Latest news with #Huntington
Associated Press
21 hours ago
- Health
- Associated Press
Novartis, BrainStorm Cell Therapeutics, Neuroplast, Rapa Therapeutics, and Longeveron Driving Innovations
DUBLIN--(BUSINESS WIRE)--Jun 27, 2025-- The 'Gene and Cell Therapies Targeting CNS Disorders Market - A Global and Regional Analysis: Focus on Drug and Region - Analysis and Forecast, 2025-2035" report has been added to offering. Global gene and cell therapies targeting central nervous system (CNS) disorders a market is on track for continued growth, driven by the aging population, and the development of more effective and comfortable solutions. The ongoing advancements in product technology and the expansion of care options will continue to shape the market's future. As demand for gene and cell therapies targeting central nervous system (CNS) disorders therapies rises, both global and regional players will play a key role in meeting the needs of individuals and healthcare systems alike, improving quality of life for people living with incontinence. The gene and cell therapies targeting central nervous system (CNS) disorders are rapidly advancing, offering potential treatments for conditions such as Parkinson's, Alzheimer's, Huntington's disease, and spinal cord injuries. Increasing incidences of CNS disorders one of the major driving factors of this market. The increasing number of patients with neurological disorders such as Alzheimer's, Parkinson's, and multiple sclerosis is creating an urgent need for innovative treatments. This drives demand for gene and cell therapies, as current treatments are often ineffective or only offer temporary relief. One of the significant drivers of the global gene and cell therapies targeting central nervous system (CNS) disorders market is the technological advancements in treatment delivery. The technological innovations in drug delivery systems, such as CRISPR and gene editing, stem cell therapy and AAV vectors for gene delivery. Furthermore, improved precision in treatment technologies such as CRISPR-Cas9 allow for precise editing of genes involved in neurological diseases. This level of precision offers hope for conditions that have a genetic root, such as Huntington's disease or spinal muscular atrophy have contributed to the market's growth. Despite the growth trajectory, several challenges continue to impact the global gene and cell therapies targeting central nervous system (CNS) disorders market. One of the primary challenges is high development and manufacturing costs. The developing and manufacturing gene and cell therapies, especially those targeting CNS disorders, is extremely expensive. The production of viral vectors for gene delivery and the preparation of cell-based therapies are both costly and complex processes. The high costs of developing these therapies can limit accessibility and affordability for patients. Additionally, these high costs can result in delayed market entry and restrictions on patient access, particularly in low- and middle-income countries. Leading players in the global gene and cell therapies targeting central nervous system (CNS) disorders market, such as Novartis, and BrainStorm Cell Therapeutics are continuously innovating to improve the effectiveness and comfort of gene and cell therapies targeting central nervous system (CNS) disorders. These companies are investing heavily in research and development to introduce new, technologically advanced therapies into the market. With a strong emphasis on user-friendly and environmentally sustainable products, these companies are shaping the future of gene and cell therapies targeting central nervous system (CNS) disorders while enhancing their market positions globally. The competitive landscape of the global gene and cell therapies targeting central nervous system (CNS) disorders market is diverse, with numerous players across different regions offering a wide range of products. Regional players and local manufacturers are expected to play an important role in the market's growth, especially as demand increases in emerging markets such as Asia-Pacific. As consumer preferences shift towards more discreet, comfortable, and affordable solutions, the gene and cell therapies targeting central nervous system (CNS) disorders market will continue to evolve, fostering new opportunities for both established and emerging companies. As the gene and cell therapies targeting central nervous system (CNS) disorders market