Latest news with #GCase
Yahoo
08-07-2025
- Health
- Yahoo
Cough Medicine May Protect Against Some of Parkinson's Worst Symptoms
An active ingredient in cough medicines since 1979 has shown promise as a treatment for neuropsychiatric symptoms in Parkinson's disease-related dementia. The drug, ambroxol, isn't approved for use in the US, Canada, or Australia, but it is commonly found in cough syrups and tablets from Europe. In a gold-standard phase 2 clinical trial, 22 participants with Parkinson's dementia who received a high daily dose of ambroxol for a year showed no worsening of key neuropsychiatric symptoms. Meanwhile, 25 patients who received the placebo displayed worsening symptoms, gaining an average of 3.73 points on an established neuropsychiatric score. Patients on ambroxol dropped an average of 2.45 points. Related: Both groups shared similar cognitive scores related to memory and language. But those taking ambroxol showed stabilization in symptoms like delusions, hallucinations, anxiety, irritability, apathy, and aberrant motor activity. Participants on ambroxol also experienced fewer falls. Though ambroxol was found to be safe, no clinically meaningful improvements in cognition were recorded. Yet there are reasons to remain optimistic. "Our goal was to change the course of Parkinson's dementia," says neurologist Stephen Pasternak from Western University in Canada. "This early trial offers hope and provides a strong foundation for larger studies." In the team's analysis, some participants carrying a high-risk GBA1 gene for Parkinson's showed improved cognitive performance on ambroxol. With a small sample size and no control group to compare outcomes to, the authors caution these results need to be followed up before drawing any conclusions. That said, high-risk GBA1 gene variants tend to result in lower activity of the enzyme Glucocerebrosidase (GCase), and this, in turn, is linked to more protein clumps in the brain, like Lewy bodies, which are associated with Parkinson's dementia. Recently, studies have found that ambroxol can significantly increase GCase activity. In the current trial, those taking the drug showed 1.5 times the amount of GCase activity. No serious adverse effects were reported by those taking ambroxol, although mild to moderate gastrointestinal issues were common and caused a few participants to drop out of the trial. The findings suggest that some of the more serious symptoms of Parkinson's disease may be managed by ambroxol if taken regularly at high doses. Because ambroxol can easily cross the blood-brain barrier, some scientists suspect the cough medicine can treat neurodegenerative conditions, like Parkinson's, Amyotrophic lateral sclerosis (ALS), Gaucher disease, neuroinflammation, or spinal cord injury. Whether that hypothesis proves to be true requires more clinical research. But the recent phase 2 trial on Parkinson's dementia gives scientists good reason to keep digging. "These findings suggest ambroxol may protect brain function, especially in those genetically at risk," says Pasternak. "It offers a promising new treatment avenue where few currently exist. If a drug like Ambroxol can help, it could offer real hope and improve lives." The study was published in JAMA Neurology. Scientists Finally Uncovered Where Gluten Reactions Start One Major Feature of Aging Might Not Be Universal After All Brain's Memory Center Never Stops Making Neurons, Study Confirms
Business Wire
11-06-2025
- Health
- Business Wire
Capsida Receives FDA IND Clearance for Its IV-Administered Gene Therapy for Parkinson's Disease Associated With GBA Mutations
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Capsida Biotherapeutics ('Capsida') today announced the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for CAP-003, its potential best-in-class intravenously (IV) administered gene therapy, to enter clinical trials for Parkinson's disease associated with GBA mutations (PD-GBA). This is the second wholly owned clinical program developed by Capsida with a cleared IND. Both programs utilize a proprietary IV-delivered, blood brain barrier-crossing engineered capsid and proprietary cargo that is detargeted from off-target tissues, like liver and dorsal root ganglia (DRG). In addition, Capsida uses a proprietary manufacturing process and CAP-003 is manufactured in Capsida's state-of-the-art wholly owned Good Manufacturing Practice (GMP) facility. 'PD-GBA is an area of substantial unmet need given the lack of approved treatments that target GCase, which is the protein encoded by the GBA gene, and provide meaningful slowing or halting of disease progression,' said Swati Tole, M.D., Chief Medical Officer of Capsida Biotherapeutics. 