Latest news with #NHP
Cision Canada
5 days ago
- Business
- Cision Canada
Rona Therapeutics Announces Breakthrough Data on Potential Annual Dosing RNAi Therapy RN026 Targeting Lipoprotein(a) in The National Lipid Association (NLA) Scientific Sessions 2025 Français
SHANGHAI, May 29, 2025 /CNW/ -- Rona Therapeutics, a global leading RNA therapy company, today announced preclinical data for its self-developed RNA interference (RNAi) therapy RN026 at the National Lipid Association (NLA) Annual Scientific Sessions. Preclinical studies revealed that the therapy, targeting lipoprotein(a) (Lp(a))—an independent risk factor for cardiovascular diseases— achieved a 99% reduction in Lp(a) with potential annual dosing regimen in non-human primate (NHP) study. Key Highlights Unparalleled Efficacy and Durability In transgenic mouse models, a single 2mg/kg subcutaneous injection resulted in 98% reduction in serum Apo(a) protein, which lasted for at least 42 days In NHP study, a single 2mg/kg subcutaneous injection achieved maximum 99% reduction of serum Lp(a); 95% Lp(a) reduction was maintained at 98 days post-dose with ~80% serum Lp(a) decreasing effect sustained till 154 days, suggesting annual dosing potential in human Precision siRNA Design Targets multiple sites inside and outside of the KIV-2 CNV region to maximize knock-down efficiency Very low hybridization related off-target risk Dual Benefits Accompanied by a 25% reduction in LDL-C levels in NHP study Repeated dose tox studies in SD rats confirmed no adverse findings About RN026 RN026 demonstrates tremendous potential as a breakthrough therapy in a range of cardiovascular diseases, i.e. ASCVD, aortic stenosis, peripheral arterial disease, with differentiated long-lasting efficacy, potent Lp(a) reduction, and excellent safety profile. There is approximately 10-20% world population that has elevated serum Lp(a) level. RN026 is poised to bring new therapeutic hope to cardiovascular patients globally. About Rona Therapeutics Rona Therapeutics is a global leader in nucleic acid innovative drug platform company, specializing in the treatment of cardiometabolic diseases, obesity and neurological diseases with proprietary oligo chemistry and delivery platform. Rona Therapeutics is committed to developing the best and first-in-class siRNA drugs with differentiation and innovation to address unmet needs and improve outcome in cardiovascular diseases, obesity, and MASH.
Yahoo
5 days ago
- Business
- Yahoo
Rona Therapeutics Announces Breakthrough Data on Potential Annual Dosing RNAi Therapy RN026 Targeting Lipoprotein(a) in The National Lipid Association (NLA) Scientific Sessions 2025
SHANGHAI, May 29, 2025 /CNW/ -- Rona Therapeutics, a global leading RNA therapy company, today announced preclinical data for its self-developed RNA interference (RNAi) therapy RN026 at the National Lipid Association (NLA) Annual Scientific Sessions. Preclinical studies revealed that the therapy, targeting lipoprotein(a) (Lp(a))—an independent risk factor for cardiovascular diseases—achieved a 99% reduction in Lp(a) with potential annual dosing regimen in non-human primate (NHP) study. Key Highlights Unparalleled Efficacy and Durability In transgenic mouse models, a single 2mg/kg subcutaneous injection resulted in 98% reduction in serum Apo(a) protein, which lasted for at least 42 days In NHP study, a single 2mg/kg subcutaneous injection achieved maximum 99% reduction of serum Lp(a); 95% Lp(a) reduction was maintained at 98 days post-dose with ~80% serum Lp(a) decreasing effect sustained till 154 days, suggesting annual dosing potential in human Precision siRNA Design Targets multiple sites inside and outside of the KIV-2 CNV region to maximize knock-down efficiency Very low hybridization related off-target risk Dual Benefits Accompanied by a 25% reduction in LDL-C levels in NHP study Repeated dose tox studies in SD rats confirmed no adverse findings About RN026 RN026 demonstrates tremendous potential as a breakthrough therapy in a range of cardiovascular diseases, i.e. ASCVD, aortic stenosis, peripheral arterial disease, with differentiated long-lasting efficacy, potent Lp(a) reduction, and excellent safety profile. There is approximately 10-20% world population that has elevated serum Lp(a) level. RN026 is poised to bring new therapeutic hope to cardiovascular patients globally. About Rona Therapeutics Rona Therapeutics is a global leader in nucleic acid innovative drug platform company, specializing in the treatment of cardiometabolic diseases, obesity and neurological diseases with proprietary oligo chemistry and delivery platform. Rona Therapeutics is committed to developing the best and first-in-class siRNA drugs with differentiation and innovation to address unmet needs and improve outcome in cardiovascular diseases, obesity, and MASH. View original content: SOURCE Rona Therapeutics View original content: 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
16-05-2025
- Business
- Yahoo
Liberate Bio Announces Presentation to Update on RAPTOR LNP Platform and CAR-M Therapeutic Programs
