Latest news with #Huntington'sDisease
Yahoo
3 days ago
- Business
- Yahoo
Huntington's Disease Clinical Trial Pipeline Analysis: 20+ Key Companies Shaping the Future of Dopamine Receptor Antagonists Therapeutics
The Huntington's disease treatment market is poised for significant growth, driven by rising prevalence rates and increased awareness globally. Advancements in gene-targeting and disease-modifying therapies, along with a robust clinical pipeline, are addressing critical unmet needs. Active government support and educational initiatives are further enhancing diagnosis and treatment uptake. Together, these factors create a strong foundation for market expansion in the coming years. New York, USA, June 03, 2025 (GLOBE NEWSWIRE) -- Huntington's Disease Clinical Trial Pipeline Analysis: 20+ Key Companies Shaping the Future of Dopamine Receptor Antagonists Therapeutics | DelveInsight The Huntington's disease treatment market is poised for significant growth, driven by rising prevalence rates and increased awareness globally. Advancements in gene-targeting and disease-modifying therapies, along with a robust clinical pipeline, are addressing critical unmet needs. Active government support and educational initiatives are further enhancing diagnosis and treatment uptake. Together, these factors create a strong foundation for market expansion in the coming years. DelveInsight's 'Huntington's Disease Pipeline Insight 2025' report provides comprehensive global coverage of pipeline Huntington's disease therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Huntington's disease pipeline domain. Key Takeaways from the Huntington's Disease Pipeline Report DelveInsight's Huntington's disease pipeline report depicts a robust space with 20+ active players working to develop 20+ pipeline Huntington's disease drugs. Key Huntington's disease companies such as Hoffmann-La Roche, Medibiofarma, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, Skyhawk Therapeutics, BPG Bio, and others are evaluating new Huntington's disease drugs to improve the treatment landscape. Promising pipeline Huntington's disease therapies, such as RG6042, MBF 015, PTC518, ANX-005, ALN-HTT02, NP001, SKY 0515, Research Programme: HDD, and others are in different phases of Huntington's disease clinical trials. In April 2025, the FDA granted a breakthrough therapy designation to AMT-130 (uniQuire) for the treatment of Huntington's disease. Previously, AMT-130 was also granted a regenerative medicine advanced therapy designation and orphan drug designation for this indication. In April 2025, Prilenia Therapeutics announced that it had entered into a collaboration and license agreement with Ferrer for the development and commercialization of pridopidine in Europe and other select markets. In March 2025, Skyhawk Therapeutics presented their novel SKY-0515 small molecule RNA splicing modulator targeting Huntington's Disease to members of the Huntington's Disease Youth Organization (HDYO), at the biannual HYDO International Congress in Prague, Czech Republic. In December 2024, Novartis announced that it had entered into a global licensing and collaboration agreement with PTC Therapeutics for PTC518, an HTT mRNA splice modulator for the treatment of Huntington's disease. Under the terms of the agreement, Novartis will pay USD 1 billion upfront and up to USD 1.9 billion in development, regulatory, and sales milestones. Novartis will also share profits in the US and pay tiered royalties on sales outside the US. In September 2024, PTC Therapeutics announced that the FDA had granted Fast Track designation to the PTC518 program for the treatment of Huntington's disease. In September 2024, the European Medicines Agency (EMA) accepted the marketing authorization application (MAA) for Prilenia Therapeutics' pridopidine for the treatment of adults with Huntington's disease. Request a sample and discover the recent advances in Huntington's disease drugs @ Huntington's Disease Pipeline Report The Huntington's disease pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage Huntington's disease drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the Huntington's disease clinical trial landscape. Huntington's Disease Overview Huntington's disease is a hereditary, progressive neurodegenerative condition marked by the gradual onset of involuntary muscle movements—affecting the hands, feet, face, and trunk—and a steady decline in cognitive abilities and memory. It impacts the central region of the brain, leading to difficulties in movement, emotional regulation, and cognitive functions. Symptoms often appear between ages 30 and 50 but can emerge as early as age 2 or as late as 80. A hallmark feature of the disease is the presence of rapid, uncontrolled movements such as muscle jerks or tics. As the condition advances, patients may experience loss of coordination, speech difficulties, memory deterioration, and worsening choreiform movements, along with personality and behavioral changes. Huntington's disease follows an autosomal dominant