
Open up new investment opportunities
Join us on our mission

Early-stage opportunity
Early-stage opportunity with near term value inflection points (phase I/II and III readout, regulatory filing, approval within 3 to 4 years).
Multidisciplinary team
Multidisciplinary team with expertise in rare disease and the ability to execute the development strategy and business plan
Clear value proposition
SB-19642 has a clear value proposition, clinical development plan and targets a defined patient population with high unmet medical needs
Strong business case
Strong business case in TUBB4A leukodystrophy estimated to be $500m.
Rapidly growing market
Rapidly growing market for ASOs. Rapidly growing market for ASOs. Rapidly growing market for ASOs.
Flexible exit scenarios
Flexible exit scenarios, plan to establish a fully integrated commercial rare disease player, with potential for earlier exit at near term inflection points
Why invest in SynaptixBio?
The opportunity
SynaptixBio has come a long way in just a few short years. We are now opening up new investment opportunities for others to join our mission and support SynaptixBio’s lifechanging work through clinical trials.

Founded in Oxford, UK (2021) SynaptixBio has licenced from the Children’s Hospital of Philadelphia (CHOP) the worldwide rights to antisense nucleotides (ASOs) that inhibit the activity of the TUBB4A gene .
The company is led by a team of experts across drug development, regulatory, and corporate development with a proven track record of successful approval, launch and M&A.
Progress and Achievements
Seed funding
In 2021, seed funding of c£2m started the process of identifying candidate ASOs. This work was performed at the Children’s Hospital of Philadelphia, the world’s leading centre for leukodystrophy research.
Private investment
Private investment of c£11m followed in 2023, then two grants from Innovate UK, the UK Government’s innovation body, amounting to £2.5m.
Global market
The global market for the gene silencing technology we use (ASOs) is predicted to grow dramatically, worth c$20 billion by 2032 according to a report from Global Market Insights Inc.
The investment by the numbers
1 in 84k
TUBB4A Leukodystrophy has a global incidence of 1 in 84,000 live births (estimated 16,000 individuals)
$500m
The Global TUBB4A Leukodystrophy market is estimated to be in excess of $500m
4-5 yrs
SynaptixBio is on an accelerated development pathway with expected approval in 4-5 years from Series A
$17.2m
The team has already raised $17.2m to fund research and pre-clinical development work.
$24m
SynaptixBio is seeking funds of $24m to complete phase 1 / 2 clinical trials.
2028
High level timelines are preparing the business for approval and launch in 2028
Let's transform lives together
If you would like to find out more about joining us on our journey email us today.
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