Dyno Therapeutics to Present Breakthrough Data on AAV Gene Delivery to the CNS, Eye, and Muscle at the 28th Annual Meeting of the American Society of Gene & Cell Therapy
WATERTOWN, Mass.--(BUSINESS WIRE)-- Dyno Therapeutics, Inc., a genetic technologies company applying artificial intelligence (AI) to enable in vivo gene delivery, today announced that it will present a broad set of data related to the design of novel capsids targeting the eye, brain, and neuromuscular system using frontier AI during a Scientific Symposium and three oral presentations at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) taking place May 13-17, 2025 at the Ernest N. Morial Convention Center in New Orleans, Louisiana.
The data will highlight Dyno's leadership in solving the biggest challenge limiting the viability and accessibility of gene therapy: safely and efficiently delivering therapeutic genetic payloads to target cells in vivo. To solve this challenge, Dyno applies AI and high-throughput in vivo data collection to design AAV capsids that are optimized for efficiency, precision targeting, and manufacturability. By extending these high-performance design capabilities and delivery technologies to therapeutic developers, Dyno aims to advance the effectiveness of next-generation gene therapies, making them applicable for a wide range of human diseases.
In the Scientific Symposium, Dyno Cofounder and CEO, Eric Kelsic, Ph.D., will detail three new AAV capsids with improved performance for targeting the central nervous system (CNS), eye, and neuromuscular system, as well as updates on the frontier AI models behind their discovery. Dr. Kelsic will also share how Dyno intends to rapidly progress these next-generation gene therapies into the clinic, add new treatments in areas of high unmet need, and improve access to transformative medicines by expanding partnerships with gene therapy developers.
Scientific Symposium
Title: Dyno Therapeutics: Leveling up genetic medicine with frontier AI and AAV vectors for CNS, eye, and muscle
Presenter: Eric Kelsic, Ph.D., Cofounder & CEO
Date and Time: Wednesday, May 14th from 12:15 – 1:15 PM CT
Location: Room 391-392
In addition to the Symposium, three research abstracts selected for oral presentation will present data on Dyno's progress enabling gene delivery for CNS, eye and neuromuscular indications:
Oral Presentations
Title: Widespread CNS Delivery With Best-In-Class Liver Detargeting Following Intravenous Injection of a Novel AAV
Presenter: Mugdha Deshpande, Ph.D.
Session: AAV Gene Transfer (A): Crossing the Blood-Brain Barrier
Date and Time: Wednesday, May 14, 2025, 1:30 PM - 1:45 PM CT
Location: New Orleans Theater C
Abstract: 2220
Title: Selective Improvement in Retinal Bipolar Cell Targeting with Intravitreal Injection of a Novel AAV Capsid in Mouse and NHP
Presenter: Amanda Miles, Ph.D.
Session: AAV Gene Transfer (B): Ocular, Neurological & Immune Cell Systems
Date and Time: Friday, May 16, 2025, 2:45 PM - 3:00 PM CT
Location: New Orleans Theater A
Abstract: 2175
Title: A Novel Neuromuscular AAV Capsid Combines Efficient Systemic Muscle and CNS Delivery with Liver Detargeting in NHP
Presenter: Megan Cramer, Ph.D.
Session: AAV Gene Transfer (C): Antibody Evasion, Cardiac & Neuromuscular Targets
Date and Time: Saturday, May 17, 2025, 11:45 AM - 12:00 PM CT
Location: New Orleans Theater A
Abstract: 2208
More details about the Scientific Symposium and oral presentations are available on the ASGCT meeting website.
