Latest news with #REVEAL
Yahoo
5 days ago
- Business
- Yahoo
Taysha Gene Therapies, Inc. (TSHA)'s TSHA-102 Shows Promising Results in Rett Syndrome Patients Aged 6+
Taysha Gene Therapies, Inc. (NASDAQ:TSHA) has announced pivotal progress in its TSHA-102 gene therapy program for Rett syndrome, following written alignment with the FDA on a single-arm, open-label pivotal trial targeting patients aged six and older. This population, shown by natural history data to have virtually zero chance of regaining developmental milestones, saw a 100% responder rate in the REVEAL Part A trial: all 10 treated patients gained or regained at least one developmental milestone post-TSHA-102, with high-dose recipients showing faster and deeper improvements. No serious adverse events or dose-limiting toxicities were reported. A scientist holding a Petri dish filled with a gene therapy sample. Taysha Gene Therapies, Inc. (NASDAQ:TSHA)'s pivotal Part B trial, expected to launch in Q3 2025, will enroll 15 females in the developmental plateau stage, using each patient as their own control and video-based, blinded milestone assessment as the primary endpoint. The FDA's support, based on robust longitudinal data from over 1,100 Rett patients, marks a major regulatory milestone. TSHA-102, a one-time AAV9 gene therapy delivering functional MECP2, is designed to address the genetic root cause of Rett syndrome, a devastating disorder with no current disease-modifying treatments. Taysha Gene Therapies, Inc. (NASDAQ:TSHA) will present further details at the IRSF Rett Syndrome Scientific Meeting in June. While we acknowledge the potential of TSHA to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than TSHA and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None. 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
Yahoo
5 days ago
- Business
- Yahoo
Taysha Gene Therapies Announces Details for Oral Presentations at the 2025 IRSF Rett Syndrome Scientific Meeting Reviewing Recent Updates from the TSHA-102 Clinical Program
Recently disclosed clinical cohort data from high (1x1015 total vg) and low dose (5.7x1014 total vg) TSHA-102 from REVEAL adolescent/adult and pediatric Phase 1/2 trials Caregiver research regarding gain/regain of developmental milestones supporting alignment with FDA on primary endpoint in the pivotal Part B trial of TSHA-102 Previously disclosed preclinical data supporting broad biodistribution across brain and spinal cord regions following lumbar intrathecal delivery of AAV9 gene therapy vectors in non-human primates Symposium on Rett syndrome natural history data findings DALLAS, June 03, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced details for three oral presentations reviewing recent updates supporting its TSHA-102 program in clinical evaluation for Rett syndrome, and a Taysha-hosted symposium on the Company's analysis of the natural history study data at the 2025 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting, taking place in Boston from June 9-11, 2025. The three presentations will also be presented during a poster session on Monday, June 9, 2025, from 5:00-7:00 PM EST. Additional details on the meeting can be found at IRSF's website. 'We are excited to present data supporting our TSHA-102 clinical program and its potential to improve function or enable achievement of developmental milestones that would significantly improve quality of life. Importantly, these findings have increased our conviction in the differentiated potential of TSHA-102 to address the high unmet needs for patients and families suffering from this devastating disease,' said Sukumar Nagendran, M.D., President and Head of Research & Development at Taysha. 'We appreciate IRSF and their ongoing partnership, as well as the individuals with Rett syndrome, their caregivers and the clinicians who contributed to the important research that has increased our understanding of the disease progression and supported our alignment with the Food and Drug Administration (FDA) on a potential path to registration.' 2025 IRSF Rett Syndrome Scientific Meeting Oral Presentation Details Title: REVEAL Adolescent/Adult and Pediatric Clinical Trial Update: Safety and Efficacy Data on TSHA-102 AAV9 Investigational Gene Therapy in Clinical Evaluation for Rett Syndrome Date/Time: Wednesday, June 11, 2025, 9:30 AM ESTSession: Industry UpdatesPresenters: Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor in Neuroscience and Pediatrics at the Université de Montréal, Director of the Rett Multidisciplinary Clinic of the CHU Sainte-Justine and Principal Investigator of the REVEAL Adolescent/Adult and Pediatric trials Title: Every Gain, Expanding Possibilities: Caregiver Insights on Meaningful Improvement in Rett Syndrome Gene Therapy (GT)Date/Time: Tuesday, June 10, 2025, 12:20 PM ESTSession: Biomarkers and Outcome MeasuresPresenters: Jenny Downs, MSc Ph.D., Program Head, Development and Disability at The Kids Research Institute Australia Title: rAAV9 Vector Biodistribution in Brain and Spinal Cord via Lumbar Intrathecal