SynaptixBio

The company's strategic focus is on a group of genetic disorders known as TUBB4A-related leukodystrophies, which disrupt the formation of myelin, the protective sheath around nerve fibers in the brain. This disruption primarily affects young children, leading to severe impairments in motor skills, speech, and swallowing. To address this, SynaptixBio is advancing a therapeutic platform based on antisense oligonucleotide (ASO) technology. This technology utilizes short synthetic DNA strands designed to bind to the mutated TUBB4A messenger RNA, effectively silencing the gene and preventing the production of the toxic protein that causes the disease. The hypothesis is that with the toxic protein suppressed, other proteins can facilitate the normal formation of myelin, potentially halting disease progression and even reversing some symptoms.

SynaptixBio operates a lean, virtual business model, with a small core team managing a network of outsourced activities, including a key sponsored research agreement with the Children's Hospital of Philadelphia (CHOP). This partnership grants SynaptixBio an exclusive worldwide license to commercialize the intellectual property developed at CHOP, a leading center for leukodystrophy research where the causative mutation for H-ABC was discovered. The company's business model is centered on advancing its lead drug candidate through clinical trials and securing regulatory approvals. Revenue generation is anticipated through the commercialization of its therapies, bolstered by strategic designations from the U.S. Food and Drug Administration (FDA). SynaptixBio has successfully obtained two Orphan Drug Designations (ODD) and two Rare Paediatric Disease Designations (RPDD) for both the most severe form of the disease, H-ABC, and a milder variant, Isolated Hypomyelination. These designations provide financial incentives, such as tax credits and grant eligibility, and can lead to a Priority Review Voucher (PRV), a valuable, sellable asset that expedites FDA review.

Since its inception, SynaptixBio has secured significant funding primarily from private investors and grants. The company has raised approximately $9.35 million over five rounds, including notable grants from UK Research and Innovation and Innovate UK. This capital is being deployed to advance its lead candidate, SB H-19642, into first-in-human clinical trials. The company has established collaborations with contract research organizations like Evotec to develop and qualify its ASO candidate, which has shown promising results in preclinical safety and efficacy studies.