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Business Wire
27-05-2025
- Business
- Business Wire
SAGA Diagnostics® Announces U.S. Commercial Launch of Pathlight™ at ASCO 2025, Setting a New Standard for Ultra-sensitive and Early MRD Detection
MORRISVILLE, N.C.--(BUSINESS WIRE)-- SAGA Diagnostics, a pioneer in blood-based cancer detection and precision medicine redefining the standard for ultra-sensitive and early molecular residual disease (MRD) detection, today announced the U.S. commercial launch of its Pathlight test for the detection of residual disease and recurrence at the 2025 American Society for Clinical Oncology (ASCO) Annual Meeting in Chicago, May 30 - June 3. Pathlight's baseline detection rate was 96% overall, and 94% in estrogen receptor-positive (ER+) breast cancer, which suggests clinical advantages over first generation ctDNA assays. Pathlight is a first-of-its-kind, multi-cancer MRD platform, initially indicated for early breast cancer, that uses structural variants (SVs) as biomarkers. SVs are well-established hallmarks of cancer, arising from and contributing to genomic instability and oncogenesis. SVs, including breakpoints and rearrangements, are highly tumor- and patient-specific and often reflect the underlying tumor biology making them informative biomarkers for tracking disease. Pathlight combines whole genome sequencing and proprietary algorithms and informatics optimized for the identification of stable SV breakpoints, generating a personalized genomic fingerprint for each tumor, which are subsequently orthogonally validated and tracked utilizing proprietary multiplex digital PCR to enable rapid, precise, efficient and quantitative MRD detection from a simple blood draw. 'By tracking structural variants — stable, unique, and tumor-defining fingerprints of each patient's cancer — Pathlight enables interception of recurrence at its most treatable and potentially curable stage,' commented Roopom Banerjee, Executive Chairman of SAGA Diagnostics. 'Our goal is to deliver the most accurate, trusted results that provide assurance and support confident, informed treatment decisions. Our published, peer reviewed data demonstrates the clinical validity of Pathlight in breast cancer and its potential power across all solid tumors and heme malignancies. SAGA has further partnered with leading clinicians, institutions, and biopharma to bring Pathlight to market and best serve patients.' The clinical validity of Pathlight was established with samples collected in the TRACER (cTdna evaluation in eaRly breAst canCER) study, published in Clinical Cancer Research 1 in January 2025. In this retrospective analysis of 100 patients with stage I–III breast cancer of all subtypes receiving standard-of-care neoadjuvant and adjuvant therapy Pathlight demonstrated best-in-class clinical performance – with 100% sensitivity and 100% specificity, and a 13.7-month lead time to recurrence as confirmed by clinical presentation or imaging. Notably, Pathlight's baseline detection rate was 96% overall, and 94% in estrogen receptor-positive (ER+) breast cancer, which suggests clinical advantages over first generation ctDNA assays. Pathlight also delivered unparalleled sensitivity – breaking the 1ppm barrier and offers the potential to detect MRD at the earliest possible opportunity, to support individualized intervention directed towards cure. 'The vast majority of breast cancer patients are diagnosed with ER+ disease, and up to 25% will experience recurrence – often many years after completing curative-intent treatment,' said David Cescon, MD, PhD, medical oncologist and clinician scientist at Princess Margaret Cancer Centre, University Health Network and senior author of the paper. 'We know that continued improvement in outcomes for people diagnosed with early breast cancer will require a shift towards more individualized treatment. There is a critical need for MRD testing that is both ultra-sensitive and ultra-specific, to enable long-term surveillance and risk-aligned treatment decisions for each patient.' Pathlight has been submitted for US Reimbursement via the MolDX Program and is analytically validated across multiple cancer types. The test is already being used successfully in clinical studies by top pharmaceutical companies and at preeminent academic institutions and national cancer centers. SAGA Diagnostics and its collaborators will be presenting three abstracts highlighting Pathlight's performance in breast cancer, ovarian cancer and soft-tissue sarcoma at ASCO: Abstract #11511| June 1 | 9:00 a.m. – 12:00 p.m. CDT Title: Potential of tumor-informed ctDNA as an early predictor for relapse in advanced ovarian cancer Abstract #5574 | June 2 | 11:30 a.m. – 1:00 p.m. CDT Title: Detecting ctDNA using personalized structural variants to forecast recurrence in localized soft tissue sarcoma (STS) Abstract #3057 | June 2 | 1:30 p.m. – 4:30 p.m. CDT Title: NeoCircle: Investigating circulating tumor DNA dynamics as a predictor of survival in primary breast cancer SAGA will also be showcasing the capabilities and partnership opportunities of Pathlight at its booth (#36150) during the conference. References Elliott MJ, Howarth K, Main S, et al. Ultrasensitive detection and monitoring of circulating tumor DNA using structural variants in early-stage breast cancer. Clin Cancer Res. 2025;31(8):1520-1532. doi:10.1158/ About Pathlight Pathlight™ is a next-generation, tumor-informed multi-cancer molecular residual disease (MRD) test, initially indicated for commercial testing in breast cancer. Pathlight uses whole genome sequencing to identify large-scale genomic changes, called structural variants (SVs), and tracks them over time using a unique combination of NGS and digital PCR, enabling industry-leading sensitivity and specificity. By optimizing for SVs that are stable, truncal biomarkers, Pathlight enables ultrasensitive recurrence detection and treatment response monitoring from early stage to metastatic disease. Developed and marketed by SAGA Diagnostics, the test has been submitted for US Reimbursement via the MolDX Program and is being used in clinical studies by multiple top 10 pharmaceutical companies and leading academic institutions and national cancer centers. For more information, About SAGA Diagnostics SAGA Diagnostics® is redefining the early detection of molecular residual disease (MRD), empowering treatment decisions with greater insight and confidence. Pathlight™, the company's flagship product, is an ultra-sensitive, blood-based, multi-cancer MRD test that is now available for commercial use in the U.S. for patients with breast cancer. SAGA is partnering with pharmaceutical and biotechnology companies, as well as commercial entities to support early through late-stage cancer development programs across a range of cancer types. SAGA's headquarters and CLIA-certified laboratory is located in the heart of the life science ecosystem in Research Triangle Park, North Carolina. SAGA Diagnostics combines world-class genomic expertise with a leadership team deeply experienced in molecular residual disease (MRD), all aligned around a compelling mission of intercepting cancer at the earliest stages when it's most treatable. For more information,
Yahoo
27-05-2025
- Business
- Yahoo
SAGA Diagnostics® Announces U.S. Commercial Launch of Pathlight™ at ASCO 2025, Setting a New Standard for Ultra-sensitive and Early MRD Detection
Pathlight is a tumor-informed, multi-cancer MRD platform with its first indication in breast cancer Published, peer-reviewed clinical study demonstrates 100% sensitivity, 100% specificity, and a 13.7-month lead time to recurrence across all subtypes and Stage I-III breast cancer Submitted for US Reimbursement via the MolDX Program MORRISVILLE, N.C., May 27, 2025--(BUSINESS WIRE)--SAGA Diagnostics, a pioneer in blood-based cancer detection and precision medicine redefining the standard for ultra-sensitive and early molecular residual disease (MRD) detection, today announced the U.S. commercial launch of its Pathlight test for the detection of residual disease and recurrence at the 2025 American Society for Clinical Oncology (ASCO) Annual Meeting in Chicago, May 30 - June 3. Pathlight is a first-of-its-kind, multi-cancer MRD platform, initially indicated for early breast cancer, that uses structural variants (SVs) as biomarkers. SVs are well-established hallmarks of cancer, arising from and contributing to genomic instability and oncogenesis. SVs, including breakpoints and rearrangements, are highly tumor- and patient-specific and often reflect the underlying tumor biology making them informative biomarkers for tracking disease. Pathlight combines whole genome sequencing and proprietary algorithms and informatics optimized for the identification of stable SV breakpoints, generating a personalized genomic fingerprint for each tumor, which are subsequently orthogonally validated and tracked utilizing proprietary multiplex digital PCR to enable rapid, precise, efficient and quantitative MRD detection from a simple blood draw. "By tracking structural variants — stable, unique, and tumor-defining fingerprints of each patient's cancer — Pathlight enables interception of recurrence at its most treatable and potentially curable stage," commented Roopom Banerjee, Executive Chairman of SAGA Diagnostics. "Our goal is to deliver the most accurate, trusted results that provide assurance and support confident, informed treatment decisions. Our published, peer