inheritance pattern, meaning only one copy of the faulty gene is needed to cause the disease. It is triggered by a mutation in a single gene on chromosome 4, which encodes the huntingtin protein. While the exact role of huntingtin remains unclear, its mutated form disrupts normal brain function, leading to involuntary movements, severe cognitive decline, and emotional disturbances such as depression and irritability. The mutation involves an expansion of a CAG repeat segment within the gene. Typically, this segment repeats 17 to 20 times in a healthy gene, but in Huntington's disease, the number of repeats rises to 40 or more. Diagnosing Huntington's disease involves a comprehensive clinical assessment, patient history, and several specialized tests. These may include blood work, genetic testing to detect mutations in the HTT gene, and brain imaging techniques such as CT scans, MRI, and EEG. CT and MRI provide detailed cross-sectional images of the brain, while EEG measures the brain's electrical activity to support diagnosis. Currently, there is no cure for Huntington's disease, and treatment is aimed at alleviating symptoms. The only two FDA-approved medications—Austedo and Xenazine—are indicated specifically for managing chorea related to Huntington's. Additional symptom management may include antidepressants, antipsychotics, and mood stabilizers to address psychiatric symptoms. Supportive therapies and multidisciplinary care are also critical in improving the quality of life for individuals affected by this out more about Huntington's disease drugs @ Huntington's Disease Treatment A snapshot of the Pipeline Huntington's Disease Drugs mentioned in the report: Drugs Company Phase MoA RoA MBF 015 Medibiofarma II HDAC1/C2 inhibitors Oral PTC518 PTC Therapeutics II HD protein inhibitors; RNA splicing modulators Oral ANX-005 Annexon II Complement C1 inhibitors Intravenous ALN-HTT02 Alnylam Pharmaceuticals I RNA interference Intrathecal NP001 Neuvivo I Macrophage modulators Unspecified SKY 0515 Skyhawk Therapeutics I RNA splicing modulators Oral Learn more about the emerging Huntington's disease therapies @ Huntington's Disease Clinical Trials Huntington's Disease Therapeutics Assessment The Huntington's disease pipeline report proffers an integral view of the emerging Huntington's disease therapies segmented by stage, product type, molecule type, route of administration, and mechanism of action. Scope of the Huntington's Disease Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Intra-articular, Intraocular, Intrathecal, Intravenous, Oral, Parenteral, Subcutaneous, Topical, Transdermal Therapeutics Assessment By Molecule Type: Oligonucleotide, Peptide, Small molecule Therapeutics Assessment By Mechanism of Action: HDAC1/C2 inhibitors, RNA interference, HD protein inhibitors, RNA splicing modulators, Macrophage modulators, Complement C1 inhibitors Key Huntington's Disease Companies: Hoffmann-La Roche, Medibiofarma, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, Skyhawk Therapeutics, BPG Bio, and others. Key Huntington's Disease Pipeline Therapies: RG6042, MBF 015, PTC518, ANX-005, ALN-HTT02, NP001, SKY 0515, Research Programme: HDD, and others. Dive deep into rich insights for new Huntington's disease treatments, visit @ Huntington's Disease Drugs Table of Contents 1. Huntington's Disease Pipeline Report Introduction 2. Huntington's Disease Pipeline Report Executive Summary 3. Huntington's Disease Pipeline: Overview 4. Analytical Perspective In-depth Commercial Assessment 5. Huntington's Disease Clinical Trial Therapeutics 6. Huntington's Disease Pipeline: Late-Stage Products (Pre-registration) 7. Huntington's Disease Pipeline: Late-Stage Products (Phase III) 8. Huntington's Disease Pipeline: Mid-Stage Products (Phase II) 9. Huntington's Disease Pipeline: Early-Stage Products (Phase I) 10. Huntington's Disease Pipeline Therapeutics Assessment 11. Inactive Products in the Huntington's Disease Pipeline 12. Company-University Collaborations (Licensing/Partnering) Analysis 13. Key Companies 14. Key Products in the Huntington's Disease Pipeline 15. Unmet Needs 16. Market Drivers and Barriers 17. Future Perspectives and Conclusion 18. Analyst Views 19. Appendix For further information on the Huntington's disease pipeline therapeutics, reach out @ Huntington's Disease Therapeutics Related Reports Huntington's Disease Epidemiology Forecast Huntington's Disease Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted Huntington's disease epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Huntington's Disease Market Huntington's Disease Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Huntington's disease companies, including Prilenia Therapeutics, Neurocrine Biosciences, SOM Biotech, Annexon Biosciences, Vaccinex, Sage Therapeutics, UniQure Biopharma, Wave life sciences, Takeda, Medesis Pharma, among others. Wilson Disease Market Wilson Disease Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Wilson disease companies, including Alexion Pharmaceuticals, Vivet