Development Strategy Used for Fundraising

SITUATION

Small virtual biotech start-up with stem-cell therapy treatment for HIV. Strong scientific expertise but limited experience in moving research phase work into early clinical (pre-IND) development. No experience in regulatory affairs to understand the dynamic nature of regenerative medicine interactions with FDA.

SOLUTION

  • Provided a review of the regulatory guidelines relevant for stem-cell therapies. Identified specific studies that would be required as part of the IND-enabling preclinical package.
  • Worked with scientific team to streamline research activities to efficiently build preclinical IND data package.
  • Prepared a high-level Phase I study design framework by reviewing ongoing, competitive Phase I studies.
  • Participated in investor due-diligence discussions, providing assurance of a clear and feasible regulatory strategy for early clinical studies.

VALUE

Established a clear development candidate target product profile for the scientific team to focus on. Broadened awareness of the preclinical IND-enabling supportive studies required for IND filing so that studies could be better designed. Phase I study designed raised important considerations with respect to manufacturing scale required for 1st patient study. Client raised $2.5 MM in additional funding to support remaining IND-enabling activities.