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SonoThera Raises $125M to Develop Ultrasound-Mediated Genetic Medicines

Biotechnology company SonoThera has raised $125 million in an oversubscribed Series B financing round. The financing was led by Vida Ventures, with participation from ARK Invest, CureDuchenne Ventures, Leaps by Bayer, Otsuka Pharmaceutical, SymBiosis, UCB Ventures SA, Vivo Capital, and existing investors ARCH Venture Partners, Alexandria Venture Investments, Duquesne Family Office, Illumina Ventures, Johnson & Johnson Innovation – JJDC, Medical Excellence Capital, RA Capital, and Vertex Ventures HC.

SonoThera will use the funds to advance its lead programs in Duchenne muscular dystrophy (DMD) and autosomal dominant polycystic kidney disease (ADPKD) in the clinic. The funds will also support efforts to expand its pipeline of targeted redosable genetic medicines across multiple organ systems and scale its proprietary platform technologies for safe, targeted therapy delivery.

The company’s platform combines a proprietary ultrasound-mediated delivery technology dubbed RIPPLE™, with a payload engineering platform dubbed PORE™. The platforms are designed to support the development of DNA and RNA therapeutics, gene editing, and gene silencing approaches. SonoThera is using its tech to develop genetic medicines that it claims will address key limitations of conventional gene therapies including delivery challenges, payload size constraints, immune responses, safety events, and difficulties with redosing. 

As Kenneth Greenberd, PhD, SonoThera’s co-founder and CEO, stated “we founded SonoThera to take a fundamentally different approach, with a platform designed to broaden the therapeutic possibilities of the field. We believe our technology has the potential to expand the range of diseases addressable by genetic medicines while enabling more precise, durable, safer, and repeatable therapies for patients.”

SonoThera has already demonstrated the targeted delivery and expression capabilities of its platform across multiple tissues, including skeletal muscle, heart, liver, kidney, adipose, and brain. It has also shown that it can deliver large payloads such as full-length dystrophin for DMD and RNA-based payloads for gene silencing applications in preclinical studies. 

The company expects to initiate its first clinical trial in DMD in 2027.

Commenting on the financing, Rajul Jain, MD, managing director at Vida Ventures, said “we believe SonoThera, with its RIPPLE delivery and PORE payload engineering technologies, has the potential to unlock opportunities in diseases with significant unmet need that have been previously inaccessible to other genetic medicine approaches.” 

In connection with the financing, Jain and Rakhshita Dhar, MS, vice president & head of Healthcare Venture Investments at Leaps by Bayer, have joined SonoThera’s Board of Directors.

The post SonoThera Raises $125M to Develop Ultrasound-Mediated Genetic Medicines appeared first on GEN – Genetic Engineering and Biotechnology News.

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<![CDATA[Delphi panel reveals how NPs and PAs can talk about long-acting injectables earlier in schizophrenia, boosting adherence and cutting relapse.]]>
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<![CDATA[Experts unpack why SSRI withdrawal hits, how to taper safely, and what protracted symptoms mean for long-term antidepressant use.]]>
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Distribution of bladder afferent activity across the sacral roots in sheep shows marked individual variation: implications for neuroprosthesis design

ObjectiveImplantable sacral anterior root stimulators enable bladder emptying after spinal cord injury but do not prevent reflex incontinence. A closed-loop neuroprosthesis that detects and inhibits reflex bladder contractions could address this, but first, reliable detection of bladder fullness from the sacral roots. Further, the distribution of afferent bladder activity between sacral roots, and the relationship between efferent and afferent activity within each root, remains unclear and must be clarified to guide implant design.MethodsElectrode books were implanted on the S1–S3 extra-dural sacral roots bilaterally in six terminally anesthetized sheep. Afferent electroneurogram (ENG) was recorded concurrently from all implanted roots during filling cystometries and correlated with bladder pressure. Each root was individually electrically stimulated and the bladder pressure response recorded. Post-mortem morphometric analysis determined fiber size distribution in each root.ResultsOverall, S2 ENG activity showed the highest correlation with bladder pressure, and electrical stimulation of S2 and S3 produced the greatest increases in bladder pressure. Fiber size distribution did not correlate with either ENG activity or bladder pressure response. Significant variation was identified between individual sheep, but notably, in four of six sheep, a single sacral root had both the highest ENG correlation to bladder pressure and the greatest bladder response to stimulation.SignificanceThis study demonstrates reliable recording of bladder afferents from sacral roots using clinically applicable electrodes. It provides the first systematic recording of bladder ENG concurrently across three pairs of sacral roots in multiple animals, and the first characterization of signal distribution between roots. Significant individual variation is identified, impacting the design of future implantable sacral neuroprostheses for bladder control.
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Artificial intelligence for autism spectrum disorder: advances in diagnosis, behavior analysis and educational support

