The startup develops molecularly targeted precision genetic medicines that utilize small molecules to modulate gene expression at the DNA level. This technology provides effective treatment options for monogenic diseases, particularly in hematology, cardiovascular, and aging-related disorders, which have not been adequately addressed by existing genomics-based therapies.
Funding
$10M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Current genomics-based therapies do not adequately address many monogenic diseases, particularly in hematology, cardiovascular, and aging-related disorders, leaving a significant unmet need for effective treatment options. Patients with sickle cell disease and thalassemia, among other hemoglobinopathies, face limited therapeutic options to manage the disease's associated morbidities.
Solution
AkiraBio is developing molecularly targeted precision genetic medicines utilizing small molecules to modulate gene expression at the DNA level, offering a potentially disease-modifying treatment. Their lead candidate, AB1, is an oral, once-daily HbF inducer currently in clinical development for sickle cell disease. AB1 aims to address all associated morbidities for all patients with sickle cell disease and thalassemia. The therapy works by depleting DNMT1, which reduces nucleotide methylation and repressor complex binding, allowing HbF gene activation and increasing the production of healthy circulating fetal red blood cells. This approach has the potential to act as a 'functional cure' for these conditions.
Target Audience
The primary target audience includes patients with sickle cell disease, thalassemia, and other hemoglobinopathies, as well as hematologists and healthcare providers specializing in these conditions.
Features
- Oral, once-daily small molecule HbF inducer
- Targets DNMT1 to reduce nucleotide methylation
- Designed to activate HbF gene expression
- Potential to increase production of healthy circulating fetal red blood cells
- Could address all associated morbidities of sickle cell disease and thalassemia
- Currently in clinical development