<![CDATA[APA’s next DSM rebrands and adds context, cross-cutting symptoms, and biomarker-ready science to sharpen diagnoses and personalize psychiatric care.]]>
STAT+: Radiopharmaceutical shows promise in post-Pluvicto setting
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Good morning. Today, we’re looking at mixed reactions to a closely watched immunology trial and growing scrutiny of a type of telehealth business model.
The need-to-know this morning
- Vera Therapeutics and the FDA reached an agreement to allow Vera to accelerate the analysis of a confirmatory Phase 3 study involving atacicept, its treatment for the chronic kidney disease IgA nephropathy. The drug is already under review for accelerated approval, with a decision expected by July 7. The new FDA agreement will allow Vera to analyze its Phase 3 study, needed for full approval, in the third quarter, rather than wait for an additional year of data to accrue.
Abivax’s positive data weighed down by cancer concerns
Abivax said yesterday that its experimental treatment for ulcerative colitis showed significant efficacy in a closely watched maintenance trial.
Kinase Droplets Activate Growth Signals, Path for Cancer Therapy
A new study published in Cell Reports titled, “Kinase condensates enrich ATP and trigger autophosphorylation,” suggests that cellular phase separation, a mechanism that organizes biomolecules into dense, liquid-like condensates, may play a previously underappreciated role in regulating kinase activity. The findings suggest that aberrant condensate formation could contribute to oncogenic signaling while also offering new opportunities for drug targeting.
“Many biological molecules have this propensity to spontaneously separate,” said Lindsay Case, PhD, assistant professor of biology at Massachusetts Institute of Technology (MIT) and corresponding author of the study. “We were really interested in asking, if we have these kinases forming droplets, what is the consequence of that in the context of signaling?”
Phase separation occurs when proteins condense into highly concentrated liquid-like droplets within cells, analogous to oil droplets separating from vinegar. Although biomolecular condensates have emerged as important organizers of cellular processes, their impact on kinase signaling has remained incompletely understood.
The researchers examined three kinases: focal adhesion kinase (FAK), Mst2, and Abl. Across all three systems, condensate formation increased kinase activity by concentrating enzymes and substrates, thereby promoting phosphorylation reactions.
For FAK, the team found that elevated protein levels were sufficient to drive droplet formation and activate downstream growth signaling. The findings raise the possibility that FAK overexpression in tumors could promote constitutive signaling through condensate formation, potentially contributing to cancer progression and metastasis.
“It was surprising that just by condensing this protein into a droplet, you can actually turn on a signaling pathway that should be turned off,” said Case. “If FAK concentration is too high, you’re always getting these droplets and you’re always signaling, regardless of what the receptors that are supposed to be controlling this are doing.”
Mst2 and Abl also phase separated at high concentrations, which led to increased activity. For Mst2, phase separation is a strategy that healthy cells use to control the Hippo signaling pathway, which promotes cell growth and survival. Phase separation can also lead both enzymes to phosphorylate additional targets, and activate different signaling pathways.
“It’s not just that you’re getting faster phosphorylation, but in those cases, the patterns of what is actually getting phosphorylated were very different inside of the droplet compared to what might be happening in a non-droplet context,” Case says. “The kinase is able to phosphorylate amino acid residues beyond the set of canonical sites that have been described before.”
Mechanistically, the team found that kinase condensates selectively concentrate ATP, the phosphate donor required for kinase activity. Positively charged regions within kinases appear to recruit negatively charged ATP molecules to support phosphorylation.
Using machine-learning analysis, the investigators predicted that approximately 45% of the roughly 500 human kinases possess the molecular features needed to form similar condensates. The findings suggest that phase separation may represent a widespread regulatory mechanism that could influence both normal cellular signaling and disease-associated kinase activity.
In future work, Case hopes to explore designing drugs that could mimic ATP’s ability to be attracted into droplets within a cell, which could reduce side effects.
The post Kinase Droplets Activate Growth Signals, Path for Cancer Therapy appeared first on GEN – Genetic Engineering and Biotechnology News.
Circio and GenAssist Collaborate on Gene Therapy for Muscle Disease and In Vivo Cell Therapy
Oslo, Norway-based Circio and Suzhou, China-based GenAssist entered into a research collaboration to develop circVec-enhanced AAV vectors specifically engineered for in vivo cell therapy and targeted, low dose systemic gene therapy.