reviewed data demonstrates the clinical validity of Pathlight in breast cancer and its potential power across all solid tumors and heme malignancies. SAGA has further partnered with leading clinicians, institutions, and biopharma to bring Pathlight to market and best serve patients." The clinical validity of Pathlight was established with samples collected in the TRACER (cTdna evaluation in eaRly breAst canCER) study, published in Clinical Cancer Research1 in January 2025. In this retrospective analysis of 100 patients with stage I–III breast cancer of all subtypes receiving standard-of-care neoadjuvant and adjuvant therapy Pathlight demonstrated best-in-class clinical performance – with 100% sensitivity and 100% specificity, and a 13.7-month lead time to recurrence as confirmed by clinical presentation or imaging. Notably, Pathlight's baseline detection rate was 96% overall, and 94% in estrogen receptor-positive (ER+) breast cancer, which suggests clinical advantages over first generation ctDNA assays. Pathlight also delivered unparalleled sensitivity – breaking the 1ppm barrier and offers the potential to detect MRD at the earliest possible opportunity, to support individualized intervention directed towards cure. "The vast majority of breast cancer patients are diagnosed with ER+ disease, and up to 25% will experience recurrence – often many years after completing curative-intent treatment," said David Cescon, MD, PhD, medical oncologist and clinician scientist at Princess Margaret Cancer Centre, University Health Network and senior author of the paper. "We know that continued improvement in outcomes for people diagnosed with early breast cancer will require a shift towards more individualized treatment. There is a critical need for MRD testing that is both ultra-sensitive and ultra-specific, to enable long-term surveillance and risk-aligned treatment decisions for each patient." Pathlight has been submitted for US Reimbursement via the MolDX Program and is analytically validated across multiple cancer types. The test is already being used successfully in clinical studies by top pharmaceutical companies and at preeminent academic institutions and national cancer centers. SAGA Diagnostics and its collaborators will be presenting three abstracts highlighting Pathlight's performance in breast cancer, ovarian cancer and soft-tissue sarcoma at ASCO: Abstract #11511| June 1 | 9:00 a.m. – 12:00 p.m. CDTTitle: Potential of tumor-informed ctDNA as an early predictor for relapse in advanced ovarian cancer Abstract #5574 | June 2 | 11:30 a.m. – 1:00 p.m. CDTTitle: Detecting ctDNA using personalized structural variants to forecast recurrence in localized soft tissue sarcoma (STS) Abstract #3057 | June 2 | 1:30 p.m. – 4:30 p.m. CDTTitle: NeoCircle: Investigating circulating tumor DNA dynamics as a predictor of survival in primary breast cancer SAGA will also be showcasing the capabilities and partnership opportunities of Pathlight at its booth (#36150) during the conference. References Elliott MJ, Howarth K, Main S, et al. Ultrasensitive detection and monitoring of circulating tumor DNA using structural variants in early-stage breast cancer. Clin Cancer Res. 2025;31(8):1520-1532. doi:10.1158/ About Pathlight Pathlight™ is a next-generation, tumor-informed multi-cancer molecular residual disease (MRD) test, initially indicated for commercial testing in breast cancer. Pathlight uses whole genome sequencing to identify large-scale genomic changes, called structural variants (SVs), and tracks them over time using a unique combination of NGS and digital PCR, enabling industry-leading sensitivity and specificity. By optimizing for SVs that are stable, truncal biomarkers, Pathlight enables ultrasensitive recurrence detection and treatment response monitoring from early stage to metastatic disease. Developed and marketed by SAGA Diagnostics, the test has been submitted for US Reimbursement via the MolDX Program and is being used in clinical studies by multiple top 10 pharmaceutical companies and leading academic institutions and national cancer centers. For more information, About SAGA Diagnostics SAGA Diagnostics® is redefining the early detection of molecular residual disease (MRD), empowering treatment decisions with greater insight and confidence. Pathlight™, the company's flagship product, is an ultra-sensitive, blood-based, multi-cancer MRD test that is now available for commercial use in the U.S. for patients with breast cancer. SAGA is partnering with pharmaceutical and biotechnology companies, as well as commercial entities to support early through late-stage cancer development programs across a range of cancer types. SAGA's headquarters and CLIA-certified laboratory is located in the heart of the life science ecosystem in Research Triangle Park, North Carolina. SAGA Diagnostics combines world-class genomic expertise with a leadership team deeply experienced in molecular residual disease (MRD), all aligned around a compelling mission of intercepting cancer at the earliest stages when it's most treatable. For more information, View source version on Contacts Media Contactmedia@ Error 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
