Latest news with #NHPs
Yahoo
14-05-2025
- Health
- Yahoo
Latus Bio Unveils AAV-Ep+ Capsid Variant Capable of Unprecedented Protein Production in the Brain
PHILADELPHIA, May 14, 2025--(BUSINESS WIRE)--Latus Bio, Inc. (Latus), a biotechnology company pioneering advances in AAV gene therapy, has announced new research published today in Science Translational Medicine, "AAVs engineered for robust brain transduction drive therapeutically relevant expression of secreted recombinant protein in NHPs and a mouse model of lysosomal storage disease." The study, led by Latus founder Beverly Davidson, PhD details the development of a novel adeno-associated virus (AAV) capsid variant - AAV-Ep+ - that demonstrates unprecedented potency in transducing cells that line the ventricles, known as ependymal cells, and cerebral neurons in mice and in non-human primates (NHPs). This advancement is potentially a significant leap forward for therapeutic gene delivery, wherein the study authors demonstrate that cells transduced with AAV-Ep+ can effectively serve as protein production depots, secreting large amounts of soluble proteins into the cerebral spinal fluid (CSF) for uptake throughout the central nervous system (CNS). This potency and distribution profile could potentially result in one-time delivery of gene therapies that encode protein treatments for lysosomal storage disorders (LSDs) as well as for other neurodevelopmental and neurodegenerative diseases that result in long term benefits for patients. The AAV-Ep+ capsid variant was identified through a massively parallelled and unbiased screen of a large-diversity AAV variant library administered to NHPs. The nominated capsid, which was isolated from tens of millions of potential candidates, displays: Remarkable tropism for cells that line the ventricular system of the brain and spinal cord of adult NHPs and mice. It also efficiently transduces neurons in cortical regions of the brain that are implicated in many diseases. Robust transduction of induced pluripotent stem cell (iPSC)-derived human neurons and mice when compared to naturally occurring AAV serotypes. This cross-species activity highlights the potential for AAV-Ep+ to deliver sustained and therapeutic expression of encoded proteins in human brain cells that could result in prolonged therapeutic benefit for patients. Low dose administration of AAV-Ep+ constructs designed to express human tripeptidyl peptidase (hTPP1) to mice deficient in TPP1 (a model of human CLN2 disease - a type of LSD) as well as to NHPs result in CSF and parenchymal tissue levels that exceeded those obtained with natural serotype capsids, reaching levels that are potentially multi-fold above therapeutic values required for CLN2 patients. "This breakthrough in AAV capsid engineering represents a critical advancement in the field of gene therapy," said Dr. Beverly Davidson, Chair of the Scientific Advisory Board of Latus Bio and corresponding author of the study. "AAV-Ep+ offers a highly efficient, low-dose solution for brain-wide protein delivery, opening new possibilities for treating neurodevelopmental diseases like CLN2 disease and beyond." The study showcases Latus' unique capsid discovery platform and ability to identify AAV capsid variants that are optimized for delivery to specific tissues and cell types, seeking to address translational shortcomings to prospectively enable better gene therapies. Latus continues to advance its pipeline of novel AAV capsid variants that target disease-relevant cell types in other regions of the central nervous system (e.g., cortex, cerebellum, spinal cord) as well as in peripheral tissues (e.g., ear, eye, heart, kidney and muscle). The Company is developing cutting-edge gene therapies that aim to transform the treatment landscape of genetically defined diseases, including many with high unmet medical needs. About Latus Bio (Latus) Latus is a biotechnology company dedicated to addressing devastating CNS and peripheral diseases via gene therapy. The Company is advancing an innovative therapeutics pipeline based on novel AAV capsid variants with potency and specificity. Latus is powered by a diverse team of visionary scientists, experienced clinicians, and leading industry executives. The Company has offices in Philadelphia, PA and in the Seaport in Boston, MA. For more information, visit and follow on LinkedIn. View source version on Contacts info@ 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
14-05-2025
- Health
- Business Wire
