Development of a biorepository to provide the research community with patient-derived, high-quality pluripotent stem cells and associated clinical information.
The company accelerates Personalized Medicine discovery by sourcing, processing and providing biosamples and associated demographic/clinical data to researchers worldwide. Its novel model, through which it recruits patients not only through partnerships with clinics and patient advocacy groups but also through a direct-to-patient, web-based platform, enables it to collect, and ultimately provide patient samples and associated data much more quickly and less costly than current market standards. Building on its existing strength, the company’s goal is to further accelerate the development of new treatments by establishing a premier center (i.e., CIRM Stem Cell Bank) for human induced pluripotent stem cell (hiPSCs) banking to provide researchers with cells/data they need for disease modeling and drug development. The company’s goal is to become a biobank that accelerates stem cell research through collaborations with local stem cell institutes and companies It has ambitious, yet realistic, growth targets over the course of the next five years. CIRM funding would be leveraged to a) extend the infrastructure to become a premier hiPSC hub that provides hiPSCs to researchers worldwide and b) become self-sustaining by mid-2016.
Statement of Benefit to California:
The company’s overall goal is to develop a collaborative research community that enhances California’s leadership and competitiveness in the field of stem cell research. It already built, and will continue doing so, several partnerships with California-based research institutes as well as companies. The main focus of the hiPSC banking will be to increase the number and quality of human cell lines available for therapeutic R&D activities such as disease modeling and drug discovery. To facilitate and further accelerate stem cell research, the biobank pursues the following plan: i) Deepen and expand relationships with California-based stem cell clusters in San Francisco, San Diego and Los Angeles. ii) Potentially provide stem cell and previously CIRM-funded, newly-formed ventures a) space and equipment and b) potential connections for follow-on funding through its ties to several venture capital firms. iii) Attract skilled workforce available to fill jobs in the private biotechnology and pharmaceutical industry.
The goal of this proposal is to establish a self-sustaining repository service for the banking and distribution of human induced pluripotent stem cell lines (hiPSC) from 3000 tissue donors for use by the scientific and medical community. Towards this end, the applicant seeks to expand its existing biobank platform by engaging three collaborating organizations to provide expertise and services associated with 1) hiPSC storage, maintenance and quality control; 2) next generation sequencing of cell lines; and 3) a suite of web-based tools for investigators to search, query, and analyze patient data. The applicant proposes to establish pre-master cell banks that can be expanded according to demand, and a batch record system for tracking key process steps prior to banking. The applicant’s current software platform will be leveraged and extended to allow clinics and the iPSC Deriver to track samples and annotate/add information to the system. Quality of the Proposed Repository - The space and equipment described in the application are more than adequate for supporting the culture and expansion of hiPSC banks, although some reviewers were concerned that their proximity to dedicated vivarium space might pose a future risk for contamination. - Reviewers considered the plans to perform karyotyping only after expansion into larger cell banks to be somewhat risky, as the patient-derived cells may harbor genetic abnormalities that could affect their stability or behavior in response to standard procedures. - The applicant’s software platform integration and dedicated personnel to this aspect of the project are major assets. Significant thought has been given to the breadth and depth of the information management program. - While the management plan appeared reasonable, a reviewer suggested that all quality control operations should report directly to the Program Director rather than through the Laboratory Director. - The sustainability plan lacked details on required budget and revenues from orders, leaving reviewers uncertain of the extent to which the repository could become self-sustaining. Feasibility - The applicant’s heavy reliance on off site collaborators for operational planning, hiPSC expertise, and major staff hiring /training poses a significant risk to overall program. - While the collaborations are admirable, the large number of moving parts within this proposal may prove challenging to coordinate, especially as information management will rely on combining existing systems with newly developed ones. - Reviewers appreciated the innovative plan to take small banks forward initially with subsequent expansion of popular lines on demand, a method that should drive down initial costs and labor requirements. Program Director (PD) and Team - The Program Director is an established scientist entrepreneur with general experience in biobanking, but limited experience with iPSCs or running larger scale banking operations. - Collaborators at the subcontracted organizations provide a useful set of talents that are well suited to the tasks proposed. - The majority of laboratory expertise relating to hiPSC resides with a key collaborator who is located off site from the main laboratory. Given this crucial role, reviewers recommended that this individual’s percent effort for Years 1 and 2 be increased. -The positions of Laboratory Director and Quality Control Manager are key to the success of this project, but are yet to be named. Appropriateness of Budget - The proposed budgets for personnel, equipment and supplies are generally reasonable, but some reviewers felt the costs related whole-genome/epigenetic data acquisition were significantly underestimated. - Reviewers considered the budget for mycoplasma testing to be excessive and suggested that alternative testing methods be explored.