MCT-8 deficiency is an ultra-rare condition with approximately 1 in 70,000 males affected by this X-linked disorder worldwide, with most cases diagnosed in early childhood. The disease is characterized by profound neurodevelopmental delays, severe motor dysfunction, and cognitive impairment, along with systemic thyroid-related complications. There are currently no approved therapies, underscoring the urgent unmet need and the potential for meaningful patient impact.
The MCT-8 deficiency program, now designated AB-101, has received Rare Pediatric Disease (RPD) designation from the U.S. Food and Drug Administration (FDA). AB-101 is designed to address both the neurological and endocrinological manifestations of the disorder, representing the first potential therapy capable of targeting both aspects of the disease.
“Securing these rights marks a defining moment for Alera Bio,” said Frank Jaeger, Founder and Chair of Alera Bio. “With AB-101, we are pursuing the first therapeutic option for MCT-8 deficiency – one that addresses not only the devastating neurological impact but also the systemic endocrine effects of the disease. This agreement positions Alera Bio to deliver innovation where no options exist today and to establish ourselves as a leader in rare neurological disease innovation. Our mission remains clear: Biology drives us. Patients define us.”
“Partnering with Alera Bio ensures this critical program will advance with the focus and resources needed to reach patients,” said Cassius Coleman, Managing Director of BLA Technology, LLC. “We are proud to see AB-101 prioritized as Alera Bio’s lead asset and are confident that this collaboration offers the best path toward meaningful impact for children and families.”
With Rare Pediatric Disease designation already granted by the FDA, Alera Bio plans to pursue Orphan Drug Designation for AB-101 as its next regulatory milestone. The company also plans to initiate preclinical development activities in Q4 2025, with the goal of advancing AB-101 into clinical studies in 2026.
About Alera Bio
Alera Bio is a privately held biotechnology company dedicated to developing transformative therapies for rare and neglected neurological diseases. Headquartered in Chicago, IL, the company was founded in 2025 with a singular goal: to improve the lives of patients suffering from devastating neurological deficits.
Alera Bio has secured exclusive rights from BLA Technology, LLC to therapeutic approaches for MCT-8 deficiency (Allan-Herndon-Dudley Syndrome). Its lead program, AB-101, has been granted Rare Pediatric Disease (RPD) designation by the FDA, underscoring the urgency of this patient population and the potential for expedited development.
Guided by a patient-first strategy, Alera Bio combines cutting-edge science with a capital-efficient development model to address critical unmet needs. The company’s approach focuses on restoring biological function, advancing therapies that target both neurological and systemic pathways, and working collaboratively with families, researchers, and regulators to accelerate meaningful solutions.
At Alera Bio, the mission is clear: Biology drives us. Patients define us.
About BLA Technology, LLC
BLA Technology, LLC is a biotechnology company advancing novel therapeutic approaches for rare diseases and thyroid-related disorders. Its intellectual property portfolio includes issued and pending patents in hypothyroidism, hyperthyroidism and MCT-8 deficiency.
Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding the development of AB-101, regulatory designations, planned preclinical and clinical activities, and the potential therapeutic impact of the program. Forward-looking statements are based on current expectations and assumptions and are subject to risks and uncertainties that could cause actual results to differ materially. Alera Bio undertakes no obligation to update forward-looking statements, except as required by law.