'We recognize the urgency for new treatment approaches, so we are working diligently to initiate the Phase 1/2 clinical trial for CAP-003 with the aim of dosing the first patient in the third quarter of this year.' About CAP-003 and the Phase 1/2 Clinical Trial In non-human primate (NHP) studies to date, a single IV infusion of CAP-003 resulted in dose-dependent increases in GCase activity in critical brain regions including the substantia nigra, frontal cortex, caudate nucleus, and putamen substantially above the established 30% efficacy threshold expected to restore enzyme activity levels back to normal in patients with PD-GBA. The NHP Good Laboratory Practices (GLP) toxicology study demonstrated a well-tolerated safety profile with no adverse histopathology. Capsida expects to dose the first patient in the Phase 1/2 clinical trial in the third quarter of this year. For more information about the Phase 1/2 clinical trial, please visit (NCT07011771). About Parkinson's disease associated with GBA mutations (PD-GBA) Mutations in GBA, the gene expressing the GCase enzyme, affect up to 15% of Parkinson's patients and are the most common genetic risk factor for PD. Post-mortem studies demonstrate an approximate 30% GCase activity deficit in patients compared to healthy individuals 1. There are no approved treatments that target GCase and there are no approved disease modifying treatments for PD. Other investigational treatments for PD-GBA have been limited by their inability to cross the blood-brain barrier and supplement GCase enzyme activity in sufficient quantities to overcome the deficit in patients and impact the disease. In an attempt to overcome these challenges, those treatments have required invasive direct brain or cerebrospinal fluid (CSF) administration, with limited results, and a significant burden for patients. About Capsida Biotherapeutics Capsida Biotherapeutics is a clinical-stage, fully integrated next-generation genetic medicines company. It has a central nervous system (CNS) pipeline consisting of disease-modifying and potentially curative treatments for rare and more common diseases across all ages. Capsida's wholly owned pipeline includes its first-in-class investigational treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), CAP-002; and potential best-in-class investigational treatment for Parkinson's disease associated with GBA mutations (PD-GBA) CAP-003. Both have received FDA Investigational New Drug (IND) clearance to initiate clinical trials. Capsida's pipeline also includes a potential best-in-class treatment for Friedreich's ataxia (FA). In addition to its wholly owned programs, the Company has validating partnerships with AbbVie, Lilly, and CRISPR Therapeutics. Capsida was founded in 2019 by lead investors Versant Ventures and Westlake Village BioPartners and originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech. Visit us at 1 Leyns, C. E, G. et al (2023). npj Parkinson's Disease, 74(9).
Yahoo
11-06-2025
- Business
- Yahoo
Capsida Receives FDA IND Clearance for Its IV-Administered Gene Therapy for Parkinson's Disease Associated With GBA Mutations
Capsida is initiating the Phase 1/2 study for CAP-003, with the first patient expected to be dosed in the third quarter of this year THOUSAND OAKS, Calif., June 11, 2025--(BUSINESS WIRE)--Capsida Biotherapeutics ("Capsida") today announced the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for CAP-003, its potential best-in-class intravenously (IV) administered gene therapy, to enter clinical trials for Parkinson's disease associated with GBA mutations (PD-GBA). This is the second wholly owned clinical program developed by Capsida with a cleared IND. Both programs utilize a proprietary IV-delivered, blood brain barrier-crossing engineered capsid and proprietary cargo that is detargeted from off-target tissues, like liver and dorsal root ganglia (DRG). In addition, Capsida uses a proprietary manufacturing process and CAP-003 is manufactured in Capsida's state-of-the-art wholly owned Good Manufacturing Practice (GMP) facility. "PD-GBA is an area of substantial unmet need given the lack of approved treatments that target GCase, which is the protein encoded by the GBA gene, and provide meaningful slowing or halting of disease progression," said Swati Tole, M.D., Chief Medical Officer of Capsida Biotherapeutics. "We recognize the urgency for new treatment approaches, so we are working diligently to initiate the Phase 1/2 clinical trial for CAP-003 with the aim of dosing the first patient in the third quarter of this year." About CAP-003 and the Phase 1/2 Clinical Trial In non-human primate (NHP) studies to date, a single IV infusion of CAP-003 resulted in dose-dependent increases in GCase activity in critical brain regions including the substantia nigra, frontal cortex, caudate nucleus, and putamen substantially above the established 30% efficacy threshold expected to restore enzyme activity levels back to normal in patients with PD-GBA. The NHP Good Laboratory Practices (GLP) toxicology study demonstrated a well-tolerated safety profile with no adverse histopathology. Capsida expects to dose the first patient