The Problem
An academic researcher at a leading Tier 1 (R1) Research University had developed a promising but highly complex gene therapy for the treatment of a rare and aggressive form of cancer, designed for systemic administration but targeted delivery. At the time of engagement, the program had languished for several years following a self-directed pre-IND meeting with FDA. Additionally, the University had never been the IND Sponsor for a first-in-human pharmaceutical product. Our team was engaged to help address FDA’s ongoing concerns and shepherd the program into clinical development.
Our Actions
Our team collaborated with multiple teams within the university, including the innovator, the study Principal Investigator, the university hospital clinical operations and quality assurance teams, and university’s Office for Research, supporting the following key deliverables:
- Our team supported the innovator in the management of their contract manufacturers, with an emphasis on resolving FDA-identified deficiencies in the characterization of the candidate study drug.
- We prepared the Investigational New Drug application, including negotiating with FDA the acceptance of Phase 0 GMP materials for the initial clinical pharmacokinetics study.
- Our team led the preparation of the study Investigator’s Brochure, Laboratory Manual, and Pharmacy Manual, closely collaborating with the university hospital teams responsible for the execution of all study procedures.
- Our team led the site initiation visit, including the training of the Principal Investigator and clinical study team staff on critical documentation and procedures.
- We developed a comprehensive set of clinical Standard Operating Procedures for the University’s R&D office, reflecting their new responsibilities as an IND sponsor.
- Our team managed all aspects of IND communications, including safety reporting, protocol and chemistry amendments, and annual reporting.
Following the IND submission, our team was asked by the University to provide clinical trial monitoring oversight and pharmacovigilance support during the entirety of the first-in-human clinical study.
Outcome
With our team’s support, our client brought a stalled program through to demonstration of pharmacokinetic proof of principle for a gene therapy product that incorporated both a novel mechanism of action and a novel mechanism of delivery, as well as developed the framework for the University to pursue its interest in maximizing innovation value through the regulatory Sponsorship of early-stage clinical trials. This strategic collaboration resulted in continued and expanded federal grant funding for the University’s broader clinical research initiatives, successful completion of out-licensing activities for multiple programs, and the designation of our team as a preferred vendor for future regulatory and clinical development support needs, a relationship that has spanned programs in numerous other therapeutic areas, including infectious disease, diagnostics, cardiorenal diseases, and neurotrauma.
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