GENETIC MEDICINE

04.05

Gene Therapy
starts with specification.

Chitosan’s cationic charge enables it to complex negatively charged nucleic acids without relying on a viral vector.

Scientist pipetting samples for gene-therapy research
GENE THERAPY · NON-VIRAL CARRIERS
PHARMACEUTICAL DEVELOPMENT PROGRAMPHARMA

01 · THE CUSTOMER PROBLEM

Gene-therapy programs need carriers that protect nucleic acids, control particle properties and support uptake without relying exclusively on viral vectors.

Mykito tunes purity, MW, DDA, charge ratio and particle architecture together, giving formulation teams a controlled starting point for DNA and RNA carrier screening.

Chitosan’s cationic charge enables it to complex negatively charged nucleic acids without relying on a viral vector.

02 · RECOMMENDED STARTING POINT

Start with the material designed for the decision.

MYKITO PLATFORMChitoGene
RECOMMENDED SAMPLEA small EndoFree MW/DDA screening set for nucleic-acid complexation studies.

Molecular weight, degree of deacetylation, charge ratio and particle architecture can be tuned together to develop non-viral DNA and siRNA delivery systems with stronger stability and transfection performance.

01

Without the shell

Controlled fungal sourcing supports a traceable, non-animal supply strategy.

02

Specification engineered

MW, DDA, purity, charge density and format are selected around the target result.

03

Application ready

Powder is only the starting point; Mykito also develops films, fibers, gels, coatings and formulations.

04

One technical partner

Material selection, formulation support and the validation plan are connected from the first sample.

03 · PERFORMANCE TARGETS

Build toward outcomes a buyer can measure.

01

Non-viral delivery

Cationic chitosan can condense DNA or RNA into polyplexes and nanoparticles for non-viral vector research.

02

In vivo siRNA development

Protected siRNA complexes can be engineered for stability, biodistribution and gene-silencing studies in vivo.

03

Transfection optimization

MW, DDA, N/P ratio, particle size and functionalization can be screened to improve uptake and transfection efficiency.

04

Targeted constructs

Reactive groups support ligand conjugation and tissue- or cell-targeting development.

04 · GO / NO-GO CRITERIA

Define the evidence before testing begins.

The first program is designed around three decision-making outputs—not an open-ended material trial.

01

Cargo binding and protection

02

Particle size, PDI and stability

03

Cytocompatibility, uptake and transfection

EndoFree purityCustom MW + DDAParticle sizeCharge ratioLigand conjugation

05 · FROM SAMPLE TO DECISION

A practical application program,
not a bag of powder.

01

Define

Agree on the function, baseline, constraints and measurable success criteria.

02

Screen

Select the MW, DDA, purity, dose and physical format that fit the application.

03

Validate

Test the winning system in the finished formulation, device, process or use environment.

AVAILABLE FORMATS
NanoparticlesPolyplexesInjectable dispersionsLyophilized complexes

Recommended first request: A small EndoFree MW/DDA screening set for nucleic-acid complexation studies.

Start the Sample Brief

06 · WHY CHITOSAN

Cationic complexation + matrix control

Protonated amino groups can interact with selected anionic actives, polymers and biological surfaces. MW, DDA, crosslinking and architecture then provide levers for adhesion, protection and release.

FORMULATION WINDOW

There is no universal concentration. Grade and dose are screened against pH, interacting ingredients, process conditions and the target performance profile.

QUALIFICATION

Route-specific purity, residuals, compatibility, stability and regulatory suitability are established for the finished system.

DEVELOPMENT CONTEXT

These application pathways describe material-development potential, not a claim that any final device, drug or therapy is approved. Biocompatibility, safety, performance, sterilization and regulatory requirements are established for the finished product and its intended use.

START AN APPLICATION PROGRAM

Move from material potential
to a defined program.

Tell us what performance, purity and format your application needs. We will help identify the right Mykito platform and a practical next step.