Cynata Therapeutics’ parallel approaches to market
08 October 2019
In 2014, Cynata Therapeutics commenced technology transfer and product development, and simultaneously commenced consultation with regulatory authorities regarding the approval pathways for its cellular therapy products.
Despite a lack of guidance because the field was emerging, Cynata Therapeutics Limited (Cynata) was able to secure regulatory approval for a world-first clinical trial, based on a sound scientific rationale. The clinical trial was completed and showed remarkable outcomes for the patients enrolled in the clinical trial. Throughout this period, Cynata’s product development generated new intellectual property (IP), which is covered by a portfolio of patent applications wholly-owned by Cynata, in addition to the intellectual property in-licensed in 2014. In short, Cynata has demonstrated significant efficiency in successfully co-developing it's product, the IP covering it, and it's regulatory pathway to market.
Challenges and opportunities
In early 2014, when the Cymerus development program began, the lack of precedents and limited guidelines made obtaining regulatory approval for Cymerus allogeneic iPSC-derived mesenchymal stem cells (MSCs) challenging. The challenges included:
- no clinical grade allogeneic iPSC line (starting material) existed worldwide
- no iPSC-derived product had ever been administered to a human subject
- no clinical trial involving allogeneic iPSC-derived cells had received regulatory clearance anywhere in the world
- no other systemic clinical use of an iPSC-derived product had even been proposed (as far as was known)
- many regulatory guidelines have limited relevance to cell-based therapies
- conventional pharmacokinetic assessments cannot be performed
- standard toxicology studies may not be logical/ feasible
- laboratories that can establish specialised animal models required for cell-based therapies often cannot comply with GLP (and vice versa)
- may not be possible to administer human cells to animals, in particular large animals
- cell-based therapies may have potential risks that do not apply to small molecules/ antibodies
However, the absence of relevant precedents and guidelines represents an opportunity as well as a challenge, because one is not constrained by prejudice expressed as “that’s how it has always been done”, and new and unconventional approaches may be acceptable.
The impact on Cynata's IP
Cynata was provided the opportunity to develop its patent portfolio in concert with its applications for regulatory approval and clinical trials. In fact, since 2014 when Cynata in-licensed its initial iPSC/ MSC technology, Cynata has developed its own technology and application of that technology such that Cynata now owns five International PCT applications, two of which have recently entered the national/ regional phase, and has another provisional application pending.
The consequence of these parallel approaches is that Cynata is now well placed in the market with both clinical trials underway and IP underpinning the technology used in those clinical trials, ultimately resulting in commercial interest from third parties.
'Like many biotech companies, Cynata was established specifically to commercialise IP licensed from an academic institution, so IP has always underpinned all of the company’s activities. We have subsequently built value by filing a number of wholly-owned patent applications to completement the in-licensed IP, and we believe this has placed the company in a very strong commercial position'
Dr Kilian Kelly, Chief Operating Officer, Cynata Therapeutics
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