Latest news with #Gaucher
Yahoo
29-07-2025
- Business
- Yahoo
Sharp Therapeutics Corp. Engages Rho Inc. to Help Advance Compounds into Clinical Trials for Gaucher Disease
Pittsburgh, Pennsylvania and Toronto, Ontario--(Newsfile Corp. - July 29, 2025) - Sharp Therapeutics Corp. (TSXV: SHRX) ("Sharp" or the "Company"), a preclinical-stage biotechnology company developing small molecule therapies to treat genetic diseases, today announced that it has engaged Rho, Inc. ("Rho"), a global contract research organization (CRO), to support Sharp's preparation and planned submission of its Investigational New Drug Application ("IND") to the U.S. Food and Drug Administration (the "FDA") for the evaluation of its clinical candidate compound ("'901") for the treatment of Gaucher disease. Gaucher disease is a genetic disorder caused by a deficiency in the enzyme glucocerebrosidase (also called beta-glucosidase or GBA1). Sharp's small molecule compound is a potential new orally available CNS-penetrant therapy for the treatment of Gaucher disease. "We are excited by the preclinical data from our Gaucher candidate, '901, and look forward to Rho's support in preparing our IND and other filings," said Scott Sneddon, PhD, JD and Chief Executive Officer of Sharp. "We intend to meet with FDA on our Phase I clinical plan, and to file for orphan drug designation this year. IND-enabling studies are also planned to commence before end of 2025. This is a key milestone for Sharp as we transition from a preclinical-stage to a clinical-stage company," he added. About '901 The '901 compound increases GBA1 activity for numerous mutations in Gaucher patient-derived cells, as well as increasing substrate turnover in a rodent model of Gaucher. Preclinical studies show the compound to be orally available and CNS-penetrant with good safety and preclinical pharmacology profiles. The compound is being targeted to all types of Gaucher with an application to GBA-associated Parkinson's disease also possible. The scientific data supporting the '901 series of compounds was presented at the GBA1 Conference in Montreal on June 5, 2025. That presentation is available on the Company's website at About Gaucher Disease Gaucher disease is a lysosomal storage disease caused by a deficiency in the GBA1 enzyme. Gaucher is the most common lysosomal storage disorder. Without this enzyme, the glucocerebroside lipid accumulates within the lysosomes of certain cells, particularly macrophages (immune cells). These overloaded cells, called "Gaucher cells," become enlarged and dysfunctional. The buildup primarily affects the spleen, liver, bone marrow, and sometimes the nervous system, leading to organ enlargement and the characteristic symptoms of the disease. This is the classic pattern of lysosomal storage diseases: enzyme deficiency → substrate accumulation → cellular dysfunction → organ pathology. About Rho, Inc. Rho is a global, privately held contract research organization (CRO) headquartered in Research Triangle Park, a biotech hub in North Carolina, US. Rho provides a full range of drug development services, from program strategy through to clinical trials and marketing applications. Since 1984, Rho has been a trusted partner to some of the most innovative pharmaceutical, biotechnology and medical device companies as well as academic and government organizations. Dedicated to service excellence and cross-functional collaboration, Rho's therapeutic expertise, employee focus and commitment to strong site relationships change what it means to work with a CRO – accelerating time to market, maximizing ROI, and delivering consistent, smarter and more efficient programs. Experience Rho by following the company on LinkedIn. About Sharp Therapeutics Corp. - First-Choice Therapies for Genetic Diseases Sharp Therapeutics is a preclinical-stage company developing small-molecule therapeutics for genetic diseases. The Company's discovery platform combines novel high throughput screening technologies, with compound libraries computational optimized based on the physics and biology of cellular trafficking defects and allosteric activation of proteins. The platform produces small molecule compounds that restore activity in mutated proteins giving the potential to treat genetic disorders with conventional pill-based medicines. For additional information on Sharp, please visit: Sharp Therapeutics Sneddon, PhD, JDCEO/CSOEmail: scott@ Caution Regarding Forward-Looking Information Certain statements contained in this press release constitute "forward-looking information" as such term is defined in applicable Canadian securities legislation. The words "may", "would", "could", "should", "potential", "will", "seek", "intend", "plan", "anticipate", "believe", "estimate", "expect" and similar expressions are intended to identify forward-looking information. All statements other than statements of historical fact may be forward-looking information. Such statements reflect Sharp's current views and intentions with respect to future events, and current information available to Sharp, and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements that may be expressed or implied by such forward-looking information to vary from those described herein should one or more of these risks or uncertainties materialize. Should any factor affect Sharp in an unexpected manner, or should assumptions underlying the forward-looking information prove incorrect, the actual results or events may differ materially from the results or events predicted. Any such forward-looking information is expressly qualified in its entirety by this cautionary statement. Moreover, Sharp does not assume responsibility for the accuracy or completeness of such forward-looking information. The forward-looking information included in this press release is made as of the date of this press release and Sharp undertakes no obligation to publicly update or revise any forward-looking information, other than as required by applicable law. Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release. To view the source version of this press release, please visit 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
