In recent years cell and gene therapies have taken the main stage in biotech as they promise to address a broad array of indications and patient populations that have historically had significantly unmet needs. Even in the face of an investment downturn, the cell and gene therapy industry saw $11.7B in investments in 2023. Building on these current trends and wins, McKinsey has highlighted the cell and gene therapy industry as a key opportunity that will continue to draw strong investor interest.
With over 2500 existing developers worldwide, and more seeking to get in on this sector, stakeholders and investors are looking for ways to navigate and succeed in this promising yet competitive sector. The promise of these treatments for both patients and investors continuously drives efforts in this space, but success in bringing cell and gene therapies to market hinges on informed portfolio management decisions.
Asset assessment for portfolio management
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It is becoming increasingly more difficult to stand out in the crowded marketplace of the cell and gene therapy sector, with that choosing the right candidates to pursue commercially is critical. The latest industry reports say that biotechs that are pursuing opportunities in the cell and gene sector, placing an emphasis on R&D initiatives, and companies with a robust clinical pipeline are well positioned to garner interest from investors.
With 1,804 ongoing clinical trials, and this number continuously rising, strategic portfolio assessments and management is critical to success. Understanding the market potential and patient demand for a product is the first step. Analyzing disease prevalence, the competitive landscape and where corresponding candidates stand in the development process allows stakeholders to allocate resources effectively and prioritize the most promising therapies. A diverse portfolio is essential, as this not only mitigates risk but maximizes opportunities for success while addressing a wide range of indications. Evaluating the existing target indications, asset modalities, and technologies in the pipeline can help assess how diversified the portfolio is.
When determining which assets to pursue, strong clinical data currently serves as one of the most powerful indicators for success in a portfolio of products. Further assessing the scalability and manufacturing feasibility of each candidate can help gain clearer insights required for strategic decision making. Consider the complexity of the manufacturing process, supply chain requirements of each, along with the required regulatory pathway to market.
Opportunities for growth and impact in cell and gene
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As the number of cell and gene therapy products in the pipeline continues to rise, there’s a growing recognition of the need to address larger patient populations and account for economic and societal considerations. Today, low- and middle-income patients still face significant health disparities and unmet medical needs. To address this, the industry should expand the view of unmet needs to prioritize those diseases that have a significant societal impact, even if they are not as financially attractive initially. Redirecting R&D and investment efforts towards the diseases most prevalent for these patient populations can help move towards more equitable care and allow companies to have a considerable impact on society, while still finding success in the sector.
When conducting a portfolio analysis, key considerations include the scalability and manufacturing feasibility, complexity of manufacturing processes and supply chain requirements. Assessing these factors early in the development process can save time and money by preventing costly delays and allows for strategic resource allocation towards other promising programs. As we look to the future, industry leaders should continue to invest in emerging technologies like gene editing, allogeneic cell therapy, and non-viral delivery methods. By continuously refining the pipeline based on patient demand and the most cutting-edge technologies, biotechs can remain at the forefront of cell and gene therapy development.
In the wake of the financial downturn the industry is seeing a correction with increased M&A activity along with rapid changes from a highly supportive regulatory environment and rebounding investor interest. With this, leveraging real-time data, risk-based decision making, and agile portfolio management practices will be key to success. In addition, embracing flexibility will allow companies to adapt swiftly to changes in the market and capitalize on emerging opportunities.
A holistic approach to portfolio management
While analyzing the program portfolio at a product level is important, aligning R&D activities and corporate strategy is critical for long term success. Focusing efforts on opportunities with high market potential, patient impact, while considering the company’s resources and capabilities can help ensure strategic alignment for the company’s larger goals. A clear understanding of the existing infrastructure and in-house manufacturing expertise helps to identify areas of strength to drive decision making. Where in-house capabilities are lacking, partnering with external stakeholders is critical. In the growing field of cell and gene therapy, expertise and specialized skills are widely dispersed, making resource and knowledge sharing essential to moving the industry forward. Forming diverse teams and partnerships that complement existing capabilities can facilitate collective problem solving to shorten timelines, increase efficiency, and ultimately improve patient outcomes.
As a critical part of asset management, financial implications should be analyzed through an in-depth cost-benefit analysis that considers development expenses, potential revenue streams, and return on investment. Forecasting models that account for several market scenarios, investment landscapes, and regulatory outcomes can help project the performance of cell and gene therapy products. With a thorough financial analysis, assessment of unique competitive advantages, and in-depth understanding of the pipeline, companies can position themselves to overcome challenges rapidly and maximize opportunities for success in bringing lifesaving therapies to market.
As cell and gene therapies continue to make headlines as a key opportunity for growth, success in this field will require both innovative tech as well as strategic foresight. Flexible portfolio management and an adaptable organization will allow companies to capitalize on exciting opportunities while fostering resilience and growth even in the face of market changes. Although highly competitive, the cell and gene therapy industry offers the opportunity for financial success as well as the chance to make an incredible difference in patient lives. With the right resources and strategy, companies can define success by driving innovation, addressing significant unmet medical needs, and ultimately improving patient outcomes.
Photo: Topp_Yimgrimm, Getty Images
Anshul Mangal is a biotech entrepreneur, experienced executive, board member, philanthropist and attorney. He is the President of Project Farma ("PF") and Precision for Medicine's Manufacturing Solutions. He is also a board member at the Alliance for Regenerative Medicines, Alliance for mRNA Medicines and IQHQ. Anshul founded and steered PF to become a prominent global consultancy specializing in biologics and advanced therapy engineering with 200+ professionals. Under Anshul's guidance, PF pioneered the industrialization of groundbreaking therapeutics, including some of the earliest commercially approved cell and gene therapies, radioligand treatments, RNA-based therapies, and cutting-edge biologics. Anshul's commitment to advancing next-generation medicines extends past PF to various boards and philanthropic organizations, including IQHQ, Alliance for Regenerative Medicines, Alliance for mRNA Medicines, Institute for Life Changing Medicines and several patient-advocacy organizations. Before his tenure at PF, Anshul was an attorney and founded several digital agencies.
John Khoury is an Executive Vice President at Project Farma. He is a 20+ year veteran and leader in the biotech/pharmaceutical industry. As a member of the Project Farma and Precision for Medicine leadership teams, John has spearheaded the firm’s growth in the gene and cell therapy space. He has led and provided strategy on key partner initiatives including make vs. buy analysis, site and vendor selection, tech transfer execution strategies, and facility start-ups. In addition to his experience in advanced therapies, John has extensive experience with small and large molecules including biologics and biosimilars. Over the past four years, he has led facility builds totaling over $500MM. During this time, he has worked with small and large biotech and pharmaceutical companies including gene and cell therapy startups and CMOs/CROs.