Therapeutics, Orphalan, among others. Wilson Disease Pipeline Wilson Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Wilson disease companies, including Alexion Pharmaceuticals, Vivet Therapeutics, Orphalan, among others. Bipolar Disorder Pipeline Bipolar Disorder Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key bipolar disorder companies, including Vanda Pharmaceuticals, Pear Therapeutics, Sunovion, Lyndra Therapeutics, among others. DelveInsight's Pharma Competitive Intelligence Service: Through its CI solutions, DelveInsight provides its clients with real-time and actionable intelligence on their competitors and markets of interest to keep them stay ahead of the competition by providing insights into the latest therapeutic area-specific/indication-specific market trends, in emerging drugs, and competitive strategies. These services are tailored to the specific needs of each client and are delivered through a combination of reports, dashboards, and interactive presentations, enabling clients to make informed decisions, mitigate risks, and identify opportunities for growth and expansion. Other Business Consulting Services Healthcare Conference Coverage Pipeline Assessment Healthcare Licensing Services Discover how a mid-pharma client gained a level of confidence in their soon-to-be partner for manufacturing their therapeutics by downloading our Due Diligence Case Study About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. Connect with us at LinkedIn CONTACT: Contact Us Shruti Thakur info@ +14699457679 in to access your portfolio
Irish Independent
15-05-2025
- Health
- Irish Independent
Huntington's Disease: ‘There's been so much progress in terms of scientific research – we've been bringing it out of the shadows'
Today is Huntington's Disease awareness day. A family history of the disease prompted war correspondent Charles Sabine to get tested, and he now campaigns to challenge stigma and work towards a treatment Today at 21:30 In January 1994, 34-year-old Emmy-winning NBC war correspondent Charles Sabine was about to fly to Prague to cover a story when he received a phone call that would change his life. His father, John, had tested positive for the gene responsible for Huntington's disease (HD), meaning Sabine and his older brother, also called John, had a 50-50 chance of inheriting the gene and developing Huntington's disease, a degenerative neurological disorder for which there is no cure. According to the HD Association of Ireland, 'HD is caused by an alteration in the huntingtin (HTT) gene on chromosome 4'. This alteration results in a 'harmful protein called mutant huntingtin' which causes cells in the brain to die, affecting 'movement, thinking and emotion', with symptoms worsening over time. It generally affects people between the ages of 35 to 50 but can occur later, or in the case of juvenile Huntington's, earlier.
Associated Press
01-05-2025
- Business
- Associated Press
DEA Unconstitutional Marijuana Hearing - MMJ to File Emergency Injunction and Suit for Irreparable Harm
'We are not just challenging DEA policy, we are defending the Constitution said Duane Boise, CEO of MMJ BioPharma Cultivation. The DEA cannot sidestep Supreme Court precedent and force us into a hearing. The agency is running a closed loop of power, Boise added, they investigate, prosecute, judge and override all in-house. That's not justice, that's a rigged game.' WASHINGTON, DC / ACCESS Newswire / May 1, 2025 / MMJ BioPharma Cultivation announced today that it will file for an emergency motion for injunctive relief in the U.S. District Court for the District of Rhode Island following a controversial ruling by a DEA Administrative Law Judge (ALJ) to vacate a previously granted stay, thereby green lighting a hearing before a DEA constitutionally flawed tribunal. The company's forthcoming lawsuit will seek to block the DEA's administrative hearing process, which MMJ contends violates the Supreme Court's ruling in Axon Enterprise, Inc. v. FTC, and represents irreparable harm to its constitutional rights. Axon and the Constitutional Crisis The Axon decision, handed down by the U.S. Supreme Court in 2023, established that federal courts may hear structural constitutional challenges to administrative agencies without requiring parties to exhaust agency proceedings first. MMJ argues that the DEA's internal administrative process-led by ALJs who are unconstitutionally insulated from presidential removal-defies this ruling. 'We are not just challenging policy; we are defending the Constitution,' said Duane Boise, CEO of MMJ BioPharma Cultivation. 