20-05-2025
- Politics
- Yahoo
Pueblo Republican launches bid to unseat Nick Hinrichsen in Colorado Senate District 3
A local healthcare practice manager with nearly 25 years of experience is venturing into politics as a 2026 Republican candidate for Colorado State Senate District 3. Dana Charles told the Chieftain she is "not a politician," but is running for a state Senate seat because she is concerned about state fees, high taxation and legislative "attacks" on rights. Charles lists affordability, parental rights, Second Amendment rights, public safety, and preserving Colorado's Taxpayer Bill of Rights (TABOR), among her priorities. "I just was so tired of questioning whether our Pueblo legislators were actually working for the people of Pueblo," she told the Chieftain. "I felt like enough was enough and I wanted to bring a voice to the state Senate that you don't have to wonder about." On her website, Charles said that recent legislation passed by members of the Colorado General Assembly has "tied the hands of law enforcement" and jeopardized the safety of Pueblo residents. She also told the Chieftain that taxes and fees have hampered residents' ability to afford living in Colorado. "Every single time we go to do absolutely anything — whether it's to renew our vehicle registration, whether it's going to the grocery store and paying bag fees — we are taxed and (made to pay fees) to death," Charles said. As a supporter of TABOR — a 1992 amendment to the state constitution limiting the amount of tax revenue Colorado government can retain and spend — Charles strongly believes Colorado does not have a revenue shortage and that Colorado residents, not state government, know how to best spend their money. In addition to having 24 years of experience in healthcare, Charles is a lifelong Pueblo resident who has raised two sons. "We have an amazing community, and I plan to tout that in every way when I'm in the state Senate. Many of the members of even our own city council, I have known for many years," Charles said. "While we may disagree on certain issues, I believe that we can communicate in a way that is positive and helps continue to move Pueblo forward." Senate District 3 includes all of Pueblo County. Senate Majority Whip Nick Hinrichsen has held the seat since February 2022. As of May 19, Charles and Hinrichsen were the only candidates who'd filed to run for Senate District 3, according to the Colorado Secretary of State's Office. Charles has already raised $11,915 in monetary contributions and has spent over $2,547, according to Transparency in Contribution and Expenditure Reporting (TRACER) information accessed May 19. Hinrichsen has received over $6,752 and spent over $5,551. City Park Bathhouse: Pueblo leaders respond to preservationists' frustrations over rejected bathhouse proposal Pueblo Chieftain reporter James Bartolo can be reached at JBartolo@ Support local news, subscribe to The Pueblo Chieftain at This article originally appeared on The Pueblo Chieftain: Pueblo Republican launches 2026 bid for Colorado Senate seat
Yahoo
15-05-2025
- Business
- Yahoo
Voyager Demonstrates ALPL Receptor-Mediated Blood-Brain Barrier Transport of Novel AAV Capsids in Molecular Therapy Publication
LEXINGTON, Mass., May 15, 2025 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, today announced the first peer-reviewed publication of data demonstrating the ability of alkaline phosphatase (ALPL) to transport a novel AAV capsid across the blood-brain barrier (BBB). The article, titled 'Highly conserved brain vascular receptor ALPL mediates transport of engineered AAV vectors across the blood-brain barrier,' was published in Molecular Therapy and can be accessed here. 'Understanding ALPL and its ability to mediate transport across the blood-brain barrier has been foundational to the evolution of our gene therapy programs, two of which are advancing towards IND filings this year with a partner,' said Mathieu Nonnenmacher, Ph.D., Vice President of Gene Therapy at Voyager. 