Latest news with #EPI-321
Yahoo
05-05-2025
- Health
- Yahoo
Epicrispr Biotechnologies Announces Five Presentations Highlighting Advances in Epigenetic Modulation and AAV Manufacturing at ASGCT 2025 Annual Meeting
- Preclinical data showcase scalable AAV manufacturing, compact gene activators, and expanded Cas effector tools for precise epigenetic modulation SAN FRANCISCO, May 05, 2025--(BUSINESS WIRE)--Epicrispr Biotechnologies, a biotechnology company focused on developing curative therapies, today announced five presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, being held May 15-17, 2025, in New Orleans, Louisiana. The presentations will highlight the company's progress across key areas, including its lead epigenetic therapeutic candidate EPI-321, novel Cas effectors for epigenome engineering, and innovations in compact modular epigenetic activators. The accepted abstracts spotlight Epicrispr's approach to modulating gene expression without altering the DNA sequence, enabling safer, tunable, and potentially reversible therapies. Two posters focus on the development and manufacturing of EPI-321, an rAAVrh74-delivered epigenetic therapy that will be initiating a first-in-human study for FSHD, a neuromuscular disease. Oral Presentation: Non-Human Primate (NHP) Safety Study of High-Dose EPI-321: A Novel AAV-Delivered Epigenetic Editing Gene Therapy for the Treatment of FSHD Presenter: Sid Boregowda, DVM, MS, Ph.D, DABT Session Title: Viral Vectors in Large Animal Models Date/Time: Wednesday, May 14, 2025, 3:45 – 5:30 PM CT Abstract Presentation Time: 4:45 – 5:00 PM CT Location: NOLA Theater B Poster Presentation Details: Directed Evolution and Characterization of Cas Effectors in Mammalian Cells for Expanded Epigenome Editing Space Presenter: Courtney Klappenbach, Senior Research Associate Poster Number: 617 Date/Time: Tuesday, May 13, 2025, 6:00 – 7:30 PM CT Location: Poster Hall I2, New Orleans Ernest N. Morial Convention Center Small Scale AAV Bioreactor Optimization Demonstrates Iterative Titer Gains of rAAVrh74 Serotype EPI-321, a CRISPR-mediated Epigenetic Therapy Presenter: James Kim, Associate Scientist Poster Number: 967 Date/Time: Tuesday, May 13, 2025, 6:00 – 7:30 PM CT Location: Poster Hall I2, New Orleans Ernest N. Morial Convention Center Compact DNA Demethylase-Activator Combination Modulators for CRISPR-mediated Epigenetic Gene Activation Presenter: Dan Hart, Ph.D, Head of Technology Development Poster Number: 1110 Date/Time: Wednesday, May 14, 2025, 5:30 – 7:00 PM CT Location: Poster Hall I2, New Orleans Ernest N. Morial Convention Center EPI-321 Development: Strategies to Establish a Scalable and Robust rAAVrh74 Upstream Manufacturing Process from 0.5 L to 1000 L Scale Presenter: Surabhi Godbole, Scientist I Poster Number: 1939 Date/Time: Thursday, May 15, 2025, 5:30 – 7:00 PM CT Location: Poster Hall I2, New Orleans Ernest N. Morial Convention Center About Epicrispr Biotechnologies Epicrispr Biotechnologies is a biotechnology company pioneering gene-modulating therapies, leading with treatments for neuromuscular diseases. The company's proprietary Gene Expression Modulation System (GEMS) enables precise, durable control of gene expression, unlocking first-in-class treatments for previously untreatable conditions. Epicrispr's lead program, EPI-321 is in clinical trials for FSHD, and the company is advancing additional gene-modulating therapies. Epicrispr also has a research collaboration with Kite Pharma to develop next-generation CAR T-cell therapies. Learn more at or follow us on LinkedIn. View source version on Contacts Investor Contact Benson Media Contact Kimberly HaKKH Sign in to access your portfolio
Yahoo
23-04-2025
- Business
- Yahoo
Epicrispr Biotechnologies Partners with Springbok Analytics to Integrate AI Muscle Analysis in First-in-Human FSHD Study
- Advanced MRI analytics to enhance early signal detection and inform development of first-in-class epigenetic therapy SOUTH SAN FRANCISCO, Calif., April 23, 2025--(BUSINESS WIRE)--Epicrispr Biotechnologies, a biotechnology company focused on developing curative therapies, today announced a partnership with Springbok Analytics to incorporate advanced, AI-driven muscle MRI imaging and analysis into its first-in-human clinical trial of EPI-321, a potential first-in-class treatment for facioscapulohumeral muscular dystrophy (FSHD). The exploratory first-in-human global trial, recently cleared to begin in the United States and New Zealand, with additional regulatory approvals pending in other countries, is designed to evaluate the safety, tolerability, and