Genetic muscle disease is an area of major unmet medical need, where current gene therapy’s high dosing requirements are associated with severe toxicity. By integrating Circio’s and GenAssist’s complementary technologies, the parties aim to develop joint next generation of AAV gene therapy candidates, according to a Circio spokesperson. The focus is on addressing genetic muscle conditions where high and broad muscle-specific expression is required at substantially lower therapeutic AAV doses than can be achieved by conventional AAV gene therapy.
“Our second-generation AAV platform establishes a new benchmark for safety, utilizing highly tissue-specific, de-targeted capsids to dramatically lower systemic dosing while eliminating off-target toxicity,” said Chunyan He, PhD, CEO of GenAssist. “Through our collaboration with Circio, we integrate their unique circular RNA technology. This partnership directly addresses the core demands of next-generation genetic medicine, overcoming the traditional dose-expression trade-off to deliver safer and more effective therapies.”
In addition, Circio and GenAssist will explore the potential of generating joint in vivo CAR T candidates for oncology and autoimmune applications. The collaboration will involve production of novel AAVs combining GenAssist´s T-cell targeting with the circVec expression cassette from Circio. The combined AAVs will subsequently be tested in vitro and in vivo, and if successful, candidates for further development will be nominated for preclinical development.
“The targeted AAVs developed by GenAssist have the ability to specifically and efficiently transduce muscle or T-cells upon systemic delivery with near-complete liver de-targeting,” added Thomas Hansen, PhD, CTO of Circio. “The partnership between Circio and GenAssist will aim to evaluate whether the enhanced circVec expression acts synergistically with these targeted capsids and promoters.
“This fits perfectly into Circio’s strategy of testing circVec in multiple tissues using different AAV variants, both internally and externally. This will allow us to identify new therapeutic avenues where circVec delivers a benefit, and forge partnerships potentially enabling multiple future development opportunities. China is a particularly interesting geography, with cutting edge science and accelerated pathways to establish early clinical data.”
The post Circio and GenAssist Collaborate on Gene Therapy for Muscle Disease and <i>In Vivo</i> Cell Therapy appeared first on GEN – Genetic Engineering and Biotechnology News.
Detection of Self-Harm in Electronic Mental Health Records Using Privacy-Preserving Local Language Models: Methodological Study
Background: Self-harm is the strongest risk factor for suicide and an important outcome for mental health care. Although prevalent in clinical populations, it is often imprecisely captured in routinely collected clinical data, where it is often recorded and stored as unstructured free text. Contemporary language models, such as GPT (OpenAI) and Gemini (Google), can analyze free-text clinical notes, but such models may violate data governance of processing sensitive patient data. Objective: This study aimed to evaluate whether a privacy-preserving language model running entirely within an institution’s secure computing infrastructure (here, the UK National Health Service [NHS]) could accurately identify the presence and timing of self-harm using electronic health records from secondary mental health care. Methods: Clinical notes were drawn from Oxford Health NHS Foundation Trust using a multistage workflow: (1) a random sample of 1000 patients with a psychiatric diagnosis, defined according to the (; codes F00–F99); (2) candidate-note identification using a Gemma3-4b language model to flag notes containing self-harm content; and (3) from those candidates, 1352 randomly sampled notes were selected for expert annotation, resulting in gold-standard corpus enriched for self-harm content. Clinical notes were annotated for the presence of self-harm and its timing (≤90 days, >90 days, or unknown). A privacy-preserving locally served 27-billion-parameter Gemma 3 language model (“Gemma3-27b”) was used as the core model. Prompts were systematically developed and refined using a labeled development set to identify self-harm and generate a structured output per clinical record. Gemma3-27b performance was compared against a strong baseline multilabel text classification model based on robustly optimized BERT pretraining approach (RoBERTa), a transformer-based language model architecture. Model performance was evaluated using precision, recall, and the -score (harmonic mean of precision and recall), with 95% CIs estimated from 1000 bootstrap samples with replacement. Results: Gemma3-27b outperformed the RoBERTa classifier across all categories, achieving Precision=0.92, Recall=0.92 (sensitivity), and -score=0.92 for notes containing self-harm, and Precision=0.97, Recall=0.97 (specificity), and -score=0.97 for notes without self-harm. For the 51 notes labeled as recent self-harm in the held-out test set, Gemma3-27b achieved Precision=0.84, Recall=0.75, and -score=0.79. The global weighted -score of Gemma3-27b across all categories was 0.88, compared to 0.85 for RoBERTa. Conclusions: With systematic prompt development on a labeled development set, but no gradient-based fine-tuning, the current Gemma3-27b language model matched or exceeded a fine-tuned RoBERTa classifier for ascertaining self-harm events and their timing. Aggregate gains were modest, while improvements were largest in the most challenging, lower-frequency timing categories. On a simplified binary recent-versus-other task, RoBERTa performed marginally better, indicating that supervised classifiers remain highly effective when the task is simplified and sufficient labeled data exist. This work demonstrates the technical feasibility of privacy-preserving self-harm detection within a secure NHS research environment.