Latus Bio Unveils AAV-Ep+ Capsid Variant Capable of Unprecedented Protein Production in the Brain
PHILADELPHIA--(BUSINESS WIRE)-- Latus Bio, Inc. (Latus), a biotechnology company pioneering advances in AAV gene therapy, has announced new research published today in Science Translational Medicine, 'AAVs engineered for robust brain transduction drive therapeutically relevant expression of secreted recombinant protein in NHPs and a mouse model of lysosomal storage disease.' "This breakthrough in AAV capsid engineering represents a critical advancement in the field of gene therapy," said Dr. Beverly Davidson, Chair of the Scientific Advisory Board of Latus Bio and corresponding author of the study. The study, led by Latus founder Beverly Davidson, PhD details the development of a novel adeno-associated virus (AAV) capsid variant - AAV-Ep+ - that demonstrates unprecedented potency in transducing cells that line the ventricles, known as ependymal cells, and cerebral neurons in mice and in non-human primates (NHPs). This advancement is potentially a significant leap forward for therapeutic gene delivery, wherein the study authors demonstrate that cells transduced with AAV-Ep+ can effectively serve as protein production depots, secreting large amounts of soluble proteins into the cerebral spinal fluid (CSF) for uptake throughout the central nervous system (CNS). This potency and distribution profile could potentially result in one-time delivery of gene therapies that encode protein treatments for lysosomal storage disorders (LSDs) as well as for other neurodevelopmental and neurodegenerative diseases that result in long term benefits for patients. The AAV-Ep+ capsid variant was identified through a massively parallelled and unbiased screen of a large-diversity AAV variant library administered to NHPs. The nominated capsid, which was isolated from tens of millions of potential candidates, displays: Remarkable tropism for cells that line the ventricular system of the brain and spinal cord of adult NHPs and mice. It also efficiently transduces neurons in cortical regions of the brain that are implicated in many diseases. Robust transduction of induced pluripotent stem cell (iPSC)-derived human neurons and mice when compared to naturally occurring AAV serotypes. This cross-species activity highlights the potential for AAV-Ep+ to deliver sustained and therapeutic expression of encoded proteins in human brain cells that could result in prolonged therapeutic benefit for patients. Low dose administration of AAV-Ep+ constructs designed to express human tripeptidyl peptidase (hTPP1) to mice deficient in TPP1 (a model of human CLN2 disease - a type of LSD) as well as to NHPs result in CSF and parenchymal tissue levels that exceeded those obtained with natural serotype capsids, reaching levels that are potentially multi-fold above therapeutic values required for CLN2 patients. "This breakthrough in AAV capsid engineering represents a critical advancement in the field of gene therapy," said Dr. Beverly Davidson, Chair of the Scientific Advisory Board of Latus Bio and corresponding author of the study. "AAV-Ep+ offers a highly efficient, low-dose solution for brain-wide protein delivery, opening new possibilities for treating neurodevelopmental diseases like CLN2 disease and beyond." The study showcases Latus' unique capsid discovery platform and ability to identify AAV capsid variants that are optimized for delivery to specific tissues and cell types, seeking to address translational shortcomings to prospectively enable better gene therapies. Latus continues to advance its pipeline of novel AAV capsid variants that target disease-relevant cell types in other regions of the central nervous system (e.g., cortex, cerebellum, spinal cord) as well as in peripheral tissues (e.g., ear, eye, heart, kidney and muscle). The Company is developing cutting-edge gene therapies that aim to transform the treatment landscape of genetically defined diseases, including many with high unmet medical needs. About Latus Bio (Latus) Latus is a biotechnology company dedicated to addressing devastating CNS and peripheral diseases via gene therapy. The Company is advancing an innovative therapeutics pipeline based on novel AAV capsid variants with potency and specificity. Latus is powered by a diverse team of visionary scientists, experienced clinicians, and leading industry executives. The Company has offices in Philadelphia, PA and in the Seaport in Boston, MA. For more information, visit and follow on LinkedIn.