in the Phase 1/2 clinical trial in the third quarter of this year. For more information about the Phase 1/2 clinical trial, please visit (NCT07011771). About Parkinson's disease associated with GBA mutations (PD-GBA) Mutations in GBA, the gene expressing the GCase enzyme, affect up to 15% of Parkinson's patients and are the most common genetic risk factor for PD. Post-mortem studies demonstrate an approximate 30% GCase activity deficit in patients compared to healthy individuals1. There are no approved treatments that target GCase and there are no approved disease modifying treatments for PD. Other investigational treatments for PD-GBA have been limited by their inability to cross the blood-brain barrier and supplement GCase enzyme activity in sufficient quantities to overcome the deficit in patients and impact the disease. In an attempt to overcome these challenges, those treatments have required invasive direct brain or cerebrospinal fluid (CSF) administration, with limited results, and a significant burden for patients. About Capsida Biotherapeutics Capsida Biotherapeutics is a clinical-stage, fully integrated next-generation genetic medicines company. It has a central nervous system (CNS) pipeline consisting of disease-modifying and potentially curative treatments for rare and more common diseases across all ages. Capsida's wholly owned pipeline includes its first-in-class investigational treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), CAP-002; and potential best-in-class investigational treatment for Parkinson's disease associated with GBA mutations (PD-GBA) CAP-003. Both have received FDA Investigational New Drug (IND) clearance to initiate clinical trials. Capsida's pipeline also includes a potential best-in-class treatment for Friedreich's ataxia (FA). In addition to its wholly owned programs, the Company has validating partnerships with AbbVie, Lilly, and CRISPR Therapeutics. Capsida was founded in 2019 by lead investors Versant Ventures and Westlake Village BioPartners and originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech. Visit us at 1Leyns, C. E, G. et al (2023). npj Parkinson's Disease, 74(9). View source version on Contacts Media Contact Inizio Evoke CommsKatherine Sign in to access your portfolio
Yahoo
03-06-2025
- Business
- Yahoo
Vanqua Bio to Present at Upcoming Scientific Conferences
CHICAGO, June 03, 2025 (GLOBE NEWSWIRE) -- Vanqua Bio, a clinical-stage biopharmaceutical company, announced that the company will present interim Phase 1 data for its lead clinical program, VQ-101, at two upcoming scientific conferences – the GBA1 Meeting 2025, which will take place in Montreal, Canada June 5-7, 2025 and the European Academy of Neurology, taking place in Helsinki, Finland June 21-24, 2025. Vanqua Bio GBA1 Conference Presentation Information: Title: VQ-101, A Small Molecule Allosteric Activator of Glucocerebrosidase (GCase) Demonstrates Robust And Sustained Target Engagement In HumansSession date and time: June 5th 11:00am EDTPresenter: Dr. Dan Ysselstein, Head of Biology Vanqua Bio EAN Conference ePresentation Information: Title: The allosteric activator of glucocerebrosidase, VQ-101, shows sustained activation of lysosomal GCase in humansSession date and time: June 23rd 2:30pm EESTPresenter: Dr. Maurizio Facheris, CMO About VQ-101 VQ-101 is a novel, orally administered, fully CNS-penetrant allosteric activator of glucocerebrosidase (GCase). VQ-101 is initially being investigated in GBA-Parkinson's (GBA-PD) and idiopathic Parkinson's disease (iPD). By restoring GCase activity to healthy levels, VQ-101 aims to address the underlying genetic mechanism of disease in GBA-PD and slow or stop the progression of disease. Initial Phase 1 results with VQ-101 demonstrated sustained lysosomal GCase activation in healthy volunteers by more than 75%. In preclinical studies in patient derived neurons, 50%+ GCase activation resulted in significant blockage of the accumulation of alpha synuclein, the pathogenic hallmark of PD. A Phase 1b study in patients with PD, with and without GBA mutations, is ongoing. About Vanqua Bio Founded in 2019 and headquartered in Chicago, Vanqua Bio is a biopharmaceutical company dedicated to discovering and developing next-generation medicines that have the potential to transform the lives of patients with neurodegenerative and inflammatory diseases. Our technology platform utilizes human genetics and patient-derived CNS cells to identify, validate, and clinically translate novel disease pathways associated with lysosomal dysfunction or aberrant activation of the innate immune system. Initially, we are targeting glucocerebrosidase (GCase) as a potential treatment for Parkinson's disease (PD). Additional programs address overactivation of the innate immune system in peripheral and central inflammatory disorders, including renal, dermatologic and neurodegenerative diseases. For more information, go to CONTACT: info@
