Operation of a Self-Sustaining California-Based Biobank of Cell Lines to Support Global Research into Genetically Complex Diseases
In response to CIRM RFA 12-04, [REDACTED] proposes to grow a California-based self-sustaining biobank of human induced pluripotent stem cell (hiPSC) lines for use in the development of new therapies. We have 27 years of experience in biorepository science and services, and have the expertise and global infrastructure to expand the supply and distribute the cell lines to international research sites. [REDACTED] will provide LN2 dry shippers and arrange for the transfer of each cell line (proposed at 100 vials) from the Deriving Laboratory (RFA 12-03) to the repository. If needed, we will train staff in the use of the shippers according to SOPs; the vials will be received and stored in the biobank according to SOPs. [REDACTED] will provide online access and researchers will be able to search the database, place orders, and submit payment. The information required from those seeking to use CIRM materials, the fee schedule, and other variables in the operation and management of the biobank, will be decided in collaboration with CIRM, the Deriver, and the Tissue Collector (RFA 12-02). As inventory is depleted, [REDACTED] will send the cell line to [REDACTED] for expansion and replenishment of the inventory. This will include those derived under CIRM 12-03 as well as from California researchers. The quality of materials in the biobank will be documented and available. The cells will be distributed in compliance with all regulations.
Statement of Benefit to California:
Our Biobanking facility was established in California in 2002 with commercial resources, demonstrating our commitment to building our business in California apart from State funding. We are already part of the business and biotech community and will not be displacing another biobanking/repository business in California. By creating a biobank of cell lines that are derived and expanded by California-based laboratories, the biobank will preserve and create additional high quality, skilled biotech laboratory jobs in California. In addition, [REDACTED] supports the entire developmental pipeline of new therapies and our goal is to provide excellent, high value service to the CIRM biobank users and correspondingly grow our California repository business along with the California pharmaceutical companies that are developing cell-based therapies. We are on the forefront of supporting the development of cell-based therapies at cryogenic temperatures and the expansion of this arena of our business will provide new jobs well beyond the laboratory for residents of California.
The goal of this proposal is to expand the capabilities of an existing biological repository towards establishing a self-sustaining, California-based biobank of human induced pluripotent cell (hiPSC) lines from 3000 tissue donors. The applicant seeks to build upon its current services and infrastructure through close collaboration with Tissue Collectors, the hiPSC Deriver, and a key subcontractor, who will be responsible for line expansion and inventory replenishment. Major project activities include generation of master cell and distribution banks, quality control testing, and modification of an existing tracking system to enable users to order cell lines and access donor-associated information. Quality of the Proposed Repository - Due to the limited information provided, reviewers were not convinced that the proposed management and sustainability plans would prove sufficient. - The applicant’s existing facility is well suited for handling, storing and distributing hiPSC lines. - It is unclear to what extent hiPSC user-established data or information could be readily incorporated back into the Repository. Feasibility - The applicant relies heavily on the ability of the Deriver to provide a significant number of lines to the repository for immediate distribution. Given the defined financial constraints on the Deriver, reviewers found this strategy to be extremely risky, if not fundamentally flawed. - Feasibility is further weakened by the critical reliance on a subcontractor whose documented experience with iPSC is minimal, and whose cell line expansion plans were poorly described. Program Director (PD) and Team - It is not clear whether the 15% commitment and monthly travel plans of the off-site Program Director would prove sufficient to successfully manage this program. - Limited details were provided about the staff and resources of the subcontracting team, or their ability to expand and test hiPSC at the capacity that would be required for this endeavor to be successful. - The program director has a broad background in cell biology and a wealth of leadership experience in industry, although direct experience with pluripotent stem cells appears lacking. Appropriateness of Budget - The budget allocated to the subcontractor for cell line expansion is poorly justified, especially given the reliance on the Deriver to provide a large number of initial lines. - Budgetary allocations for storage and distribution activities appears reasonable, but details are lacking on the costs anticipated for quality assessments.