<![CDATA[New DOE RISE rule cuts federal loan limits for graduate nursing, PAs, and social work—threatening workforce diversity and care access. ]]>
<![CDATA[FDA issues CRL for CTx-1301 in ADHD.]]>
STAT+: Pharmalittle: We’re reading about a Lilly threat to 340B hospitals, Ebola vaccine funding, and more
Rise and shine, everyone, another busy day is on the way. And it is getting off to a pleasant start here on the Pharmalot campus, where clear blue skies and comfy breezes are greeting us. Who could ask for anything more? Actually, we could — it is time to reheat the kettle for another cuppa stimulation. Our choice today is old-fashioned green tea. And here is a helpful tip — a teaspoon of honey enhances the flavors splendidly. Of course, you are invited to join us. For the full experience, we are now hawking replicas — take a look. Meanwhile, here are a few tidbits to help you along. As always, do keep in touch. We appreciate feedback, criticism, and tips….
Eli Lilly has told about 50 hospitals participating in a federal drug discount program to submit comprehensive claims data over the next five days or they will no longer receive the mandated price breaks, STAT writes. The move comes after the company announced a policy last January demanding such data in a bid to reduce what it calls duplicate discounts paid to participating hospitals. For the past few years, more than 2,300 hospitals have complied with the demand, but Lilly said that some of the larger hospitals systems around the U.S. have refused to do so, despite recent follow-up letters regarding the policy that went into effect on Feb. 1. Up to 1,000 have so far not complied.
The U.S. Food and Drug Administration is scheduled to have a closed-door roundtable this week with over a dozen groups advocating for rare disease treatments, Bloomberg Law reports. The meeting, scheduled for Wednesday, will connect FDA Acting Commissioner Kyle Diamantas and other senior agency officials with at least 10 rare disease groups for a private conversation about advancing treatments that often struggle to meet the agency’s rigorous approval process. The meeting comes as the rare disease community has sought more clarity from regulators amid turmoil over recent decisions for rare drugs. Under former Commissioner Marty Makary, who resigned last month, several initial rejections were issued to companies seeking to market rare disease drugs and gene therapies.
Low-Cost, Portable Biotech Tools Improve Access to Bioresearch and Diagnostics
A global research team headed by scientists at University of Toronto’s Leslie Dan Faculty of Pharmacy has demonstrated the effectiveness of a suite of low-cost, portable biotechnology tools that are designed to improve access to laboratory research and diagnostics in resource-limited settings.
The newly reported study highlights how decentralized biomanufacturing tools and freeze-dried reagents can help researchers produce high-value biological materials locally—reducing reliance on fragile international supply chains and expanding access to life sciences innovation globally.
“For labs in low- and middle-income countries [LMICs], access to high-quality supplies and equipment is a chronic problem,” says research lead Keith Pardee, PhD, associate professor at the Leslie Dan Faculty of Pharmacy Pardee. “Shipping can take a long time, it’s expensive, and products often require a cold chain to retain their effectiveness. This research is in response to those challenges to develop tools that are more accessible for labs in lower-resource settings and improve research equity.”
Pardee, alongside collaborators including Camila González, PhD, at the Universidad de los Andes, Bogotá, Fernán Federici, PhD, at Millennium Institute for Integrative Biology (iBio), Santiago, and Lindomar Pena, PhD, at Aggeu Magalhães Institute (IAM), Oswaldo Cruz Foundation (Fiocruz), Recife, reported on the study in Science Advances. In their paper, “International multisite implementation of distributed cell-free protein biomanufacturing to advance health and research equity,” the authors concluded, “This study lays the foundation for fundamental shifts in biotechnology manufacturing practices in LMICs and developing nations, moving from reliance on centralized and outsourced production facilities to adopting decentralized, local production platforms.”
“Emerging biotechnologies hold transformative potential to strengthen economic and health security, while benefiting the planet,” the authors wrote. However, they pointed out, “Access to advanced tools, such as molecular diagnostics, life-saving treatments, and biomanufacturing infrastructure, remains largely concentrated in wealthier regions, thereby restricting access to transformative solutions for communities that need them most.”
