Company will deliver three oral abstracts and host an Industry Symposium

Cambridge, MA – April 27, 2021 – Dyno Therapeutics, a biotechnology company applying artificial intelligence (AI) to gene therapy, today announced that it will deliver three oral presentations and an Industry Symposium at the 24^th annual meeting of the American Society of Gene and Cell Therapy (ASGCT) being held as a virtual meeting on May 11–14, 2021.

Details for the oral presentations are as follows:

Title: Efficient Design of Optimized AAV Capsids using Multi-property Machine Learning Models Trained across Cells, Organs and Species (Abstract #23)
Presenter:  Eric Kelsic, Ph.D., CEO and Co-founder, Dyno Therapeutics
Session: Development of AAV Capsid Variants
Time: 5:45 – 6:00pm EST on Tuesday, May 11

Title: Risk-Adjusted Selection for Validation of Sequences in AAV Design Using Composite Sampling (Abstract #24)
Presenter: Lauren Wheelock, Ph.D., Scientist I, Machine Learning, Dyno Therapeutics
Session: Development of AAV Capsid Variants
Time: 6:00 – 6:15pm EST on Tuesday, May 11

Title: AAV Capsid Property Estimation Is Improved by Combining Single-Molecule
ID Tags and Hierarchical Bayesian Modeling of Experimental Processes (Abstract #190)
Presenter: Kathy Lin, Ph.D., Sr. Scientist, Computational Biology, Dyno Therapeutics
Session: Novel AAV Biology and Platform Technologies
Time: 6:15 – 6:30pm EST on Thursday, May 13

Dyno will also host an Industry Symposium entitled “Building Dyno Therapeutics” from 5:15 – 6:45pm EST on Thursday, May 13. During this interactive session, employees throughout the company will describe the breakthroughs that enabled Dyno’s approach to AAV capsid engineering, how the company is inventing new methods for machine learning and quantitative high-throughput in vivo experimentation, and the story of developing a world-class team and culture alongside groundbreaking science.

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 with optimized properties including improved safety, manufacturability and applicability for treating a broader range of diseases.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput 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.

Scientific leaders bring deep expertise in machine learning and AAV gene therapy

Cambridge, MA – April 26, 2021 – Dyno Therapeutics, a biotech company applying artificial intelligence (AI) to gene therapy, today announced the expansion of the company’s Scientific Advisory Board (SAB) with the addition of Debora Marks, Ph.D., of Harvard Medical School and the Broad Institute, and Nicole Paulk, Ph.D., of the University of California San Francisco. 

 

Dr. Marks and Dr. Paulk will play an instrumental role in providing scientific insight and expert guidance, complementing the company’s diverse and interdisciplinary team that is focused on applying machine learning to Adeno-Associated Virus (AAV) capsid engineering for improved gene therapy delivery. They join existing members of Dyno’s SAB, George Church, Ph.D., Robert Winthrop Professor of Genetics at Harvard Medical School and a Core Faculty member at Harvard’s Wyss Institute for Biologically Inspired Engineering, and Tomas Bjorklund, Ph.D., Associate Professor of Neuroscience at Lund University. 

 

“We are honored and delighted to welcome Professors Debora Marks and Nicole Paulk to Dyno’s SAB. These preeminent scientists bring world-leading expertise in the areas of machine learning and AAV gene therapy which will accelerate Dyno’s design of best-in-class capsids that solve unmet medical needs,” said Eric Kelsic, Ph.D., co-founder and CEO of Dyno Therapeutics. “Debora offers unique insights based on her pioneering work applying machine learning to protein structure and design. Nicole’s deep scientific expertise in AAV gene therapy research, development and manufacturing will be invaluable for guiding our platform to generate products that help millions of patients.”

Debora S. Marks, Ph.D. is an Associate Professor of Systems Biology at Harvard Medical School, and an Associate Member of the Broad Institute of Harvard and MIT. Dr. Marks has led a distinguished career in academia and industry, and is an expert in the application of machine learning algorithms for protein design and engineering. She earned her B.Sc. in Mathematics from the University of Manchester, U.K., and her Ph.D. in Mathematical Biology from Humboldt University, Germany. 

