USA – MASSACHUSETTS Trends and Developments Contributed by: Patti Melick, Toby Bannon, Katherine Rubino and Kelsey Loomis, Wiggin and Dana LLP
increasingly demonstrate meaningful patient outcomes, the business and legal side of the equation has become a critical contributor to these scientific advancements. The appeal of cell and gene therapies is broad, with 2025 bringing a variety of deals, including notable deals such as AstraZeneca in its licence agreement with Algen Biotechnologies and its acquisition of EsoBiotec; Kite Pharma in its licence agreement with Pregene; and Eli Lilly in its collaborations with MeiraGTx and Seamless Therapeutics and its acquisitions of Verve Therapeutics and Adverum Biotechnologies. Additionally, there were notable terminations of deals in 2025, including Genen- tech’s agreement with Adaptive Biotechnologies, Kite Pharma’s agreement with Shoreline Biosciences, and Carisma Therapeutics’ unwinding from its Moderna relationship and failed reverse merger with OrthoCel- lix. Still, as the cell and gene therapy space continues to calibrate its momentum and optimism, the focus on deal terms and risk allocation has become increasingly important. The regulatory landscape has also experienced some calibration and innovators must remain mindful of how updated guidance may translate into diligence and deal making realities. Recent FDA guidance has promoted more flexible chemistry, manufacturing, and controls approaches with an aim to accelerate development timelines for cell and gene therapy play- ers. It remains to be seen if this approach will relieve some pressure from the development process. In the meantime, innovators can still expect to see challeng- es and, if those innovators become licensors, to see robust diligence into their manufacturing know-how, processes and capabilities, often including related representations, warranties, indemnities, and technol- ogy transfer provisions. Investor and strategic partner expectations are also evolving in parallel. While single-asset and single-indi- cation deals remain attractive, parties are often also seen evaluating the durability and utility of underly- ing platforms. High-profile collaborations such as the decade-plus evolving relationship between Vertex and CRISPR Therapeutics [1 , 2 ] (which notably gave rise to CASGEVY) serve as an example of how parties are considering broad relationship to structure the division of labour, governance, cost-sharing, and profit alloca- tion to drive continued innovation. These structures
reflect the recognition of the evolution of risk during – and perhaps even more notably in the cell and gene therapy space than other technologies – after devel- opment. Reimbursement and patient access considerations are also an important focus during deal negotiations. Products that are potentially curative, single- or minimal-administration therapies introduce payment models and logistical challenges that often differ from other therapies, thus impacting the manner of think- ing through royalties, milestones and diligence obliga- tions. The challenges encountered by bluebird bio [1 , 2 ] (now Genetix Biotherapeutics) commercialising Lyf- genia (including scrutiny on high price and long treat- ment process leading to slow uptake) underscores how regulatory approval does not automatically trans- late into sustainable market success, a distinction that is increasingly reflected in transaction modeling and, as in a related manner, influences agreement terms including diligence obligations (including post- approval diligence) and milestone structures. The Massachusetts ecosystem benefits from strong alignment between policy support, capital formation, and operational infrastructure. Organisations such as the Massachusetts Life Sciences Center continue to invest in foundational capacity, while industry groups like MassBio have elevated manufacturing scale- up as a priority. With well over 100 companies and research institutions in Massachusetts working in the cell and gene therapy space, including the headquar- ters or major hubs of powerhouses like bluebird bio (now Genetix Biotherapeutics), Vertex, Sarepta, Editas and more, Massachusetts demonstrates how scien- tific, financial, and political support can drive innova- tion and dealmaking in this space. In sum, these dynamics underscore that success in cell and gene therapy is no longer defined solely by scientific breakthrough, but by the ability to thought- fully structure the commercial and legal frameworks that bring those breakthroughs to patients, resulting in success not only for those patients but for industry partners as well. As manufacturing complexity, reim- bursement models, and platform durability continue to shape investment and collaboration decisions, deal architecture is becoming an essential tool for man-
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