evolves, emerging trends such expansion of cell-based therapies and focuses on personalized or precision medicine. This trend allows treatments to be more specific and effective for individual patients, reducing side effects and improving patient outcomes. Companies Featured Key Topics Covered: Executive Summary 1. Global Gene and Cell Therapies Targeting CNS Disorders Market: Industry Outlook 1.1 Overview 1.2 Regulatory Landscape 1.3 Global Gene and Cell Therapies Targeting CNS Disorders Market, Patent Landscape 1.3.1 By Country 1.3.2 By Year 1.4 Global Gene and Cell Therapies Targeting CNS Disorders Market, Clinical Trials Landscape 1.5 Key Trends 1.6 Market Dynamics 1.6.1 Overview 1.6.1.1 Impact Analysis 1.6.2 Market Drivers 1.6.3 Market Restraints 1.6.4 Market Opportunities 2. Global Gene and Cell Therapies Targeting CNS Disorders Market, by Drugs, $Million, 2023-2035 2.1 Gene Therapy Drugs 2.2 Cell Therapy Drugs 3. Global Gene and Cell Therapies Targeting CNS Disorders, by Region, $Million, 2023-2035 3.1 North America 3.1.1 Key Findings in North America 3.1.2 Market Dynamics 3.1.3 Market Sizing and Forecast 3.1.3.1 North America Gene and Cell Therapies Targeting CNS Disorders Market, By Country 3.1.3.1.1 U.S. 3.1.3.1.2 Canada 3.2 Europe 3.2.1 Key Findings in Europe 3.2.2 Market Dynamics 3.2.3 Market Sizing and Forecast 3.2.3.1 Europe Gene and Cell Therapies Targeting CNS Disorders Market, By Country 3.2.3.1.1 Germany 3.2.3.1.2 U.K. 3.2.3.1.3 France 3.2.3.1.4 Italy 3.2.3.1.5 Spain 3.2.3.1.6 Rest-of-Europe 3.3 Asia-Pacific 3.3.1 Key Findings in Asia-Pacific 3.3.2 Market Dynamics 3.3.3 Market Sizing and Forecast 3.3.3.1 Asia-Pacific Gene and Cell Therapies Targeting CNS Disorders Market, By Country 3.3.3.1.1 Japan 3.3.3.1.2 China 3.3.3.1.3 India 3.3.3.1.4 Rest-of-Asia-Pacific 3.4 Rest-of-the-World 3.4.1 Key Findings in Rest-of-the-World 3.4.2 Market Dynamics 3.4.3 Market Sizing and Forecast 4. Global Gene and Cell Therapies Targeting CNS Disorders Market: Competitive Landscape and Company Profiles 4.1 Competitive Landscape 4.1.1 New Offerings 4.1.2 Mergers and Acquisitions 4.1.3 Partnerships, Alliances, and Business Expansion 4.1.4 Funding Activities 4.1.5 Regulatory Approvals 4.2 Company Profiles 4.2.1 Overview 4.2.2 Product Portfolio 4.2.3 Target Customers 4.2.4 Key Professionals 4.2.5 Analyst View 5. Research Methodology For more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. View source version on CONTACT: Laura Wood, Senior Press Manager [email protected] For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 KEYWORD: INDUSTRY KEYWORD: GENERAL HEALTH NEUROLOGY HEALTH GENETICS PHARMACEUTICAL SOURCE: Research and Markets Copyright Business Wire 2025. PUB: 06/27/2025 09:33 AM/DISC: 06/27/2025 09:33 AM
Scotsman
4 days ago
- Health
- Scotsman
Labour-led cuts plan is shameful badge of dishonour for capital
Angus Robertson at Redhall Walled Garden This week I was pleased to join Edinburgh and Lothian MSPs at Redhall Walled Garden along with Thrive Collective, the partnership of community-based mental health providers that delivers prevention-focused support to people across the city – the very services now facing the axe under Edinburgh's new mental health plans. Sign up to our daily newsletter Sign up Thank you for signing up! Did you know with a Digital Subscription to Edinburgh News, 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... We stood in united and urgent opposition to the Labour-led proposals from the Edinburgh Integration Joint Board (EIJB) to slash almost all community mental health funding in the capital. If these cuts are implemented, Edinburgh could become the only capital city in Europe without local government-funded mental health support. That would be a shameful badge of dishonour for Scotland's capital, and one we must do everything possible to prevent. Advertisement Hide Ad Advertisement Hide Ad Let's be clear: The proposed £2.2 million cut to block-funded mental health contracts would devastate services that meet people where they are – often those with severe mental illness, people living under Compulsory Treatment Orders, or with conditions like Huntington's. Organisations such as Redhall Walled Garden and those within the Thrive Collective have long delivered vital early intervention services, keeping people out of hospital, in employment and engaged with family and community life. Now they are facing an existential threat. What's most disturbing is that this is not an isolated misjudgment, but