 

Dr. Debora Marks commented, “I’m very excited to start working with Dyno’s world-class team and I’m particularly impressed by Dyno’s use of machine learning as a systematic approach to solving gene therapy’s biggest challenge: efficient, safe and precise gene delivery to target tissues.”

 

Nicole Paulk, Ph.D. is an Assistant Professor of AAV Gene Therapy at the University of California San Francisco Department of Biochemistry & Biophysics. She is a pioneer in next-generation AAV gene delivery and gene editing platforms. She has developed therapies for gene repair and gene transfer for numerous rare diseases, and has applied high-throughput comparative proteomic and epigenomic approaches to address challenges in fundamental AAV biology. She earned her B.S. in Medical Microbiology from Central Washington University, and her Ph.D. in Viral Gene Therapy from Oregon Health & Science University.

 

Dr. Nicole Paulk said, “I am eager to contribute my expertise to Dyno’s efforts to design novel AAV vectors that overcome the limitations of current vectors, ultimately to expand the opportunities for gene therapy to help more patients.”

 

About Dyno Therapeutics

 

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput 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.

Cambridge, MA – February 11, 2020 – Dyno Therapeutics, a biotech company applying artificial intelligence (AI) to gene therapy, today announced a publication in Nature Biotechnology that demonstrates the use of artificial intelligence to generate an unprecedented diversity of adeno-associated virus (AAV) capsids towards identifying functional variants capable of evading the immune system, a factor that is critical to enabling all patients to benefit from gene therapies. The research was conducted in collaboration with Google Research, Harvard’s Wyss Institute for Biologically Inspired Engineering and the Harvard Medical School laboratory of George M. Church, Ph.D., a Dyno scientific co-founder. The publication is entitled “Deep diversification of an AAV capsid protein by machine learning.”

 

It is estimated that up to 50-70% of the human population have pre-existing immunity to natural forms of the AAV vectors currently being using to deliver gene therapies. This immunity renders a large portion of patients ineligible to receive gene therapies which rely upon these capsids as the vector for delivery. Overcoming the challenge of pre-existing immunity to AAV vectors is therefore a major goal for the gene therapy field.

 

“The approach described in the Nature Biotechnology paper opens a radically new frontier in capsid design. Our study clearly demonstrates the potential of machine learning to guide the design of diverse and functional sequence variants, far beyond what exists in nature,” said Eric Kelsic, Ph.D., Dyno’s CEO and co-founder.  “We continue to expand and apply the power of artificial intelligence to design vectors that can not only overcome the problem of pre-existing immunity but also address the need for more effective and selective tissue targeting. At Dyno, we are making rapid progress to design novel AAV vectors that overcome the limitations of current vectors, improving treatments for more patients and expanding the number of diseases treatable with gene therapies.”

 

The Nature Biotechnology paper describes the rapid production of a large library of distinct AAV capsid variants designed by machine learning models. Nearly 60% of the variants produced were determined to be viable, a significant increase over the typical yield of <1% using random mutagenesis, a standard method of generating diversity.

 

“The more we change the AAV vector from how it looks naturally, the more likely we are to overcome the problem of pre-existing immunity,” added Sam Sinai, Ph.D., Dyno co-founder and Machine Learning Team Lead. “Key to solving this problem, however, is also ensuring that capsid variants remain viable for packaging the DNA payload. With conventional methods, this diversification is time- and resource-intensive, and results in a very low yield of viable capsids. In contrast, our approach allows us to rapidly unlock the full potential diversity of AAV capsids to develop improved gene therapies for a much larger number of patients.

 

This research builds upon previous work published in Science in which a complete landscape of single mutations around the AAV2 capsid was generated followed by evaluation of the functional properties important for in vivo delivery. In parallel with these works, Dyno has established collaborations with leading gene therapy companies Novartis, Sarepta Therapeutics, Roche and Spark Therapeutics to develop next-generation AAV gene therapy vectors with a goal of expanding the utility of gene therapies for ophthalmic, muscle, central nervous system (CNS) and liver diseases.

