce HFCs, targeting a 15% cap on production and use by 2036. This includes allowances for essential uses like inhaler propellants, but the message is clear: the clock is ticking. A complete phase-out of HFCs is foreseen by 2050. Likewise, national healthcare systems (HCS) have started decarbonizing, and inhalers are a key focus. Decarbonization policies include updated clinical guidelines, greener procurement policies, and growing patient awareness—all supporting a shift to low-carbon inhaler options. But why wait for regulation to act? Real climate leadership in pharma means anticipating change, not reacting to it. It means embedding sustainability into product development, supply chains, and patient engagement strategies. It means recognizing that innovation is not just about new molecules, it’s about new models of care that are resilient, equitable, and low-carbon. At Chiesi, we believe patients shouldn’t have to choose between their health and the planet’s Our transition to a carbon minimal pMDI portfolio reflects our commitment to shared value: where innovation drives not only business growth, but also patient health, community well-being, and environmental sustainability. To protect both patients and the planet, Chiesi has invested more than €350M to reduce the climate impact of its pMDI products. A non-persistent, next-generation propellant, HFA 152a, has been adopted to replace the current high-global warming potential propellant, HFA 134a, reducing pMDIs’ carbon footprint by up to 90%, in the same range as dry powder inhalers (DPIs). But this is more than a product upgrade: it’s a systemic shift. To reinforce supply security and European resilience, Chiesi is also investing €430 million in a new manufacturing site in Nerviano (Milan), set to open by 2030. This facility will produce carbon minimal inhalers (next generation propellant pMDIs and DPIs), and sterile biological products through a fully European-based supply chain. We now have the knowledge, the technology, and the momentum to transform respiratory care into a model of sustainability. What’s needed now is a collective will. When healthcare professionals, policymakers, industry leaders, and patients come together with a shared purpose, we can build a future where health and sustainability go hand in hand. So let’s answer the question of when we need to act with a powerful, collective now . Because every breath counts and every “yes” to change helps to protect it. Author Michelle Soriano, Executive VP, Chiesi AIR Franchise Michelle Soriano is a global healthcare leader with over 20 years of experience in strategic leadership across vaccines, established products, and specialty care. Currently, as Executive Vice President at Chiesi Group, she leads the global Respiratory Strategy, overseeing portfolio management, pipeline development, and resource allocation. Beyond her corporate role, Michelle serves on the Harvard Graduate School Alumni Association Council and on the Honorary Board of the Science Club for Girls (SCFG), where she helps guide the organization’s vision and champions STEM mentorship initiatives. She holds a PhD in Biological and Biomedical Sciences from Harvard University and a bachelor’s degree in biomedical research from the National Autonomous University of Mexico. Previous Next