Latest news with #ASGCT2025
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17-05-2025
- Business
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Fractyl Health Unveils New Rejuva® Smart GLP-1™ Pancreatic Gene Therapy Preclinical Data Highlighting Durable Potency and Safety with Limited Systemic GLP-1 Exposure at ASGCT 2025
Data suggests that single dose of RJVA-001 leads to durable metabolic improvements with low systemic GLP-1 exposure in db/db mouse model of T2D— potentially simultaneously addressing durability, adherence, and tolerability challenges seen with current GLP-1 drugs Endoscopic ultrasound-guided delivery achieves targeted pancreatic expression in a large animal model with no toxicity observed to date Data show nutrient-responsive GLP-1 secretion in human beta cells and human islets, demonstrating that Rejuva mimics natural hormone regulation rather than constant drug-driven stimulation Rejuva advancing toward first-in-human studies; first CTA module submission for RJVA-001 expected by June 2025 BURLINGTON, Mass., May 17, 2025 (GLOBE NEWSWIRE) -- Fractyl Health, Inc. (Nasdaq: GUTS) (the Company), a metabolic therapeutics company focused on pattern-breaking approaches that treat root causes of obesity and type 2 diabetes (T2D), today announced an oral presentation of new preclinical data from its Rejuva smart GLP-1 gene therapy platform at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting. The data highlight RJVA-001's potential to deliver durable, nutrient-responsive GLP-1 secretion from pancreatic beta cells—mimicking natural hormone regulation with low circulating levels of GLP-1, offering a potentially profound mechanistic advantage over pharmacologic GLP-1 drugs. RJVA-001 also showed strong efficacy, targeted delivery, and a favorable safety profile, reinforcing its readiness for first-in-human studies. The data were featured in an oral presentation at ASGCT titled 'Endoscopic Ultrasound-Guided Delivery of Human Glucagon-like Peptide-1 Pancreatic Gene Therapy: Safety and Feasibility in a Porcine Model.' 'RJVA-001 represents a fundamentally different approach to treating metabolic disease - one that delivers the power of GLP-1 in a more natural manner,' said Professor Randy Seeley, Ph.D., Henry King Ransom Professor of Surgery and Director of Michigan Nutrition Obesity Research Center at Michigan School of Medicine. 'The ability to drive durable metabolic effects with physiologic hormone levels, from a one-time treatment, would be a scientific breakthrough with huge potential implications for patients. This has the potential to reshape how we think about treating T2D and obesity at scale.' Key Data Presented at ASGCT 2025: Single-dose RJVA-001 led to durable, dose-dependent metabolic improvements in a well-established diabetic model: In db/db mice, treatment with RJVA-001 led to sustained reductions in blood sugar, improved fasting insulin levels, and improvements in body weight over six weeks— supporting Rejuva's potential for sustained disease modification on both blood sugar and body weight control. Treatment with RJVA-001 resulted in a >200 mg/dL reduction in fasting blood sugar, a >2-fold increase in fasting insulin levels, and prevention of weight gain, compared to a ~20% increase seen in vehicle-treated controls. These results demonstrate broad-based metabolic improvement in insulin secretion, weight gain, and blood sugar control in this gold-standard model of T2D. RJVA-001 achieved glycemic control and prevented weight gain with significantly lower systemic GLP-1 exposure than required by GLP-1 drugs to achieve similar effects: Circulating GLP-1 levels were more than 5-fold lower than those seen with pharmacologic GLP-1 drugs and were comparable to levels observed after gastric bypass surgery—suggesting a substantially lower risk of GLP-1-related side effects such as nausea and vomiting. In db/db mice, circulating levels of active GLP-1 were 10-20 pM with RJVA-001, compared to 50-150 pM typically reported with pharmacologic GLP-1 drugs1. Data show nutrient- and dose-responsive GLP-1 secretion in transduced human beta cells and islets, demonstrating that Rejuva mimics native hormone regulation rather than constant drug-driven stimulation: RJVA-001 activated glucose-dependent expression of GLP-1 in both in vitro and ex vivo models, consistent with physiologic endocrine function. In transduced human beta cells, GLP-1 secretion more than doubled— and GLP-1 bioactivity increased >3-fold — when shifting from low glucose to high glucose conditions. These results demonstrate tight nutrient gating and dose-responsive control of RJVA-001 drug action. Adaptive expression based on disease state: In db/db mice, RJVA-001 drove higher GLP-1 expression in diabetic animals than in healthy controls at the same dose, demonstrating the platform's ability to adapt to metabolic need. RJVA-001-treated healthy mice maintained normal weight and