Infusion in Nonnhuman Primates (NHP): Assessing the Administration Route Leveraged in TSHA-102 Rett Syndrome Clinical Trials Date/Time: Tuesday, June 10, 2025, 10:10 AM ESTSession: Model Systems Presenters: Fred Porter, Ph.D., Chief of Staff and Technical Operations Officer of Taysha Taysha-Hosted Symposium: Gain and Regain of Developmental Milestones: How Natural History Insights are Redefining the Therapeutic Development for Rett SyndromeDate/Time: Tuesday, June 10, 2025, 7:30 AM ESTPresenter: Jeffrey Neul, M.D., Ph.D., Director, Vanderbilt Kennedy Center, Annette Schaffer Eskind Chair, Professor at the Vanderbilt University Medical Center, who served as Administrative Head of the Rett Syndrome Natural History Study About TSHA-102TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the European Commission and Innovative Licensing and Access Pathway designation from the Medicines and Healthcare products Regulatory Agency. About Rett SyndromeRett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K. About Taysha Gene TherapiesTaysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company's management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as 'anticipates,' 'believes,' 'expects,' 'intends,' 'projects,' 'plans,' and 'future' or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of TSHA-102, including the reproducibility and durability of any favorable results initially seen in patients dosed to date in clinical trials, including with respect to functional milestones, and our other product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, including the timing of initiating additional trials, reporting data from our clinical trials and making regulatory submissions, communications from the FDA on the regulatory pathway for TSHA-102, and the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2024, and our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, which are available on the SEC's website at Additional information will be made available in other filings that we make from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law. Company Contact:Hayleigh Collins Senior Director, Corporate Communications and Investor Relations Taysha Gene Therapies, Media Contact:Carolyn HawleyInizio 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
Yahoo
5 days ago
- Business
- Yahoo
Taysha Gene Therapies Announces Details for Oral Presentations at the 2025 IRSF Rett Syndrome Scientific Meeting Reviewing Recent Updates from the TSHA-102 Clinical Program
Recently disclosed clinical cohort data from high (1x1015 total vg) and low dose (5.7x1014 total vg) TSHA-102 from REVEAL adolescent/adult and pediatric Phase 1/2 trials Caregiver research regarding gain/regain of developmental milestones supporting alignment with FDA on primary endpoint in the pivotal Part B trial of TSHA-102 Previously disclosed preclinical data supporting broad biodistribution across brain and spinal cord regions following lumbar intrathecal delivery of AAV9 gene therapy vectors in non-human primates Symposium on Rett syndrome natural history data findings DALLAS, June 03, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced details for three oral presentations reviewing recent updates supporting its TSHA-102 program in clinical evaluation for Rett syndrome, and a Taysha-hosted symposium on the Company's analysis of the natural history study data at the 2025 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting, taking place in Boston from June 9-11, 2025. The three presentations will also be presented during a poster session on Monday, June 9, 2025, from 5:00-7:00 PM EST. Additional details on the meeting can be found at IRSF's website. 'We are excited to present data supporting our TSHA-102 clinical program and its potential to improve function or enable achievement of developmental milestones that would significantly improve quality of life. Importantly, these findings have increased our conviction in the differentiated potential of TSHA-102 to address the high unmet needs for patients and families suffering from this devastating disease,' said Sukumar Nagendran, M.D., President and Head of Research & Development at Taysha. 'We appreciate IRSF and their ongoing partnership, as well as the individuals with Rett syndrome, their caregivers and the clinicians who contributed to the important research that has increased our understanding of the disease progression and supported our alignment with the Food and Drug Administration (FDA) on a potential path to registration.' 