Cell and Gene Therapy

Astellas and Dyno Therapeutics Announce Research Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Skeletal and Cardiac Muscle

Astellas and Dyno Therapeutics Announce Research Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Skeletal and Cardiac Muscle
Astellas Pharma Inc. and Dyno Therapeutics, Inc. announced an option and license agreement was signed on November 23 to develop next-generation adeno-associated virus vectors for gene therapy directed to skeletal and cardiac muscle using Dyno's CapsidMap™ platform.

Dyno's CapsidMap platform represents a transformative approach applying in vivo experimental data and machine learning to create novel AAV capsids – the cell-targeting protein shells of viral vectors – designed to optimize tissue targeting and immune-evading properties, in addition to improving packaging capacity and manufacturability. Unlike traditional approaches, CapsidMap is uniquely well-suited to simultaneously optimize capsids for delivery across multiple organs, with the goal of enabling more effective whole-body treatment for many diseases.

With the establishment of the Astellas Gene Therapies Center of Excellence following the 2020 acquisition of Audentes Therapeutics Inc., Astellas is a leader in genetic medicines, working alongside its world-renowned partners to build a portfolio of potentially life-changing gene therapies. This research collaboration combines Dyno's AI-powered AAV vector engineering capabilities with Astellas Gene Therapies global leadership in AAV-based pipeline assets.

"Through our efforts in gene therapy and the Astellas Gene Therapies Center of Excellence, Astellas strives to identify, develop and deliver transformative gene-based therapies for patients with genetic diseases who currently have few or no effective treatment options. Our principal focus is on developing adeno-associated virus delivered therapies for the treatment of well-defined serious diseases. We are dedicated to delivering novel approaches and utilizing new technologies that can deliver transformational value for patients."

Naoki Okamura, Chief Strategy Officer and Chief Financial Officer, Chief Business Officer at Astellas

"We are so happy to be partnering with Astellas, a world leader in developing gene therapies. Dyno and Astellas each bring unique strengths to this collaboration, together enabling more rapid creation of new therapies for patients with great unmet need," said Dyno's CEO and co-founder Eric D. Kelsic, Ph.D. "This partnership demonstrates the flexibility of Dyno's platform to precisely design the delivery properties of gene therapy vectors towards multi-organ and disease-specific profiles, applying the scientific insights we are rapidly learning across all our partnered and internal vector engineering programs using CapsidMap."

Under the terms of the agreement, Dyno will design novel AAV capsids with improved functional properties for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities, including manufacturing, of gene therapy product candidates using the novel capsids. Dyno will receive an $18 million upfront payment and be eligible to receive additional payments during the research phase of the collaboration as well as clinical and sales milestone payments and royalties for any resulting products. The aggregate potential value of future milestone and royalty payments to Dyno exceeds $235 million per product and over $1.6 billion in total value.

About Astellas
Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients.

About Astellas Gene Therapies
Astellas integrated its wholly owned subsidiary, Audentes Therapeutics, Inc. as of April 1, 2021 and established "Astellas Gene Therapies" within the organization as an Astellas Center of Excellence to develop genetic medicines with the potential to deliver transformative value for patients. Based on an innovative scientific approach and industry leading internal manufacturing capability and expertise, we are currently exploring three gene therapy modalities: gene replacement, exon skipping gene therapy, and vectorized RNA knockdown and hope to also advance additional Astellas gene therapy programs toward clinical investigation. We are based in San Francisco, with manufacturing and laboratory facilities in South San Francisco and Sanford, North Carolina.

About Dyno Therapeutics
Dyno Therapeutics is a pioneer in applying artificial intelligence and quantitative in vivo experiments to gene therapy. The company's proprietary CapsidMap™ platform rapidly discovers and systematically optimizes Adeno-Associated Virus capsid vectors that significantly outperform current approaches for in vivo gene delivery, thereby expanding the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts. 

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PR Newswire | October 10, 2023

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Vidofludimus calcium shows a separation in serum NfL over placebo in this interim analysis, an effect also seen across different subgroups," stated Prof. Jens Kuhle, M.D., Ph.D., Senior Physician, Head of Neuroimmunology Unit and Multiple Sclerosis Centre, University Hospital Basel, Switzerland. "Particularly remarkable, the non-active progressive MS population, which represents the highest unmet medical need in MS, also showed differences in NfL levels over this relatively short observation period in favor of vidofludimus calcium. Meanwhile, although longer follow-up is needed, the GFAP data set also shows a potential promising early signal. Overall, the interim biomarker data further support vidofludimus calcium's possible activity beyond an anti-inflammatory effect, which may be related to its potent Nurr1 activation." "The clear separation observed in serum NfL for vidofludimus calcium over placebo in the PMS patient population represents another major step forward for, what potentially could be, a first-in-class Nurr1 activator for MS," commented Daniel Vitt, Ph.D., Chief Executive Officer and President of Immunic. "Although no head-to-head data is available, it is encouraging to see that vidofludimus calcium's improvement in NfL over placebo appears at least as good as, and is in fact numerically higher than that observed with historical studies of other therapeutic approaches for PMS. We believe that, if the top-line CALLIPER data, expected in April of 2025, continue to show a neuroprotective effect, we may be able to position vidofludimus calcium as the first oral treatment option for non-active SPMS. Additionally, the drug's first-in-class ability to activate Nurr1, a known neuroprotective target, should also significantly benefit our ongoing phase 3 ENSURE program in relapsing MS where prevention of disability progression independent of relapse activity (PIRA), serves as a key outcome." "We are very pleased to see such strong improvements in serum NfL for vidofludimus calcium over placebo in the overall PMS population of this interim analysis, as well as across all PMS subtypes and in patients with and without disease activity, and with and without MRI activity. We even saw evidence in non-active SPMS, a population where the medical need for new therapies is high as there is currently no relevant treatment available in the US," added Andreas Muehler, M.D., Chief Medical Officer of Immunic. "Finally, we were also excited to see an encouraging early signal with GFAP. 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