blood sugar, reinforcing the potential safety of this nutrient-responsive smart GLP-1. At equal dosing, db/db mice had more than twice the circulating GLP-1 levels of healthy mice, with no effect on weight or blood sugar in the healthy group. These results support the physiologic, selective, and adaptive action of RJVA-001 based on the body's metabolic state. Endoscopic ultrasound–guided delivery of RJVA-001 in large animals showed targeted pancreatic expression with no observed toxicity: In Yucatan pigs, device deployment was completed in an average procedure time of <20 minutes using a standard clinical endoscopic ultrasound technique. RJVA-001 localized to the pancreas with minimal systemic distribution and no adverse safety findings, even at doses exceeding projected clinical levels. Biodistribution studies showed vector copy number of 7 vector genomes/nucleus in the targeted splenic lobe of the pancreas (equivalent to the body and tail of the human pancreas) versus <0.2 vector genomes/nucleus in the liver. These results indicate profound de-targeting of the liver with local administration with the Rejuva catheter and ultrasound-guided route of administration. No acute or longer-term serum or histopathological evidence of toxicity was observed post-procedure. Serum lipase, neurofilament-light, troponin I, and ALT all remained below the upper limit of normal or below detection, indicating the absence of pancreatic, neuronal, cardiac, and liver toxicity, respectively. No on-target or off-target organ inflammation or other histopathological findings were observed in the study. 'We believe RJVA-001 represents a transformative advance in metabolic medicine,' said Dr. Harith Rajagopalan, Co-Founder and Chief Executive Officer of Fractyl Health. 'These data suggest that a one-time, smart GLP-1 can restore physiologic signaling in the pancreas and achieve durable disease modification in diabetes and obesity - without the high levels of systemic drug exposure that causes side effects with current GLP-1 therapies. RJVA-001 has demonstrated superior potency, durability, and convenience in preclinical models, along with a potentially improved tolerability profile than GLP-1 drugs. We believe the Rejuva platform has the potential to usher in a category-closing modality for T2D and obesity. We look forward to our upcoming CTA submission for RJVA-001 and, pending regulatory authorization, preliminary human data in 2026.' The full ASGCT presentation is available on the Fractyl Health website in the Presentations & Publications section. About Fractyl HealthFractyl Health is a metabolic therapeutics company focused on pattern-breaking approaches that treat root causes of obesity and T2D. Despite advances in treatment over the last 50 years, obesity and T2D continue to be rapidly growing drivers of morbidity and mortality in the 21st century. Fractyl Health's goal is to transform metabolic disease treatment from chronic symptomatic management to durable disease-modifying therapies that target the organ-level root causes of disease. Fractyl Health is based in Burlington, MA. For more information, visit or About Rejuva®Fractyl Health's Rejuva platform focuses on developing next-generation adeno-associated virus (AAV)-based, locally delivered gene therapies for the treatment of obesity and T2D. The Rejuva platform is in preclinical development and has not yet been evaluated by regulatory agencies for investigational or commercial use. Rejuva leverages advanced delivery systems and proprietary screening methods to identify and develop metabolically active gene therapy candidates targeting the pancreas. The program aims to transform the management of metabolic diseases by offering novel, disease-modifying therapies that address the underlying root causes of disease. The Company plans to submit the first Clinical Trial Application (CTA) module for RJVA-001 in type 2 diabetes to regulators by June 2025, and if the CTA is authorized, the Company expects to dose the first patients with RJVA-001 and report preliminary data in 2026. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the promise and potential impact of our preclinical or clinical trial data, the design, initiation, timing and results of clinical enrollment and any clinical studies or readouts, the content, information used for, timing or results of any investigational new drug (IND)-enabling studies, IND applications or Clinical Trial Applications, communications with regulators, the potential launch or commercialization of any of our product candidates or products, the potential treatment population or benefits for any of our product candidates or products, and our strategic and product development objectives and goals, including with respect to enabling long-term control over obesity and type 2 diabetes without the burden of chronic therapies, redefining the future of metabolic disease treatment, positioning our Company at the forefront of the global opportunity for metabolic care, and the timing of any of the foregoing. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause the Company's actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the Company's limited operating history; the incurrence of significant net losses and the fact that the Company expects to continue to incur significant net losses for the foreseeable future; the Company's need for substantial additional financing; the Company's ability to continue as a going concern; the restrictive and financial covenants in the Company's credit agreement; the lengthy and unpredictable regulatory approval process for the Company's product candidates; uncertainty regarding its clinical studies; the fact that the Company's product candidates may cause serious adverse events or undesirable side effects or have other properties that may cause it to suspend or discontinue clinical studies, delay or prevent regulatory development, prevent their regulatory approval, limit the commercial profile, or result in significant negative consequences; additional time may be required to develop and obtain regulatory approval or certification for the Company's Rejuva gene therapy candidates; the Company's reliance on third parties to conduct certain aspects of the Company's preclinical studies and clinical studies; the Company's reliance on third parties for the manufacture of the materials for its Rejuva gene therapy platform for preclinical studies and its ongoing clinical studies; the regulatory approval process of the FDA, comparable foreign regulatory authorities and lengthy, time-consuming and inherently unpredictable, and even if we complete the necessary clinical studies, we cannot predict when, or if, we will obtain regulatory approval or certification for any of our product candidates, and any such regulatory approval or certification may be for a more narrow indication than we seek; and the potential launch or commercialization of any of Company's product candidates or products and our strategic and product development objectives and goals, and the other factors discussed under the caption 'Risk Factors' in our Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (the SEC) on May 13, 2025 and in our other filings with the SEC. These forward-looking statements are based on management's current estimates and expectations. While the Company may elect to update such forward-looking statements at some point in the future, the Company disclaims any obligation to do so, even if subsequent events cause its views to change. Contacts Media Contact Jessica Cotrone, Corporate Communications jcotrone@ 978.760.5622 Investor Contact Brian Luque, Head of Investor Relations and Corporate DevelopmentIR@ 951.206.1200 _________________1 Smits MM, Holst JJ. Endogenous glucagon-like peptide (GLP)-1 as alternative for GLP-1 receptor agonists: Could this work and how? Diabetes Metab Res Rev. 2023 Nov;39(8):e3699. doi: 10.1002/dmrr.3699. Epub 2023 Jul 24. PMID: 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
17-05-2025
- Business
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Fractyl Health Unveils New Rejuva® Smart GLP-1™ Pancreatic Gene Therapy Preclinical Data Highlighting Durable Potency and Safety with Limited Systemic GLP-1 Exposure at ASGCT 2025
Data suggests that single dose of RJVA-001 leads to durable metabolic improvements with low systemic GLP-1 exposure in db/db mouse model of T2D— potentially simultaneously addressing durability, adherence, and tolerability challenges seen with current GLP-1 drugs Endoscopic ultrasound-guided delivery achieves targeted pancreatic expression in a large animal model with no toxicity observed to date Data show nutrient-responsive GLP-1 secretion in human beta cells and human islets, demonstrating that Rejuva mimics natural hormone regulation rather than constant drug-driven stimulation Rejuva advancing toward first-in-human studies; first CTA module submission for RJVA-001 expected by June 2025 BURLINGTON, Mass., May 17, 2025 (GLOBE NEWSWIRE) -- Fractyl Health, Inc. (Nasdaq: GUTS) (the Company), a metabolic therapeutics company focused on pattern-breaking approaches that treat root causes of obesity and type 2 diabetes (T2D), today announced an oral presentation of new preclinical data from its Rejuva smart GLP-1 gene therapy platform at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting. The data highlight RJVA-001's potential to deliver durable, nutrient-responsive GLP-1 secretion from pancreatic beta cells—mimicking natural hormone regulation with low circulating levels of GLP-1, offering a potentially profound mechanistic advantage over pharmacologic GLP-1 drugs. RJVA-001 also showed strong efficacy, targeted delivery, and a favorable safety profile, reinforcing its readiness for first-in-human studies. The data were featured in an oral presentation at ASGCT titled 'Endoscopic Ultrasound-Guided Delivery of Human Glucagon-like Peptide-1 Pancreatic Gene Therapy: Safety and Feasibility in a Porcine Model.' 