Clinical Trials Insight


short supply at some points.” This fragility is exacerbated by the fact that CGTs aren’t manufactured once but repeatedly through the trial. To mitigate such scenarios, supply chain managers now proactively draw contingency plans to ensure sourcing from alternative vendors, if required. That can be harder to achieve for reagents when compared with lab consumables or equipment. “While there may be equivalent reagents, manufacturers often design their processes with a preferred reagent,” says Rochlin. If a switch is later desired, the alternatives would need to be qualified by regulatory bodies, lengthening clinical trial timelines, and they may not behave the same. “Thinking that through early is extremely helpful in planning clinical processes,” says Rochlin. Too often, however, the most precious resources are the patients themselves. Most CGTs under development are autologous. Even allogeneic therapies, Rochlin explains, are typically manufactured for each patient in early clinical trials. Moreover, the patient populations for CGT trials are smaller. Since these therapies often target patients suffering from advanced-stage cancers or rare diseases, patients’ cells can be in short supply. For autologous treatments, samples are transported from the patient to the manufacturing site, and the therapies are delivered back to the patient. CGT manufacturers must be agile enough to respond to the unpredictable availability of suitable patients. Once identified, patients need to undergo conditioning regimens or pre-treatments before the therapy can be administered. “The patient’s pre-treatments and the product release criteria of the therapy need to be aligned for delivery on the appropriate day,” says Blair McNeill, head of cell therapy for pharmaceutical company Sumitomo Pharma America. For patients with life-debilitating indications, it can be a matter of life and death. The return journey is highly susceptible to lags at multiple points. For example, a delay in apheresis at the hospital can propagate down the chain. “Delivering it to the patient on time requires a very close coordination of logistics and manufacturing,” says McNeill.


Just-in-time delivery


Cells are viable for 24 to 48 hours from collection, and cell therapies derived from them, in turn, have a shelf life of 24 to 96 hours. Consequently, for autologous therapies, the windows for apheresis and therapy infusion are exceedingly short. Gene therapies and allogeneic cell therapies can be frozen, but are often not during early-stage clinical trials and must be administered within a similar time frame. The short timeline necessitates the adoption of just-in-time (JIT) delivery. JIT helps manufacturers across industries save inventory costs by producing


www.worldpharmaceuticals.net


The importance of building a strong intellectual property portfolio for cell and gene therapy companies


Cell and gene therapy (CGT) companies are working at the frontier of modern medicine, aiming to deliver transformative treatments for patients with life-threatening conditions. However, turning breakthrough technologies into marketable therapies requires more than innovation alone, as a carefully planned intellectual property (IP) strategy is required. From early-stage platform development through to clinical trials, each phase presents unique opportunities to build and strengthen a robust IP portfolio. The ability for a CGT start-up to attract investment is dependent on the strength and exclusivity of its IP portfolio, according to one of Mathys & Squire’s speakers at the London Biotechnology Show 2025. They also mentioned that the product is rarely in its final form at launch, causing investors to look at IP as a sign of long-term value and competitive advantage. Therefore, a strong IP allows companies to secure multiple rounds of fundraising over several years, which is critical in a field with long development timelines and high capital needs. These start-ups are encouraged to actively grow their IP portfolios by filing patents for platform modifications and delivery systems, offering 20 years of protection from competitors. Early collaboration with patent attorneys is vital to not only help write and submit patents, but to advise on the strategic evolution of the portfolio. This guidance ensures the IP supports both scientific advancement and commercial positioning, further attracting investors while providing scientific success in the real world. As development progresses, CGT companies must improve their platforms to create a strong asset ready to undergo clinical trials. Additionally, preclinical research is crucial as it identifies optimal dose levels and safety profiles for targeted indications. Once identified, companies must protect this information with IP filings to strengthen the company’s IP portfolio, opening doors for licensing deals, partnerships and continued investment. When entering clinical stages, the company’s focus shifts to protecting areas


particularly important in CGT, such as clinically relevant formulations, administration methods, and patient-specific applications. At this stage, new IP filings can secure long- term exclusivity and cover how the product will be marketed once approved. Strong, well-targeted IP can raise a company’s value to buyers since it gives them control over a product that’s close to being ready for the market. For CGT companies, IP is not just protection, it is a strategic fortress which provides


opportunities for business growth while enabling innovation and fuelling funding. Building a strong IP portfolio can dramatically elevate a company’s valuation and position in the


market, turning early-stage science into a high-value market-ready asset. By George El-Helou, MSc, pharma analyst, GlobalData


goods only when needed. The extremely low viability and astronomical costs of CGTs, with a single dose usually costing millions of dollars, make this benefit highly pronounced. But it also amplifies the challenges of CGT supply chains, such as leaving them more exposed to material shortages or delays.


The major hurdle for the timely delivery of these therapies is their low tolerance for temperature excursions. Deviations from the ideal temperature at any point during storage, processing, or transport can severely lower their effectiveness. “But if it’s a product that can be cryogenically frozen, some of those logistics become easier,” says McNeill. Cryogenic shippers can maintain temperatures as low as -150°C for up to ten days and feature real- time temperature monitoring to ensure the product stays within the acceptable range at all times. The extended shelf-life, though, often comes at the cost of viability as the toxicity from cryoprotective agents further diminishes the low cell count and viability. Advances in cryopreservation packaging could retain sample integrity longer. To this end, McNeill adds, “there


65


Page 1  |  Page 2  |  Page 3  |  Page 4  |  Page 5  |  Page 6  |  Page 7  |  Page 8  |  Page 9  |  Page 10  |  Page 11  |  Page 12  |  Page 13  |  Page 14  |  Page 15  |  Page 16  |  Page 17  |  Page 18  |  Page 19  |  Page 20  |  Page 21  |  Page 22  |  Page 23  |  Page 24  |  Page 25  |  Page 26  |  Page 27  |  Page 28  |  Page 29  |  Page 30  |  Page 31  |  Page 32  |  Page 33  |  Page 34  |  Page 35  |  Page 36  |  Page 37  |  Page 38  |  Page 39  |  Page 40  |  Page 41  |  Page 42  |  Page 43  |  Page 44  |  Page 45  |  Page 46  |  Page 47  |  Page 48  |  Page 49  |  Page 50  |  Page 51  |  Page 52  |  Page 53  |  Page 54  |  Page 55  |  Page 56  |  Page 57  |  Page 58  |  Page 59  |  Page 60  |  Page 61  |  Page 62  |  Page 63  |  Page 64  |  Page 65  |  Page 66  |  Page 67  |  Page 68  |  Page 69  |  Page 70  |  Page 71  |  Page 72  |  Page 73  |  Page 74  |  Page 75  |  Page 76  |  Page 77  |  Page 78  |  Page 79  |  Page 80  |  Page 81  |  Page 82  |  Page 83  |  Page 84  |  Page 85