'Building on our first-generation capsids, such as VCAP-102, which is featured in this paper, we have evolved next-generation capsids with even stronger brain transduction and liver de-targeting, as well as stealth capsids with immune-evading capabilities.' The Molecular Therapy paper outlines the generation of novel, cross-species AAV capsid VCAP-102, which demonstrates 20- to 400-fold increased gene transfer across multiple brain regions relative to AAV9 in both rodents and non-human primates (NHP), and the identification of ALPL as the primary receptor used by VCAP-102 to cross the BBB. In addition, the confirmation that the ALPL capsid family binds and demonstrates transcytosis with human ALPL in a cell barrier in vitro model suggests clinical translatability. As previously announced, Voyager presented next-generation and stealth-capsid data at the American Society of Gene & Cell Therapy's (ASGCT) 28th annual meeting. In multiple NHP studies utilizing a variety of payloads, a single intravenous 3e13 vg/kg dose of Voyager's second-generation CNS capsids transduced up to 98% of dopaminergic neurons in substantia nigra, up to 94% of motor neurons in the spinal cord, up to 66% of neurons in the thalamus, up to 43% of neurons in the motor cortex, and 87-99% of astrocytes broadly across brain regions. 'In addition to speeding the evolution of novel capsid families, we are leveraging our work with ALPL and other receptors to deliver diverse classes of non-viral candidates into the CNS,' said Todd Carter, Ph.D., Chief Scientific Officer of Voyager Therapeutics. 'We believe this multi-modality approach, encompassing both viral and non-viral CNS delivery, is critical to addressing unmet needs in neurological disease.' About the TRACER™ Capsid Discovery PlatformVoyager's TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of novel AAV capsids to enable gene therapy. Voyager has leveraged TRACER to create multiple families of novel capsids that, following intravenous delivery in preclinical studies, harness the extensive vasculature of the central nervous system (CNS) to cross the blood-brain barrier and transduce a broad range of CNS regions and cell types. In cross-species preclinical studies (rodents and multiple non-human primate species), intravenous delivery of TRACER-generated capsids resulted in widespread payload expression across the CNS at relatively low doses, enabling selection of multiple development candidates in Voyager's wholly-owned and partnered gene therapy programs for neurologic diseases. About Voyager TherapeuticsVoyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; and Neurocrine Biosciences, Inc. For more information, visit Voyager Therapeutics® is a registered trademark, and TRACER™ is a trademark, of Voyager Therapeutics, Inc. Forward-Looking StatementsThis press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as 'will,' 'anticipated,' 'expect,' 'believe,' 'anticipate,' 'potential,' 'may,' or 'continue,' and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding Voyager's ability to advance its AAV-based gene therapy programs and non-viral CNS delivery programs, including the potential for Voyager's novel TRACER capsids to achieve desired results in humans, including neuronal and glial transduction across multiple brain regions, and ALPL-mediated transcytosis similar to the results demonstrated in rodents and NHPs; potential clinical translatability in humans; increased patient eligibility to receive AAV gene therapies; and expectations for advancement of gene therapy product candidates under the collaboration programs, including anticipated submission of IND filings and initiation of clinical trials in two partnered programs are forward looking. All forward-looking statements are based on estimates and assumptions by Voyager's management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain and subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the continued development of Voyager's technology platforms, including Voyager's TRACER platform and its non-viral discovery platform; Voyager's scientific approach and program development progress, and the restricted supply and increased costs of critical research components; the development by third parties of capsid identification platforms that may be competitive to Voyager's TRACER capsid discovery platform; Voyager's ability to create and protect intellectual property rights associated with the TRACER capsid discovery platform, the capsids identified by the platform, and development candidates for Voyager's pipeline programs; the timing, initiation, conduct and outcomes of Voyager's preclinical and clinical studies; the availability of data from clinical trials; the expectations and decisions of regulatory authorities; the availability or commercial potential of product candidates under collaborations; the success of Voyager's product candidates; the willingness and ability of Voyager's collaboration partners to meet obligations under collaboration agreements with Voyager; the