CMC

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

When a client's project's timeline was in jeopardy due to a lack of communication between the vendor and the client, DHC’s project management services were added to support overall project governance.

Precision is key, especially in a(n international) tech transfer. DHC provided Person in Plant support in managing dose preparation training of clinical sites for this client's flagship product.

This case study considers DHC’s proprietary quantitative modeling software, and how its adaptive approach provides clients with highly-customized probability-weighted forecasting.

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.

Nonsensical, counterintuitive, or otherwise "off" data is likely to signal a problem. A team with remediation experience, like Dark Horse's, can perform a failure analysis to identify and remediate the source(s) of the issue. 

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.

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.

Take a look at how sometimes a quantitative review can yield the excellent news that your program is in better shape than you realized.

This client requested assistance in codifying their manufacturing process in preparation for an international technology transfer and global regulatory filings.

This case study exemplifies proactive project management of cross-functional program details; specifically, those that occur at the interface of CMC and clinical operations.

This client requested design and outsourcing of pre/nonclinical efficacy and safety studies to demonstrate comparability of a changing clinical stage manufacturing process.

This client required CDMO selection assistance (a key element of manufacturing support) to provide a clear path for their non/preclinical stage gene therapy program.

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.

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

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 example demonstrates that it is rarely too late to ask for a expert overview. A thorough review of the facility design documents yielded many design improvements that were possible even at this late stage, which provided significant operational improvements to benefit the facility long-term.

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.

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’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).

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.

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.

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.

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.

Assistance with the design/use of studies demonstrating biological relevance of a lead candidate in vitro potency assay demonstrates analytical and non/preclinical core compentencies.

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.