2025 IRSF Rett Syndrome Scientific Meeting Oral Presentation Details Title: REVEAL Adolescent/Adult and Pediatric Clinical Trial Update: Safety and Efficacy Data on TSHA-102 AAV9 Investigational Gene Therapy in Clinical Evaluation for Rett Syndrome Date/Time: Wednesday, June 11, 2025, 9:30 AM ESTSession: Industry UpdatesPresenters: Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor in Neuroscience and Pediatrics at the Université de Montréal, Director of the Rett Multidisciplinary Clinic of the CHU Sainte-Justine and Principal Investigator of the REVEAL Adolescent/Adult and Pediatric trials Title: Every Gain, Expanding Possibilities: Caregiver Insights on Meaningful Improvement in Rett Syndrome Gene Therapy (GT)Date/Time: Tuesday, June 10, 2025, 12:20 PM ESTSession: Biomarkers and Outcome MeasuresPresenters: Jenny Downs, MSc Ph.D., Program Head, Development and Disability at The Kids Research Institute Australia Title: rAAV9 Vector Biodistribution in Brain and Spinal Cord via Lumbar Intrathecal Infusion in Nonnhuman Primates (NHP): Assessing the Administration Route Leveraged in TSHA-102 Rett Syndrome Clinical Trials Date/Time: Tuesday, June 10, 2025, 10:10 AM ESTSession: Model Systems Presenters: Fred Porter, Ph.D., Chief of Staff and Technical Operations Officer of Taysha Taysha-Hosted Symposium: Gain and Regain of Developmental Milestones: How Natural History Insights are Redefining the Therapeutic Development for Rett SyndromeDate/Time: Tuesday, June 10, 2025, 7:30 AM ESTPresenter: Jeffrey Neul, M.D., Ph.D., Director, Vanderbilt Kennedy Center, Annette Schaffer Eskind Chair, Professor at the Vanderbilt University Medical Center, who served as Administrative Head of the Rett Syndrome Natural History Study About TSHA-102TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the European Commission and Innovative Licensing and Access Pathway designation from the Medicines and Healthcare products Regulatory Agency. About Rett SyndromeRett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K. About Taysha Gene TherapiesTaysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company's management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as 'anticipates,' 'believes,' 'expects,' 'intends,' 'projects,' 'plans,' and 'future' or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of TSHA-102, including the reproducibility and durability of any favorable results initially seen in patients dosed to date in clinical trials, including with respect to functional milestones, and our other product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, including the timing of initiating additional trials, reporting data from our clinical trials and making regulatory submissions, communications from the FDA on the regulatory pathway for TSHA-102, and the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2024, and our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, which are available on the SEC's website at Additional information will be made available in other filings that we make from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law. Company Contact:Hayleigh Collins Senior Director, Corporate Communications and Investor Relations Taysha Gene Therapies, Media Contact:Carolyn HawleyInizio
Yahoo
29-05-2025
- Business
- Yahoo
Taysha Gene Therapies, Inc. (TSHA)'s TSHA-102 Shows Promising Results in Rett Syndrome Patients Aged 6+
Taysha Gene Therapies, Inc. (NASDAQ:TSHA) has announced pivotal progress in its TSHA-102 gene therapy program for Rett syndrome, following written alignment with the FDA on a single-arm, open-label pivotal trial targeting patients aged six and older. This population, shown by natural history data to have virtually zero chance of regaining developmental milestones, saw a 100% responder rate in the REVEAL Part A trial: all 10 treated patients gained or regained at least one developmental milestone post-TSHA-102, with high-dose recipients showing faster and deeper improvements. No serious adverse events or dose-limiting toxicities were reported. A scientist holding a Petri dish filled with a gene therapy sample. Taysha Gene Therapies, Inc. (NASDAQ:TSHA)'s pivotal Part B trial, expected to launch in Q3 2025, will enroll 15 females in the developmental plateau stage, using each patient as their own control and video-based, blinded milestone assessment as the primary endpoint. The FDA's support, based on robust longitudinal data from over 1,100 Rett patients, marks a major regulatory milestone. TSHA-102, a one-time AAV9 gene therapy delivering functional MECP2, is designed to address the genetic root cause of Rett syndrome, a devastating disorder with no current disease-modifying treatments. Taysha Gene Therapies, Inc. (NASDAQ:TSHA) will present further details at the IRSF Rett Syndrome Scientific Meeting in June. While we acknowledge the potential of TSHA to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than TSHA and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None. 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
Techday NZ
21-04-2025
- Business
- Techday NZ
Gurucul launches self-driving SIEM to automate security ops