biological activity of a single intravenous dose of EPI-321 in adults with FSHD. Through this collaboration, Springbok's advanced MRI-based muscle analytics will be integrated into the study to detect early, objective insights of muscle-level changes and generate high-resolution data. "High-quality, objective data is essential in early clinical development, especially when advancing a first-in-class therapeutic," said Amber Salzman, Ph.D., CEO, Epicrispr Biotechnologies. "Springbok's automated muscle analysis gives us a detailed view of how EPI-321 may be impacting muscle tissue, strengthening our ability to assess early signals and shape future development." Springbok's proprietary technology uses proprietary AI to segment and quantify dozens of individual muscles from MRI scans, analyzing key biomarkers such as muscle volume, fat infiltration, and inflammation. These high-resolution analytics allow for rapid interpretation of complex imaging data to deliver actionable insights within hours and supporting data-driven clinical decisions from the earliest stages of development. While MRI is an exploratory biomarker in this first-in-human trial, and not a primary study endpoint, the ability to detect subtle, muscle-level changes may be critical in shaping the design of future studies. In conditions like FSHD, where conventional functional endpoints may take longer to reflect therapeutic effect, quantitative muscle imaging offers an accelerated path to understanding biological response. "Quantitative imaging has the potential to dramatically capture more information in early-stage trials in neuromuscular disease," said Scott Magargee, CEO and Co-Founder, Springbok Analytics. "We're proud to support Epicrispr as it pioneers a new therapeutic modality for FSHD and advances the role of imaging biomarkers in clinical development." Springbok's Contributions to the EPI-321 Study Include: Streamlined Imaging Protocols: Enabling whole-body MRI to assess muscle volume and fat infiltration in under 45 minutes. High-Resolution Analytics: Automated segmentation and quantification of muscle volume, fat content, and inflammation across dozens of muscle groups. Scalability & Support: A robust quality management system and turnkey onboarding processes to support rapid deployment across global study locations. Epicrispr is advancing EPI-321 with support from a $68 million Series B financing led by Ally Bridge Group and SOLVE FSHD. EPI-321 is the first clinical application of epigenetic gene modulation in neuromuscular disease, and data generated through this study will help define imaging biomarkers for later-phase trials and support regulatory engagement. About Epicrispr Biotechnologies Epicrispr Biotechnologies is a biotechnology company pioneering gene-modulating therapies, leading with treatments for neuromuscular diseases. The company's proprietary Gene Expression Modulation System (GEMS) enables precise, durable control of gene expression, unlocking first-in-class treatments for previously untreatable conditions. Epicrispr's lead program, EPI-321 is in clinical trials for FSHD, and the company is advancing additional gene-modulating therapies. Epicrispr also has a research collaboration with Kite Pharma to develop next-generation CAR T-cell therapies. Learn more at or follow us on LinkedIn. About Springbok Analytics Springbok Analytics is a leading muscle health analytics company dedicated to advancing health and performance outcomes through innovative, AI-driven solutions that deliver a clearer, more comprehensive view of muscle health. Built on more than 15 years of research and scientific validation, Springbok's FDA-cleared technology transforms MRI data into personalized, 3D visualizations of muscle health. These detailed analyses provide precise metrics, including individual muscle volume and composition, fat infiltration, asymmetries, scar tissue, edema, and tendon morphology. By offering a more accurate and complete understanding of musculoskeletal health, Springbok enhances diagnostic accuracy, treatment monitoring, research capabilities, and performance optimization. View source version on Contacts Investor ContactBenson Media ContactKimberly HaKKH Springbok Media Sign in to access your portfolio
Business Wire
23-04-2025
- Health
- Business Wire
Epicrispr Biotechnologies Partners with Springbok Analytics to Integrate AI Muscle Analysis in First-in-Human FSHD Study