 

About CapsidMap™ for Designing Optimized AAV Gene Therapies

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dyno’s proprietary CapsidMap™ platform overcomes the limitations of today’s gene therapies on the market and in development. Today’s treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery efficiency, immunity, payload size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology to engineer capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dyno’s massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the design of novel capsids optimized for gene therapy.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput 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.

Collaboration combines Dyno’s AI-powered AAV vector capabilities with Roche and Spark Therapeutics’ leading-edge gene therapy capabilities
Dyno is eligible to receive milestone payments that may exceed $1.8 billion

Cambridge, MA – October 14, 2020 – Dyno Therapeutics, a biotech company applying artificial intelligence (AI) to gene therapy, today announced a collaboration and license agreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) to apply Dyno’s CapsidMap™ platform for the development of next-generation adeno-associated virus (AAV) vectors for gene therapies for central nervous system (CNS) diseases and liver-directed therapies for the portfolio of both Roche and Spark Therapeutics, a member of the Roche Group.

Dyno’s proprietary CapsidMap™ platform represents a transformative approach to the identification of novel AAV capsids – the cell-targeting protein shell of viral vectors – optimizing tissue targeting and immune-evading properties, in addition to improving packaging capacity and manufacturability.

Under the terms of the agreement, Dyno will be responsible for the design of novel AAV capsids with improved functional properties for gene therapy, while Roche and Spark Therapeutics will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the novel capsids. Dyno will receive an undisclosed upfront payment and if successful, is 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 payments to Dyno may exceed $1.8 billion.

“This new partnership represents Dyno’s largest collaboration to date, and we are excited to work with Roche and Spark Therapeutics to expand the frontier of gene therapies for the central nervous system and liver. The Dyno, Roche and Spark teams share a bold vision for gene therapy and believe that enhancing vectors that deliver these therapies is key to developing new treatments for patients in need,” stated Dyno’s CEO and co-founder Eric Kelsic, Ph.D. “Partnering is a fundamental element of Dyno’s business strategy, and the continuing interest by leading gene therapy developers is accelerating our growth plans and positive impact on patients.”

“We strongly believe in the potential of gene therapy and are excited to bring together experts from Roche, Spark and Dyno to develop next-generation gene therapies. Dyno’s innovative AI-powered approach to designing optimized AAV vectors will further complement and build on our progress in gene therapy. We look forward to leveraging Dyno’s technology to develop new, innovative treatments for patients across CNS and liver-directed therapies,” said James Sabry, Head of Roche Pharma Partnering.

About CapsidMap™ for Designing AAV Gene Therapies

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dyno’s proprietary CapsidMap™ platform overcomes the limitations of today’s gene therapies on the market and in development. Today’s treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery, immunity, packaging size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology for the design of novel capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dyno’s massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the discovery and optimization of synthetic AAV capsids.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput 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.

Dyno Therapeutics has been named Startup of the Year by Xconomy, an award that recognizes the most innovative new company with a focus on those advancing creative solutions to the challenges of biology, to break onto the scene this year.

“We are incredibly honored to be selected as Xconomy’s Startup of the Year and recognized for our pioneering approach to gene therapy,” said Eric Kelsic, PhD, chief executive officer of Dyno Therapeutics. “Key to Dyno’s rapid ascent has been our systematic focus on solving gene therapy’s biggest challenge, the efficient, safe and precise gene delivery to target tissues. Just as essential to our success is our diverse team of the best problem solvers who are driving cutting-edge science toward improving patient health.”

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput 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 recently came out of stealth and earlier this year, signed deals valued in excess of $2 billion with gene therapy leaders Novartis and Sarepta.

Since 2017, the mission of Xconomy’s annual awards programs has been to provide the industry a moment to pause, recognize, and honor the very best the life sciences has to offer – the people, companies, and organizations that challenge the status quo and never accept what works as good enough.

 

 

 

Dyno sponsors ASGCT virtual career fair 

Cambridge, Mass., May 12, 2020 – Dyno Therapeutics, a biotechnology company applying artificial intelligence (AI) to gene therapy, today announced two presentations and a poster at the American Society of Gene and Cell Therapy (ASGCT) conference being held as a virtual meeting on May 12-15, 2020.

