Programs in the Pipeline
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.
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.
Utilizing CRISPR-Cas9
CRISPR-Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. CRISPR Cas9 was adapted from the natural defence mechanisms of bacteria and archaea (the domain of single-celled microorganisms). These organisms use CRISPR-derived RNA and various Cas proteins, including Cas9, to fend off attacks by viruses and other foreign bodies.
Understanding
β-Hemoglobinopathies
The β-Hemoglobinopathies are life-threatening disorders that are causing a global health burden. They are the most common monogenetic disorders worldwide and lead to deformed and defective red blood cells. There are ~6 million patients and 560 million carriers worldwide.
The Science behind our Therapy
W increase production of beneficial haemoglobins, i.e., haemoglobins that are not affected by the β-Hemoglobinopathies. Unlike our competitors who increase Fetal Hemoglobin, we increase Adult Hemoglobin 2 (HbA2) as it is identical in form and function as regular Adult Hemoglobin.