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Epicrispr Biotechnologies, a biotechnology company focused on developing curative therapies, today announced a partnership with Springbok Analytics to incorporate advanced, AI-driven muscle MRI imaging and analysis into its first-in-human clinical trial of EPI-321, a potential first-in-class treatment for facioscapulohumeral muscular dystrophy (FSHD). The exploratory first-in-human global trial, recently cleared to begin in the United States and New Zealand, with additional regulatory approvals pending in other countries, is designed to evaluate the safety, tolerability, and biological activity of a single intravenous dose of EPI-321 in adults with FSHD. Through this collaboration, Springbok's advanced MRI-based muscle analytics will be integrated into the study to detect early, objective insights of muscle-level changes and generate high-resolution data. 'High-quality, objective data is essential in early clinical development, especially when advancing a first-in-class therapeutic,' said Amber Salzman, Ph.D., CEO, Epicrispr Biotechnologies. 'Springbok's automated muscle analysis gives us a detailed view of how EPI-321 may be impacting muscle tissue, strengthening our ability to assess early signals and shape future development.' Springbok's proprietary technology uses proprietary AI to segment and quantify dozens of individual muscles from MRI scans, analyzing key biomarkers such as muscle volume, fat infiltration, and inflammation. These high-resolution analytics allow for rapid interpretation of complex imaging data to deliver actionable insights within hours and supporting data-driven clinical decisions from the earliest stages of development. While MRI is an exploratory biomarker in this first-in-human trial, and not a primary study endpoint, the ability to detect subtle, muscle-level changes may be critical in shaping the design of future studies. In conditions like FSHD, where conventional functional endpoints may take longer to reflect therapeutic effect, quantitative muscle imaging offers an accelerated path to understanding biological response. 'Quantitative imaging has the potential to dramatically capture more information in early-stage trials in neuromuscular disease,' said Scott Magargee, CEO and Co-Founder, Springbok Analytics. 'We're proud to support Epicrispr as it pioneers a new therapeutic modality for FSHD and advances the role of imaging biomarkers in clinical development.' Springbok's Contributions to the EPI-321 Study Include: Streamlined Imaging Protocols: Enabling whole-body MRI to assess muscle volume and fat infiltration in under 45 minutes. High-Resolution Analytics: Automated segmentation and quantification of muscle volume, fat content, and inflammation across dozens of muscle groups. Scalability & Support: A robust quality management system and turnkey onboarding processes to support rapid deployment across global study locations. Epicrispr is advancing EPI-321 with support from a $68 million Series B financing led by Ally Bridge Group and SOLVE FSHD. EPI-321 is the first clinical application of epigenetic gene modulation in neuromuscular disease, and data generated through this study will help define imaging biomarkers for later-phase trials and support regulatory engagement. About Epicrispr Biotechnologies Epicrispr Biotechnologies is a biotechnology company pioneering gene-modulating therapies, leading with treatments for neuromuscular diseases. The company's proprietary Gene Expression Modulation System (GEMS) enables precise, durable control of gene expression, unlocking first-in-class treatments for previously untreatable conditions. Epicrispr's lead program, EPI-321 is in clinical trials for FSHD, and the company is advancing additional gene-modulating therapies. Epicrispr also has a research collaboration with Kite Pharma to develop next-generation CAR T-cell therapies. Learn more at or follow us on LinkedIn. About Springbok Analytics Springbok Analytics is a leading muscle health analytics company dedicated to advancing health and performance outcomes through innovative, AI-driven solutions that deliver a clearer, more comprehensive view of muscle health. Built on more than 15 years of research and scientific validation, Springbok's FDA-cleared technology transforms MRI data into personalized, 3D visualizations of muscle health. These detailed analyses provide precise metrics, including individual muscle volume and composition, fat infiltration, asymmetries, scar tissue, edema, and tendon morphology. By offering a more accurate and complete understanding of musculoskeletal health, Springbok enhances diagnostic accuracy, treatment monitoring, research capabilities, and performance optimization.