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
North Wales Chronicle
04-07-2025
- Sport
- North Wales Chronicle
Jesse Evans digs deep for Grimes glory
Noel Meade's talented dual-purpose performer was unable to get involved when contesting the Copper Horse Stakes at Royal Ascot a fortnight ago but having previously impressed over hurdles at Ballinrobe, the nine-year-old was among the market principals for this Grade Three contest at 7-2. With 5-2 favourite Gaucher faltering on the run to the final flight, it was Barry Connell's veteran Enniskerry who looked most likely to emerge victorious after taking over the lead, but Jesse Evans dug deep on the run-in for Donagh Meyler and passed the post a half-length to the good. 'He's been great and has brought the owners all over the place and was in Ascot not so long ago. He's a yard favourite,' said Meyler. 'He's ground it out and is as tough as nails. He had a nice weight on the ratings and had match fitness on his side which definitely paid off there. 'He's toughed it out the whole way up the straight. I'm not sure what plans are but he's won a Graded race today and we'll celebrate that.' Meade was at Bellewstown, from where he told Racing TV: 'I'm absolutely thrilled – he's a little diamond. He's not very big, but he wears his heart on his sleeve and he rarely runs a bad race. 'Jumping helps him as he's a very slick jumper and he's a marvellous horse. He's won a lot of money – Flat, fences and hurdles.' On future plans, he added: 'I don't think he'd get home in the Galway Plate. We'll probably enter him in case the ground came up really good, but any time we've run him over further than two miles, it didn't work. 'I'd say it's unlikely we'll run him in the Plate and I don't think we'll run him in the Galway Hurdle again either. He's run well in it before, but he's at the top of the handicap and it would be very hard for him. 'If he went to Galway there's a chase there he might run in instead, but we'll see.'
South Wales Argus
03-07-2025
- Sport
- South Wales Argus
Jesse Evans digs deep for Grimes glory
Noel Meade's talented dual-purpose performer was unable to get involved when contesting the Copper Horse Stakes at Royal Ascot a fortnight ago but having previously impressed over hurdles at Ballinrobe, the nine-year-old was among the market principals for this Grade Three contest at 7-2. With 5-2 favourite Gaucher faltering on the run to the final flight, it was Barry Connell's veteran Enniskerry who looked most likely to emerge victorious after taking over the lead, but Jesse Evans dug deep on the run-in for Donagh Meyler and passed the post a half-length to the good. 'He's been great and has brought the owners all over the place and was in Ascot not so long ago. He's a yard favourite,' said Meyler. 'He's ground it out and is as tough as nails. He had a nice weight on the ratings and had match fitness on his side which definitely paid off there. 'He's toughed it out the whole way up the straight. I'm not sure what plans are but he's won a Graded race today and we'll celebrate that.' Meade was at Bellewstown, from where he told Racing TV: 'I'm absolutely thrilled – he's a little diamond. He's not very big, but he wears his heart on his sleeve and he rarely runs a bad race. 'Jumping helps him as he's a very slick jumper and he's a marvellous horse. He's won a lot of money – Flat, fences and hurdles.' On future plans, he added: 'I don't think he'd get home in the Galway Plate. We'll probably enter him in case the ground came up really good, but any time we've run him over further than two miles, it didn't work. 'I'd say it's unlikely we'll run him in the Plate and I don't think we'll run him in the Galway Hurdle again either. He's run well in it before, but he's at the top of the handicap and it would be very hard for him. 'If he went to Galway there's a chase there he might run in instead, but we'll see.'
Leader Live
03-07-2025
- Sport
- Leader Live
Jesse Evans digs deep for Grimes glory
Noel Meade's talented dual-purpose performer was unable to get involved when contesting the Copper Horse Stakes at Royal Ascot a fortnight ago but having previously impressed over hurdles at Ballinrobe, the nine-year-old was among the market principals for this Grade Three contest at 7-2. With 5-2 favourite Gaucher faltering on the run to the final flight, it was Barry Connell's veteran Enniskerry who looked most likely to emerge victorious after taking over the lead, but Jesse Evans dug deep on the run-in for Donagh Meyler and passed the post a half-length to the good. 'He's been great and has brought the owners all over the place and was in Ascot not so long ago. He's a yard favourite,' said Meyler. 'He's ground it out and is as tough as nails. He had a nice weight on the ratings and had match fitness on his side which definitely paid off there. 'He's toughed it out the whole way up the straight. I'm not sure what plans are but he's won a Graded race today and we'll celebrate that.' Meade was at Bellewstown, from where he told Racing TV: 'I'm absolutely thrilled – he's a little diamond. He's not very big, but he wears his heart on his sleeve and he rarely runs a bad race. 'Jumping helps him as he's a very slick jumper and he's a marvellous horse. He's won a lot of money – Flat, fences and hurdles.' On future plans, he added: 'I don't think he'd get home in the Galway Plate. We'll probably enter him in case the ground came up really good, but any time we've run him over further than two miles, it didn't work. 'I'd say it's unlikely we'll run him in the Plate and I don't think we'll run him in the Galway Hurdle again either. He's run well in it before, but he's at the top of the handicap and it would be very hard for him. 'If he went to Galway there's a chase there he might run in instead, but we'll see.'
