SynaptixBio

Activities: Research & Development

Trending Articles

  1. NHS, MHRA and Medicines UK launch Project Revive to tackle medicine shortages NHS England, the MHRA and Medicines UK have launched Project Revive, a collaborative pilot designed to address critical medicines shortages by fast-tracking dormant...
  2. Isopod Rapid Cleanrooms: the ideal elite controlled environment pod solution for Life Sciences and Engineering start-ups The Guardtech Group’s quick-assembly cleanroom pods combine superior performance, build quality and cost-effectiveness to give new applications the perfect platform to get...
  3. You need to be a subscriber to read this article.
    Click here to find out more.
    Delivering precision: why advanced therapies are redefining pharmaceutical logistics As advanced therapies narrow tolerances and personalise care, pharmaceutical logistics is being redefined as a precision-driven extension of treatment delivery, writes Michael...
  4. Nose-to-brain delivery outperforms systemic BBB technologies according to MetP Pharma The pioneering company has published preclinical data that strongly supports the advantages of its proprietary nose-to-brain drug delivery technology compared with systemic...
  5. AstraZeneca strengthens weight management pipeline with CSPC Pharmaceuticals collaboration The pair have signed a strategic collaboration covering eight obesity and type 2 diabetes programmes, including a clinical-ready GLP-1/GIP asset, AI-driven peptide discovery...

Upcoming event

High Shear = Mixing Success

19 February 2026 | Virtual See all

Related Content

You need to be a subscriber to read this article.
Click here to find out more.
Research & Development

Solving the rare disease puzzle

Dan Williams, founder at SynaptixBio, explores the complex challenges of diagnosing and treating rare diseases, their impact on patients and healthcare systems and how small biotech firms are reshaping therapeutic innovation
Regulatory

New NICE thresholds: good news for rare disease drugs but process remains complex

Recent changes introduced by NICE will ultimately see more ultra-rare disease drugs get to market, but Dan Williams, CEO at SynaptixBio, says the process remains challenging
Research & Development

SynaptixBio selects H-ABC antisense oligonucleotide for clinical progression

The company will assess the safety and efficacy of SB H-19642 for the treatment of Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum (H-ABC)
Research & Development

SynaptixBio and Evotec collaborate to develop novel antisense oligonucelotides for H-ABC

The companies will look to discover novel antisense oligonucleotides targeting mutant TUBB4A for the treatment of H-ABC

SynaptixBio awarded FDA Orphan Drug Designation

SynaptixBio are now aiming to develop a treatment for the second variant of TUBB4A leukodystrophy
Research & Development

Biotech company awarded prestigious Innovate UK grant to expand search for rare disease therapies

SynaptixBio, the only company developing a treatment for a rare but deadly disease, will receive almost half a million pounds from the UK’s national innovation agency
Subscribe now