Lentiviral vectors
Used to test durable genetic payload delivery and disease-relevant readouts in experimental systems.
Therapeutic Development
Developing therapeutic tools for inherited cerebral small vessel disease.
We are developing and testing novel lentiviral, non-integrating lentiviral, AAV, and antisense oligonucleotide approaches to target inherited CSVD, with early work focused on building reliable experimental evidence.
Therapeutic toolkit
Different modalities answer different biological questions.
Lentiviral vectors can support stable delivery of genetic payloads in experimental systems, while non-integrating lentiviral vectors are designed to reduce genomic integration while retaining useful delivery properties. AAV vectors offer a compact delivery system with strong relevance for in vivo gene therapy research. ASOs provide a flexible way to modulate RNA through knockdown, splice modulation, or target-specific transcript control.
Together, these tools allow the team to compare how different therapeutic ideas perform in vitro and in vivo before any concept is considered for a translational route.
Non-integrating LV
Supports delivery experiments where reduced integration is an important design consideration.
Adeno-associated virus
Provides an in vivo-relevant vector platform for compact therapeutic payloads and translational testing.
Antisense oligonucleotides
Opens RNA-targeted opportunities including knockdown, splice modulation, and allele-informed approaches.
Design platform
shCREATE supports the design of siRNA, shRNA, and ASO tools.
shCREATE is a software platform created by Dr Favour Felix-Ilemhenbhio to support RNA-targeting design work. It can help researchers generate candidate small interfering RNA (siRNA), short hairpin RNA (shRNA), and antisense oligonucleotide (ASO) sequences for experimental testing.
Small interfering RNA
Short RNA molecules that can reduce expression of a target transcript, useful for testing whether lowering a gene product changes a disease readout.
Short hairpin RNA
Vector-expressed RNA hairpins that can provide longer-term knockdown experiments in cell and model systems.
Antisense oligonucleotide
Synthetic oligonucleotides that can modulate RNA through knockdown, splice switching, or other transcript-focused mechanisms.
Roadmap
Keep the science close to the disease, the evidence, and the people affected.
CSVD Therapeutics is building a practical translational path for inherited cerebral small vessel disease. The work begins with defined genetic causes, uses disease-relevant models to test candidate interventions, and prioritizes the evidence needed before any treatment concept can be responsibly moved toward patients.
Start with a defined cause
Focus on inherited CSVD where variants in COL4A1 or NOTCH3 give a precise biological starting point.
Test disease-relevant systems
Use cellular and vascular readouts to ask whether candidate therapies change measurable disease biology.
Develop credible interventions
Evaluate approaches such as plasmid systems, viral vectors, ASOs, CRISPR strategies, and drug repurposing where appropriate.
Build readiness for patients
Connect biomarkers, safety, delivery, natural history, and patient priorities into the evidence package needed for future trials.