Yahoo
14-05-2025
- Health
- Yahoo
Latus Bio Unveils AAV-Ep+ Capsid Variant Capable of Unprecedented Protein Production in the Brain
PHILADELPHIA, May 14, 2025--(BUSINESS WIRE)--Latus Bio, Inc. (Latus), a biotechnology company pioneering advances in AAV gene therapy, has announced new research published today in Science Translational Medicine, "AAVs engineered for robust brain transduction drive therapeutically relevant expression of secreted recombinant protein in NHPs and a mouse model of lysosomal storage disease." The study, led by Latus founder Beverly Davidson, PhD details the development of a novel adeno-associated virus (AAV) capsid variant - AAV-Ep+ - that demonstrates unprecedented potency in transducing cells that line the ventricles, known as ependymal cells, and cerebral neurons in mice and in non-human primates (NHPs). This advancement is potentially a significant leap forward for therapeutic gene delivery, wherein the study authors demonstrate that cells transduced with AAV-Ep+ can effectively serve as protein production depots, secreting large amounts of soluble proteins into the cerebral spinal fluid (CSF) for uptake throughout the central nervous system (CNS). This potency and distribution profile could potentially result in one-time delivery of gene therapies that encode protein treatments for lysosomal storage disorders (LSDs) as well as for other neurodevelopmental and neurodegenerative diseases that result in long term benefits for patients. The AAV-Ep+ capsid variant was identified through a massively parallelled and unbiased screen of a large-diversity AAV variant library administered to NHPs. The nominated capsid, which was isolated from tens of millions of potential candidates, displays: Remarkable tropism for cells that line the ventricular system of the brain and spinal cord of adult NHPs and mice. It also efficiently transduces neurons in cortical regions of the brain that are implicated in many diseases. Robust transduction of induced pluripotent stem cell (iPSC)-derived human neurons and mice when compared to naturally occurring AAV serotypes. This cross-species activity highlights the potential for AAV-Ep+ to deliver sustained and therapeutic expression of encoded proteins in human brain cells that could result in prolonged therapeutic benefit for patients. Low dose administration of AAV-Ep+ constructs designed to express human tripeptidyl peptidase (hTPP1) to mice deficient in TPP1 (a model of human CLN2 disease - a type of LSD) as well as to NHPs result in CSF and parenchymal tissue levels that exceeded those obtained with natural serotype capsids, reaching levels that are potentially multi-fold above therapeutic values required for CLN2 patients. "This breakthrough in AAV capsid engineering represents a critical advancement in the field of gene therapy," said Dr. Beverly Davidson, Chair of the Scientific Advisory Board of Latus Bio and corresponding author of the study. "AAV-Ep+ offers a highly efficient, low-dose solution for brain-wide protein delivery, opening new possibilities for treating neurodevelopmental diseases like CLN2 disease and beyond." The study showcases Latus' unique capsid discovery platform and ability to identify AAV capsid variants that are optimized for delivery to specific tissues and cell types, seeking to address translational shortcomings to prospectively enable better gene therapies. Latus continues to advance its pipeline of novel AAV capsid variants that target disease-relevant cell types in other regions of the central nervous system (e.g., cortex, cerebellum, spinal cord) as well as in peripheral tissues (e.g., ear, eye, heart, kidney and muscle). The Company is developing cutting-edge gene therapies that aim to transform the treatment landscape of genetically defined diseases, including many with high unmet medical needs. About Latus Bio (Latus) Latus is a biotechnology company dedicated to addressing devastating CNS and peripheral diseases via gene therapy. The Company is advancing an innovative therapeutics pipeline based on novel AAV capsid variants with potency and specificity. Latus is powered by a diverse team of visionary scientists, experienced clinicians, and leading industry executives. The Company has offices in Philadelphia, PA and in the Seaport in Boston, MA. For more information, visit and follow on LinkedIn. View source version on Contacts info@ 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