'RJVA-001 represents a fundamentally different approach to treating metabolic disease - one that delivers the power of GLP-1 in a more natural manner,' said Professor Randy Seeley, Ph.D., Henry King Ransom Professor of Surgery and Director of Michigan Nutrition Obesity Research Center at Michigan School of Medicine. 'The ability to drive durable metabolic effects with physiologic hormone levels, from a one-time treatment, would be a scientific breakthrough with huge potential implications for patients. This has the potential to reshape how we think about treating T2D and obesity at scale.' Key Data Presented at ASGCT 2025: Single-dose RJVA-001 led to durable, dose-dependent metabolic improvements in a well-established diabetic model: In db/db mice, treatment with RJVA-001 led to sustained reductions in blood sugar, improved fasting insulin levels, and improvements in body weight over six weeks— supporting Rejuva's potential for sustained disease modification on both blood sugar and body weight control. Treatment with RJVA-001 resulted in a >200 mg/dL reduction in fasting blood sugar, a >2-fold increase in fasting insulin levels, and prevention of weight gain, compared to a ~20% increase seen in vehicle-treated controls. These results demonstrate broad-based metabolic improvement in insulin secretion, weight gain, and blood sugar control in this gold-standard model of T2D. RJVA-001 achieved glycemic control and prevented weight gain with significantly lower systemic GLP-1 exposure than required by GLP-1 drugs to achieve similar effects: Circulating GLP-1 levels were more than 5-fold lower than those seen with pharmacologic GLP-1 drugs and were comparable to levels observed after gastric bypass surgery—suggesting a substantially lower risk of GLP-1-related side effects such as nausea and vomiting. In db/db mice, circulating levels of active GLP-1 were 10-20 pM with RJVA-001, compared to 50-150 pM typically reported with pharmacologic GLP-1 drugs1. Data show nutrient- and dose-responsive GLP-1 secretion in transduced human beta cells and islets, demonstrating that Rejuva mimics native hormone regulation rather than constant drug-driven stimulation: RJVA-001 activated glucose-dependent expression of GLP-1 in both in vitro and ex vivo models, consistent with physiologic endocrine function. In transduced human beta cells, GLP-1 secretion more than doubled— and GLP-1 bioactivity increased >3-fold — when shifting from low glucose to high glucose conditions. These results demonstrate tight nutrient gating and dose-responsive control of RJVA-001 drug action. Adaptive expression based on disease state: In db/db mice, RJVA-001 drove higher GLP-1 expression in diabetic animals than in healthy controls at the same dose, demonstrating the platform's ability to adapt to metabolic need. RJVA-001-treated healthy mice maintained normal weight and blood sugar, reinforcing the potential safety of this nutrient-responsive smart GLP-1. At equal dosing, db/db mice had more than twice the circulating GLP-1 levels of healthy mice, with no effect on weight or blood sugar in the healthy group. These results support the physiologic, selective, and adaptive action of RJVA-001 based on the body's metabolic state. Endoscopic ultrasound–guided delivery of RJVA-001 in large animals showed targeted pancreatic expression with no observed toxicity: In Yucatan pigs, device deployment was completed in an average procedure time of <20 minutes using a standard clinical endoscopic ultrasound technique. RJVA-001 localized to the pancreas with minimal systemic distribution and no adverse safety findings, even at doses exceeding projected clinical levels. Biodistribution studies showed vector copy number of 7 vector genomes/nucleus in the targeted splenic lobe of the pancreas (equivalent to the body and tail of the human pancreas) versus <0.2 vector genomes/nucleus in the liver. These results indicate profound de-targeting of the liver with local administration with the Rejuva catheter and ultrasound-guided route of administration. No acute or longer-term serum or histopathological evidence of toxicity was observed post-procedure. Serum lipase, neurofilament-light, troponin I, and ALT all remained below the upper limit of normal or below detection, indicating the absence of pancreatic, neuronal, cardiac, and liver toxicity, respectively. No on-target or off-target organ inflammation or other histopathological findings were observed in the study. 'We believe RJVA-001 represents a transformative advance in metabolic medicine,' said Dr. Harith Rajagopalan, Co-Founder and Chief Executive Officer of Fractyl Health. 