Dyno receives $18 million upfront and is eligible for milestone and royalty payments potentially exceeding $1.6 billion

TOKYO and CAMBRIDGE, Mass., Dec. 2, 2021 – Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., “Astellas”) and Dyno Therapeutics, Inc. (President and CEO: Eric Kelsic, Ph.D., “Dyno”) today announced an option and license agreement was signed on November 23 to develop next-generation adeno-associated virus (AAV) 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,” said Naoki Okamura, Chief Strategy Officer and Chief Financial Officer, Chief Business Officer at Astellas. “We are dedicated to delivering novel approaches and utilizing new technologies that can deliver transformational value for patients.” 

“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. For more information, please visit our website at https://www.astellas.com/en. 

 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 (AI) and quantitative in vivo experiments to gene therapy. The company’s proprietary CapsidMap™ platform rapidly discovers and systematically optimizes Adeno-Associated Virus (AAV) 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. Visit www.dynotx.com for additional information.

About CapsidMap™ for Designing Optimized AAV Gene Therapies
Dyno’s CapsidMap™ platform overcomes the limitations of gene therapies on the market and under development today by optimizing capsids, the cell-targeting protein shells of Adeno-Associated Virus (AAV) vectors. Current gene therapies primarily use a small number of naturally occurring capsids that are limited by delivery efficiency, pre-existing immunity, payload size, and manufacturing challenges. CapsidMap works in two stages, first by measuring capsid properties in high-throughput using next-generation DNA library synthesis and DNA sequencing. With these vast quantities of in vivo data, CapsidMap then generates improved capsid sequences by applying advanced search algorithms that leverage machine learning. Dyno’s comprehensive map of capsid sequence space and AI‑powered tools thereby accelerate the design of AAV gene therapies towards optimizing manufacturability and applicability for treating a broader range of diseases.

Astellas Cautionary Notes
In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management’s current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas’ intellectual property rights by third parties. 

Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice. 

 

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Contacts for inquiries or additional information: 

Astellas Pharma Inc.
Corporate Advocacy & Relations
TEL: +81-3-3244-3201 

Dyno Therapeutics
Nisha Deo 
Head of Communications 
TEL: +1-857-242-0593  

 

Dyno empowers a diverse team of the best problem-solvers to drive cutting-edge science toward improving patient health. Our mission is to build the ideal capsid and solve the challenges of vivo gene delivery. We call ourselves AAViators and are engaged in Collective Innovation as we aim to maximize our impact on patients through the development of groundbreaking technologies.

Our journey so far has been one of rigor and joy. We are so honored that the 2021 NEVY Awards Academy has nominated Dyno for the “Emerging Company of the Year” Award. Recently we’ve also been recognized with disruptive potential in the biopharma industry.

Our data driven approach is a key enabler and differentiator. With our CapsidMapTM Platform, Dyno is building the world’s most informative and diverse library of capsid measurements, and an algorithm that learns to efficiently explore sequence space in search of the best capsids for  therapeutic delivery. (Read more about how our platform is delivering the promise of gene therapy here)

Our other core differentiator is our focus on productive teamwork. Everytime a new AAViator accepts an offer to join the team we reiterate our foundation of trust, healthy conflict, commitment, accountability and attention to results. Onboarding at Dyno starts by reading Patrick Lencioni’s The Five Dysfunctions of a Team: A Leadership Fable, which enables discussions about these aspects of productive teamwork across the whole company. For example, this common language helps us explore the importance of being vulnerable and open about our weaknesses and mistakes, in order to build the trust required for teams to flourish. In recognizing the importance of trust we also realize how difficult it is to achieve.

We must therefore be open to give and receive feedback (both positive and negative) so that we improve trust at an individual, team, and organizational level. Similar best-practices apply for each concept all the way through attention to results (for more explanation, I highly recommend reading Lencioni’s book).

Scientific breakthroughs can emerge from the most uncanny places. We are structured in a way that makes it easier for anyone (regardless of hierarchy or discipline) at any level to propose a change to our platform, any of which could eventually lead to a scientific breakthrough.

Today we are over 60 full time AAViators. We continue to climb to meet the needs of every potential partner, and in the process hope to justify the accolades we have received. As we reach 150 strong we look forward to welcoming those with fresh ideas interested in catalyzing our Collective Innovation.

If you are looking for a hard working team-focused company where you can connect your creative ideas to real-world impact contact me directly or take a look at our Careers page!