Alera Bio is developing AB-101, a proprietary formulation that restores normal thyroid hormone transport to treat MCT-8 deficiency, a rare disorder causing both endocrine imbalance and severe neurological impairment. The dual-action therapy normalizes systemic thyroid levels while penetrating the central nervous system to improve cognitive function and neurodevelopment in pediatric patients. Alera Bio also offers patient‑focused support resources for families and clinicians managing this condition.
Funding
Funding not disclosed
Founders
Product
Problem
Patients with MCT-8 deficiency suffer from systemic thyroid hormone transport defects that cause both endocrine imbalances and severe neurological impairments, leading to developmental delays and reduced quality of life. Existing treatments do not address the underlying transport deficiency, leaving a critical unmet medical need for therapies that can normalize thyroid hormone levels and improve neurodevelopmental outcomes.
Solution
Alera Bio is developing AB-101, a proprietary formulation designed to restore normal thyroid hormone transport in tissues affected by MCT-8 deficiency. The compound targets both peripheral endocrine symptoms and central neurological deficits, aiming to normalize thyroid hormone levels throughout the body while enhancing cognitive function and neurodevelopment. By addressing the root cause of the disorder, AB-101 seeks to provide a disease-modifying therapy that improves overall patient health and daily functioning. The development program follows a patient‑first approach, incorporating safety and efficacy considerations specific to children and their caregivers.
Target Audience
Primary customers are pediatric neurologists, endocrinologists, and rare‑disease specialists treating children with MCT-8 deficiency, as well as healthcare systems and patient advocacy groups focused on rare neurological disorders.
Features
- Proprietary formulation that simultaneously normalizes systemic thyroid hormone levels and penetrates the central nervous system
- Dual-action mechanism targeting endocrine and neurological pathways associated with MCT-8 deficiency
- Preclinical data indicating improvement in cognitive function and neurodevelopmental markers
- Safety profile optimized for pediatric use, with dosing designed for long‑term administration
- Integrated patient support resources, including education and caregiver assistance programs