'These data suggest that a one-time, smart GLP-1 can restore physiologic signaling in the pancreas and achieve durable disease modification in diabetes and obesity - without the high levels of systemic drug exposure that causes side effects with current GLP-1 therapies. RJVA-001 has demonstrated superior potency, durability, and convenience in preclinical models, along with a potentially improved tolerability profile than GLP-1 drugs. We believe the Rejuva platform has the potential to usher in a category-closing modality for T2D and obesity. We look forward to our upcoming CTA submission for RJVA-001 and, pending regulatory authorization, preliminary human data in 2026.' The full ASGCT presentation is available on the Fractyl Health website in the Presentations & Publications section. About Fractyl HealthFractyl Health is a metabolic therapeutics company focused on pattern-breaking approaches that treat root causes of obesity and T2D. Despite advances in treatment over the last 50 years, obesity and T2D continue to be rapidly growing drivers of morbidity and mortality in the 21st century. Fractyl Health's goal is to transform metabolic disease treatment from chronic symptomatic management to durable disease-modifying therapies that target the organ-level root causes of disease. Fractyl Health is based in Burlington, MA. For more information, visit or About Rejuva®Fractyl Health's Rejuva platform focuses on developing next-generation adeno-associated virus (AAV)-based, locally delivered gene therapies for the treatment of obesity and T2D. The Rejuva platform is in preclinical development and has not yet been evaluated by regulatory agencies for investigational or commercial use. Rejuva leverages advanced delivery systems and proprietary screening methods to identify and develop metabolically active gene therapy candidates targeting the pancreas. The program aims to transform the management of metabolic diseases by offering novel, disease-modifying therapies that address the underlying root causes of disease. The Company plans to submit the first Clinical Trial Application (CTA) module for RJVA-001 in type 2 diabetes to regulators by June 2025, and if the CTA is authorized, the Company expects to dose the first patients with RJVA-001 and report preliminary data in 2026. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the promise and potential impact of our preclinical or clinical trial data, the design, initiation, timing and results of clinical enrollment and any clinical studies or readouts, the content, information used for, timing or results of any investigational new drug (IND)-enabling studies, IND applications or Clinical Trial Applications, communications with regulators, the potential launch or commercialization of any of our product candidates or products, the potential treatment population or benefits for any of our product candidates or products, and our strategic and product development objectives and goals, including with respect to enabling long-term control over obesity and type 2 diabetes without the burden of chronic therapies, redefining the future of metabolic disease treatment, positioning our Company at the forefront of the global opportunity for metabolic care, and the timing of any of the foregoing. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause the Company's actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the Company's limited operating history; the incurrence of significant net losses and the fact that the Company expects to continue to incur significant net losses for the foreseeable future; the Company's need for substantial additional financing; the Company's ability to continue as a going concern; the restrictive and financial covenants in the Company's credit agreement; the lengthy and unpredictable regulatory approval process for the Company's product candidates; uncertainty regarding its clinical studies; the fact that the Company's product candidates may cause serious adverse events or undesirable side effects or have other properties that may cause it to suspend or discontinue clinical studies, delay or prevent regulatory development, prevent their regulatory approval, limit the commercial profile, or result in significant negative consequences; additional time may be required to develop and obtain regulatory approval or certification for the Company's Rejuva gene therapy candidates; the Company's reliance on third parties to conduct certain aspects of the Company's preclinical studies and clinical studies; the Company's reliance on third parties for the manufacture of the materials for its Rejuva gene therapy platform for preclinical studies and its ongoing clinical studies; the regulatory approval process of the FDA, comparable foreign regulatory authorities and lengthy, time-consuming and inherently unpredictable, and even if we complete the necessary clinical studies, we cannot predict when, or if, we will obtain regulatory approval or certification for any of our product candidates, and any such regulatory approval or certification may be for a more narrow indication than we seek; and