Cell One has extensive experience of being engaged to assist companies to assess both the CGT industry at large (including current market snapshot analysis as well as providing forecasts of trends that will impact the industry) as well as specific CGT companies, including both CGT Companies and CGT Service Providers. The following is a sample of engagements that Cell One has completed:

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Validation of Internal Systems and Processes for a North American Government-Funded CGT CDMO

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Challenge: The client has recently completed construction and validation of a new GMP C> manufacturing facility that was built within an existing office building. In preparation for both the local regulatory agency and FDA GMP audit the client was looking for an independent expert to undertake a GMP audit of the facility.

Cell One Actions: Cell One undertook a full GMP audit assessing the facilities compliance with local, FDA and EMA GMP requirements. At the end of the audit Cell One delivered a full audit report that included: detailed report on non-conformities with risk assessments; corrective actions; and overall opinion.

Outcome for Client: The audit report provided a validation of the internal systems and processes were in accordance with the GMP requirements of the local regulator, FDA and EMA and supported the client’s preparation for a successful audit.

 

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Leading European Private Equity Firm Looking for Expansion of Domain Knowledge

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Challenge: The client already had significant understanding and investments in the small and large molecule CDMO sector and needed to develop a deeper understanding of the global CGT CDMO, Viral Vector, and tools market for a USD $1B roll-up investment.

Cell One Actions: Cell One completed an in-depth market analysis across CGT, CDMO, viral vector and tools markets, including providing a high-level identification of potential targets, market and company challenges, risks, and opportunities.

Outcome for Client: The client built an understanding of global CGT CDMO market and tools providers, including vector manufacturers, to enable planning of future investment strategy.

 

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Leading US Private Equity Firm Looking for Due Diligence support for a CGT CDMO Acquisition

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Challenge: The client had agreed terms to acquire a global CGT CDMO, with operations in US, Japan, and Europe with an acquisition cost of$500M. As part of its due diligence of the acquisition, the client sought out Cell One as an expert in CGT development and CDMO market to evaluate the business and market.

Cell One Actions: Cell One completed due diligence on target acquisition focusing on facilities, equipment, quality, business development and analysis of prospects.

Outcome for Client: The completed due diligence formed part of the total due diligence process and provided information in a timely manner to support Go/No-Go decision.

 

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Leading US Private Equity Firm Looking for Due Diligence support for a CGT Tools Market Acquisition

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Challenge: The client was considering investment in new cold chain containers for CGT products and was looking for expert advice and evaluation of the opportunity.

Cell One Actions: Cell One completed a detailed evaluation of the market, company, competitors, and technology and provided the client with a detailed report highlighting the market opportunity, the competitiveness, and uniqueness of the technology as well as assessment of financial feasibility and potential risks of an investment.

Outcome for Client: The final report provided expert analysis of an investment which was used to support discussions at the firm’s Investment Committee.

 

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Address Challenges with Manufacturing Strategy

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Challenge: The client was encountering issues with the manufacturing strategy for gene therapy products.

Cell One Actions: Cell One undertook a deep dive into product, process development, assays, analytics, assessment of facility and facility constraints leading to development of manufacturing strategies.

Outcome for Client: The new manufacturing process recommended by Cell One was adopted and successfully implemented.

 

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Identification of Approach to Manufacturing for CGT Developer

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Challenge: The client was focusing on commencing clinical trials in the US for their gene therapy products. The current in-house manufacturing of plasmid, vectors & product was not approved by the FDA.

Cell One Actions: Cell One completed a full assessment of the technology, program and local clinical trial results and provided expert guidance on product deployment strategy including action to achieve compliance with FDA regulations.

Outcome for Client: Report findings and recommendations resulted in board recommending evaluation and assessment of partnership models.

 

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Assist CGT Developer with Response to FDA Complete Response Letter

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Challenge: The client submitted IND to FDA and received a CRL with the client having to submit a reply within mandated timelines.

Cell One Actions: Cell One team contributed to the development of response strategy, and worked with clients and other external consultants to complete response to Complete Response Letter

Outcome for Client: Client was able to provide its response to the FDA’s Complete Response Letter for its IND within the FDA-provided timelines and received IND approval for the FDA.

 

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Evaluate the Opportunity for a CGT Developer to expand into GLP Services, including Funding Sources

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Challenge: A CGT developer was searching for non-dilutive funding sources and wanted support an evaluation and advice on a strategy to expand into GLP.

Cell One Actions: Cell One provided detailed guidance on regulatory requirements, commercial/operational advice, market opportunities, and financial feasibility for an investment in a GLP facility focusing on: i) gene sequencing; ii) diagnostics; and iii) personal medicine.

Outcome for Client: The finalized report was integrated into the final business plan that was presented to the board for Go/No-Go decision, and the business plan recommended by Cell One was ultimately implemented.

 

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Address Starting Material Supply Challenges

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Challenge: The starting material for the company’s platform was human tissue that had to be sourced in a regulatory compliant process.

Cell One Actions: Cell One partnered with internal stakeholders to design a supply chain strategy that met global regulatory requirements for sourcing of starting materials, human tissue sourced.

Outcome for Client: Final report used as foundation for implemented supply chain that has achieved regulatory compliance.

 

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Research and Recommendation of Business Plan to Develop CDMO Service Offerings

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Challenge: The client had an existing strong business in cell collection services and saw expansion into CGT CDMO services as the natural evolution of the company.

Cell One Actions: Cell One developed a detailed business plan for expansion of services into global CGT CDMO.

Outcome for Client: The business plan was used to support the Go/No-Go investment analysis and board decision for expansion in CGT CDMO services. The business plan was ultimately used as the foundation to implement the client’s expansion into CGT CDMO services.

 

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Research and Recommendation of Business Plan to Expand CDMO Capabilities to External Clients

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Challenge: Evaluate feasibility and competency of using in-house C> manufacturing resources to enter the global C> CDMO market.

Cell One Actions: Cell One developed a detailed business plan evaluating the financial and operational feasibility of using existing in-house C> manufacturing assets and resources to enter the global CGT CDMO market, as well as providing extensive training to client teams on operating and CGT CDMO, and difference between expectation of in-house projects and client projects.

Outcome for Client: Gained in-depth understanding of client operations and created business plan used in discussions with board and investors to support Go/No-Go decisions.

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