Globe and Mail
14-05-2025
- Business
- Globe and Mail
Editas Medicine Reports New In Vivo Data Highlighting the Potential of Editas' Gene Upregulation Strategy in HSCs at the American Society of Gene and Cell Therapy Annual Meeting
Data demonstrate therapeutically relevant editing levels using a clinically validated strategy, supporting its development as a novel, in vivo approach to treating sickle cell disease and beta thalassemia CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today shared new in vivo data demonstrating therapeutically relevant levels of HBG1/2 promoter editing in hematopoietic stem cells (HSCs) with a single dose of proprietary targeted lipid nanoparticle (tLNP) in humanized mice and non-human primates (NHPs). This clinically validated approach targeting HBG1/2 promoters to upregulate fetal hemoglobin (HbF) is in pre-clinical development as a potential transformative in vivo gene editing medicine for the treatment of sickle cell disease and beta thalassemia. The Company reported these data in a presentation available today and will detail the data in an oral presentation today at 1:30 p.m. CT/2:30 p.m. ET at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in New Orleans, LA, and virtually. In these studies, the Company's proprietary tLNP formulation delivered HBG1/2 promoter editing cargo to HSPCs and/or HSCs in humanized mice (mice engrafted with human CD34+ cells) and in NHPs. In an ongoing NHP study, administration of a single intravenous dose of Editas Medicine's proprietary tLNP demonstrated high efficiency HSC delivery and achieved up to 47% HBG1/2 editing levels. In a study with humanized mice, administration of a single dose achieved 48% editing of HBG1/2 in long-term HSCs. Both studies exceeded the predicted editing threshold of ≥25% required for therapeutic benefit. In addition to achieving therapeutically relevant editing levels, preliminary biodistribution data in NHPs with Editas' tLNP shows significant de-targeting of the liver in contrast to standard LNPs. 'These findings are very encouraging and further support our approach to developing a potentially first- and best-in-class in vivo gene edited medicine for the treatment of sickle cell disease and beta thalassemia,' said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. 'We believe that translating these preclinical results to the clinic will address the continuing significant unmet need for a transformative gene edited medicine with the potential to improve the lives of people living with sickle cell disease and beta-thalassemia around the world.' Editas Medicine's in vivo HSC program targets HBG1/2 promoters to mimic naturally occurring mechanisms of hereditary persistence of fetal hemoglobin (HPFH) and utilizes proprietary AsCas12a to edit with high efficiency and minimize off-target editing. Editing the HBG1/2 promoters with AsCas12a with the investigational medicine reni-cel led to robust increases in fetal hemoglobin (HbF) and total hemoglobin (Hb) in clinical trials. Oral Presentation Details: Title: In Vivo Delivery of HBG1/2 Promoter Editing Cargo to HSC of Humanized Mouse and Non-Human Primate with Lipid Nanoparticles Session Date and Time: Wednesday, May 14, 2025, 1:30 p.m. – 1:45 p.m. CT Session Title: Translational Applications of Base and Prime Editors Room: 265-268 Final Abstract Number: AMA353 Additional Editas Medicine presentations are below. Abstracts can be accessed on the ASGCT website, and the presentations will be posted on the Editas Medicine website during the conference. Poster Presentations: Title: Design and Development of Improved LNP Targeting Ligands for In Vivo Hematopoietic Stem Cell Editing Session Date and Time: Tuesday, May 13, 2025, 6:00 p.m. – 7:30 p.m. CT Session Title: Tuesday Poster Reception Presentation Room: Poster Hall, Hall 12 Final Abstract Number: AMA245 Title: Design of Chemically Modified AsCas12a Guide RNAs for Increased Potency of LNP-Delivered Gene Editing Cargos Session Date and Time: Tuesday, May 13, 2025, 6:00 p.m. – 7:30 p.m. CT Session Title: Tuesday Poster Reception Presentation Room: Poster Hall, Hall 12 Final Abstract Number: AMA420 Title: In Vivo Gene Editing and Disease-Associated Biomarker Reduction for Multiple Liver Targets in Non-human Primate Using AsCas12a Nuclease Delivered by LNP Session Date and Time: Wednesday, May 14, 2025, 5:30 p.m. – 7:00 p.m. CT Session Title: Wednesday Poster Reception Presentation Room: Poster Hall, Hall 12 Final Abstract Number: AMA640 Title: In Vivo CRISPR Editing of Genetic Regulatory Regions Results in Functional Upregulation of Target Protein and Meaningful Reduction of Disease-Associated Biomarker in Mice Session Date and Time: Wednesday, May 14, 2025, 5:30 p.m. – 7:00 p.m. CT Session Title: Wednesday Poster Reception Presentation Room: Poster Hall, Hall 12 Final Abstract Number: AMA351 About Editas Medicine As a pioneering gene editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of in vivo medicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision in vivo gene editing medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute's Cas12a patent estate and Broad Institute and Harvard University's Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit
The Independent
17-03-2025
- Health
- The Independent
Scientists may have finally developed pill to cure Ebola
Scientists have found that an oral antiviral drug successfully prevents monkeys from dying after an Ebola infection, an advance that may prevent future outbreaks of the deadly virus. The deadly Ebola virus is highly lethal to humans and nonhuman primates (NHPs) with death rates close to 90 per cent of infected individuals. For instance, the 2013–2016 West African Ebola virus disease (EVD) epidemic caused 11,325 deaths among 28,600 infected people while another outbreak between 2018 and 2020 in Democratic Republic of Congo (DRC) and Uganda killed 2299 individuals out of 3481 infection cases. While antibody-based treatments have shown some success in animal and human trials, the combination of cold-chain transport and storage requirements pose substantial challenges to contain outbreaks, researchers say. Thus, there is a need for developing oral pills that can be rapidly and widely deployed to save lives and contain the spread of the virus in resource-limited regions. 'Oral antivirals have several advantages over parenterally administered drugs, including potential to be easier to supply, store, distribute, and administer,' scientists explained. The drug Obeldesivir (ODV) was previously found to have broad activity across several RNA viruses – including the filovirus family to which Ebola belongs – when given 24 hours after exposure. However, in the previous research, scientists used intramuscular delivery of the virus into macaques, which causes a much faster disease course that makes it harder to track the drug's effect. In the latest study, published in the journal Science Advances, researchers found that ODV protects 100 per cent of rhesus macaques exposed to the highly potent Makona variant of Ebola virus via mucosal administration. Five rhesus and five crab-eating macaques received daily ODV for 10 days starting 24 hours post-exposure, while three monkeys served as controls. Scientists found that ODV provided 100 per cent protection against death for rhesus macaques and 80 per cent protection for the crab-eating monkeys. Since the disease progressed slower under the latest virus administration route, researchers could also explore ODV's mechanisms of action. They found that the treated monkeys had higher expression of proteins that support activation of the immune system's T cells. Monkeys provided ODV also appeared to have better anti-inflammatory action and dampening of any severe immune reaction. Overall, researchers say the results support the potential of ODV as an oral postexposure prophylaxis. 'These findings suggest that ODV treatment affords the opportunity for the development of adaptive immunity while mitigating excessive inflammation, potentially preventing fatal outcomes,' they wrote. Scientists hope to further understand how a delayed ODV treatment would affect immune responses. 'However, even if ODV is only effective when given between 24 hours after EBOV exposure and before the onset of illness, it may have utility in combating EBOV outbreaks and for subjects with known exposures to EBOV, including accidents in the clinic or laboratories,' they wrote.
