Expert consulting services to fast track your gene therapy company to clinical trials
If you are in developing stages for AAV based gene therapies or have already chosen a clinical candidate or are getting ready to perform IND enabling studies, AAV GTx Consultation can be of help. Reach out for a free initial communication.
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Learn More- 01
Vector Design and Clinical Candidate Selection
Promoter design and promoter enhancement (regulatory elements); sufficient expression levels in target cells; efficient and cost effective production of therapeutic vector require careful design and review.
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Design of IND enabling studies and GLP Tox studies
IND enabling studies and selection of appropriate animal models, design and execution of efficacy studies and GLP toxicology studies and biodistribution.
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Functional Potency Assay Design
The beauty and strength of gene therapy is the cell specific promoter, the challenge for functional potency assay typically is the cell specific promoter. Often the time needed and complexity to design such assay(s) is underestimated.
About AAV GTx Consultation
AAV GTx Consultation specializes in guiding AAV gene therapy companies through the IND submission process. Our expertise in GLP study design, functional assay development, and preparation of IND documentation helps clients in the ophthalmology and otology fields achieve successful IND approvals.
AAV GTx Consultation can assist with optimization of vector design, and development toward developmental clinical candidate selection, and assist in working with CRO and CDMO companies, and assist in preparation of the IND submission documents.
Adrian M. Timmers, Ph.D. CEO-Founder
IND Application Design
A successful IND (investigational new drug) application will have three major components addressing CMC (chemistry, manufacturing and control), pharmacology and toxicology, and clinical study protocol. The team at AAV GTx Consultation can assist you in every aspect of an IND application and help navigate and work on preparation of several additional critical components (e.g. functional potency assays).
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Clinical Trials Today
AAV GTx Consultation can assist with optimization of vector design, and development toward developmental clinical candidate selection, and assist in working with CRO and CDMO and preparation of the IND submission documents.
Reach out for Consultation Now