SynaptixBio Ltd.

SynaptixBio Ltd. is a rare disease biotech company dedicated to developing life-saving therapies for severe leukodystrophies. They are focused on addressing a specific subset of leukodystrophies caused by mutations in the TUBB4A gene, which disrupts the formation of myelin sheaths surrounding nerve fibres in the brain. SynaptixBio aims to silence the expression of the mutated gene using antisense oligonucleotide (ASO) technology, which has shown promise in treating other genetic disorders.

SynaptixBio was founded in 2021 by experienced biopharmaceutical leaders with a strong focus on rare disease advocacy. Their team includes scientists from the Children's Hospital of Philadelphia (CHOP), where the causative mutation for TUBB4A leukodystrophy was discovered. They have established a partnership with CHOP to translate their ground-breaking research into a transformative therapy and are aiming to launch clinical trials in 2024. Their mission is driven by the desire to improve the quality of life and extend the survival of patients with this devastating disease.

SynaptixBio is a rare disease biotech company focused on developing lifesaving therapies for severe leukodystrophies, a group of genetic disorders affecting the brain's white matter. Their mission is to disrupt current treatment practices for these devastating conditions by developing a therapy that addresses the root cause of the disease and has the potential to be curative. Their efforts are driven by a team of experienced leaders, scientists, and clinicians who are passionate about finding new and effective treatments for this type of rare and underserved disease.

SynaptixBio promotes a culture of collaboration, innovation, and dedication to finding cures for rare diseases. They are driven by a shared mission to improve the lives of patients and their families. The company is comprised of experienced leaders and scientists, with a strong emphasis on supporting and advocating for patients.