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Actio gains $66m to advance small molecule therapeutics pipeline
Actio gains $66m to advance small molecule therapeutics pipeline

Yahoo

time2 days ago

  • Business
  • Yahoo

Actio gains $66m to advance small molecule therapeutics pipeline

US-based Actio Biosciences has closed a Series B financing round, raising $66m to advance the genetics-driven small molecule therapeutics pipeline aimed at rare and common diseases. The funding will primarily be used to propel the development of the company's lead oral programmes, ABS-1230 and ABS-0871. The round was jointly led by new investor Regeneron Ventures and current investor Deerfield Management. Other participants were current investors Canaan, Euclidean Capital and Droia Ventures. ABS-1230 is a selective potassium channel subfamily T member 1 (KCNT1) inhibitor designed to address KCNT1-related epilepsy, a severe paediatric epileptic encephalopathy. The company is preparing to launch the healthy volunteer segment of a Phase I trial of the therapy in the second half of 2025. The company then plans to expand into a Phase Ib proof-of-concept trial in patients with KCNT1-related epilepsy in early 2026. The US Food and Drug Administration (FDA) has awarded ABS-1230 both orphan drug and rare paediatric designations. ABS-0871, another small molecule in Actio's pipeline, inhibits transient receptor potential vanilloid 4 (TRPV4) and is being developed for the treatment of the rare inherited neurological disorder, Charcot-Marie-Tooth disease type 2C (CMT2C). Actio is progressing this therapy through the healthy volunteer phase of a Phase I trial and aims to move into a Phase Ib trial in individuals with TRPV4+ CMT2C in 2026. The US regulator has granted orphan drug, fast track and rare paediatric drug designations to the therapy. Actio Biosciences CEO and co-founder David Goldstein stated: 'We have made tremendous progress across our pipeline – executing a precision medicine strategy that targets the root causes of disease through genetically informed drug development. 'ABS-1230 and ABS-0871 have the potential to be transformative disease-modifying therapies in their respective rare indications, and growing evidence supports expansion into broader indications. This new funding from industry-leading investors speaks to the value of our approach and provides us with important resources to continue advancing our programmes.' "Actio gains $66m to advance small molecule therapeutics pipeline" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data

Early Surgical Referral Key in Pharmacoresistant Epilepsy
Early Surgical Referral Key in Pharmacoresistant Epilepsy

Medscape

time2 days ago

  • Health
  • Medscape

Early Surgical Referral Key in Pharmacoresistant Epilepsy

OTTAWA — Surgery can achieve improvements in seizure activity in patients (particularly younger patients) with epilepsy that is refractory to drug therapies, according to a presentation at the Canadian Neurological Sciences Federation (CNSF) Congress 2025. Roy Dudley, MD, PhD, a pediatric neurosurgeon at Montreal Children's Hospital and Montreal Neurological Institute and associate professor of pediatric surgery at McGill University, Montreal, underlined the importance of early referral for surgery. An ad hoc task force of the International League Against Epilepsy defined drug-resistant epilepsy as the persistence of seizures despite at least two syndrome-adapted antiseizure drugs used at efficacious daily doses. Many patients with pharmacoresistant epilepsy have tried more than two medications, said Dudley. Age influences responsiveness to surgery, and very young pediatric patients are good surgical candidates because of their neuroplasticity, he stressed. 'Pretty aggressive surgery, such as an anatomical hemispherectomy, can be performed in a child,' said Dudley, noting a case of a 2-year-old child who became seizure-free after such a surgery. 'The goal is to make a child seizure-free, but if we can decrease seizures significantly, that is important. If a young child continues to have seizures, their neurological development will be poor.' Evolution in Technology Citing published research, Dudley pointed out that intraoperative MRI (iMRI) has not been helpful in surgical cases where lesions are poorly defined and the boundaries of the lesions are not apparent. 'These are lesions that extend beyond what is seen on iMRI,' he said. 'The iMRI does not help you to see these lesions, which is what drove us to using new tools.' One of those new tools is robot-assisted stereoelectroencephalography (SEEG), which accurately pinpoints the locations from which epileptic seizures originate. 'Using a robot makes it more precise to implant the electrodes because human error is taken out of the procedure,' said Dudley. Robot-assisted SEEG pinpoints seizure locations more rapidly. Other tools that are currently used for epilepsy surgery include magnetoencephalography, EEG, functional MRI, and advanced MRI voxel-based post-processing. Pool of Surgical Candidates Gerald A. Grant, MD, Allan H. Friedman Distinguished Professor of Neurosurgery and chair of Neurosurgery at Duke University School of Medicine in Durham, North Carolina, agreed that younger age increases the benefits of surgery in a patient with drug-resistant epilepsy. 'There is a greater chance for plasticity or rewiring of the brain if we treat as young as possible, which is also why it is so gratifying as a pediatric neurosurgeon to treat children with severe epilepsy, since we can have such enormous impact on their lives,' he told Medscape Medical News. 'We have advocated to identify the children who might benefit most from surgical intervention as young as possible, since the longer we wait, the greater the impact on long-term neurological development.' Grant noted that working with neurologists is key to expanding the pool of candidates for epilepsy surgery. 'If a child fails two or more seizure medications, they may be a candidate for surgical intervention and should be referred early to a comprehensive epilepsy center. Partnering with the community neurologists is key, since there are many children with severe medically refractory epilepsy who are surgical candidates who, unfortunately, are not referred until much later in their course or even into adulthood.' Artificial intelligence (AI) also might play a role in referrals to surgery. 'We are now testing an AI tool embedded in our electronic medical record at Duke to look at every child or adult getting evaluated by a neurologist and flagging the chart if, based on the AI algorithm, they potentially could benefit from a referral for surgical intervention,' said Grant.

