Regulatory Affairs

Development of novel, potentially groundbreaking manufacturing equipment and consumables for cell therapies is a complicated dance. It is a delicate balance of conflicting development and commercial priorities against customer requirements and the need to get to market quickly. In this case a client came to DHC concerned with the progress of their internal development program and worried about the maturity of their integrated bioreactor system for the manufacture of CAR-T and next generation cell therapies.

Experienced regulatory writers at DHC can provide a range of support from full authorship of regulatory submissions to gap analysis and editing of existing documents. A simple regulatory writing request can occasionally uncover unexpected gaps. In this example, as part of meeting package development and remediation of another consultancy’s work, Dark Horse experts identified a path to accomplish the client’s goals in a way that met the crucial initial timeline constraints and set them up for clarity in how to achieve their ultimate regulatory goals.

Development of a CAR-T cell therapy licensed from an academic institution required support in developing a pre-IND and IND-enabling nonclinical strategy.

An emergent situation calls for simultaneous problem-solving on multiple fronts.
This client approached DHC for interim leadership support as they faced a critical clinical supply shortage—driven by manufacturing and comparability challenges—for their clinical stage lipid nanoparticle (LNP)-based drug product.

Our clients may experience a variety of situations that lead to a leadership gap within their organizations. Senior staff at Dark Horse possess years of leadership experience and can help fill the gap and keep operations moving forward. With senior staff available for nearly any technical or strategic need (and the ability for each of them to call upon 40+ technical SMEs for additional support), this has become a common request for DHC.

Requests for a roadmap to IND are common queries at DHC. This case study combines the experiences of a range of client projects to extrapolate the elements of a typical roadmap. In this type of engagement, DHC’s cross-functional expertise provides clients with an integrated understanding of the major activities, timelines, costs and value inflection points on the path to IND. Project deliverables are valuable both for internal planning and for fundraising activities.

DHC was asked by an investor client to support due diligence on two cutting-edge CGT assets that the investor was considering combining in order to spin out a new cell therapy company. DHC reviewed quality, CMC, and regulatory information relating to both assets, performed onsite evaluations of each, evaluated manufacturing contract terms, and reviewed the new entity’s planned timelines, budgets, and milestones.

DHC was engaged to provide strategic guidance in building a “hotel”-style manufacturing facility, which requires the ability to provide flexible GMP clean room suites for direct use by a wide range of early-stage manufacturing clients.

An academic organization was planning to retrofit a U.S.-based facility to meet their needs for both HCT/P manufacturing as well as multi-product early-stage cell therapies.

In this example, an international tools and tech company requested a market analysis of the gene-editing and advanced therapies space as well as a facility buildout plan to manufacture a critical component for gene therapy applications.

A pure-play gene therapy client whose product was just being spun out of academia brought DHC in early to provide long-term guidance, conceptualizing and building a dev process that would stand up to the challenges of future trial phases and into commercial production.

This case study follows the experience of a client who is new to CGT (though otherwise practiced in late stage processes) and requires CRL remediation to prepare for BLA resubmission.

Combine BLA rejections and late-stage product delays with the rising proof of comparability burden as therapies approach a regulatory hurdle, and an expert comparability review becomes a near-necessity.

Dark Horse offers varying levels of analytical support, ranging from high-level strategic oversight to detailed tactical operational support. In this case, the client required a broad scope of guidance while preparing to file for licensure.

When a company develops a product for use in a cell and gene therapy process, one of the steps is to get that product (complete with any consumables for use) approved. This client asked for assistance in the associated regulatory process.

Clinical development of CGT products is increasingly an international endeavor, requiring an integrated global regulatory strategy.

This study highlights a custom piece of manufacturing equipment for a cell therapy client. In addition to engineering oversight, DHC also provided regulatory and PM support.

DHC’s support for this client's gene-modified cell therapy platform demonstrated comparability of the improved manufacturing process (thus streamlining associated filings with global regulators).