A key factor is the reliance on centralized bioproduction systems, “… which require sophisticated, capital-intensive infrastructure and cold supply chains that are often unavailable in resource-limited settings.” Access to healthcare is similarly affected by the availability of biomanufacturing capacity and biologistics, which can slow delivery of diagnostics, delay disease control programs, and limit the ability to carry out life sciences research.
For their newly reported work the team focused on synthetic biology and cell-free systems—technologies that isolate and freeze-dry the molecular machinery needed to produce proteins commonly used in life sciences research. Because the reagents are freeze-dried, they can be shipped and stored without refrigeration, then reactivated simply by adding water. “One promising avenue to improving access is cell-free protein synthesis (CFPS), which offers the potential to empower communities through affordable, low-burden, on-site production of critical bioreagents, diagnostic tools, and therapeutic agents,” the researchers stated.
They paired these systems with low-cost, adaptable hardware, including a 3D-printed hand-powered centrifuge developed by postdoctoral fellow Mohammad Simchi, PhD, at the Leslie Dan Faculty of Pharmacy. Together, the technologies enabled teams to produce a range of research proteins and diagnostic tools in diverse settings, from conventional laboratories to remote field locations.
“With efficient, low-cost systems in place, rapid on-site production of high-value bioproducts for research, including growth factors, vaccines, and diagnostic enzymes, became achievable within a single day and at a fraction of the typical cost,” they commented.
Using the platform, researchers successfully produced growth factors used in life sciences research and therapeutics, as well as a SARS-CoV-2 vaccine candidate tested in mice and diagnostic tools targeting several clinically relevant pathogens. Using molecular, cell-based, animal model, and clinical sample testing, the bioproducts were validated through proof-of-concept studies and multisite clinical trials. “Direct comparisons with high-cost commercial reagents, the current gold standards, demonstrated similar performance, efficiency, precision, and reproducibility,” the investigators further noted.
First author Severino Jefferson Ribeiro da Silva, PhD, a postdoctoral fellow in Pardee’s lab, said, “Our work shows that it is possible to produce high-value bioreagents on site, essentially anywhere. Through this work, we demonstrated our tools across diverse international settings while maintaining performance comparable to commercial products.”
The authors say that, to their knowledge, the study is the first to translate cell-free biomanufacturing from laboratory to real-world use, across multiple geographic settings, including those that historically have had limited access to the bioeconomy. “By prioritizing accessibility, affordability, and reproducibility, we show that cell-free biomanufacturing is a transformative tool for expanding global research capacity and, ultimately, health equity and participation in the bioeconomy.”
A key component of the project involved testing the systems in a variety of environments across Canada and internationally. Da Silva travelled to the Algonquin Highlands to evaluate diagnostic tools for tick-borne pathogens and tuberculosis, while graduate student Quinn Matthews travelled to the Yukon where he produced and purified proteins using the portable system on a mountain outside Whitehorse.
Collaborators in Chile, Brazil, Colombia, and India also tested the systems, helping ensure the technologies addressed the practical realities faced by researchers in different regions. The project involved extensive international collaboration, including regular meetings, student exchanges and knowledge sharing among participating teams.
Da Silva says the research team experienced first-hand many of the logistical challenges their collaborators routinely face, including lengthy customs delays and damaged shipments containing critical reagents.
“Those experiences highlighted how dependent many researchers and labs still are on fragile international supply chains. If a shipment is delayed, an entire project can stop,” says da Silva. “This work makes it possible to reduce that dependency by enabling local production of key proteins directly at the point of need.”
The researchers say the long-term goal is to help research labs in remote and underserved regions gain access to high-quality diagnostics, research reagents and biomanufacturing capabilities produced closer to home, strengthening resilience against future supply chain disruptions while empowering their research capacity and address local healthcare needs. “With their low cost and operational simplicity, we see these platforms and similar disruptive technologies … as part of a new generation of tools that will help shape a future in which bioreagents, advanced diagnostics, and life-saving therapeutics are accessible to all.”
Da Silva added, “This work is really about access and scientific empowerment. Many labs worldwide have the expertise and ideas to conduct life sciences and applied science research, but they face major challenges accessing key bioreagents and essential materials. Decentralized biomanufacturing could help reduce those barriers and make research and diagnostics more accessible globally.”
The post Low-Cost, Portable Biotech Tools Improve Access to Bioresearch and Diagnostics appeared first on GEN – Genetic Engineering and Biotechnology News.
Hampshire and Isle of Wight adopts continuing healthcare platform
Hampshire and Isle of Wight Integrated Care Board (HIOW ICB) has gone live with the IEG4 digital continuing healthcare solution.