the potential launch or commercialization of any of Company's product candidates or products and our strategic and product development objectives and goals, and the other factors discussed under the caption 'Risk Factors' in our Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (the SEC) on May 13, 2025 and in our other filings with the SEC. These forward-looking statements are based on management's current estimates and expectations. While the Company may elect to update such forward-looking statements at some point in the future, the Company disclaims any obligation to do so, even if subsequent events cause its views to change. Contacts Media Contact Jessica Cotrone, Corporate Communications jcotrone@ 978.760.5622 Investor Contact Brian Luque, Head of Investor Relations and Corporate DevelopmentIR@ 951.206.1200 _________________1 Smits MM, Holst JJ. Endogenous glucagon-like peptide (GLP)-1 as alternative for GLP-1 receptor agonists: Could this work and how? Diabetes Metab Res Rev. 2023 Nov;39(8):e3699. doi: 10.1002/dmrr.3699. Epub 2023 Jul 24. PMID: while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data
Yahoo
13-05-2025
- Business
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Countable Labs Launches Category-defining, Highly Sensitive PCR Platform — Countable PCR — Finally Quantifying Rare Events, Even From Limited Samples
PALO ALTO, Calif., May 13, 2025--(BUSINESS WIRE)--Countable Labs (formerly Enumerix) enters the genomics market with the launch of Countable PCR, redefining what's possible with PCR for rare event detection. By delivering 10 times more sensitivity and 10 times lower CVs than digital PCR, Countable PCR gives scientists the confidence they've been looking for when quantifying rare events, even from limited samples. "Scientists have always been forced to compromise," says Eleen Shum, founding scientist & VP of Product at Countable Labs. "Quantitative PCR offers speed but sacrifices accuracy. Digital PCR makes strides towards precision but brings complexity with not a lot of cost savings. And, Next Generation Sequencing is powerful but comes with significant overhead. Countable PCR ends that compromise." At the heart of Countable PCR's new technology is the ability to truly isolate single molecules within over 30 million compartments in a 3D gel matrix. From there, detecting and counting up to one million molecules is simple, direct, and precise. And, to cut time and cost from current workflows, Countable PCR allows scientists to go from experiment idea to results much faster with the introduction of its Universal Multiplexing Kit — a quicker, cost-effective way to develop multiplex assays in three days and not months. "This isn't just about quantifying rare events," says Jocelyn Davé, CEO of Countable Labs. "It's about removing friction from genomics workflows so applications that were previously too slow, too complex, or too expensive to scale can finally become routine." Countable Labs debuts Countable PCR at ASGCT 2025 from May 13 - 17 in New Orleans. To learn more about Countable PCR, visit About Countable Labs Countable Labs is redefining PCR with single-molecule precision and category-defining sensitivity, overcoming the limitations of legacy technologies. Countable PCR delivers 10 times the sensitivity and precision of digital PCR in a single reaction. Plus, it speeds up assay development with simplified multiplexing. These breakthroughs unlock high-sensitivity applications that were previously impractical due to cost and complexity. View source version on Contacts John Valdez, VP of Marketingjvaldez@ 650.468.0054
Associated Press
13-05-2025
- Business
- Associated Press
Tenaya Therapeutics to Highlight Capsid Engineering, Gene Editing and Manufacturing Research at the ASGCT 28th Annual Meeting
SOUTH SAN FRANCISCO, Calif., May 13, 2025 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced that it will present five abstracts at the upcoming American Society of Gene and Cell Therapy (ASGCT 2025) 28th Annual Meeting taking place May 13-17, 2025 in New Orleans, LA. The abstracts being presented at ASGCT 2025 capture the outcome of efforts to advance Tenaya's core capabilities in novel capsid engineering, identification, design and optimization of cardiomyocyte-targeting genetic medicines, and manufacturing of adeno-associated virus (AAV) gene therapies. Abstract highlights Details of Tenaya's ASGCT 2025 presentations are as follows: Tuesday, May 13, 2025 Poster Abstract Session - 6:00 pm – 7:30 pm Wednesday, May 14, 2025 Poster Abstract Session – 5:30 pm – 7:30 pm Following the conference, Tenaya's presentations will be available in the 'Our Science' section of the company's website. About Tenaya Therapeutics Tenaya Therapeutics is a clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Tenaya employs a suite of integrated internal capabilities, including modality agnostic target validation, capsid engineering and manufacturing, to