About Dyno Therapeutics
Dyno Therapeutics is building high-performance genetic technologies that transform patient health by overcoming the in vivo gene delivery challenge for therapeutic developers. Dyno's platform integrates frontier AI with high-throughput experimentation to accelerate the optimization of AAV capsids that significantly outperform existing gene delivery vectors. Just as exponential breakthroughs in computer chip technology powered the AI revolution, Dyno's versatile platform leverages foundational advances from in vivo multiplexing technologies and high-performance computing to address therapeutic grand challenges. Dyno is partnering with leading gene therapy developers, including Astellas, Roche, and Sarepta, and with technology companies including NVIDIA. Visit www.dynotx.com for additional information.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
2 hours ago
- Yahoo
SOLVE FSHD and Modalis Announce Strategic Collaboration to Develop an Innovative CRISPR-Based Epigenome Editing Treatment for Facioscapulohumeral Muscular Dystrophy
VANCOUVER, British Columbia & TOKYO & WALTHAM, Mass., June 08, 2025--(BUSINESS WIRE)--SOLVE FSHD, a venture philanthropy organization dedicated to accelerating treatments for facioscapulohumeral muscular dystrophy (FSHD), and Modalis Therapeutics Corporation (TSE 4883; "Modalis"), a CRISPR-based epigenome editing therapeutics company focused on rare genetic diseases, today announced a strategic collaboration to develop an innovative therapy for FSHD, a debilitating muscular disorder affecting approximately 1 million individuals worldwide. The novel therapy leverages Modalis's proprietary CRISPR-GNDM® (Guide Nucleotide-Directed Modulation) technology, which can dynamically modulate gene expression without introducing double-strand DNA breaks. SOLVE FSHD will provide strategic funding to support the development of Modalis's MDL-103 program. MDL-103 is an innovative therapeutic solution that continuously suppresses the expression of the DUX4 gene, the toxic disease-causing gene for FSHD, which becomes abnormally activated due to epigenetic changes in the D4Z4 repeat region on chromosome 4. MDL-103 is designed to have durable activity over long periods of time under the control of a strong, muscle-specific promoter, and is delivered to the muscles of patients using a muscle-tropic AAV delivery system. Modalis's CRISPR-GNDM® technology has the potential to transform the treatment of FSHD by epigenetically silencing the expression of DUX4. "SOLVE FSHD is pleased to partner with Modalis and to add them to our diverse portfolio of collaborators that are advancing potential therapies for FSHD," stated Eva Chin, Executive Director of SOLVE FSHD. "SOLVE FSHD identified Modalis as a company committed to finding a cure for this debilitating condition. We were impressed by their unique approach to targeting the epigenetic cause of FSHD, using a platform technology that has shown promise in other neuromuscular diseases. We believe that the support from SOLVE FSHD will allow Modalis to accelerate the advancement of MDL-103 into clinical trials." "We are delighted to be working in partnership with SOLVE FSHD and greatly appreciate the invaluable support for the development of MDL-103," said Haru Morita, CEO of Modalis. "This strategic collaboration is a strong validation of Modalis's CRISPR-GNDM® technology and our MDL-103 program. As a pioneer in this technology, we have demonstrated promising long-term drug efficacy in mouse models, shown durable target engagement and safety in non-human primates, and exhibited excellent biodistribution in neuromuscular disorders. We believe that MDL-103, which incorporates CRISPR-GNDM® technology with a muscle tropic AAV delivery system, has significant potential as a breakthrough treatment for FSHD." About SOLVE FSHD SOLVE FSHD is a venture philanthropic organization established to catalyze innovation and accelerate key research in finding a cure for FSHD. Established by renowned Canadian entrepreneur and philanthropist, Chip Wilson, the Wilson family has committed $100 million to kick-start funding into projects that support the organizations' mission to solve FSHD by 2027. The goal of SOLVE FSHD is to find a solution that can slow down or stop muscle degeneration, increase muscle regeneration and strength, and improve the quality of life for those living with FSHD, visit About Modalis Therapeutics Corporation Modalis was founded in 2016 and conducts research and development activities in Massachusetts, USA. Modalis is a pioneering leader in the field of epigenetic medicine. Modalis develops therapeutics for patients suffering from serious genetic disorders such as neuromuscular diseases, CNS diseases, and cardiomyopathies. Modalis's proprietary CRISPR-GNDM® technology is capable of specifically up or down modulating the expression of disease-relevant genes without introducing double-strand DNA breaks. For more information, visit View source version on Contacts SOLVE FSHDAlexandra Grant, House of Wilsonalexandrag@ Modalis Therapeutics CorporationCorporate Planning Departmentmedia@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
2 hours ago
- Business Wire
SOLVE FSHD and Modalis Announce Strategic Collaboration to Develop an Innovative CRISPR-Based Epigenome Editing Treatment for Facioscapulohumeral Muscular Dystrophy