Yahoo
26-03-2025
- Health
- Yahoo
Epicrispr banks $68M to test epigenetic editing on rare muscle disease
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. Epicrispr Biotechnologies has raised $68 million in pursuit of a first-of-its-kind genetic medicine for a rare neuromuscular disorder called facioscapulohumeral muscular dystrophy. EPI-321, the startup's lead program, uses CRISPR tools to stop errant expression of a gene implicated in the muscle-wasting condition. Epicrispr will start a Phase 1 trial this year in New Zealand, according to the company's Wednesday statement. The San Francisco Bay Area biotech's Series B round was led by Ally Bridge Group and involved Solve FSHD, an advocacy group formed by Lululemon Athletica founder Chip Wilson. FSHD is a rare neuromuscular disorder estimated to affect about 870,000 people worldwide. The disease is characterized by progressive muscle weakness that begins in the face, back and upper arms and can leave people in wheelchairs or with debilitating pain and fatigue. Though there are no available medications for FSHD, drugmakers in recent years have zeroed in on a gene called DUX4. In FSHD, a genetic error causes DUX4 to be overexpressed, eventually resulting in muscle degeneration and atrophy. Biotech companies have been working on various ways, from small molecule drugs to gene therapies, to stop that from happening. One high-profile effort, an oral drug developed by Fulcrum Therapeutics and Sanofi, failed in Phase 3 testing last year. But other companies, including Avidity Biosciences, Novartis, Arrowhead Pharmaceuticals and Dyne Therapeutics have drugs in development as well. There are currently more than a dozen active DUX4-targeting drug programs, according to the nonprofit FSHD Society. Epicrispr says its approach is unique among that group. The company is using CRISPR tools to turn genes on or off instead of altering DNA directly. In FSHD, it's harnessing CRISPR to bind a precise region of the DUX4 gene and make a chemical modification. The hope is doing so might stop expression of the encoded protein, without the health risks associated with cutting into DNA. According to Amber Salzman, the company's CEO, that strategy has shown potential in preclinical tests to impact muscle function and block the DUX4 protein from 'seeping out.' 'We're going after the absolute root cause' of the disease, she said. 'It's a really, really different approach.' Salzman has for years worked on genetic disorders as a biotech executive and patient advocate. While at GSK many years ago, her son and two nephews were diagnosed with a rare disease called adrenoleukodystrophy. She connected with prominent gene therapy researcher Jim Wilson, met several other experts in the field and started the nonprofit Stop ALD Foundation. One of Salzman's nephews died from ALD in 2004. But her son and second nephew received a treatment that was later approved as Skysona. In the meantime, Salzman worked at multiple biotech startups, including eye gene therapy developer Adverum Biotechnologies. In 2021, a recruiter gauged her interest in leading Epicrispr, which was then known as Epic Bio. By then, the startup had already started working on FSHD — a disease that affected her husband's family, Salzman said. That, and the potential to use epigenetic editing against a wide range of diseases, convinced her to take the job. 'All of a sudden, I found a company that had addressed all the limitations I'd come across in genetic medicine,' she said. Epicrispr raised a $55 million Series A round in 2022. Along with FSHD, it's working on drugs for heterozygous familial hypercholesterolemia, alpha-1 antitrypsin deficiency, a pair of eye diseases and certain undisclosed blood cancers. All of its work is preclinical. The company was co-founded by Stanford researcher Stanley Qi, who worked closely with gene editing pioneer Jennifer Doudna at UC Berkeley.
Yahoo
26-03-2025
- Business
- Yahoo
Epicrispr Biotechnologies Secures $68 Million Series B to Initiate Clinical Trial for First-in-Class Disease-Modifying Epigenetic Neuromuscular Therapy for FSHD
- Financing led by Ally Bridge Group, with participation from SOLVE FSHD, a venture philanthropy organization - Lead program, EPI-321, is the first clinical application of epigenetic modulation in neuromuscular diseases, with a first-in-human trial in New Zealand to commence in 2025 as part of a broader global clinical strategy SOUTH SAN FRANCISCO, Calif., March 26, 2025--(BUSINESS WIRE)--Epicrispr Biotechnologies, a biotechnology company focused on developing curative therapies, today announced it has secured $68 million in the first close of its Series B financing. The proceeds will support the clinical development of EPI-321, a first-in-class, disease-modifying therapy for facioscapulohumeral muscular dystrophy (FSHD), a genetic neuromuscular disease. The Series B financing was led by Ally Bridge Group, with participation from SOLVE FSHD, the venture philanthropy organization founded by Chip Wilson, founder of Lululemon Athletica and FSHD patient, along with other new and existing investors. The financing will support Epicrispr's upcoming clinical trial of EPI-321, as well as