CAMBRIDGE, Mass., May 16, 2025--(BUSINESS WIRE)--Liberate Bio, Inc., a biotechnology company developing medicines which efficiently and selectively deliver payloads to immune cells while bypassing the live, announced an oral presentation by Melissa Deck - Director of Platform, demonstrating the success of Liberate's RAPTOR™ screening platform and NHP data for the company's first lead candidates at the Oligonucleotide and Peptide Therapeutics 2025 (TIDES) being held May 19th-22nd, 2025 in San Diego, California. "We built the RAPTOR platform to address key limitations of existing delivery vehicles and to expand the potential of oligonucleotide therapeutics beyond the liver. We are thrilled to have reached this critical inflection point," said Walter R. Strapps, Ph.D., Chief Scientific Officer of Liberate. "The presentation shows that our lead lipids robustly deliver mRNA cargo to monocytes and macrophages in non-human primates. This has enabled our first therapeutic program, in vivo CAR-M to treat solid tumors." Key Highlights from the Presentation: Liberate Bio has built RAPTOR™, a robust nanoparticle screening platform for evaluating and identifying extrahepatic delivery vehicles directly in non-human primates (NHPs). RAPTOR™ has generated lipid nanoparticles (LNPs) with significant delivery to bone marrow and expression of mRNA cargo in monocytes, macrophages and other cell-types relevant to in vivo CAR therapy. At the same time, Liberate's lead candidates show a five -old reduction in liver accumulation of cargo. TIDES Presentation Details: TITLE: "Innovating Nucleic Acid Delivery: RAPTOR, Artificial Intelligence, and the Future of CAR-M" SESSION TITLE: Delivery of Macromolecules PRESENTATION TYPE: Oral PRESENTER: Melissa K. Deck, Director of Platform, Liberate Bio. DATE AND TIME: Wednesday, 21 May, 10:45am PT About Liberate Bio Our vision at Liberate Bio is to achieve the extraordinary—delivering genetic medicines that transcend liver-based limitations, liberating patients from disease. Today's genetic medicines are limited by the availability of safe, effective delivery vehicles that direct nucleic acid therapeutics to any organ other than the liver. To realize the full potential of genetic medicines, we use our proprietary screening platform to rapidly evaluate rational and artificial intelligence-designed nanoparticles, reducing the cycle time for identifying novel vehicles from years to months. Our initial lead candidates are allowing us to pursue multiple therapeutic areas based on delivery to various bone marrow derived cells including HSCs, monocytes and macrophages. We have built the team and a suite of tools that allows us to rapidly move RNA therapeutics to the clinic by significantly increasing the translatability of the preclinical work. For more information about the company's technologies, team, and mission, visit View source version on Contacts Walter Strapps, CSOwstrapps@ 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
16-05-2025
- Business
- Business Wire
Liberate Bio Announces Presentation to Update on RAPTOR LNP Platform and CAR-M Therapeutic Programs
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Liberate Bio, Inc., a biotechnology company developing medicines which efficiently and selectively deliver payloads to immune cells while bypassing the live, announced an oral presentation by Melissa Deck - Director of Platform, demonstrating the success of Liberate's RAPTOR™ screening platform and NHP data for the company's first lead candidates at the Oligonucleotide and Peptide Therapeutics 2025 (TIDES) being held May 19th-22nd, 2025 in San Diego, California. 'We built the RAPTOR platform to address key limitations of existing delivery vehicles and to expand the potential of oligonucleotide therapeutics beyond the liver. We are thrilled to have reached this critical inflection point,' said Walter R. Strapps, Ph.D., Chief Scientific Officer of Liberate. 'The presentation shows that our lead lipids robustly deliver mRNA cargo to monocytes and macrophages in non-human primates. This has enabled our first therapeutic program, in vivo CAR-M to treat solid tumors.' Key Highlights from the Presentation: Liberate Bio has built RAPTOR™, a robust nanoparticle screening platform for evaluating and identifying extrahepatic delivery vehicles directly in non-human primates (NHPs). RAPTOR™ has generated lipid nanoparticles (LNPs) with significant delivery to bone marrow and expression of mRNA cargo in monocytes, macrophages and other cell-types relevant to in vivo CAR therapy. At the same time, Liberate's lead candidates show a five -old reduction in liver accumulation of cargo. TIDES Presentation Details: TITLE: 'Innovating Nucleic Acid Delivery: RAPTOR, Artificial Intelligence, and the Future of CAR-M' SESSION TITLE: Delivery of Macromolecules PRESENTATION TYPE: Oral PRESENTER: Melissa K. Deck, Director of Platform, Liberate Bio. About Liberate Bio Our vision at Liberate Bio is to achieve the extraordinary—delivering genetic medicines that transcend liver-based limitations, liberating patients from disease. Today's genetic medicines are limited by the availability of safe, effective delivery vehicles that direct nucleic acid therapeutics to any organ other than the liver. To realize the full potential of genetic medicines, we use our proprietary screening platform to rapidly evaluate rational and artificial intelligence-designed nanoparticles, reducing the cycle time for identifying novel vehicles from years to months. Our initial lead candidates are allowing us to pursue multiple therapeutic areas based on delivery to various bone marrow derived cells including HSCs, monocytes and macrophages. We have built the team and a suite of tools that allows us to rapidly move RNA therapeutics to the clinic by significantly increasing the translatability of the preclinical work. For more information about the company's technologies, team, and mission, visit
