Latest news with #GBA1Meeting2025
Associated Press
03-06-2025
- Business
- Associated Press
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 Humans Session date and time: June 5th 11:00am EDT Presenter: 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 humans Session date and time: June 23rd 2:30pm EEST Presenter: 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 [email protected]
Cision Canada
02-06-2025
- Business
- Cision Canada
Congruence Therapeutics Announces Oral Presentation on Novel GCase Activators and Correctors for Parkinson's Disease with GBA1 Mutations at the GBA1 Meeting 2025
- , June 3, 2025 /CNW/ -- Congruence Therapeutics, a computationally-driven biotechnology company building a unique pipeline of correctors for diseases of protein misfolding, including MC4R-deficient genetic obesity, GBA-driven Parkinson's disease and Alpha-1 antitrypsin deficiency, announced today its participation at the GBA1 Meeting 2025, being held June 5-7, 2025 in Montreal, Canada. Congruence is also a sponsor of the meeting. "We are pleased to present our science on novel GCase activators and correctors for GBA1 Parkinson's Disease at this important scientific congress," said Sharath Hegde, Ph.D., Chief Scientific Officer of Congruence. "We are developing small molecules discovered leveraging our Revenir ™ platform that are designed to correct biological deficits resulting from mutations in the GBA1 gene. We look forward to advancing the most potent orally active and brain-penetrant allosteric GCase activator and corrector molecules for future evaluation in people living with Parkinson's disease." Oral presentation details: Title:" Discovery of Small Molecule Therapeutics for GBA1-PD" Session: 2- Small Molecules Targeting GBA1 Session Date and Time: Thursday, June 5 th, 2025, 11:40am-12:00pm Presenting Author: Indranath Ghosh, PhD, Director, Medicinal Chemistry, Congruence Therapeutics About Congruence Therapeutics Congruence is a computationally-driven biotechnology company building a unique pipeline of transformative small molecule correctors rationally designed to rescue aberrant protein function. Our proprietary scalable platform, Revenir™, captures the biophysical features of proteins across their conformational ensembles, in order to identify novel allosteric and cryptic pockets which are virtually screened to generate novel chemical matter. Congruence has leveraged its proprietary discovery engine to launch a pipeline of wholly owned drugs directed to high-value, genetically validated, difficult-to-drug targets spanning a variety of indications including genetic obesity, GBA Parkinson's Disease, and α1-Antitrypsin Deficiency. Congruence is further deploying its discovery platform to support two recently executed multi-target research collaborations with large pharmaceutical companies focused on the discovery of small molecule correctors for the treatment of solid tumors and metabolic diseases. For more information, please visit Media Contact Amy Conrad Juniper Point [email protected] 858-366-3243 SOURCE Congruence Therapeutics
Yahoo
02-06-2025
- Business
- Yahoo
Congruence Therapeutics Announces Oral Presentation on Novel GCase Activators and Correctors for Parkinson's Disease with GBA1 Mutations at the GBA1 Meeting 2025
-Congruence is optimizing several GCase-targeting compounds for GBA-1 Parkinson's disease- MONTREAL, June 3, 2025 /CNW/ -- Congruence Therapeutics, a computationally-driven biotechnology company building a unique pipeline of correctors for diseases of protein misfolding, including MC4R-deficient genetic obesity, GBA-driven Parkinson's disease and Alpha-1 antitrypsin deficiency, announced today its participation at the GBA1 Meeting 2025, being held June 5-7, 2025 in Montreal, Canada. Congruence is also a sponsor of the meeting. "We are pleased to present our science on novel GCase activators and correctors for GBA1 Parkinson's Disease at this important scientific congress," said Sharath Hegde, Ph.D., Chief Scientific Officer of Congruence. "We are developing small molecules discovered leveraging our Revenir™ platform that are designed to correct biological deficits resulting from mutations in the GBA1 gene. We look forward to advancing the most potent orally active and brain-penetrant allosteric GCase activator and corrector molecules for future evaluation in people living with Parkinson's disease." Oral presentation details: Title: "Discovery of Small Molecule Therapeutics for GBA1-PD"Session: 2- Small Molecules Targeting GBA1Session Date and Time: Thursday, June 5th, 2025, 11:40am-12:00pmPresenting Author: Indranath Ghosh, PhD, Director, Medicinal Chemistry, Congruence Therapeutics About Congruence TherapeuticsCongruence is a computationally-driven biotechnology company building a unique pipeline of transformative small molecule correctors rationally designed to rescue aberrant protein function. Our proprietary scalable platform, Revenir™, captures the biophysical features of proteins across their conformational ensembles, in order to identify novel allosteric and cryptic pockets which are virtually screened to generate novel chemical matter. Congruence has leveraged its proprietary discovery engine to launch a pipeline of wholly owned drugs directed to high-value, genetically validated, difficult-to-drug targets spanning a variety of indications including genetic obesity, GBA Parkinson's Disease, and α1-Antitrypsin Deficiency. Congruence is further deploying its discovery platform to support two recently executed multi-target research collaborations with large pharmaceutical companies focused on the discovery of small molecule correctors for the treatment of solid tumors and metabolic diseases. For more information, please visit Company ContactCharles GrubsztajnChief Operating Officercgrubsztajn@ Media ContactAmy ConradJuniper Pointamy@ 858-366-3243 View original content to download multimedia: SOURCE Congruence Therapeutics View original content to download multimedia: Sign in to access your portfolio
Yahoo
29-05-2025
- Business
- Yahoo
Gain Therapeutics Announces Oral Presentation at 2nd GBA1 Meeting
BETHESDA, Md., May 29, 2025 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) ('Gain', or the 'Company'), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced an oral presentation at the GBA1 Meeting 2025, being held June 5th-7th in Montreal, Canada. Details of the oral presentation are as follows: Date: Thursday, June 5, 2025 Time: 11:20 p.m. EST Presenter: Jonas Hannestad, M.D., Ph.D., Chief Medical Officer, Gain Therapeutics About Gain Therapeutics, Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain's lead drug candidate, GT-02287 is currently being evaluated for the treatment of Parkinson's disease with or without a GBA1 mutation in a Phase 1b clinical trial. GT-02287 has further potential in Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease. Gain has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors. Gain's unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced Magellan™ platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders and oncology. Forward-Looking StatementsThis release contains 'forward-looking statements' made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are typically preceded by words such as 'believes,' 'expects,' 'anticipates,' 'intends,' 'will,' 'may,' 'should,' or similar expressions. These forward-looking statements reflect management's current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct or that those goals will be achieved, and you should be aware that actual results could differ materially from those contained in the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, statements regarding: the development of the Company's current or future product candidates including GT-02287; expectations regarding the completion and timing of results from a Phase 1b clinical study for GT-02287; expectations regarding the timing of patient enrollment for a Phase 1b clinical study for GT-02287; the timing of any submissions to the FDA or other regulatory bodies and agencies; and the potential therapeutic and clinical benefits of the Company's product candidates. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the Company's business in general, please refer to the Company's Form 10-K for the year ended December 31, 2024. All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this release. We have no obligation, and expressly disclaim any obligation, to update, revise or correct any of the forward-looking statements, whether as a result of new information, future events or otherwise. Investors:Gain Therapeutics, Inc. Apaar Jammu Manager, Investor Relations and Public Relationsajammu@ LifeSci Advisors LLCChuck PadalaManaging Directorchuck@ Media:Russo Partners LLC Nic Johnson and Elio 846-9256Error 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