VANCOUVER, British Columbia & TOKYO & WALTHAM, Mass.--(BUSINESS WIRE)-- SOLVE FSHD, a venture philanthropy organization dedicated to accelerating treatments for facioscapulohumeral muscular dystrophy (FSHD), and Modalis Therapeutics Corporation (TSE 4883; 'Modalis'), a CRISPR-based epigenome editing therapeutics company focused on rare genetic diseases, today announced a strategic collaboration to develop an innovative therapy for FSHD, a debilitating muscular disorder affecting approximately 1 million individuals worldwide. The novel therapy leverages Modalis's proprietary CRISPR-GNDM ® (Guide Nucleotide-Directed Modulation) technology, which can dynamically modulate gene expression without introducing double-strand DNA breaks. SOLVE FSHD will provide strategic funding to support the development of Modalis's MDL-103 program. MDL-103 is an innovative therapeutic solution that continuously suppresses the expression of the DUX4 gene, the toxic disease-causing gene for FSHD, which becomes abnormally activated due to epigenetic changes in the D4Z4 repeat region on chromosome 4. MDL-103 is designed to have durable activity over long periods of time under the control of a strong, muscle-specific promoter, and is delivered to the muscles of patients using a muscle-tropic AAV delivery system. Modalis's CRISPR-GNDM ® technology has the potential to transform the treatment of FSHD by epigenetically silencing the expression of DUX4. 'SOLVE FSHD is pleased to partner with Modalis and to add them to our diverse portfolio of collaborators that are advancing potential therapies for FSHD,' stated Eva Chin, Executive Director of SOLVE FSHD. 'SOLVE FSHD identified Modalis as a company committed to finding a cure for this debilitating condition. We were impressed by their unique approach to targeting the epigenetic cause of FSHD, using a platform technology that has shown promise in other neuromuscular diseases. We believe that the support from SOLVE FSHD will allow Modalis to accelerate the advancement of MDL-103 into clinical trials.' 'We are delighted to be working in partnership with SOLVE FSHD and greatly appreciate the invaluable support for the development of MDL-103,' said Haru Morita, CEO of Modalis. 'This strategic collaboration is a strong validation of Modalis's CRISPR-GNDM ® technology and our MDL-103 program. As a pioneer in this technology, we have demonstrated promising long-term drug efficacy in mouse models, shown durable target engagement and safety in non-human primates, and exhibited excellent biodistribution in neuromuscular disorders. We believe that MDL-103, which incorporates CRISPR-GNDM ® technology with a muscle tropic AAV delivery system, has significant potential as a breakthrough treatment for FSHD.' About SOLVE FSHD SOLVE FSHD is a venture philanthropic organization established to catalyze innovation and accelerate key research in finding a cure for FSHD. Established by renowned Canadian entrepreneur and philanthropist, Chip Wilson, the Wilson family has committed $100 million to kick-start funding into projects that support the organizations' mission to solve FSHD by 2027. The goal of SOLVE FSHD is to find a solution that can slow down or stop muscle degeneration, increase muscle regeneration and strength, and improve the quality of life for those living with FSHD, visit Modalis was founded in 2016 and conducts research and development activities in Massachusetts, USA. Modalis is a pioneering leader in the field of epigenetic medicine. Modalis develops therapeutics for patients suffering from serious genetic disorders such as neuromuscular diseases, CNS diseases, and cardiomyopathies. Modalis's proprietary CRISPR-GNDM ® technology is capable of specifically up or down modulating the expression of disease-relevant genes without introducing double-strand DNA breaks. For more information, visit
Yahoo
7 hours ago
- Yahoo
Jim Cramer Says He 'Personally Would Not Own Altria (MO)'
We recently published a list of . In this article, we are going to take a look at where Altria Group, Inc. (NYSE:MO) stands against other stocks that Jim Cramer discusses. Answering a caller's query about Altria Group, Inc. (NYSE:MO), Cramer commented: 'Alright, but here's, okay, so I'm going to give you two answers to this because I've been trying to change my mind about some things…. I personally would not own Altria. Why? Because I don't like what they do. Is it a superior stock better than most? The answer is, as I was writing How to Make Money in Any Market, the answer is yes. So you've got two answers. It's up to you. I couldn't own it for my trust. I couldn't live with myself.' A close-up of an assembly line with a blend of tobacco products. Altria (NYSE:MO) produces and markets a range of tobacco and nicotine products, including cigarettes, cigars, smokeless tobacco, nicotine pouches, and e-vapor devices, sold under brands like Marlboro, Black & Mild, Copenhagen, and NJOY ACE. Andvari Associates stated the following regarding Altria Group, Inc. (NYSE:MO) in its Q1 2025 investor letter: 'Last year, Andvari made its first investments in tobacco companies with the purchase of Philip Morris International and Altria Group, Inc. (NYSE:MO). At the time of our purchase, Philip Morris and Altria had underperformed the S&P 500 over the prior 5- and 10-year periods. Both traded at low valuations and with high dividend yields. But thanks to following the industry o and on for 10+ years, and thanks to many discussions with long-time shareholders of the companies, Andvari felt the time was right to make the plunge. The timing could not have been much better for us as both companies have so far contributed positively to Andvari's recent overall performance. Overall, MO ranks 8th on our list of stocks that Jim Cramer discusses. While we acknowledge the potential of MO as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an extremely cheap AI stock that is also a major beneficiary of Trump tariffs and onshoring, see our free report on the best short-term AI stock. READ NEXT: 20 Best AI Stocks To Buy Now and 30 Best Stocks to Buy Now According to Billionaires. Disclosure: None. This article is originally published at Insider Monkey. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
