Now that you know the value of a Pre-IND meeting, it is important to consider if you are at the right stage to request one.
A readiness assessment can help identify any gaps or areas of concern in the development of your AAV gene therapy product and enable your team to address these issues proactively.
Please see below for a checklist to guide you in assessing if you are ready for a Pre-IND meeting. Completing the elements in the checklist is the first step to increasing the likelihood of a successful meeting and eventual IND submission.
Note that these are best practice recommendations for when a Pre-IND meeting would be most useful to a sponsor. You may have completed or not yet completed many of the tasks in the stages listed below and still be granted a Pre-IND meeting, so do your best to check off as many items on this list to ensure you are at the optimal stage of readiness for the Pre-IND!
Table: Readiness Assessment for Pre-IND meeting: Check to confirm completion of these tasks for optimal stage of readiness
Area | Tasks |
|---|
Pre-clinical | Completed dose range finding studies and planning confirmatory GLP dose range studies Completed pilot in vivo pharmacology and toxicology studies and planning GLP pivotal pharmacology/toxicology studies Planning GLP biodistribution studies
|
CMC | Preliminary defined processes, testing and specifications for Master Cell Bank (MCB), Working Cell Bank (WCB) and Drug Substance (DS) and Drug Product (DP) manufacturing Specifically, tests for safety should be identified and qualification/validation are in process. Potency tests are typically not expected to be completed at Pre-IND, but ideas on these types of tests should be presented Near completion or completion of GMP lots. At a minimum, engineering runs representative of GMP production should be in process Preliminary testing and design of stability studies for DS, DP, and Point of Care (POC) delivery (e.g., reconstitution of frozen product and administration through syringe)
|
Clinical | Ideally, a completed clinical study synopsis should be prepared and included in the Pre-IND meeting package. If a final synopsis is not ready, at a minimum, a clinical study concept sheet which includes details on the population, inclusion/exclusion criteria, endpoints, dosing and dose escalation regimen, dose rationale, safety, and efficacy (if applicable) measurements and analysis, study time points and overall duration
|
Patient Engagement | |
Prior to proceeding, let us do one final check to ensure you are ready to initiate the Pre-IND meeting preparation.
Have you checked all the readiness assessment requirements?
Adeno-associated virus (AAV) - A member of the parvovirus family of single-stranded small DNA viruses that require a helper virus such as adenovirus or herpes simplex virus for replication. AAV vectors are the leading platform for gene delivery for the treatment of a variety of human diseases.
Gene Therapy is an approach that treats genetic disorders by providing new DNA to certain cells or correcting the DNA
Good Laboratory Practice (GLP) - is a quality system concerned with the organizational process and the conditions under which non-clinical health and environmental safety studies are planned, performed, monitored, recorded, archived and reported.
Good Manufacturing Practice (GMP) - Compilation of various guidelines/guidance documents/directives issued and elaborated by international organizations and institutions, in collaboration with pharmaceutical industry and several national regulatory authorities in different regions and countries, in order to be guaranteed the highest standards of efficacy, quality and safety in any process that involves the manufacture of health products.
Drug Substance (DS) - The main ingredient in a medicine that causes the desired effect of the medicine. Some medicines contain more than one drug substance that act in different ways in the body. Also called active pharmaceutical ingredient, API, and pharmacologic substance.
Drug Product (DP) - A dosage form that contains one of more active and/or inactive ingredients