Latest news with #RettSyndrome
Scotsman
03-07-2025
- Entertainment
- Scotsman
Glasgow mum pays tribute to music therapy charity at star-studded awards bash
Rhiannon met pop star Rick Astley at the awards ceremony. | Rhiannon Lowden The night raised a huge amount of money for a good cause. Sign up to our daily newsletter – Regular news stories and round-ups from around Scotland direct to your inbox Sign up Thank you for signing up! Did you know with a Digital Subscription to The Scotsman, you can get unlimited access to the website including our premium content, as well as benefiting from fewer ads, loyalty rewards and much more. Learn More Sorry, there seem to be some issues. Please try again later. Submitting... A Glasgow mum has spoken in front of a star-studded audience at a glittering awards ceremony that has already raised over £715,000 for Nordoff and Robbins, the UK's largest music therapy charity. Rhiannon Louden took to the stage in front of music royalty - including David Gilmour, Sophie Ellis-Bextor, Mika, The Last Dinner Party, The Corrs, Idles, Tony Christie, Rick Astley and Radiohead's Jonny Greenwood - at the O2 Silver Clef Awards. Advertisement Hide Ad Advertisement Hide Ad She shared the ways that music therapy has helped her three-year-old daughter Poppy who lives with Rett Syndrome - a rare genetic neurological disorder that causes severe physical and mental disabilities. Speaking to the audience at the event at London's JW Marriott Grosvenor House, hosted by Scottish DJ Edith Bowman, Rhiannon said: 'It's more than just therapy. Poppy has found a place, just for her, in a world where so few of them exist. A place where she is understood, where she can engage and communicate with everyone around her. Most importantly we've found hope and pure joy. 'Every child deserves a bright future, and Poppy's is brighter because of music therapy.' Rhiannon onstage at the Nordoff and Robbins O2 Silver Clef Awards 2025. | John Marshall - JM Enternational It was the 49th time the awards have been held, with over £17 million for Nordoff and Robbins since 1976, and David Gilmour paid tribute to the 'wonderful' charity's work while collecting the prestigious O2 Silver Clef Award. Advertisement Hide Ad Advertisement Hide Ad The Pink Floyd star said 'My relationship with them goes back 50 years – thank you to them for this award and for the miraculous work they do, and have done since then.' Funds raised at the event will directly support children and adults across the UK who are autistic, living with dementia, or have learning difficulties, brain injuries, mental health challenges or life-limiting illnesses – helping them to connect and communicate through music. You can find out more about Nordoff and Robbins and how you can support them here.
Yahoo
30-06-2025
- Business
- Yahoo
Cantor Fitzgerald Reiterates 'Overweight' Rating on Taysha (TSHA) for Positive TSHA-102 Clinical Data
Taysha Gene Therapies, Inc. (NASDAQ:TSHA) is one of the 10 best healthcare penny stocks to buy according to analysts. On June 13, Cantor Fitzgerald reiterated its 'Overweight' rating and $13.00 price target for Taysha. An MRI scan of a patient with a brain disorder, depicting the company's successful treatments for central nervous system disorders. According to their research note, three key factors played a key role in the analysts' decision. First, Cantor Fitzgerald's representatives met with Taysha's management team at the International Rett Syndrome Foundation (IRSF) Scientific Meeting. The occasion provided an opportunity for the analysts to fine-tune their outlook for the company. Secondly, the affirmation was based on recent positive Phase 1/2 clinical data and a regulatory update for TSHA-102. TSHA-102 is an adeno-associated virus (AAV)-based gene therapy in development for Rett syndrome. The data presented at the IRSF meeting was a reprise presentation of previously released results. Third, Cantor Fitzgerald noted strong positive sentiment among stakeholders. The sentiment analyzed includes that of investors and key opinion leaders regarding Taysha's developments. However, the analysts were quick to acknowledge that the sentiment was not reflected in the stock's current market valuation. Taysha Gene Therapies, Inc. (NASDAQ:TSHA) is a clinical-stage biotechnology company. It develops AAV-based gene therapies for rare monogenic diseases of the central nervous system. Its lead programs include TSHA-102 for Rett syndrome and TSHA-120 for giant axonal neuropathy, among others, targeting conditions such as CLN1, CLN7, and Angelman syndrome. While we acknowledge the potential of TSHA as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: Goldman Sachs Energy Stocks: 10 Stocks to Buy and 10 Best AI Stocks to Buy According to Billionaire David Tepper. Disclosure: None.
News.com.au
24-06-2025
- Health
- News.com.au
WTF: Treating neurodevelopmental diseases differently – why NTI164 offers new hope
What are ASD and Rett syndrome? How do they affect children and families? Why is there a need for new treatment options in neurodevelopmental care? And what excites Professor Fahey most about NTI164's future? In this interview, Professor Michael Fahey, a leading Paediatric Neurologist and Clinical Geneticist at Monash Medical Centre, joins host Tylah Tully to answer all these questions and more, delving into his experience with Neurotech's (ASX:NTI) NTI164 drug in treating ASD and Rett syndrome. As the first clinician to prescribe NTI164 during its clinical program, Professor Fahey shares powerful patient outcomes and his hope for a treatment that "enables people to be their best" without the side effects of conventional therapies. He explains how NTI164 is showing early promise, with families reporting transformative changes. "People were more alert, more able to participate... Families would tell us that they went out to dinner as a family for the first time," he said. Professor Fahey believe the early clinical success really underscores the real-world impact of the drug could have, beyond clinical metrics. Watch the video to learn more about the NTI164 drug candidate and Professor Fahey's key takeaways from the clinical program. This video was developed in collaboration with Neurotech, a Stockhead advertiser at the time of publishing.
Yahoo
03-06-2025
- 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
03-06-2025
- 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