part of a broader pattern. From Westminster to the City Chambers, Labour appears committed to carrying on the austerity agenda of the Conservatives. Cut quietly, cut deep and only reconsider when public outrage makes the cost too high. It's a cynical, backwards approach to public service and one that is failing the people of Edinburgh. Labour in Edinburgh, backed by their coalition partners, has demonstrated that they are no better than the service-slicing Tories who came before them. The SNP warned about this trajectory and, sadly, those warnings are now being realised. In the EIJB, SNP Councillor Vicky Nicolson, along with only three others, voted against these devastating proposals. Her powerful speech before the vote reminded us that small investments in community support prevent far greater costs – social, human and financial – down the line. Vicky's experience in community-based prevention work shows exactly what we risk losing: support that prevents homelessness, keeps families together, and stops people falling into crisis. But such outcomes are only possible if the resources – the staff, community spaces, third sector organisations – still exist. Once dismantled, these services will not be easily rebuilt. Advertisement Hide Ad Advertisement Hide Ad The consultation process was, frankly, a disgrace. Rushed through in four weeks with a belated and inadequate easy-read version, many participants – including people with learning disabilities – said they could not provide an informed response. Their voices were not heard. Worse still, there has been little attempt to reflect their input in the final plans. The third sector, again, was brought in late and left feeling tokenised, not trusted as the experts they are. Organisations like Health All Round and Big Hearts are among those already bruised by earlier EIJB decisions. Now, the axe swings again. At the very moment when mental health support should be expanding to meet rising demand and cost-of-living pressures, Labour has chosen to retreat. The SNP will continue to fight these short-sighted, harmful cuts – in Edinburgh and in every part of government. We will always stand up for the poorest and most vulnerable, protect prevention and early intervention, and ensure Scotland's capital is a place where mental health support is a right, not a privilege. Angus Robertson is SNP MSP for Edinburgh Central and Constitution, External Affairs and Culture Secretary
Los Angeles Times
17-06-2025
- Health
- Los Angeles Times
Huntington's Disease: Genetics, Symptoms, and Hope for the Future
Huntington's disease (HD) is one of those rare conditions that affects not just the patient but the entire family—medically, emotionally and genetically. This inherited brain disorder causes gradual breakdown of nerve cells especially in the parts of the brain involved in movement, thinking and mood regulation. Although rare, affecting 3 to 7 people per 100,000 globally, its impact is profound and relentless. With no cure in sight, HD is at the center of intense research to understand its molecular roots and develop targeted therapies [1]. Huntington's disease is inherited in an autosomal dominant pattern, meaning if a person inherits one copy of the defective gene they will develop the disease. The culprit is a genetic mutation in the hd gene (also known as the huntingtin gene), where a DNA segment—specifically a CAG trinucleotide repeat—is abnormally expanded. Normally this segment is repeated 10 to 35 times. In HD it's repeated 36 times or more, sometimes even in the 100s [3] [4] [5] [6]. Huntington's disease is caused by a genetic mutation in the huntingtin gene, specifically the HD mutation involving expanded CAG repeats. This expanded repeat leads to the creation of a toxic version of a protein called huntingtin which misfolds and accumulates in brain cells. Over time these protein clumps disrupt cellular function and lead to death of neurons—especially in the striatum and cortex, areas responsible for motor control and cognition [4] [5]. The loss of nerve cells in these regions is what causes the symptoms of Huntington's disease. Different genetic variants can influence the age of onset and progression of the disease. Researchers have identified two distinct disease phases: an early phase where the brain seems to compensate for the damage and a later phase where symptoms are more visible and rapid neurodegeneration [7]. Understanding the disease process at the molecular level is key