At Ariya Bio, we are on a mission to harness the power of CRISPR-Cas9 and hematopoietic stem cells to develop transformative genomic medicines.
Our unique approach
At Ariya Bio, we are pioneering a next-generation gene therapy platform, harnessing blood stem cells, also known as Hematopoietic Stem Cells (HSCs) as living, in-vivo biotherapeutic protein factories. The CRISPR-Cas-based cell and gene therapy platform we are developing is versatile and promises to be easily reprogrammable. We combine two powerful technologies: hematopoietic stem cell therapies and CRISPR-Cas gene editing to develop transformative genomic medicines.
We harvest the patients' own HSCs by apheresis, purify them and either insert small DNA sequences, a healthy working copy of the affected gene or a therapeutic protein to cure the disease. The patient then receives conditioning to deplete the diseased hematopoietic stem cells to make space for the gene-corrected therapeutic HSC population. The gene-corrected stem cells are then infused back into the patient intravenously. These cells then engraft, expand, and produce therapeutic blood cells.
Our platform is applicable to a wide range of diseases and promises to bring curative genomic medicines to patients.
Discover Our Programs
Beta-Hemoglobinopathies
Beta-Hemoglobinopathies are caused by mutations in the Beta-Globin gene affecting the structure and function of hemoglobin.
We have two candidates, ARY001 and ARY002 in pre-clinical development.
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Multiple Assets - Undisclosed
Thanks to Ariya Bio's transformative CRISPR-Cas-based gene therapy platform, hematopoietic stem cells and their downstream lineages can be harnessed to express biotherapeutic proteins in vivo. This gives us great flexibility in which indications to target.
Ariya Bio in the Press
ETH Zurich molecular biologist Mandy Boontanrart is researching gene therapies that could be used to cure two of the most common types of inherited anaemia. She has now developed a promising approach for so-called beta-hemoglobinopathies.
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This study presents the important finding that gene editing could be used to activate δ-globin expression to treat disorders of red blood cell synthesis. The evidence supporting the claims of the authors is convincing…
Mandy Boontanrart, PhD, a postdoc at ETH Zurich in Switzerland, has a particularly personal motivation for her research on a novel genetic therapy for sickle cell disease…
Our Team
Our diverse team of researchers is working every day to bring cutting-edge therapeutics to patients around the world.