Treatment for a previously untreatable disease to improve quality of life for children, caregivers, and families.
Ceres Brain Therapeutics is developing a drug candidate for a rare neurological disease that causes severe intellectual disabilities, autistic behaviors, and seizures. The drug could be made available by 2027.
Ceres Brain produced 60 kg of the CBT101 formulation in this mixer for use in a nasal spray. (c) ceres brain
X-linked creatine transporter deficiency is an inherited disease linked to a genetic mutation on the X chromosome that prevents creatine (an amino acid that provides energy to cells) from reaching neurons. With an initial clinical trial scheduled for 2024, Ceres Brain Therapeutics is currently the company in the world closest to developing a drug for this disease.
The drug, CBT101, is a pro-creatine that, thanks to a chemical vector graft, can cross the blood-brain barrier, supplying neurons with creatine. Animal tests have shown very significant improvement in cognitive behavior.
Ceres Brain Therapeutics has a portfolio of six patents, including four CEA patents. As part of an R&D agreement with the CEA, the startup is deepening its understanding of the mechanism of its drug candidate and developing new candidates targeted at other rare neurological diseases.
Key figure: 16,000
Estimated number of children worldwide affected by congenital creatine transporter deficiency
Technology:
- Active ingredient coupled with a chemical vector to cross the blood-brain barrier and neuronal membrane
- Nasal administration to reach the brain via the olfactory and trigeminal nerves
Year founded: 2019
CEA institute: Frédéric-Joliot Institute