to developing new treatments. Huntington's disease symptoms usually emerge between 30-50 years and progress over 15-20 years. The classic triad of symptoms includes: The range of disease symptoms includes symptoms of HD and symptoms of Huntington's disease, covering motor, cognitive and behavioral changes throughout the course of the illness. Research in 2025 has shown that changes in how the striatum connects with the hippocampus may explain some of the memory difficulties HD patients experience, especially those involving spatial awareness [10]. Also worth noting are lesser-known symptoms like loss of smell in advanced disease stages [11] and subtle sex-based differences in disease expression. For example, male patients may have lower levels of 17β-estradiol and reduced number of DARPP-32+ neurons, which may influence disease severity [12]. Other symptoms like sleep disturbances and weight loss may also appear as the disease advances. As the disease progresses symptoms worsen over time with increasing severity of movement disorders, cognitive decline and behavioral changes. Motor symptoms especially uncontrolled movements and balance problems can lead to physical injury from falls or accidents, further impacting quality of life. Juvenile Huntington's disease is a rare form that affects children and adolescents, often presenting with unique features like seizures and more rapid progression compared to adult-onset cases. Huntington's disease diagnosis is confirmed by a genetic test showing 36 or more CAG repeats in the HTT gene. However, the diagnostic process also involves: Presymptomatic genetic testing is available for individuals without known family history of HD. While this can provide clarity, it raises ethical challenges, especially around mental health support and family planning [9]. Beyond the faulty gene itself, scientists have found a cascade of biological disruptions that drive HD. Imaging studies show that HD affects brain structure and function, leading to progressive changes in key brain regions and chemical systems: Huntington's disease affects both physical and mental health, leading to movement disorders, cognitive decline and psychiatric symptoms that worsen over time. Each of these changes leads to neuronal death, providing multiple targets for therapeutic intervention. Individuals who inherit the genetic mutation will develop Huntington's disease. There's no cure for HD yet, but several treatments can help manage symptoms: More exciting, however, are the experimental approaches in development: For updates on these trials, you can follow progress via resources like the HD portal, or the European Huntington's Disease Network. People with Huntington's disease benefit from tailored care and support, and their involvement in research and clinical trials is crucial for developing better therapies. Living with Huntington's isn't just about managing symptoms—it's about navigating a complex emotional and social landscape. Many patients grapple with: Family members are often deeply involved in caregiving, emotional support, and making important decisions throughout the course of the disease. Because of this, a team-based approach to care is critical. Neurologists, psychiatrists, genetic counselors, social workers, therapists, and genetic counseling services all play a part in supporting HD patients and their loved ones. A genetic counselor is a healthcare professional who guides patients through the genetic testing process, explains inheritance patterns, and answers questions about the benefits and risks of testing. Huntington's disease is a devastating diagnosis, but the landscape is slowly shifting. Thanks to advances in genetic research, brain imaging, and experimental therapies, we're moving closer to more personalized and effective treatments. Until then, early diagnosis, supportive care, and active participation in research remain key to improving quality of life for those affected by HD. [1] Stoker, T. B., Mason, S. L., Greenland, J. C., Holden, S. T., Santini, H., & Barker, R. A. (2022). Huntington's disease: diagnosis and management. Practical neurology, 22(1), 32–41. [2] Walker F. O. (2007). Huntington's disease. Lancet (London, England), 369(9557), 218–228. [3] Kim, A., Lalonde, K., Truesdell, A., Gomes Welter, P., Brocardo, P. S., Rosenstock, T. R., & Gil-Mohapel, J. (2021). New Avenues for the Treatment of Huntington's Disease. International journal of molecular sciences, 22(16), 