Jazmyn Gourdeas fundraiser after Zivan Radmanovic Bali shooting
Jazmyn Gourdeas fundraiser after Zivan Radmanovic Bali shooting

Daily Telegraph

time4 days ago

  • Daily Telegraph

Jazmyn Gourdeas fundraiser after Zivan Radmanovic Bali shooting

Don't miss out on the headlines from Breaking News. Followed categories will be added to My News. The wife of Bali shooting victim Zivan Radmanovic is still in Indonesia, a close friend has revealed in a desperate public plea for support. Mr Radmanovic, 32, was shot and killed at a villa at the Badung Regency north of Kuta on Saturday. His wife Jazmyn Gourdeas was in the villa but not physically injured. A second man Sanar Ghanim, 34, was also shot during the attack. An image shows Mr Ghanim being tended to by paramedics with his feet and face covered in blood. Now, a fundraiser has been launched for Ms Gourdeas, with close family friend Jamie Lee revealing new details about the aftermath of the tragedy. 'This sudden and devastating loss has left Jazmyn a single mother to six beautiful children, aged from just 9 months to 13 years,' the fundraiser states. 'While only married for just under a year, the couple were deeply in love and shared a family together. A GoFundMe has been set up for Jazmyn Gourdeas after the shooting of Zivan Radmanovic. Picture: Supplied 'Now Jazmyn faces the unimaginable challenge of caring for their large family alone, including three children diagnosed with epilepsy who require ongoing medication, therapies and frequent medical appointments.' Ms Lee said Ms Gourdeas remained in Indonesia as the investigation into the killing progresses, and her family were taking care of the children in Australia. The financial burden facing Ms Gourdeas would now be 'overwhelming', she added. 'Zivan was a kind, hardworking man devoted to his family,' she said. 'His loss leaves a huge hole in their lives, but together, we can help Jazmyn and her children find a way forward. 'Your donation – no matter the size – will help provide stability, care and comfort to a grieving family facing an uncertain future.' As of Wednesday morning, $13,800 had been raised from a $20,000 target. Indonesian police have arrested two people over the shooting of Mr Radmanovic and Mr Ghanim. Sanar Ghanim receives treatment after the shooting. Picture: Supplied Media organisation Nusa Bali reported that one of the alleged offenders was arrested in Jakarta, while another person was captured abroad and was being taken to the Indonesian capital. Indonesian National Police Chief General Listyo Sigit Prabowo held a press conference in Bali on Tuesday at the Jembrana Police Headquarters. 'Regarding the shooting involving Australian citizens, I have received a report from the Bali Police Chief. One suspect has been secured in Jakarta, and another is on the way from overseas,' the police chief told media. 'A more detailed statement will be released by Bali Police. 'What I can say for now is that this case was solved through co-operation between our Criminal Investigation Agency (Bareskrim), Immigration, and the Australian Federal Police (AFP).'. General Sigit did not release any information about identity of the alleged shooters but told media that Bali Regional Police would provide more detailed information. It was reported there were two alleged offenders who might be Australian and the gunmen used firearms during the attack. It was not clear what the motive was behind the attack. Bali Regional Police Chief Daniel Adityajaya said his department would provide an update later. 'Once the evidence and documentation are complete, Bali Police will issue an official statement to the public and media,' he said. Originally published as Fundraiser launched for family of Bali shooting victim Zivan Radmanovic