'The DEA cannot sidestep Supreme Court precedent and force us into a hearing that, by their own partial admission, is unconstitutional.' From Delay to Damage MMJ has been seeking DEA approval since 2018 to manufacture pharmaceutical-grade cannabis for FDA clinical trials aimed at treating Huntington's Disease and Multiple Sclerosis. Despite: ...MMJ has waited more than 2,300 days, well beyond the 60-day review period mandated by the Medical Marijuana and Cannabidiol Research Expansion Act (MCREA). Now, with the DEA insisting on proceeding with a hearing before an ALJ system MMJ contends is structurally unconstitutional, the company is seeking judicial intervention to prevent what it sees as an 'illegal and biased process.' Alleged Irreparable Harm The emergency injunction will argue that forcing MMJ to participate in this proceeding now would cause: 'This is a direct threat to the right of every American business to a fair process under the law,' Boise added. 'We will not submit to an unconstitutional tribunal.' What's Next MMJ's legal team plans to file the emergency motion and accompanying complaint in the coming days. The action seeks: MMJ's leadership emphasized that this fight is not just about one company-but about accountability at the highest levels of government. 'We are going to federal court to say enough is enough,' said Boise. 'It's time the DEA follows the law-not rewrites it.' MMJ is represented by attorney Megan Sheehan. CONTACT: Madison Hisey [email protected] 203-231-8583 SOURCE: MMJ International Holdings press release
South China Morning Post
28-04-2025
- Health
- South China Morning Post
Scientists in Hong Kong offer new hope into treatment of Huntington's Disease
A research team at the Chinese University of Hong Kong (CUHK) has discovered a new underlying mechanism and possible therapeutic target for Huntington's Disease, a rare, incurable disease that can cause twitching movements and cognitive decline. Advertisement The team of the School of Life Sciences at CUHK revealed on Monday that they had found a 4.5-fold increase in the level of a protein called PAPD5 in the brains of patients with Huntington's Disease compared to healthy individuals, which contributed to neuronal apoptosis, or nerve cell death. Their research showed that blocking the activity of PAPD5 could reduce these harmful effects, opening up new possibilities for developing medication. The research paper was published in the international journal Nature Communications on April 9. 'We provide a new angle to understand the underlying mechanism. This PAPD5-mediated pathway was first uncovered in our study. It has not been reported before,' said Stephen Chen Zhefan, the assistant professor at the School of Life Sciences at CUHK and the lead author of the paper. Advertisement 'In addition to strengthening our understanding of the disease mechanisms, we also propose a new target for the future, a therapeutic development against Huntington's Disease.'
Yahoo
26-04-2025
- Health
- Yahoo
Family affected by Huntington's disease brings awareness to Siouxland
SIOUX CITY, Iowa (KCAU) — A family with a rare genetic disease is bringing awareness to Siouxland. Deb Conley has Huntington's Disease. According to the Huntington Study Group, four to five people out of 100,000 have the neurological disorder. 'Huntington's is kind of described as a combination of having Alzheimer's, Parkinson's and ALS all together in one,' Angie Conley, Deb's daughter-in-law, said. It is a genetic disorder, and Deb Conley's father and grandfather passed away from the disease. Norm Waitt Sr. YMCA opens new Early Learning and Youth Development Center 'It most commonly occurs to people in their thirties and forties, is when they start showing symptoms,' Angie said. 'Symptoms can be either kind of psychically and then they can have like some subtle personality changes. Other people show more cognitive executive skills type of changes. The career part of it, which is kind of your motor symptoms, you can have loss of coordination and movement, swallowing, talking, that type of thing.' There's a 50 percent chance a child of a parent with Huntington's will inherit the disease. Deb's son — Angie's husband — was recently diagnosed. 'He wasn't showing a lot of like the motor symptoms that spurred more recent,' Angie said. 'It was really difficult, but it also explained a lot for him. And it kind of put things into perspective for our family.' Despite the challenges the family faces, they say hope gets them through each day. 'It's a little more of a depressing type of disease, you know, having the disease if I didn't have my faith,' Deb said. The Conleys want to inform others about the disease and help fund research. On May 3, there will be walk for Huntington's disease awareness in Le Mars at Cleveland Park. 2nd annual Breakfast by the Bridge event raises awareness of youth homelessness 'We are doing a walk for hope for Huntington's disease,' Angie said. 'It's put on by the Huntington's Disease Society of America.' Research into Huntington's disease has improved how we understand the condition. 'It's changed over the years. when my grandpa was diagnosed, an autopsy was the only way you could find out. but now we've advanced so far in knowing what gene it is,' Deb said. Click this link if you want to know more about the Huntington's Disease Society of America's Iowa Statewide Team Hope Walk and want to register for the walk in Le Mars. Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.