8363. [4] Ghosh, R., & Tabrizi, S. J. (2018). Huntington disease. Handbook of clinical neurology, 147, 255–278. [5] McColgan, P., & Tabrizi, S. J. (2018). Huntington's disease: a clinical review. European journal of neurology, 25(1), 24–34. [6] Bates, G. P., Dorsey, R., Gusella, J. F., Hayden, M. R., Kay, C., Leavitt, B. R., Nance, M., Ross, C. A., Scahill, R. I., Wetzel, R., Wild, E. J., & Tabrizi, S. J. (2015). Huntington disease. Nature reviews. Disease primers, 1, 15005. [7] Hong, E. P., MacDonald, M. E., Wheeler, V. C., Jones, L., Holmans, P., Orth, M., Monckton, D. G., Long, J. D., Kwak, S., Gusella, J. F., & Lee, J. M. (2021). Huntington's Disease Pathogenesis: Two Sequential Components. Journal of Huntington's disease, 10(1), 35–51. [8] Wolf, B., Schwarzer, A., Côté, A. L., Hampton, T. H., Schwaab, T., Huarte, E., Tomlinson, C. R., Gui, J., Fisher, J. L., Fadul, C. E., Hamilton, J. W., & Ernstoff, M. S. (2012). Gene expression profile of peripheral blood lymphocytes from renal cell carcinoma patients treated with IL-2, interferon-α and dendritic cell vaccine. PloS one, 7(12), e50221. [9] Rodríguez-Arribas, M., Yakhine-Diop, S. M. S., Pedro, J. M. B., Gómez-Suaga, P., Gómez-Sánchez, R., Martínez-Chacón, G., Fuentes, J. M., González-Polo, R. A., & Niso-Santano, M. (2017). Mitochondria-Associated Membranes (MAMs): Overview and Its Role in Parkinson's Disease. Molecular neurobiology, 54(8), 6287–6303. [10] Glikmann-Johnston, Y., Delagneau, G., Barta, T., Stout, J. C., & Razi, A. (2025). Neural Mechanisms of Object Location Memory in Huntington's Disease. Movement disorders : official journal of the Movement Disorder Society, 10.1002/mds.30232. Advance online publication. [11] Bylsma, F. W., Moberg, P. J., Doty, R. L., & Brandt, J. (1997). Odor identification in Huntington's disease patients and asymptomatic gene carriers. The Journal of neuropsychiatry and clinical neurosciences, 9(4), 598–600. [12] Bode, F. J., Stephan, M., Suhling, H., Pabst, R., Straub, R. H., Raber, K. A., Bonin, M., Nguyen, H. P., Riess, O., Bauer, A., Sjoberg, C., Petersén, A., & von Hörsten, S. (2008). Sex differences in a transgenic rat model of Huntington's disease: decreased 17beta-estradiol levels correlate with reduced numbers of DARPP32+ neurons in males. Human molecular genetics, 17(17), 2595–2609.
Yahoo
17-06-2025
- Business
- Yahoo
Skyhawk Therapeutics Announces First Patient Dosed in Phase 2/3 FALCON-HD Trial of SKY-0515 for Huntington's Disease
SKY-0515 is an oral small molecule designed to reduce the production of both huntingtin (HTT) and PMS1 proteins—two key drivers of HD pathology Initiation of the FALCON-HD trial follows promising Phase 1 results demonstrating up to 72% reduction in HTT mRNA in healthy volunteers The SKY-0515 Phase 1 trial in patients with Huntington's disease reached full enrollment ahead of schedule BOSTON, June 18, 2025 /PRNewswire/ -- Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule therapies designed to modulate critical RNA targets, today announced that the first patient has been dosed in its Phase 2/3 FALCON-HD trial evaluating SKY-0515, an investigational oral RNA splicing modulator for the treatment of Huntington's disease (HD). SKY-0515 is designed to reduce the production of both HTT and PMS1 proteins—two key drivers of HD pathology. In a Phase 1 study in healthy volunteers, SKY-0515 demonstrated dose-dependent HTT mRNA reduction, achieving an average of 72% lowering at the highest dose tested. The compound was generally well tolerated across all doses. Additionally, the Company's Phase 1 trial in HD patients, which began in January 2025, completed enrollment ahead of schedule in March 2025. "Dosing the first patient in our FALCON-HD trial marks a significant milestone in our mission to develop a disease-modifying therapy for Huntington's patients," said Bill Haney, Founder and Chief Executive Officer of Skyhawk Therapeutics. "Building on our compelling Phase 1 data, we are eager to assess SKY-0515's potential to make a meaningful difference in the lives of patients affected by this devastating condition." FALCON-HD is a Phase 2/3 randomized, double-blind, placebo-controlled, dose ranging study to evaluate the pharmacodynamics, safety, and efficacy of SKY-0515 in participants with Stage 2 and early Stage 3 HD. The trial includes multiple sites across Australia and New Zealand. The initial dosing took place at Flinders Medical Centre in Adelaide, Australia. "We are pleased to participate in this important clinical