Harnessing the convergence of exponential advances in AI, genome sequencing and DNA synthesis technologies, Dyno’s approach to enabling safe and highly efficient in vivo gene delivery accelerates the development of optimized gene therapies

Dyno’s LEAPSM technology achieves super-human design of capsids that perform dramatically better than all prior variants, supporting earlier in vivo validation of high-performance DNA delivery

WATERTOWN, Mass., May 9, 2024 – Dyno Therapeutics, Inc., a techbio company pioneering applications of artificial intelligence to engineer AAV capsids that expand the potential of genetic medicine, today hosted a Scientific Symposium at the 27th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting being held in Baltimore, MD.

The Scientific Symposium showcased Dyno’s approach to applying artificial intelligence to engineering novel adeno-associated virus (AAV) capsids for optimized in vivo delivery. The Symposium, titled “AAV Capsid Design in the Era of AI,” covered Dyno’s approach to solving one of the most difficult challenges of gene therapy: safely and efficiently delivering therapeutic DNA to target organs within a patient’s body.

“The present AI revolution in biotech was prefaced by decades of exponential change in the tech industry which catalyzed the personal computer revolution by putting a computer on every desk in every home, then into every pocket and on every wrist. Now the convergence of exponential change in AI, genome sequencing, and DNA synthesis technologies is driving a similar trend, this time with respect to the cost of in vivo gene delivery,” said Eric Kelsic, Ph.D., Founder and Chief Executive Officer of Dyno Therapeutics.

“Today gene therapies can cost several millions of dollars per dose,” Kelsic continued. “Dyno is committed to reducing the cost of delivering therapeutic DNA down to zero, which will be key to making the next generation of transformative gene therapies even more affordable. Historical trends suggest that dramatic cost reductions will actually grow the overall gene therapy market, supporting a robust ecosystem of gene therapy developers and eventually enabling billions of patients to benefit from life-changing gene therapies.”

Using generative AI, Dyno recently increased the efficiency of capsid-mediated in vivo DNA delivery in the eye by 80-fold with the Dyno eCap™ 1 capsid, and to the brain by 100-fold with the Dyno bCap™ 1 capsid. Building on these initial successes, Kelsic shared how Dyno’s AI-powered methods achieved two breakthroughs, in Low-Shot Efficient Accelerated Performance (LEAP), and in the design of synthetic capsid serotypes:

  • Capsids designed with LEAP dramatically outperformed capsids designed by humans without AI-assistance, improving in vivo delivery efficiency and potentially enabling lower manufacturing costs: Dyno developed LEAP technology to efficiently generate capsid sequences with enhanced performance beyond any of the capsids in the training data. LEAP now enables Dyno to rapidly advance better capsids into in vivo validation studies, resulting in faster engineering and more effective use of R&D resources. Vitally, LEAP also helps to bring down the cost of in vivo delivery by yielding capsids that more precisely deliver more therapeutic DNA per dollar of production cost.
  • Applying AI to capsid diversification generated diverse synthetic serotypes, towards expansion of patient populations who can benefit from gene therapy: Pre-existing immunity to natural AAV capsids can make anywhere from 20-80% of gene therapy patients ineligible for treatment. Dyno’s AI-powered methods successfully designed synthetic capsids that differ substantially from natural capsid sequences while still remaining functional for production and transduction of human cell lines. These results demonstrate the potential of these AI methods to broaden the number of patients who can benefit from gene therapy using synthetic capsids for gene delivery. Growing the demand for gene therapies by increasing the size of the addressable patient population is one additional way to accelerate an exponential reduction in the cost of gene therapies, since higher product revenues enable more profits to be invested in innovation that further reduces costs, thereby enabling more patients to benefit from future therapies.

Kelsic added that these breakthroughs in applying AI to the design of high-performance biological sequences will benefit from Dyno’s collaboration with NVIDIA, announced earlier in the day. The work will help advance design capabilities for gene therapies and other sequence-based medicines with potential for transformative patient impact. The collaboration will focus on scaling Dyno’s pioneering “lab-in-the-loop” sequence design approach using NVIDIA’s optimized cloud and BioNeMo platforms.

About Dyno Therapeutics

Dyno Therapeutics is solving the in vivo gene delivery challenge while partnering with gene therapy developers to maximize patient impact. Dyno’s platform combines AI with high-throughput experimentation to accelerate the design of AAV capsids with properties that significantly outperform current in vivo gene delivery vectors, with the goal of expanding the range of diseases treatable with genetic medicines. Dyno has partnered with leading gene therapy developers, including Astellas, Novartis, Roche, and Sarepta, and is broadly open to partnering across therapeutic areas. Dyno was founded in 2018 and is located in Watertown, Massachusetts. Visit www.dynotx.com for additional information.

Media Contact:
Alice Tirard
Dyno Therapeutics

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