Epilepsy May Signal Future Dementia Risk
Epilepsy May Signal Future Dementia Risk

Medscape

time4 days ago

  • Health
  • Medscape

Epilepsy May Signal Future Dementia Risk

Epilepsy prevalence was significantly higher among people with frontotemporal dementia (FTD) than among those with Alzheimer's disease (AD), a new study showed, with epilepsy occurring in some cases up to 10 years before an FTD diagnosis. METHODOLOGY: In a case-control study in Finland, epilepsy prevalence and antiseizure medicine ( ASM) purchases were compared between 245 patients with FTD (mean age at diagnosis, 65 years; 49% women), more than 1300 patients with AD (mean age, 72 years; 59% women), and more than 2400 individuals matched for age, sex, and location to act as the healthy control group (mean age, 65 years; 49% women). Data were collected from 2010 to 2021, and epilepsy cases were identified using codes from the International Statistical Classification of Diseases and Related Health Problems, 10th Revision (ICD-10). Purchases of ASMs were tracked throughout the study period, and epilepsy prevalence was evaluated at four timepoints: 10 years before the diagnosis of FTD, 5 years before diagnosis, at diagnosis, and 5 years after diagnosis. TAKEAWAY: Both epilepsy prevalence and ASM purchases were significantly greater in patients with FTD than in those in the healthy control group at all timepoints ( P < .001 for both measures). < .001 for both measures). Epilepsy prevalence was significantly higher in patients with FTD than in patients with AD 10 years before ( P = .03), 5 years before ( P = .002), and 5 years after ( P = .05) the diagnosis of FTD. = .03), 5 years before ( = .002), and 5 years after ( = .05) the diagnosis of FTD. The prevalence of ASM use was also significantly higher among patients with FTD than among patients with AD ( P = .004 at 5 years before FTD diagnosis and P < .001 at other timepoints). = .004 at 5 years before FTD diagnosis and < .001 at other timepoints). After adjusting for age at the time of FTD diagnosis, the risk for mortality was not significantly different between patients with both FTD and epilepsy and those without epilepsy. IN PRACTICE: 'It is noteworthy that epilepsy occurred in some patients with FTD already 10 years before their dementia diagnosis, and it was more common in all the examined stages of the disease than previous international studies have reported,' lead investigator Annemari Kilpeläinen, MD, Institute of Clinical Medicine-Neurology, University of Eastern Finland, Kuopio, Finland, said in a press release. The researchers added that 'enhancing knowledge of the comorbidity between epilepsy and FTD could lead to more precise and comprehensive diagnostics and treatment of these conditions as well as new pathophysiologic findings.' SOURCE: The study was published online on June 2 in JAMA Neurology . LIMITATIONS: This study was limited by reliance on ICD-10 codes for classification of epilepsy types and included only two major provinces in Finland. Additionally, epilepsy diagnoses recorded before 1998 may have been missed. DISCLOSURES: This study was part of the Real-World Data Project on Neurodegenerative Diseases, funded by Roche OY. Additional funding was provided by the Kuopio University Hospital, the Finnish Brain Foundation, the Uulo Arhio Foundation, the Finnish Medical Foundation, the Finnish Cultural Foundation, the Wihuri Foundation, and the Sigrid Juselius Foundation. Several investigators reported having ties with various organizations and pharmaceutical companies. Full details are provided in the original article.

What Happens In The Brain Just Before You Die? A Psychologist Explains
What Happens In The Brain Just Before You Die? A Psychologist Explains