generate a portfolio of genetic medicines aimed at the treatment of both rare genetic disorders and more prevalent heart conditions. Tenaya's pipeline includes TN-201, a gene therapy for MYBPC3 -associated hypertrophic cardiomyopathy (HCM), TN-401, a gene therapy for PKP2 -associated arrhythmogenic right ventricular cardiomyopathy (ARVC), TN-301, a small molecule HDAC6 inhibitor intended for heart failure with preserved ejection fraction (HFpEF), and multiple early-stage programs in preclinical development. For more information, visit Forward-Looking Statements This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Words such as 'will,' 'potential,' 'may,' and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, the presentation of data covering Tenaya's capabilities in novel capsid engineering, identification, design and optimization of cardiomyocyte-targeting genetic medicines, and manufacturing of AAV gene therapies; the therapeutic potential of Tenaya's prime editing technology in a humanized RBM20 murine model of DCM; TN-501 as a potential treatment for PLN R14del-associated DCM; the commercial potential of Tenaya's manufacturing process to support the development of more cost-effective AAV gene therapies. The forward-looking statements contained herein are based upon Tenaya's current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including but not limited to: availability of data at the referenced times; risks associated with the process of discovering, developing and commercializing therapies that are safe and effective for use as human therapeutics; Tenaya's ability to develop, initiate or complete preclinical studies and clinical trials, and obtain approvals, for any of its product candidates; Tenaya's continuing compliance with applicable legal and regulatory requirements; Tenaya's ability to raise any additional funding it will need to continue to pursue its business and product development plans; Tenaya's reliance on third parties; Tenaya's manufacturing, commercialization and marketing capabilities and strategy; the loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenaya's ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled 'Risk Factors' in documents that Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Contact Michelle Corral VP, Corporate Communications and Investor Relations Tenaya Therapeutics [email protected] Investors AnneMarie Fields Stern IR [email protected] Media Wendy Ryan Ten Bridge Communications [email protected]
Yahoo
13-05-2025
- Business
- Yahoo
Tenaya Therapeutics to Highlight Capsid Engineering, Gene Editing and Manufacturing Research at the ASGCT 28th Annual Meeting
Advancements Lay the Groundwork for Future Genetic Medicines for Rare and Prevalent Forms of Heart Disease SOUTH SAN FRANCISCO, Calif., May 13, 2025 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced that it will present five abstracts at the upcoming American Society of Gene and Cell Therapy (ASGCT 2025) 28th Annual Meeting taking place May 13-17, 2025 in New Orleans, LA. The abstracts being presented at ASGCT 2025 capture the outcome of efforts to advance Tenaya's core capabilities in novel capsid engineering, identification, design and optimization of cardiomyocyte-targeting genetic medicines, and manufacturing of adeno-associated virus (AAV) gene therapies. Abstract highlights : Building on previous research that showed the superior cardiomyocyte-targeting and robust transduction attributes of AAV serotype 9 (AAV9) compared to other naturally occurring capsids, Tenaya's researchers combined high throughput in vivo experimental screening with multiple in silico screening tools to efficiently identify various novel capsid candidates. Novel capsids were then compared to one another and to AAV9 in murine and non-human primate models. Select top performing capsids were further evaluated and found to outperform AAV9 in terms of cardiomyocyte targeting and efficient in vivo cardiac gene therapy. Tenaya researchers will share efforts to develop prime editing, a precision gene editing technique, aimed at cardiomyocytes. The machinery required for prime editing exceed the capacity of a single AAV capsid, so Tenaya researchers created a prototype utilizing one of the company's engineered AAV capsids, a dual cassette arrangement delivered via two AAV capsids, and cardiomyocyte-specific regulatory elements. The therapeutic potential of this prime editing prototype was studied in an RBM20 murine model of DCM. The prime editing prototype successfully achieved correction of the mutated RBM20 allele, improving cardiac function and reversing disease in the mouse model. A humanized mouse model of RBM20-mutant DCM was also developed and validated by Tenaya researchers. Testing of the prototype in this model demonstrated effective cardiac editing of the humanized allele using the in vivo dual vector prime editing approach. : Tenaya previously presented