continued advancement of the company's broader pipeline. Epicrispr also announced clinical trial application (CTA) approval from New Zealand's Medsafe to initiate a first-in-human trial of EPI-321, the first epigenetic therapy to enter the clinic for a neuromuscular disease. The study is expected to begin in 2025, and will evaluate the safety, tolerability, pharmacodynamics, and biological activity of a single intravenous dose of EPI-321 in adults with FSHD. "FSHD is one of the most common adult muscular dystrophies, with estimates of up to 1 million patients affected worldwide. But patients have no disease-modifying therapy for this progressive disease," said Dr. Richard Roxburgh, Associate Professor of Medicine at the University of Auckland and principal investigator for the EPI-321 clinical trial, which is planned to be conducted in partnership with Pacific Clinical Research Network, a leading clinical research center in New Zealand. "We look forward to advancing this clinical trial which could, with a single treatment, permanently address the disease's underlying cause, and are hopeful that it will pave the way for new standards for therapies in genetic diseases." EPI-321 is an investigational one-time gene-modulating therapy designed to silence aberrant expression of DUX4, a gene that is incorrectly activated in FSHD and leads to progressive muscle degeneration. Delivered systemically via a clinically validated AAV vector, EPI-321 has demonstrated robust suppression of DUX4 expression and protection of muscle tissue in preclinical models. EPI-321 has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations. "We are developing a first-in-class, one-time epigenetic therapy that targets the genetic root cause of FSHD," said Amber Salzman, Ph.D., CEO, Epicrispr Biotechnologies. "The Series B financing and regulatory clearance to begin our first-in-human trial marks a pivotal milestone as we become a clinical-stage company. With a strong investor syndicate and recent FDA designations recognizing EPI-321's potential, our team is laser-focused on advancing EPI-321 into the clinic to provide a much-needed therapy to patients and families in desperate need." "With a robust body of data validating the potential of EPI-321 and the GEMS platform broadly, Epicrispr has shown itself to be a leading epigenetic editing company," said Andrew Lam, Pharm.D., Managing Director, Head of Biotech Private Equity, Ally Bridge Group. "We are proud to lead this investment in Epicrispr's future, and we look forward to partnering with their leadership to support their continued success." "As someone living with FSHD, I know the devastating impact of this disease and the urgent need for treatments that target its root cause," said Chip Wilson, founder and Chairman of SOLVE FSHD. "We commend Epicrispr's commitment in advancing EPI-321 for FSHD and are glad to be part of this financing to support its transition to the clinic." Concurrent with the funding, Epicrispr has expanded its Board of Directors with the addition of Andrew Lam, Pharm.D., of Ally Bridge Group, Eric Crombez, M.D., Chief Medical Officer of Ultragenyx, and Jennifer King, Ph.D., former SVP of Business Development at Intellia Therapeutics and an expert in rare diseases and strategic partnerships. About EPI-321 EPI-321 is an investigational epigenetic therapy that aims to address the underlying molecular mechanisms of FSHD with a one-time dose. It has been granted FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations. Following intravenous administration, EPI-321 is directed to muscle tissue within a single AAV vector, which has been clinically validated for muscle delivery. Preclinical studies on EPI-321 have demonstrated its ability to robustly suppress pathological expression of the DUX4 gene and reduce muscle cell death. A first-in-human clinical trial of EPI-321 is planned for 2025. About Epicrispr Biotechnologies Epicrispr Biotechnologies is a biotechnology company pioneering gene-modulating therapies, leading with treatments for neuromuscular diseases. The company's proprietary Gene Expression Modulation System (GEMS) enables precise, durable control of gene expression, unlocking first-in-class treatments for previously untreatable conditions. Epicrispr's lead program, EPI-321 is in clinical trials for FSHD, and the company is advancing additional gene-modulating therapies. Epicrispr also has a research collaboration with Kite Pharma to develop next-generation CAR T-cell therapies. Learn more at or follow us on LinkedIn. About Ally Bridge Group Ally Bridge Group is a global healthcare investment firm focused on private and public high-impact life science innovation. Founded in 2013 by Frank Yu, the firm has led or co-led over $6 billion in healthcare transactions. The firm's mission is to generate superior risk-adjusted returns for investors guided by the core principle of selective investment in healthcare innovation that addresses unmet medical needs. Ally Bridge Group has offices in New York and Hong Kong. For more information, visit or follow us on LinkedIn. View source version on Contacts Investor ContactBenson Media ContactKimberly HaKKH Sign in to access your portfolio