IntroductionArtificial intelligence has become an increasingly relevant field of research in the study of Autism Spectrum Disorder (ASD), offering novel technological approaches for the analysis, detection, and support of individuals on the autism spectrum. The aim of this study was to systematically review recent scientific literature examining the application of artificial intelligence in ASD.MethodsThe review was conducted following the PRISMA 2020 guidelines. Searches were performed in PubMed, Scopus, Dialnet, and Google Scholar, including studies published between 2019 and 2025. After applying predefined inclusion and exclusion criteria, 18 empirical studies were included in the final analysis. Methodological quality and risk of bias were assessed using Joanna Briggs Institute critical appraisal tools adapted to the methodological design of each study.ResultsCurrent research focuses primarily on four areas: early detection and diagnostic support, automated analysis of behavioral and social patterns, AI-based educational technologies, and communication support systems. Although the reviewed studies demonstrate promising advances in machine learning, computer vision, and natural language processing, important methodological limitations remain, particularly regarding external validation, dataset representativeness, and heterogeneity of performance indicators.DiscussionOverall, artificial intelligence shows considerable potential for supporting diagnosis, education, and communication in ASD; however, greater methodological robustness, transparency, and ethical safeguards remain necessary before broader implementation in real clinical and educational settings.
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Head circumference assessment in pediatric MRI: a pilot study of manual measurement methods and automated segmentation-based alternatives

PurposeHead circumference (HC) is an important clinical parameter in neuropediatrics, but it is often missing or outdated in referral information. This can lead to subjective, reader-dependent estimation during MRI interpretation. We first aimed to compare magnetic resonance imaging (MRI)-based methods for HC measurement against the tape measure (ground truth), and second to establish an automated alternative.MethodsIn 23 children (mean age 4.5 years, range 0.5–17 years), HC was prospectively measured with a tape measure (ground truth) on the day of MRI. MRI-based HC measurements were derived from 3D T1-weighted MPRAGE and followed a two-step workflow: measurement plane selection and circumference measurement within that plane. Plane selection was performed using visual-based, rule-based, atlas-based [(infant) FreeSurfer], or neural network (nn)-based methods. Circumference measurement was performed using manual ellipsoid, manual contour, automated ellipsoid, or automated contour methods. The relative technical error of measurement (r-TEM; acceptable < 1.5%) and intraclass correlation coefficient (ICC; two-way mixed ANOVA model) were used to assess accuracy and consistency with the tape measure.ResultsVisual-based with manual ellipsoid/contour and rule-based with manual ellipsoid/contour showed acceptable accuracy (r-TEM 0.73%–1.12%). Visual-based with automated ellipsoid and rule-based with automated ellipsoid also demonstrated acceptable accuracy (r-TEM 0.77% and 0.68%). Atlas-based with automated ellipsoid achieved the lowest r-TEM (0.55%), followed by nn-based with automated ellipsoid (r-TEM 0.75%). In contrast, automated contour approaches showed unacceptable accuracy (r-TEM 3.42%–4.21%). Seven nn-based measurements with automated ellipsoid/contour were spurious. ICCs were high across all methods (0.993–0.997); however, manual contour and automated ellipsoid were associated with overfitting issues.ConclusionThe developed, fully automated algorithm based on (infant) FreeSurfer provides precise and reliable head circumference measurements from pediatric MRI scans with acceptable overall accuracy and excellent consistency with manual measurements using a tape (gold standard). Our algorithm simplifies the head circumference measurement process and provides a reproducible, reader-independent value that enhances the interpretation of neuroradiological findings. Further studies should be conducted to validate with larger sample sizes and to develop deep neural network algorithms for segmentation.
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Sexual function in women with complex PTSD: a comparative study

BackgroundPosttraumatic stress disorder (PTSD) is associated with impaired sexual functioning in women, but the effects of complex PTSD (CPTSD) remain unclear. This study tested three hypotheses: (1) women with CPTSD would report lower overall sexual function than women with PTSD and trauma-exposed controls; (2) the pain domain would show the largest group differences; and (3) CPTSD symptom severity would be negatively associated with sexual function, while PTSD symptom severity would not.MethodsA cross-sectional study included 386 Mexican female university students (18–55 years) who completed the Female Sexual Function Index (FSFI), International Trauma Questionnaire (ITQ), and Adverse Childhood Experiences Questionnaire (ACE-IQ). Group differences were assessed using ANOVA with post-hoc comparisons. Multivariable linear regressions examined whether CPTSD severity predicted sexual function independently of PTSD severity and age. A sensitivity analysis excluded women aged 45 and older.ResultsFSFI total scores differed significantly across groups (F = 3.52, p = 0.031). Women with CPTSD reported lower overall sexual function (M = 26.56, SD = 6.41) than trauma-exposed controls (M = 28.86, SD = 5.03; p = 0.047). In the pain domain, women with CPTSD reported greater sexual pain than controls (F = 6.35, p = 0.002). Multivariable regressions showed that CPTSD severity predicted lower FSFI total scores (β = -0.22, p < 0.001), independent of age and PTSD severity (adjusted R2 = 0.028). For sexual pain, the bivariate association with CPTSD (rho = -0.16, p < 0.01) did not persist after adjusting for age; age was the only significant predictor (β = 0.02, p = 0.007). Sensitivity analyses yielded unchanged results.ConclusionsCPTSD severity is associated with poorer overall sexual function, independent of age and PTSD severity. However, the association with sexual pain did not persist after accounting for age. Clinical and research implications are discussed.
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Logotherapy techniques to unlock resilience among Ukrainian refugees: a pre-post quasi-experimental design

