SynaptixBio
SynaptixBio is a pioneering biotechnology company dedicated to developing advanced gene silencing therapies for rare and deadly neurodegenerative diseases, including TUBB4A leukodystrophy. With a mission to transform lives, the company leverages cutting-edge antisense oligonucleotide technology and collaborates with leading research centers to bring innovative treatments to clinical trials, aiming to provide hope for patients with currently incurable conditions.
Industries
Nr. of Employees
small (1-50)
Products
Preclinical ASO therapeutic candidate for TUBB4A-related leukodystrophy
A preclinical antisense oligonucleotide therapeutic candidate developed to reduce production of a toxic protein produced by a mutated TUBB4A gene, intended to address hypomyelination and related neurological deficits.
Preclinical ASO therapeutic candidate for TUBB4A-related leukodystrophy
A preclinical antisense oligonucleotide therapeutic candidate developed to reduce production of a toxic protein produced by a mutated TUBB4A gene, intended to address hypomyelination and related neurological deficits.
Services
Sponsored research and academic licensing
Sponsorship of academic research and exclusive licensing of academic intellectual property to translate laboratory findings into clinical development candidates.
Clinical development sponsorship and oversight
Sponsorship and oversight of clinical trials including clinical strategy, site partnerships, medical monitoring and regulatory compliance to progress candidates to first-in-human studies.
Sponsored research and academic licensing
Sponsorship of academic research and exclusive licensing of academic intellectual property to translate laboratory findings into clinical development candidates.
Clinical development sponsorship and oversight
Sponsorship and oversight of clinical trials including clinical strategy, site partnerships, medical monitoring and regulatory compliance to progress candidates to first-in-human studies.
Expertise Areas
- Antisense oligonucleotide therapeutics
- Preclinical development and candidate selection
- Clinical trial design and medical oversight for paediatric rare diseases
- Regulatory strategy for orphan and rare-paediatric designations
Key Technologies
- Antisense oligonucleotides (ASO) therapeutics
- Next-generation sequencing (NGS)
- Juvenile and species-appropriate animal models
- Non-clinical (in vitro/in vivo) efficacy and toxicology studies