possibility or the timing of Voyager's receipt of program reimbursement, development or commercialization milestones, option exercise, and other payments under Voyager's existing licensing or collaboration agreements; the ability of Voyager to negotiate and complete licensing or collaboration agreements with other parties on terms acceptable to Voyager and the third parties; the success of programs controlled by third-party collaboration partners in which Voyager retains a financial interest; the ability to attract and retain talented directors, employees, and contractors; and the sufficiency of Voyager's cash resources to fund its operations and pursue its corporate objectives. These statements are also subject to a number of material risks and uncertainties that are described in Voyager's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. ContactsTrista Morrison, tmorrison@ Investors: Sarah McCabe, smccabe@ Brooke Shenkin, brooke@ 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 Upturn
28-04-2025
- Health
- Business Upturn
Voyager Next-Generation CNS Capsids Featured at ASGCT 28th Annual Meeting
By GlobeNewswire Published on April 29, 2025, 01:30 IST – Oral presentation on tau silencing gene therapy VY1706, which has previously shown up to 73% knockdown of tau mRNA in NHPs in the CNS following a single IV dose of 1.3e13 vg/kg – – Featured data also include anti-amyloid gene therapy for Alzheimer's disease, as well as multiple presentations on Voyager's continued enhancements to its highly BBB penetrant novel capsids – LEXINGTON, Mass., April 28, 2025 (GLOBE NEWSWIRE) — Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, today announced eight oral and poster presentations at the upcoming American Society of Gene & Cell Therapy's (ASGCT) 28th annual meeting taking place in New Orleans, May 13-17, 2025. 'Voyager continues to raise the bar with our TRACER capsids. In multiple studies utilizing a variety of payloads, our capsids have transduced 43%-98% of neurons and 87-99% of astrocytes broadly across brain regions following a single intravenous 3e13 vg/kg dose in non-human primates,' said Todd Carter, Ph.D., Chief Scientific Officer of Voyager Therapeutics. 'Our new data at ASGCT build on our strong foundation in developing gene therapies for Alzheimer's disease: our tau silencing gene therapy VY1706, which will be featured in an oral presentation, has previously shown up to 73% knockdown of tau mRNA in NHPs following a single IV dose of 1.3e13 vg/kg, and we will also present data from our anti-amyloid gene therapy program. Our data also feature continued enhancements such as immune evasion to potentially increase the percentage of the population who could benefit from these treatments. With two INDs expected this year and another next year, we look forward to assessing and hopefully validating the performance of our capsids in humans.' Anti-Tau and Anti-Amyloid Gene Therapies for Alzheimer's Disease Oral Presentation: Intravenous delivery of VY1706, a CNS penetrant AAV gene therapy for Alzheimer's disease, provides broad tau lowering in NHP. Rajeev Sivasankaran, Ph.D., VP, Head of Neuroscience. Thursday, May 15, 2025, 8:50 a.m. – 9:15 a.m. CT Cross-species BBB-penetrant IV-delivered AAV gene therapy provides broad and robust CNS tau lowering in tauopathy mouse models and non-human primate (#559). Hechen Bao, Ph.D., Scientist II, Neuroscience. Tuesday, May 13, 2025, 6:00 p.m. – 7:30 p.m. CT One-time delivery of a vectorized anti-amyloid antibody for increased and sustained CNS expression and target engagement (#541). Cassandra Retzlaff, Ph.D., Scientist II, Neuroscience. Tuesday, May 13, 2025, 6:00 p.m. – 7:30 p.m. CT Reducing Immunogenicity and Enhancing Developability and Manufacturing of Capsids Oral Presentation: Discovery of AAV9-derived CNS capsids evading pre-existing neutralizing antibodies. Damien Maura, Ph.D., Senior Scientist II, Capsid Discovery. Wednesday, May 14, 2025, 2:15 p.m. – 2:30 p.m. CT Machine-learning for AAV9 mutant-capsid screening for both production and ALPL-mediated transduction efficiency (#1911). Daniel Cox, Ph.D., Senior Scientist, Data Sciences. Thursday, May 15, 2025, 5:30 p.m. – 7:00 p.m. CT Assessment of two HEK293 cell line cloning strategies to improve AAV yield (#1954). Hung-Lun Hsu, Ph.D., Scientist II, Process Development. Thursday, May 15, 2025, 5:30 p.m. – 7:00 p.m. CT Enabling large-scale implementation of anion exchange chromatography for full capsid enrichment of a novel adeno-associated viral vector (#1455). Tom Elich, B.S., Senior Engineer II, Process Development. Wednesday, May 14, 2025, 5:30 p.m. – 7:00 p.m. CT An alternative to detergent lysis: Promoting rAAV release to media by optimizing osmolality, pH and harvest timing (#1477). Christian Gagnon, M.S., Senior Associate Engineer, Process Development. Wednesday, May 14, 2025, 5:30 p.m. – 7:00 p.m. CT Presentations will be available on Voyager's website at: About the TRACER™ Capsid Discovery Platform Voyager's TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of novel AAV capsids to enable gene therapy. Voyager has leveraged TRACER to create multiple families of novel capsids that, following intravenous delivery in preclinical studies, harness the extensive vasculature of the central nervous system (CNS) to cross the blood-brain barrier and transduce a broad range of CNS regions and cell types. In cross-species preclinical studies (rodents and multiple non-human primate species), intravenous delivery of TRACER-generated capsids resulted in widespread payload expression across the CNS at relatively low doses, enabling selection of multiple development candidates in Voyager's wholly-owned and partnered gene therapy programs for neurologic diseases. About Voyager Therapeutics Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; and Neurocrine Biosciences, Inc. For more information, visit Voyager Therapeutics ® is a registered trademark, and TRACER™ is a trademark, of Voyager Therapeutics, Inc. Forward-Looking Statements This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as 'will,' 'expect,' 'potential,' 'believe,' 'could,' or 'continue,' and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding Voyager's ability to advance its AAV-based gene therapy programs such as tau mRNA knock-down program with VY1706 and its anti-amyloid gene therapy program, including expectations for Voyager's achievement of preclinical and clinical development milestones for its potential development candidates for treating Alzheimer's disease; the potential for Voyager's novel TRACER capsids to achieve desired results in humans, including achievement of a higher therapeutic index and increased patient eligibility to receive AAV gene therapies by immune evasion; Voyager's expectation to advance gene therapy product candidates through IND filings under its internal and partnered programs; and the ability of Voyager's improvements in manufacture to enable increased yields and large-scale development of AAV gene therapies are forward looking. All forward-looking statements are based on estimates and assumptions by Voyager's management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain and subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the expectations and decisions of regulatory authorities; the timing, initiation, conduct and outcomes of Voyager's preclinical and clinical studies; the availability of data from clinical trials; the availability or commercial potential of product candidates under collaborations; the success of Voyager's product candidates; the willingness and ability of Voyager's collaboration partners to meet obligations under collaboration agreements with Voyager; the continued development of Voyager's technology platforms, including Voyager's TRACER platform and its non-viral platform technology; Voyager's scientific approach and program development progress, and the restricted supply and increased costs of critical research components; the development by third parties of capsid identification platforms that may be competitive to Voyager's TRACER capsid discovery platform; Voyager's ability to create and protect intellectual property rights associated with the TRACER capsid discovery platform, the capsids identified by the platform, and development candidates for Voyager's pipeline programs; the possibility or the timing of Voyager's receipt of program reimbursement, development or commercialization milestones, option exercise, and other payments under Voyager's existing licensing or collaboration agreements; the ability of Voyager to negotiate and complete licensing or collaboration agreements with other parties on terms acceptable to Voyager and the third parties; the success of programs controlled by third-party collaboration partners in which Voyager retains a financial interest; the ability to attract and retain talented directors, employees, and contractors; and the sufficiency of Voyager's cash resources to fund its operations and pursue its corporate objectives. These statements are also subject to a number of material risks and uncertainties that are described in Voyager's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. Contacts Trista Morrison, [email protected] Investors: Sarah McCabe, [email protected] Media: Brooke Shenkin, [email protected] Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same. GlobeNewswire provides press release distribution services globally, with substantial operations in North America and Europe.
