Transforming Lives

Pioneering Enzyme Replacement Therapies for lysosomal storage diseases, using AI and synthetic biology to meet patients' unmet clinical needs.

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About ZYTHERA

Next-generation enzyme replacement therapies for lysosomal storage diseases.

ZYTHERA is at the forefront of developing groundbreaking treatments for lysosomal storage diseases (LSDs), a diverse group rare metabolic disorders caused by enzyme deficiencies that lead to the accumulation of harmful substances in cells. Our mission is to transform the lives of patients by delivering highly effective and innovative enzyme replacement therapies (ERT).

We are committed to researching, developing, and commercializing state-of-the-art enzyme replacement therapies that address the unmet medical needs of those suffering from lysosomal storage diseases. Our focus is on delivering therapies that are not only safe and effective but also accessible to patients worldwide.

Our platform

Transforming lives through Advanced Enzyme Replacement Therapies

The ZYTHERA platform combines generative artificial intelligence for protein engineering, with a high-throughput microbial screening platform and downstream mammalian biologics production system to move from computational design to lead candidate identification in as little as 4-weeks.

Generative Protein Engineering

Our propietary generative AI model uses protein sequence information to learn high-order amino acid relationships to design variant libraries of a target enzyme. Our variants are prioritized using our in-silico characterisation workflows to bring into the lab the most promising candidates.

We have developed a highly efficient, microliter-scale, protein screening platform based on P. pastoris, a widely used yeast in the biotech industry, which allows us to screen hundreds of enzymes in as little as 2 weeks.

Efficient production

Our Chinese Hamster Ovary (CHO) system integrates landing pads at genomic loci, which are designed to ensure single copy genomic integration of target enzymes to facilitate stable production of proteins with human-like biochemical properties.

Our Team

Experts in Next-generation Therapeutics Engineering

Photo of Giovanni Stracquadanio, PhD Giovanni Stracquadanio, PhD CEO and Co-founder
Photo of Giovanni Stracquadanio, PhD Giovanni Stracquadanio, PhD CEO and Co-founder Bio Dr Giovanni Stracquadanio is an Personal Chair in Engineering Biology at the University of Edinburgh. He is an UKRI EPSRC fellow and co-director of the Edinburgh Genome Foundry. He has more than 15 years of experience in AI, computational biology and microbiology to address biomedical challenges. He was co-author of the first synthetic eukaryotic chromosome and has characterised the role of high-frequency inherited mutations in the p53 pathway. He is now working on engineering new enzyme replacement therapies for Fabry diseases using AI and microbial expression systems.
Photo of Susan Rosser, PhD Susan Rosser, PhD Co-founder
Photo of Susan Roser, PhD Susan Rosser, PhD Co-founder Bio Dr Susan Rosser is a Professor of Synthetic Biology at the University of Edinburgh. She is Director of the Edinburgh Mammalian Synthetic Biology Research Centre, Co-director of the Edinburgh Genome Foundry. Her research focuses on developing tools for synthetic biology approaches for pathway and genome engineering in bacteria, yeast and mammalian cell systems. The applications of her work include rapid cell line engineering for biologics production. Read more Close
Photo of Eve Miller-Hodges, MD PhD Eve Miller-Hodges, MD PhD Medical Advisor
Photo of Michelle Levene Eve Miller-Hodges, MD PhD Medical Advisor Bio Eve Miller-Hodges is a Senior Clinical Lecturer in Inherited Metabolic Disorders and Renal Medicine at the University of Edinburgh with experience across all aspects of translational research, including developmental biology, clinical trials and data science. She helped establish clinical care for LSD patients in Scotland and now works in the Scottish Inherited Metabolic Disorders Service which provides specialist management of all patients with lysosomal storage disorders in Scotland. Read more Close
Photo of Michelle Levene Michelle Levene Commercial Champion
Photo of Michelle Levene Michelle Levene Commercial Champion Bio Michelle Levene is Commercial Champion at Zythera, leading commercial strategy and business development. With over 20 years of experience in life sciences, Michelle has delivered successful clinical development programs, managed rare disease product launches, driven cross-functional teams, and supported major acquisitions and spin-outs. She is passionate about building partnerships that enable patient access to transformative treatments and excels at navigating complex commercial challenges. Read more Close

Research

Our ground-breaking research

ZYTHERA stems from ground-breaking research conducted in the Stracquadanio and Rosser lab at the University of Edinburgh and published in international, high-impact, peer-reviewed journals

Dirichlet latent modelling enables effective learning and sampling of the functional protein design space

Dirichlet latent modelling enables effective learning and sampling of the functional protein design space

Lobzaev, E. and Stracquadanio, G., 2024. Nature Communications, 15(1), p.9309.

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Protein engineering using variational free energy approximation

Protein engineering using variational free energy approximation

Lobzaev, E., Herrera, M.A., Kasprzyk, M. and Stracquadanio, G., 2024. Nature Communications, 15(1), p.10447.

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News and Media

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