The ongoing Russian-Ukrainian war has triggered a significant refugee crisis, resulting in widespread trauma, displacement, and mental health challenges among affected populations. This study aimed to explore the potential usefulness of logotherapy, a meaning-centered therapy, in addressing the mental health needs of Ukrainian refugees. The research was conducted in a naturalistic scenario of a group of 20 Ukrainian refugees residing in Europe who received a tailored logotherapy or meaning-centered psychological support, compared with controls who did not. Meaning-oriented techniques, namely, Socratic Dialogue, Modification of Attitude, Paradoxical Intention, and Dereflection were used during the intervention. Participants were assessed in a pre-post quasi-experimental design using validated self-report measures for anxiety (Generalized Anxiety Disorder-7), depression (Beck Depression Inventory), and general health (General Health Questionnaire-12). The improvements were clearly observed as reductions in their self-reported anxiety and depressive symptoms after the meaning-centeres psychological support. Overall, these preliminary findings indicate that logotherapy may be a promising and feasible approach to psychological support for refugee populations. However, given its pilot nature and quasi-experimental design, causal conclusions cannot be drawn, and further research using larger, randomized, and methodologically rigorous designs is warranted to examine long-term effects and broader applicability.
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Affect before diagnosis: applying affective neuroscience to psychiatry

Jaak Panksepp spent nearly five decades mapping the primary-process affective systems of the mammalian brain across different species, producing a framework of considerable empirical power that is functionally invisible within psychiatry. Psychiatry has not built upon that literature in human contexts. Consequently, Affect has never occupied the foundational place in psychiatry that the evidence warrants. This paper attempts to close that gap and assembles twelve converging lines of evidence for subcortical primacy of Affect, including evidence not previously synthesized for this purpose: pseudobulbar affect, gelastic and dacrystic epilepsy, double dissociation of volitional and emotional facial expression, affective blindsight, and neonatal emotional behavior. The evidence shows that Affect is generated subcortically, the cortex modulates rather than creates it, and when cortical regulation is removed or impaired, affective states persist or intensify. From this evidence, personality is best understood as an individual’s position in a configuration space defined by subcortical Affect generation parameters and cortical regulatory capacity, with psychopathology occurring when signal intensity chronically exceeds or overwhelms regulatory capacity. This framework generates a specific clinical prediction: conditions that co-occur with personality pathology at rates incompatible with independent etiology are expressions of the same affective architecture through different conditioned channels, not independent diseases. Evidence confirms this directly: when personality pathology improves, its comorbid conditions decline substantially; when it does not, they persist. Further, the affective-regulatory framework transforms the clinical encounter by replacing a character verdict with a neurobiologically grounded account in which regulatory capacity is buildable and recovery is construction rather than correction.
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Psychological distress experiences among patients living with HIV: a qualitative systematic review

BackgroundHIV patients endure psychological distress throughout the phases of diagnosis, treatment, and rehabilitation, which considerably affects their quality of life. A thorough comprehension of patients’ psychological distress experiences is crucial for healthcare workers to devise focused solutions for enhanced care. This study aims to consolidate evidence concerning psychological distress in HIV patients and elucidate its sources and influencing factors.DesignA systematic review and meta-synthesis of qualitative studies.MethodsA thorough literature review was performed across eight electronic databases from inception to June 2025.Qualitative studies were selected if pertinent to the psychological distress experiences of HIV patients. The quality of qualifying studies was assessed utilizing JBI qualitative research quality evaluation standard. The thematic synthesis method was utilized to amalgamate the findings.ResultsA total of 1583 studies were identified through database research, and 11 studies were incorporated into the review. Three analytical themes emerged from the findings: individual factors including disease diagnosis and emotional dilemma; environmental factors including economic hardship, social environment and limited access to health support; interpersonal factors including decline in marital intimacy and insufficient support from family and friends.ConclusionPsychological distress is a subjective phenomenon influenced by the interaction of individual, environmental, and interpersonal factors, commonly seen in HIV patients. Effective therapeutic care requires understanding psychological distress prevalence and causes. Individualized, sustainable, and adaptive psychosocial care interventions should be taken to alleviate psychological distress, improve patients’ mental health and facilitate their reintegration into society.Systematic Review Registration:https://www.crd.york.ac.uk/PROSPERO/view, identifier CRD420251050269.