trial and to have dosed the first patient here at Flinders," said Dr. Karyn Boundy, FRACP, Neurologist, Principal Investigator at Flinders Medical Centre. "Given the lack of approved disease-modifying treatments for Huntington's disease, we are hopeful that SKY-0515 could offer a new therapeutic option for patients." "As Skyhawk kicks off their Phase 2/3 FALCON-HD trial in Australia and New Zealand, the international Huntington's community looks forward to expansion worldwide," said Ed Wild, professor of neurology at the University College London. "SKY-0515's unique ability to reduce both HTT and PMS1 could meaningfully enhance therapeutic impact beyond that of lowering HTT alone." About the FALCON-HD TrialFALCON-HD (NCT06873334) is a Phase 2/3 randomized, double-blind, placebo-controlled, dose ranging study to evaluate the pharmacodynamics, safety, and efficacy of SKY-0515 in participants with Stage 2 and early Stage 3 HD. The trial plans to enroll 120 subjects across 10 sites in Australia and New Zealand. Eligible patients will receive a once-daily oral dose of SKY-0515 at one of three dose levels, or placebo, for a treatment period of at least 12 months. The trial aims to assess the potential of SKY-0515 to modulate RNA splicing and reduce the production of huntingtin (HTT) and PMS1 proteins, which are implicated in the pathology of Huntington's disease. Additional information about FALCON-HD, including participating sites and eligibility criteria, can be found at and About SKY-0515SKY-0515 is an orally administered small molecule RNA splicing modulator developed through Skyhawk's proprietary platform. It is designed to reduce production of both huntingtin (HTT) and PMS1 proteins, two key contributors to Huntington's disease. SKY-0515 has shown robust, dose-dependent HTT mRNA reduction—up to 72%—in healthy volunteer studies, with favorable safety and tolerability. SKY-0515 is currently being evaluated in a Phase 2/3 clinical trial. About Skyhawk TherapeuticsSkyhawk Therapeutics is a clinical-stage biotechnology company focused on the discovery and development of novel small molecule therapies designed to modulate critical RNA targets and revolutionize patient treatment for some of the world's most intractable diseases. Skyhawk's discovery expertise is rooted in its proprietary drug discovery platform, which assesses, identifies, and tests RNA splicing targets and small molecules across a broad range of therapeutic areas and disease states. Skyhawk has built collaborations with multiple pharma partners that leverage Skyhawk's novel platform across disease areas including neurodegenerative disease, autoimmune disease, and oncology. For more information visit Skyhawk ContactsKyle Dow, VP Corporate Logo - View original content: SOURCE Skyhawk Therapeutics 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
Business Wire
16-06-2025
- Business
- Business Wire
PTCT Investors Have Opportunity to Join PTC Therapeutics, Inc. Fraud Investigation with the Schall Law Firm
LOS ANGELES--(BUSINESS WIRE)-- The Schall Law Firm, a national shareholder rights litigation firm, announces that it is investigating claims on behalf of investors of PTC Therapeutics, Inc. ('PTC' or 'the Company') (NASDAQ: PTCT) for violations of the securities laws. The investigation focuses on whether the Company issued false and/or misleading statements and/or failed to disclose information pertinent to investors. PTC issued a press release on May 5, 2025, 'announc[ing] results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients.' According to the Company, 'the study met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels (p<0.0001) at Week 12 and favorable safety and tolerability.' However, analysts who reviewed the data believe that the Company would need to conduct a Phase 3 study to determine if the treatment actually slows HD. Based on this news, shares of PTC fell by more than 18.6% on the same day. If you are a shareholder who suffered a loss, click here to participate. We also encourage you to contact Brian Schall of the Schall Law Firm, 2049 Century Park East, Suite 2460, Los Angeles, CA 90067, at 310-301-3335, to discuss your rights free of charge. You can also reach us through the firm's website at or by email at bschall@ The Schall Law Firm represents investors around the world and specializes in securities class action lawsuits and shareholder rights litigation. This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and rules of ethics.
