AB-101
A New Hope for
Patients Suffering
from AHDS
Background
​​MCT-8 deficiency is characterized by impaired thyroid hormone transport, leading to elevated circulating triiodothyronine (T3) levels in the blood and inadequate delivery of thyroid hormones to the brain. This imbalance causes both neurological (e.g., developmental delays, hypotonia, spasticity) and endocrinological symptoms (e.g., abnormal thyroid function tests).​
Our Lead Program
AB-101 designed to address both the endocrinological
AND the neurological symptoms of MCT-8 deficiency
​Our lead product is expected to modulate thyroid hormone activity and support critical metabolic pathways.
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Our unique product can is expected to bypass the dysfunctional MCT-8 transporter and directly enter brain cells. This will help regulate thyroid hormone signaling in tissues where MCT-8 transport is impaired, potentially reducing neurodevelopmental symptoms and stabilizing peripheral thyroid hormone levels.
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Additionally, our lead product can play an important role in converting thyroid hormone precursors into their active forms, which can help maintain optimal thyroid function. We believe it also supports brain health by aiding in the synthesis of neurotransmitters, which may alleviate some of the neurological deficits in MCT-8 deficiency.
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We believe that our product supports the synthesis of neurotransmitters and myelin, both of which are critical for neurodevelopment and brain function. By enhancing methylation and neurotransmitter pathways, it may help mitigate developmental delays and cognitive deficits in MCT-8 deficiency patients.
Why our Approach is Unique
Scientific Rationale: A Multi-Pathway Approach to Correcting Thyroid Dysfunction and Neurological Deficits in MCT-8 Deficiency
Our investigational therapy is designed to specifically address the biochemical and neurological disruptions caused by MCT-8 deficiency by:
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Tissue-Specific Effects: Ensuring targeted delivery to critical tissues like the brain and muscles, maximizing therapeutic outcomes.
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Promoting Neurogenesis and Neuroplasticity: Supporting the growth of new neurons and the formation of adaptive neural pathways, ultimately improving cognitive function and emotional well-being.
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Targeting Key Neurochemical Pathways: Modulating thyroid hormone activity, dopamine synthesis, serotonin balance, and oxidative stress to create a balanced neurochemical environment.
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Decreasing Neurotoxicity: Protecting neurons from the toxic effects of dysregulated neurotransmitter metabolism and oxidative damage.
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Our lead product offers a promising multi-faceted approach for MCT-8 deficiency by addressing both neurological and endocrinological symptoms. While further scientific validation is needed, this therapeutic strategy could provide significant improvements in quality of life for patients with this rare condition.
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Disclaimer: This therapy is currently in development and is not approved by the FDA for commercial use.