Details for the presentations and poster are as follows:

Presentations

Title: Artificial intelligence powered design of synthetic AAV capsids without pre-existing immunity for the universal treatment of all patients

Presenter:  Eric Kelsic, Ph.D., Chief Executive Officer and Co-founder, Dyno Therapeutics

Education Session Title: Synthetic Biology Meets Immunology: DNA and RNA Tools

Date and Time: Tuesday May 12, 2020; 2:40 – 3:15 p.m.

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Title:  Massively Parallel Deep Diversification of AAV Capsid Proteins by Machine Learning

Presenter:  Sam Sinai, Ph.D., Lead Machine Learning Scientist and Co-founder, Dyno Therapeutics

Date and Time: Wednesday May 13, 2020; 4:30 – 4:45 p.m.

Oral Abstract Session Title: Vector and Cell Engineering, Production or Manufacturing III

Abstract Number: 541

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Poster

Title: Accurately Quantifying Transduction within Barcoded AAV Capsid Libraries via Tracking of Single-Molecule ID Tags

Presenters: Kathy Lin, Ph.D., Computational Biology Scientist, and Jeff Gerold, Ph.D., Head of Data Science, Dyno Therapeutics

Date and Time: Thursday, May 14, 2020; 5:30 – 6:30 p.m.

Poster Session Title: AAV Vectors – Virology and Vectorology

Abstract Number: 1006

This poster will be available under the Publications section of the Dyno Therapeutics website at the time of the presentation at www.dynotx.com.

 

ASGCT Virtual Career Fair

Also at ASGCT 2020, Dyno is the main sponsor of the virtual career fair. Dyno is actively recruiting as the company continues to grow its current team of 20 employees and expects to double in the next year.  Current listings can be found at the ASGCT Career Fair website and at www.dynotx.com.

About CapsidMap™ for Designing AAV Gene Therapies 

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dyno’s proprietary CapsidMap™ platform overcomes the limitations of today’s gene therapies on the market and in development. Today’s treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery, immunity, packaging size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology for the design of novel capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dyno’s massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the discovery and optimization of synthetic AAV capsids.

Dyno’s technology platform builds on certain intellectual property developed in the lab of George Church, Ph.D., who is Robert Winthrop Professor of Genetics at Harvard Medical School (HMS), a Core Faculty member at Harvard’s Wyss Institute for Biologically Inspired Engineering, and a co-founder of Dyno. Several of the technical breakthroughs that enabled Dyno’s approach to optimize synthetic AAV capsid engineering were described in a November 2019 publication in the journal Science, based on work conducted by Dyno founders and members of the Church Lab at HMS and the Wyss Institute. Dyno has an exclusive option to enter into a license agreement with Harvard University for this technology.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experimentation to gene therapy. The company’s proprietary CapsidMap™ platform is designed to rapidly discover and systematically optimize superior Adeno-Associated Virus (AAV) capsid vectors with delivery properties that significantly improve upon current approaches to gene therapy and expand 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.

Collaboration focuses on gene therapies for serious eye diseases and leverages Dyno’s CapsidMap™ artificial intelligence platform to design AAV vectors

Financial terms include upfront consideration, research funding, license fees, clinical and sales milestone payments, and royalties to develop AAV vectors to address an extensive range of ophthalmic gene therapies 

Cambridge, Mass., May, 11, 2020 – Dyno Therapeutics, a biotechnology company applying artificial intelligence (AI) to gene therapy, today announced a collaboration with Novartis to develop improved Adeno-Associated Virus (AAV) vectors for research, development, and commercialization of gene therapies for ocular diseases. The partnership will allow the parties to utilize Dyno’s CapsidMap™ artificial intelligence platform along with Novartis expertise in gene therapy development and global commercialization to deliver innovative gene therapies to patients with serious diseases of the eye.