research on the development of a Cas9 gene editing therapy, TN-501, designed to specifically inactivate the mutant phospholamban (PLN)-R14del allele, and thereby eliminate the "poison peptide" effects of the mutant protein while preserving wild-type function. Preclinical studies in murine models demonstrated that a low dose of a murine surrogate of TN-501 effectively prevented mutant PLN protein aggregation, reduced cardiac fibrosis, improved cardiac function, and significantly enhanced survival to wild-type levels. Doses tested were well tolerated and anti-Cas9 antibody responses and Cas9 T-cell activation were low. Tenaya successfully established Sf9/rBV-based manufacturing processes at the 1000L scale for its clinical-stage AAV gene therapy programs and subsequently also established a proprietary HEK293-based manufacturing process. Tenaya's HEK393 process utilizes a single plasmid expression system, as well novel transfection reagents, to improve overall yield while lowering overall costs compared to current commercially available options, which may support the development of more cost-effective AAV gene therapies. Details of Tenaya's ASGCT 2025 presentations are as follows: Tuesday, May 13, 2025Poster Abstract Session - 6:00 pm – 7:30 pm Abstract: #523Title: A Humanized RBM20 Mouse Model Exhibits Dilated Cardiomyopathy Phenotypes and Enables Development of In Vivo Prime Editing for Treating Human RBM20 Cardiomyopathy PatientsPresenting author: Wenjing Liang, Ph.D., Scientist Abstract: #952Title: High Productivity HEK293 AAV Production Platform Enabled by Novel Transfection Reagents and Proprietary Plasmid Expression SystemsPresenting author: Charles Feathers, Senior Manager, Process Development Wednesday, May 14, 2025Poster Abstract Session – 5:30 pm – 7:30 pm Abstract: #1022Title: TN-501 Gene Editing Therapy for PLN-R14del-Associated CardiomyopathyPresenting author: Huanyu Zhou, Ph.D., Associate Director Abstract: #1028Title: Developing In Vivo Prime Editing as a Potential Treatment Option for Heart DiseasePresenting author: Lindsey Rollosson, Research Associate II Abstract: #1396 Title: Engineering Novel AAV Capsids for Cardiac Gene DeliveryPresenting author: Ze Cheng, Ph.D., Principal Scientist Following the conference, Tenaya's presentations will be available in the 'Our Science' section of the company's website. About Tenaya TherapeuticsTenaya Therapeutics is a clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Tenaya employs a suite of integrated internal capabilities, including modality agnostic target validation, capsid engineering and manufacturing, to generate a portfolio of genetic medicines aimed at the treatment of both rare genetic disorders and more prevalent heart conditions. Tenaya's pipeline includes TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), TN-301, a small molecule HDAC6 inhibitor intended for heart failure with preserved ejection fraction (HFpEF), and multiple early-stage programs in preclinical development. For more information, visit Forward-Looking StatementsThis press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Words such as 'will,' 'potential,' 'may,' and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, the presentation of data covering Tenaya's capabilities in novel capsid engineering, identification, design and optimization of cardiomyocyte-targeting genetic medicines, and manufacturing of AAV gene therapies; the therapeutic potential of Tenaya's prime editing technology in a humanized RBM20 murine model of DCM; TN-501 as a potential treatment for PLN R14del-associated DCM; the commercial potential of Tenaya's manufacturing process to support the development of more cost-effective AAV gene therapies. The forward-looking statements contained herein are based upon Tenaya's current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including but not limited to: availability of data at the referenced times; risks associated with the process of discovering, developing and commercializing therapies that are safe and effective for use as human therapeutics; Tenaya's ability to develop, initiate or complete preclinical studies and clinical trials, and obtain approvals, for any of its product candidates; Tenaya's continuing compliance with applicable legal and regulatory requirements; Tenaya's ability to raise any additional funding it will need to continue to pursue its business and product development plans; Tenaya's reliance on third parties; Tenaya's manufacturing, commercialization and marketing capabilities and strategy; the loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenaya's ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled 'Risk Factors' in documents that Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Contact Michelle CorralVP, Corporate Communications and Investor RelationsTenaya TherapeuticsIR@ InvestorsAnneMarie FieldsStern IR MediaWendy RyanTen Bridge Communicationswendy@