Forbes

time5 days ago

  • Health
  • Forbes

What Happens In The Brain Just Before You Die? A Psychologist Explains

Do our lives really flash before our eyes in death? A once-in-a-lifetime brain scan offers a glimpse ... More at what the mind might be doing in our final moments. Death is perhaps the only thing we are promised in life. And yet, despite how natural and inevitable it is, it remains one of the most elusive subjects in modern neuroscience. Of course, we know what death is. We know what it does to the body: how it stills the lungs, quiets the heart and brings all systems to a halt. What we don't know, however, is how the mind behaves in those final moments. For decades, we've relied on folklore and personal accounts of near-death experiences for clues. Some people describe brilliant white lights, others recall feelings of pure peace — and many say they saw nothing at all. But among the most enduring and cross-cultural reports is the idea that your 'life flashes before your eyes,' a phenomenon researchers call a 'life review.' Until recently, this has remained unprovable; half folklore, half hope. But a 2022 study published in Frontiers in Aging Neuroscience caught something remarkable by complete accident: the first-ever glimpse of the brain in the moments just before, and just after, death. In those few seconds, something happened that looks strikingly similar to a life review. This study did not begin with death in mind. Rather, the team of neuroscientists were conducting a routine EEG on an 87-year-old Canadian man with epilepsy. The initial purpose of this scan was to monitor his brain activity, in the hopes of better understanding his seizures. But during the recording, the patient suffered a sudden heart attack. As the authors of the study explained, 'After discussion with the patient's family and in consideration of the Do-Not-Resuscitate (DNR) status of the patient, no further treatment was administered and the patient passed away.' In total, the team of researchers captured approximately 15 minutes of brain activity leading up to, during and just after the patient's death. And, in turn, what was supposed to be standard data collection became something the first known recording of a human brain during the process of dying. This rare and accidental event allowed the researchers to observe, in real time, what happens to the brain as it shuts down. But it was the final 30 seconds before and after the heart patient's heart stopped that something phenomenal happened. 'Just before and after the heart stopped working, we saw changes in a specific band of neural oscillations,' said Dr. Ajmal Zemmar, one of the leading neurosurgeons within the study, in a statement to Frontiers in Psychology. More specifically, the EEG recorded an abnormal surge in gamma waves. These are the highest-frequency among all brainwaves, and they primarily occur within the hippocampus (the brain's memory center). Typically, these oscillations are associated with memory retrieval, dreaming and deep meditation. This spike in gamma wave activity — in tandem with other oscillations such as delta, theta, alpha and beta — created a brainwave pattern that looked remarkably similar to what we see when someone is deeply remembering something, or dreaming vividly. In light of this unprecedented finding, Zemmar suggests that, 'Through generating oscillations involved in memory retrieval, the brain may be playing a last recall of important life events just before we die.' Zemmar notes that this idea — that our brains might perform a kind of memory replay at the end of life — aligns with what thousands of people have described during near-death experiences. In a 2014 study from OMEGA - Journal of Death and Dying, researchers analyzed dozens of these near-death experiences. A consistent theme that emerged was the blurring of 'boundaries between space, time and perception.' The researchers explain that individuals often experience intense feelings of 'joy, happiness, peace, and unconditional love' in these moments. And, often, these are accompanied by something of a panoramic life review. Fascinatingly, researchers believe that humans aren't the only species whose brains seem to do this. Similar brain wave activity has been observed in rats. A 2013 study published in Proceedings of the National Academy of Sciences showed that dying rodents also experience a sharp increase in gamma oscillations just before death. A 2019 study in Frontiers of Cellular Neuroscience supported the same findings, too. Given these cross-species similarities, Zemmar and his colleagues suggest something spectacular: that no matter the species, mammals may spend their last moments in deep mental reverie. That consciousness, in its final seconds, could be universal in its inward retreat. It's also likely that brainwaves aren't the only mechanisms involved in this process. It's widely believed that the brain releases large amounts of dimethyltryptamine (DMT) at the time of death. This naturally occurring psychedelic compound can be found in many plants and animals — and it occurs endogenously within humans. In fact, a 2018 study in Frontiers in Psychology found that people who were administered this hallucinogen reported experiences that were uncannily similar to that of a near-death experience: vivid imagery, feelings of transcendence, timelessness and unparalleled emotional insight. Of course, this scan doesn't prove that life reviews are a definitive aspect of dying, nor that they happen to everyone. It was a single case. The patient had epilepsy, which can influence gamma activity on its own. We don't know whether he was truly reliving memories or simply dreaming. But, while we can't say for certain what exactly the brain is doing when we die, we can speculate that these phenomena — the surge in gamma waves, the potential DMT release, the reports of life reviews, the similar findings in animals — may all point to a final burst of meaning-making. That, even as the body begins to shut down, the mind might still be hard at work to give us one last vivid, beautiful experience. That said, these findings don't diminish the depth of loss, nor do they make grieving any easier in any way. Zemmar himself has spoken openly about how indescribably difficult grief can be. But, in his statement, he leaves us with wisdom that's less scientific, and instead more comforting: 'Something we may learn from this research is: although our loved ones have their eyes closed and are ready to leave us to rest, their brains may be replaying some of the nicest moments they experienced in their lives.' Near-death experiences often give rise to 'ego deaths.' Take this science-backed test to find out if you've experienced one before: Ego Dissolution Scale

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