“We are delighted to be collaborating with Novartis,” stated Eric D. Kelsic, Ph.D., CEO and Co‑founder of Dyno Therapeutics. “Many eye diseases are ideally suited to being treated with gene therapies, and more opportunities can be opened with new and improved AAV vectors. With their extensive ophthalmologic expertise, Novartis is an ideal partner to leverage Dyno’s platform to design AI-powered vectors to expand the impact of gene therapies for ocular diseases. This collaboration is a major step forward in our plan to realize the potential of Dyno’s CapsidMap platform for gene therapies to improve patient health.”

Under the terms of the agreement, Dyno will be responsible for using AI technology and its suite of machine learning and experimental tools for the design and discovery of novel AAV capsids, the cell-targeting protein shell of viral vectors, with improved functional properties for gene therapy. Novartis will be responsible for conducting preclinical, clinical, and commercialization activities for the gene therapy product candidates created with the novel AAV capsids. Dyno will receive upfront consideration plus committed research funding and license fees. Additionally, Dyno will be eligible to receive clinical, regulatory and sales milestone payments. Dyno will also receive royalties on worldwide net sales of any commercial products developed through the partnership.

About CapsidMap™ for Designing AAV Gene Therapies 

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dyno’s proprietary CapsidMap™ platform overcomes the limitations of today’s gene therapies on the market and in development. Today’s treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery, immunity, packaging size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology for the design of novel capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dyno’s massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the discovery and optimization of synthetic AAV capsids.

Dyno’s technology platform builds on certain intellectual property developed in the lab of George Church, Ph.D., who is Robert Winthrop Professor of Genetics at Harvard Medical School (HMS), a Core Faculty member at Harvard’s Wyss Institute for Biologically Inspired Engineering, and a co-founder of Dyno. Several of the technical breakthroughs that enabled Dyno’s approach to optimize synthetic AAV capsid engineering were described in a November 2019 publication in the journal Science, based on work conducted by Dyno founders and members of the Church Lab at HMS and the Wyss Institute. Dyno has an exclusive option to enter into a license agreement with Harvard University for this technology.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experimentation to gene therapy. The company’s proprietary CapsidMap™ platform is designed to rapidly discover and systematically optimize superior Adeno-Associated Virus (AAV) capsid vectors with delivery properties that significantly improve upon current approaches to gene therapy and expand 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.

— Agreement leverages Sarepta’s leadership in gene therapy for neuromuscular and cardiovascular diseases and Dyno’s CapsidMap artificial intelligence platform to design AAV vectors — 

CAMBRIDGE, Mass., May 11, 2020 – Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and Dyno Therapeutics, Inc., a biotech company applying artificial intelligence (AI) to gene therapy, today announced an agreement to develop next-generation Adeno-Associated Virus (AAV) vectors for muscle diseases, using Dyno’s CapsidMap™ platform.

AI and machine learning technologies have the potential to deliver enhanced vectors for gene therapies. Dyno’s proprietary CapsidMap platform opens up new ways to identify novel capsids – the cell-targeting protein shell of viral vectors – that could offer improved muscle targeting and immune-evading properties, in addition to advantages in packaging and manufacturing.

“Sarepta’s world-leading gene therapy engine is founded on three pillars: developing a broad portfolio of programs to treat rare diseases; our first-in-class manufacturing expertise; and investment in advancing and further improving the science of gene therapy to help patients in need of more options. To that end, our agreement with Dyno provides us with another valuable tool to develop next-generation capsids for gene therapies to treat rare diseases,” said Doug Ingram, Sarepta’s President and Chief Executive Officer. “By leveraging Dyno’s AI platform and Sarepta’s deep expertise in gene therapy development, our goal is to advance next-generation treatments with improved muscle-targeting capabilities.”

Under the terms of the agreement, Dyno will be responsible for the design and discovery of novel AAV capsids with improved functional properties for gene therapy and Sarepta will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the novel capsids. If successful, Dyno could receive over $40 million in upfront, option and license payments during the research phase of the collaboration. Additionally, if Sarepta develops and commercializes multiple candidates for multiple muscle diseases, Dyno will be eligible for additional significant future milestone payments. Dyno will also receive royalties on worldwide net sales of any commercial products developed through the collaboration.