Business Wire
14-05-2025
- Business
- Business Wire
Capsida Presents New GLP Toxicology Data Supporting Recent FDA IND Clearance of Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Capsida Biotherapeutics ('Capsida') today announced the presentation of new non-human primate (NHP) GLP toxicology data for CAP-002, its wholly owned first-in-class, intravenously (IV) administered investigational gene therapy for syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE). These data were the foundation for the Investigational New Drug (IND) application for CAP-002 that was recently cleared by the U.S. Food and Drug Administration (FDA). Capsida will deliver these data in an oral presentation today at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), taking place May 13-17, 2025, in New Orleans and virtually. The three-month cohort data from the NHP GLP toxicology study demonstrate dose-dependent brain-wide expression of STXBP1 and simultaneous detargeting of the liver and dorsal root ganglia (DRGs). CAP-002 was delivered as a single dose via IV infusion at 3.8E13 vg/kg, 5.9E13 vg/kg, and 7.4E13 vg/kg. There was a dose-dependent increase in average cargo RNA levels in the brain, with the 3.8E13 vg/kg dose of CAP-002 demonstrating a 207-fold increase over cargo RNA levels previously shown for 2.5E13 vg/kg of IV-delivered adeno-associated virus 9 (AAV9). Together with pharmacology data in a mouse model of STXBP1-DEE, these NHP GLP toxicology results show that all doses of CAP-002 tested have the potential to fully correct seizures and meaningfully correct cognitive and motor dysfunction in patients. The 3.8E13 vg/kg dose of CAP-002 also demonstrated robust 10x and 13x detargeting of liver and DRGs respectively, compared to a 2.5E13 vg/kg dose of AAV9. CAP-002 was well tolerated with no adverse clinical pathology or histopathology findings throughout the central nervous system, DRGs, or peripheral organs, including the liver. CAP-002 is the first IV-administered, AAV blood brain barrier-crossing genetic medicine program entering a human clinical trial. CAP-002 is manufactured in Capsida's state-of-the-art wholly owned facility using a proprietary manufacturing process. Capsida received FDA Orphan Drug Designation in October 2024 and is now initiating study start-up activities for the SYNRGY Phase 1/2a clinical trial, with the first patient expected to be dosed in the third quarter of this year. 'These GLP toxicology data established the potential of CAP-002 to safely treat STXBP1-DEE and were a critical part of the data package to enable the FDA IND clearance of this first-in-class IV investigational gene therapy,' said Swati Tole, M.D., Chief Medical Officer of Capsida Biotherapeutics. 'Our focus is on initiating our Phase 1/2a SYNRGY clinical trial with the aim of providing a safe, disease-modifying treatment for STXBP1-DEE patients and their families.' Presentation Details Oral Presentation: Title: Systemic Gene Therapy CAP-002 Demonstrates Potential for Disease-Modifying Treatment of Seizures and Motor and Cognitive Deficits of STXBP1-DEE Using an Engineered, CNS-Targeted AAV Date and Time: Session: Viral Vectors in Large Animal Models Location: New Orleans Theater B Presenter: Nicholas Flytzanis, Ph.D., Founder, Chief Research and Innovation Officer, Capsida Abstracts can be found at and the presentation will be available under the 'Publications' section of the Capsida website. About STXBP1-DEE Syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE) is estimated to affect up to one in 26,000 births globally, equating to approximately 5,000 pediatric patients in the U.S. and Europe. The STXBP1 protein is present in every neuron and is essential for normal neurotransmission. Mutations in the STXBP1 gene are associated with early-onset seizures, severe developmental delay and intellectual disability, motor abnormalities, and a risk of sudden unexpected death in epilepsy (SUDEP). There are no approved treatments for STXBP1-DEE. About Capsida Biotherapeutics Capsida Biotherapeutics is a fully integrated next-generation genetic medicines company with 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 treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), which has received Investigational New Drug (IND) clearance to initiate clinical trials from the U.S. Food and Drug Administration (FDA). Capsida's pipeline also includes potential best-in-class treatments for Parkinson's disease associated with GBA mutations (PD-GBA) and Friedreich's ataxia (FA). The PD-GBA program is also on track to enter clinical development this quarter. 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