Gurucul has announced the introduction of a Self-Driving SIEM powered by advanced AI capabilities, aiming to streamline and enhance security operations through automation and improved workflows. The latest upgrade to Gurucul's Unified Data and Security Analytics Platform, REVEAL, incorporates multiple AI agents across the entire threat management lifecycle. These autonomous features are designed to reduce engineering, maintenance, and operational tasks, providing what Gurucul describes as a "self-driving" SIEM that enables engineers, analysts, and investigators to focus on higher-value activities while AI manages repetitive work. Gurucul's Data Optimiser now includes new pipeline AI agents that autonomously discover, classify, normalise and filter data. According to the company, these features help customers control and optimise their security data fabric, with smart filtering reducing storage and processing costs. The reliance on manual data management is minimised, encompassing onboarding and tuning, which is intended to reduce costs by at least 40%. AI agents within the platform have been developed to detect evolving attack chains independently. This allows them to inform new machine learning detection models, signatures and rules, proactively optimising existing models and recommending additional data sources for expanded detection use cases. These AI-driven processes support real-time content creation of models and rules. The platform also introduces an AI virtual analyst to provide security teams with expert guidance and contextual insights. By automatically triaging alerts and appending relevant information, such as attack blast radius, MITRE ATT&CK framework alignment, and external threat intelligence, Gurucul's system aims to reduce analysts' workload. The AI component benefits from adaptive learning, refining its outputs based on historical data, prior detections and analyst feedback. Natural Language Processing (NLP) capabilities have been integrated to enable more efficient searching and content creation, including incident reports. AI-powered orchestration and response have also been upgraded, allowing the platform to dynamically modify and execute response playbooks based on real-time threat data. This is intended to ensure that automated incident response remains tailored to the specific policy and threat characteristics of each incident. Gurucul's Sme AI copilot, originally launched in August 2023, has received several updates, including advanced prompts, new prompt books, enhanced natural language search functions, and improved incident analysis and reporting. These upgrades are meant to provide deeper insights with reduced investigation times. Neda Pitt, Chief Information Security Officer at BELK, stated, "This is yet another reason why I chose to replace my legacy SIEM with the Gurucul platform. Even before these AI enhancements, the platform outperformed any other SIEM I've encountered. Now, with these Agentic AI capabilities, I'm glad I made the bet on the future of the industry. Gurucul is paving the path toward the autonomous SOC. My analysts have upleveled their productivity, and I can't wait for them to get to the next level with AI removing the busy work." Stewart Alpert, Chief Information Security Officer and Chief Technology Officer at Hornblower, added, "Gurucul has redefined what I expect from an AI-powered Next-Gen SIEM. It operates as a true force multiplier for my SOC — prioritised alerts, high-efficacy detections deeply aligned with our threat posture, and proactive responses. Another capability that truly stands apart is in its risk-based approach to identity: the precision with which they score and surface risky users has become a cornerstone of our insider threat programme." Jason Elmore, Chief Executive Officer at Tuearis Cyber, commented, "As an MSSP, we are always looking for ways to maximise value for our customers while optimising the workflows for our analysts and reducing costs. We picked Gurucul because they provide a truly differentiated platform that not only helped reduce our data management costs leveraging Snowflake but also helped us stay ahead of the threat landscape with advanced detection capabilities. It's innovations like these new Agentic AI capabilities that validate our decision to switch to Gurucul. They continue to offer modernisation that increases value delivery for our customers while maximising the output of our SOC." Saryu Nayyar, Chief Executive Officer at Gurucul, said, "Alert overload, the sophistication of threats and operational bottlenecks are some of the most pressing challenges in security operations today. We have built a small army of Agentic AI agents that go to work for you across the entire data and threat lifecycle, boosting analyst workflows to address these critical pain points." "We are flipping the idea of the resource-intensive traditional SIEM on its head to optimise SecOps resources and reduce time spent on data management, detection engineering, false positives, triage, investigation and response. We're continuing to disrupt the status quo, set the bar high, and solve real customer problems. These ground-breaking advancements with purpose-built AI use cases are helping SOC teams do their critical work efficiently with swift responses against modern threats." Gurucul's platform upgrade, including the enhanced Sme AI copilot and Agentic AI multi-agent workflows, has been made available since December 2024 as part of release v12.4.