“This agreement is a major step forward in our plan to realize the potential of Dyno’s AI platform for gene therapies to improve patient health. We are excited to work with Sarepta to create gene therapies with improved properties to address a range of muscle-related diseases,” stated Dyno’s CEO and co-founder Eric D. Kelsic, Ph.D. “The success of the gene therapies developed through this collaboration with Sarepta will rely on AI-powered vectors that allow gene therapies to be safely and precisely targeted to the muscle tissue.”

About CapsidMap™ for Designing AAV Gene Therapies 

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dyno’s proprietary CapsidMap™ platform overcomes the limitations of today’s gene therapies on the market and in development. Today’s treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery, immunity, packaging size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology for the design of novel capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next-generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dyno’s massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the discovery and optimization of synthetic AAV capsids.

Dyno’s technology platform builds on certain intellectual property developed in the lab of George Church, Ph.D., who is Robert Winthrop Professor of Genetics at Harvard Medical School (HMS), a Core Faculty member at Harvard’s Wyss Institute for Biologically Inspired Engineering, and a co-founder of Dyno. Several of the technical breakthroughs that enabled Dyno’s approach to optimize synthetic AAV capsid engineering were described in a November 2019 publication in the journal Science, based on work conducted by Dyno founders and members of the Church Lab at HMS and the Wyss Institute. Dyno has an exclusive option to enter into a license agreement with Harvard University for this technology. 

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experimentation to gene therapy. The company’s proprietary CapsidMap™ platform is designed to rapidly discover and systematically optimize superior Adeno-Associated Virus (AAV) capsid vectors with delivery properties that significantly improve upon current approaches to gene therapy and expand 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 Sarepta Therapeutics

At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook. 

Sarepta Therapeutics Forward-looking Statements

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the potential of artificial intelligence and machine learning technologies to deliver enhanced vectors for gene therapies; the potential of the CapsidMap™ platform to offer improved muscle targeting and immune-evading properties, in addition to advantages in packaging and manufacturing; the agreement between Sarepta and Dyno Therapeutics providing a valuable tool to develop next-generation capsids for gene therapies to treat rare disease; the parties’ goal to advance next-generation treatments with improved muscle-targeting capabilities; the parties’ responsibilities under the agreement and potential payments to Dyno Therapeutics; and the potential of AI-powered vectors to allow gene therapies to be safely and precisely targeted to the muscle tissue.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and Dyno Therapeutics may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; the results of research may not be consistent with past results or may not be positive or may otherwise fail to meet regulatory approval requirements for the safety and efficacy of product candidates; possible limitations of company financial and other resources; manufacturing limitations that may not be anticipated or resolved for in a timely manner; regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sarepta’s product candidates; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K for the year ended December 31, 2019 and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review Sarepta’s 2019 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q filed with the SEC as well as other SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Dyno’s CapsidMap™ platform designed to overcome the limitations of current vectors, improving gene therapies and expanding the range of treatable diseases   

Dyno could potentially receive over $2 billion in payments under existing collaboration agreements

Initial R&D programs and collaborations focus on discovery of gene therapy AAV vectors for ophthalmic, muscle, central nervous system, and liver diseases

Cambridge, Mass., May 11, 2020 – Dyno Therapeutics, Inc., a biotechnology company applying artificial intelligence (AI) to gene therapy, today announced the company’s launch from stealth mode with its proprietary platform, CapsidMap™. The platform enables the design of novel Adeno-Associated Virus (AAV) vectors that significantly improve upon current approaches to gene therapy and expand the scope of accessible diseases. Through its R&D and collaborations with biopharmaceutical companies, Dyno has active programs focused on novel gene therapy vectors for ophthalmic, muscle, central nervous system (CNS), and liver diseases. The company could potentially receive well over $2 billion in upfront payments, research support, option fees, as well as pre-clinical, clinical, regulatory and sales milestones under its collaboration agreements.

“At Dyno, we see a vast opportunity to expand the treatment landscape for gene therapies. The success of gene therapy relies on the ability of vectors to safely and precisely deliver a gene to the intended target cells and tissues,” said Eric Kelsic, Ph.D., CEO and Co-founder of Dyno Therapeutics. “Our approach addresses the major limitations of naturally occurring AAV vectors and creates optimized, disease-specific vectors for gene therapies with great curative potential. Our portfolio of R&D programs and newly-announced collaborations with leading gene therapy developers reflect the applicability of our AI-powered approach to improve treatments for patients and expand the number of treatable diseases with gene therapies.”

Dyno’s technology platform builds on certain intellectual property developed in the lab of George Church, Ph.D., who is Robert Winthrop Professor of Genetics at Harvard Medical School (HMS) and a Core Faculty member at Harvard’s Wyss Institute for Biologically Inspired Engineering. Several of the technical breakthroughs that enable Dyno’s approach to AAV capsid engineering were described in a November 2019 publication in the journal Science, based on work conducted by the company’s founders and members of the Church Lab at HMS and the Wyss Institute. Dyno has an exclusive option to enter into a license agreement with Harvard University for this technology. Church is a co-founder of Dyno and Chairman of the company’s Scientific Advisory Board.

The CapsidMap platform applies Dyno’s proprietary artificial intelligence technology to discover and design novel AAV capsids, the cell-targeting protein shell of viral vectors. CapsidMap systematically generates and then evaluates millions of new AAV variants at an unprecedented scale, dramatically accelerating the identification of improved AAV vectors. CapsidMap uses advanced machine learning search algorithms, combined with high-throughput experiments generating massive quantities of in vivo data, to accelerate the creation of superior synthetic AAV capsids.

The company launched in late 2018 with a $9 million financing co-led by Polaris Partners and CRV. Alan Crane, a co-founder of Dyno and Entrepreneur Partner at Polaris Partners, and Dylan Morris, General Partner at CRV, have joined Dyno’s board of directors, with Alan Crane serving as Dyno’s Executive Chairman. Dyno does not anticipate the need for additional fundraising at this time based on the significant financial resources made available from collaborations.

Alan Crane stated, “We invested in Dyno because we believe that the company’s platform represents a paradigm shift in the development of gene therapies. Gene therapies have the potential to cure diseases that are not adequately treated by existing small molecule and antibody therapeutics. As an industry, we often know which genes we want to administer for treatment, but we can’t effectively get them to the target tissues and cell types. AAV capsids with improved tropism, immunogenicity, packaging size, and manufacturing features will expand treatments to more patients.”

Dyno is building a multi-disciplinary team of experts in business, gene therapy, and machine learning. In addition to Eric Kelsic, George Church, and Alan Crane, the company’s founders include Sam Sinai, Ph.D., Lead Machine Learning Scientist, Adrian Veres, Ph.D., Scientific Advisor, and Tomas Bjorklund, Ph.D., a scientific advisor of Dyno who is Associate Professor at Lund University and a leader in AAV capsid engineering.

About CapsidMap™ for Designing AAV Gene Therapies 

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dyno’s proprietary CapsidMap™ platform overcomes the limitations of today’s gene therapies on the market and in development. Today’s treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery, immunity, packaging size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology for the design of novel capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dyno’s massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the discovery and optimization of synthetic AAV capsids.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experimentation to gene therapy. The company’s proprietary CapsidMap™ platform is designed to rapidly discover and systematically optimize superior Adeno-Associated Virus (AAV) capsid vectors with delivery properties that significantly improve upon current approaches to gene therapy and expand 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.

Dyno Therapeutics founders and collaborators demonstrate machine-guided AAV capsid engineering with the potential to transform gene therapy

CAMBRIDGE, Mass.–December 2, 2019–Dyno Therapeutics, a biotechnology company pioneering use of artificial intelligence in gene therapy, today announced a publication in the journal Science that demonstrates the power of a comprehensive machine-guided approach to engineer improved capsids for gene therapy delivery. The research was conducted by Dyno co-founders Eric D. Kelsic, Ph.D. and Sam Sinai, Ph.D., together with colleague Pierce Ogden, Ph.D., at Harvard’s Wyss Institute for Biologically Inspired Engineering and the Harvard Medical School laboratory of George M. Church, Ph.D., a Dyno scientific co-founder. The publication, entitled “Comprehensive AAV capsid fitness landscape reveals a viral gene and enables machine-guided design,” is available here.¹

Improved AAV vector capsid for gene therapy engineered with a new machine-guided approach shows, in red, improvements in efficiency of viral production based on the average effect of insertions at all possible amino acid positions, with white showing neutral and blue showing deleterious positions. (Left: capsid viewed from outside, Right: cut-out to reveal inner positions). Credit: Eric Kelsic, Dyno Therapeutics. (Photo: Business Wire)

 

AAV capsids are presently the most commonly used vector for gene therapy because of their established ability to deliver genetic material to patient organs with a proven safety profile. However, there are only a few naturally occurring AAV capsids, and they are deficient in essential properties for optimal gene therapy, such as targeted delivery, evasion of the immune system, higher levels of viral production, and greater transduction efficiency. Starting at Harvard in 2015, the authors set out to overcome the limitations of current capsids by developing new machine-guided technologies to rapidly and systematically engineer a suite of new, improved capsids for widespread therapeutic use.

In the research published in Science, the authors demonstrate the advance of their unique machine-guided approach to AAV engineering. Previous approaches have been limited by the difficulty of altering a complex capsid protein without breaking its function and by the general lack of knowledge regarding how AAV capsids interact with the body. Historically, rather than addressing this challenge directly, the most popular approaches to capsid engineering have taken a roundabout solution: generating libraries of new capsids by making random changes to the protein. However, since most random changes to the capsid actually result in decreased function, such random libraries contain few viable capsids, much less improved ones. Recognizing the limitation of conventionally generated capsid libraries, the authors implemented a machine-guided approach that gathered a vast amount of data using new high-throughput measurement technologies to teach them how to build better libraries and, ultimately, lead to synthetic capsids with optimized delivery properties.

Focusing on the AAV2 capsid, the authors generated a complete landscape of all single codon substitutions, insertions and deletions, then measured the functional properties important for in vivo delivery. They then used a machine-guided approach, leveraging these data to efficiently generate diverse libraries of AAV capsids with multiple changes that targeted the mouse liver and that outperformed AAVs generated by conventional random mutagenesis approaches. In the process, the authors’ systematic efforts unexpectedly revealed the existence of a previously-unrecognized protein encoded within the sequence of all the most popular AAV capsids, which they termed membrane-associated accessory protein (MAAP). The authors believe that the protein plays a role in the natural life cycle of AAV.

“This is just the beginning of machine-guided engineering of AAV capsids to transform gene therapy,” underscores co-author Sam Sinai, Ph.D., Lead Machine Learning Scientist and co-founder of Dyno Therapeutics. “The success of the simple linear models used in this study has led us to pursue more data and higher capacity machine learning models, where the potential for improvement in capsid designs feels boundless.”

“The results in the Science publication demonstrate, for the first time, the power of linking a comprehensive set of advanced techniques – large scale DNA synthesis, pooled in vitro and in vivo screens, next-generation sequencing readouts, and iterative machine-guided capsid design – to generate optimized synthetic AAV capsids,” explains co-first and co-corresponding author Eric D. Kelsic, Ph.D., CEO and co-founder of Dyno Therapeutics. “At Dyno, our team is committed to advancing these technologies to identify capsids that meet the urgent needs of patients who can benefit from gene therapies.”

About Dyno Therapeutics
Dyno Therapeutics is a pioneer in applying artificial intelligence to gene therapy. The company’s powerful and proprietary genetic engineering platform is designed to rapidly and systematically develop improved AAV capsids that redefine the gene therapy landscape. Dyno was founded by experienced biotech entrepreneurs and leading scientists in the fields of synthetic biology, gene therapy, and machine learning. The company is located in Cambridge, Massachusetts. For additional information, please visit the company website at www.dynotx.com

¹ Ogden P.J., Kelsic E.D., Sinai S., Church G.M. Comprehensive AAV capsid fitness landscape reveals a viral gene and enables machine-guided design. Science. 2019 Nov 29;366(6469):1139-43. doi: 10.1126/science.aaw2900

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