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2026 Biotech Global Forum
Overview
With over 20 speakers, GenScript’s fourth Biotech Global Forum brought together pioneers and leaders in cell and gene therapy for engaging presentations and lively discussions on a wide range of topics vital to the advancement of these innovative drugs. The event provided a platform for patient advocates facilitating access to novel therapies, innovators charting the course of future therapies, industry leaders and investors with deep insight into the sector, and supply-chain and manufacturing experts working on expanding and expediting the delivery of high-quality therapies to patients.
A recurring theme throughout the discussions is the need for effective communication about the novelty of highly personalized immunotherapies, such as CAR-T cells, and their capacity to provide cures. Increasing awareness among the general public and payers alike about the mechanisms of action and efficacy of these innovative therapies is of utmost urgency. Another prevailing theme discussed was the need for manufacturing optimization, standardization, and process automation to ensure high quality and consistency in producing cellular therapies. The expanding opportunities for indications besides cancer, including autoimmune diseases, are expected to create heavy demands for these specialized therapies. Nevertheless, the consensus among experts is that supply chain and manufacturing capabilities are not yet equipped to meet these new demands.
By addressing manufacturing challenges, particularly the quality of starting biomaterials and facing out manual processes, among others, experts agreed that patients’ access to effective therapies can be significantly improved. Overall, the ultimate goal is reducing cost while facilitating the distribution of these therapies with the potential for point-of-care cell therapies in the future.
In a heartfelt presentation, Tom Whitehead shared the inspiring journey of his daughter Emily, who became a pioneer in pediatric cancer treatment through innovative immunotherapy. Whitehead, the president and co-founder of the Emily Whitehead Foundation, recounted the family's harrowing experience after Emily was diagnosed with acute lymphoblastic leukemia shortly after her fifth birthday.
Initially, Emily's prognosis was grim, with doctors suggesting hospice care after multiple relapses. However, a breakthrough came when the Children's Hospital of Philadelphia offered her a chance to participate in a groundbreaking T-cell therapy trial, led by Dr. Stephan Grupp. This experimental treatment, which had never been tried on a child, ultimately resulted in Emily achieving complete remission just 23 days after her treatment.
Whitehead emphasized the importance of this treatment not only for Emily but for the future of cancer research, stating that her case has changed the landscape of pediatric cancer therapy. He highlighted the emotional rollercoaster the family endured, including a near-fatal reaction to the treatment that left Emily in a coma for 14 days. Remarkably, she woke up on her seventh birthday and has remained cancer-free for nearly 13 years.
The Whitehead family's story has garnered significant media attention, including a feature in The New York Times and a short film directed by Oscar-winner Ross Kauffman. Through the Emily Whitehead Foundation, they aim to provide hope and support to families facing similar challenges, advocating for access to advanced treatments and raising awareness about the potential of personalized cell immunotherapy.
Tom Whitehead concluded his presentation by expressing gratitude for the advancements in cancer research and the ongoing efforts of medical professionals, urging continued support for funding and awareness initiatives. The foundation has already assisted families in over 50 countries, demonstrating the global impact of Emily's story and the importance of innovative cancer treatments.
Dr. Carl June, a prominent figure in immunology and the development of CAR-T cell therapies, delivered a compelling presentation on the evolution and future of CAR-T technology at the recent GenScript Biotech Global Forum. He began by recounting the historical context of CAR-T cell development, highlighting the pivotal moment in 2009 when Zelig Eshhar created the first redirected T-cell that did not require HLA binding. This innovation laid the groundwork for the CAR (chimeric antigen receptor) technology that has since transformed cancer treatment.
June shared the remarkable story of Emily Whitehead, the first pediatric patient treated with CAR-T cells in 2012, whose successful outcome garnered significant media attention and ultimately contributed to the FDA's approval of CAR-T therapies in 2017. He emphasized the rapid advancements in the field, noting that over 1,000 clinical trials are currently underway, exploring various cell types and combinations to enhance treatment efficacy.
A key focus of June's presentation was the differentiation in patient responses to CAR-T therapy, particularly in relation to the persistence of CAR-T cells and their effectiveness in combating blood cancers. He presented unpublished data indicating that patients with long-term B-cell aplasia—an absence of B-cells due to effective CAR-T action—exhibited better outcomes. This finding suggests a complex interplay between different immune cell types, particularly CD4 and CD8 T-cells, in sustaining an effective anti-cancer response.
June also discussed ongoing innovations in CAR-T therapies, including the development of "armored" CAR-T cells that secrete cytokines to enhance immune responses and dual CAR-T therapies targeting multiple antigens simultaneously. These advancements aim to address the challenges posed by solid tumors, which have historically been more resistant to CAR-T treatments compared to blood cancers.
The presentation concluded with a look at the expanding applications of CAR-T technology beyond oncology, including potential treatments for autoimmune diseases, infectious diseases, and organ transplantation. June highlighted the promising results from trials in autoimmune conditions, where CAR-T cells have shown the ability to reset the immune system, offering hope for curative therapies.
Overall, Dr. June's insights underscore the transformative potential of CAR-T cell therapies in modern medicine, with ongoing research poised to further enhance their efficacy and broaden their applications. The field is at a critical juncture, with significant innovations on the horizon that could redefine treatment paradigms for various diseases.
Dr. Peter Marks from the FDA outlined significant advancements and regulatory efforts aimed at enhancing the delivery and development of gene therapies, particularly CAR T-cell therapies and CRISPR technologies. With 22 gene therapies currently approved in the U.S., including CAR T-cells and genetically modified stem cells, the FDA is refining its approach to ensure safety while fostering innovation.
A key highlight was the FDA's reassessment of CAR T-cell therapies, which previously carried a box warning due to concerns about potential secondary malignancies related to treatment with engineered T cells. Recent data, however, indicates that the risk is much lower than initially thought, prompting plans to update labeling and encourage further development, especially for autoimmune diseases.
Peter emphasized the potential shift towards decentralized manufacturing of CAR T-cells, facilitated by advancements in automated systems. This could enhance accessibility and reduce costs, particularly for smaller indications and solid tumors. The integration of CRISPR technology is also poised to revolutionize the field, allowing for multiple edits in T-cells and potentially enabling in vivo production of CAR T-cells, which could further lower costs and improve patient outcomes.
The FDA is actively working to streamline regulatory processes for gene therapies, particularly for rare diseases, where treatment options are scarce. A pilot program with the European Medicines Agency aims to harmonize review processes, potentially accelerating the availability of therapies across multiple regions.
Additionally, the FDA is leveraging lessons learned from the COVID-19 pandemic to enhance communication with companies, aiming for faster development timelines for pediatric rare diseases. The ongoing pilot program includes several products targeting life-altering conditions in children, with the goal of expediting their path to market.
Looking ahead, Peter expressed optimism about the future of gene therapy, highlighting the potential for a significant increase in the number of therapies available for various diseases over the next decade. The combination of advanced manufacturing technologies, regulatory innovations, and the application of artificial intelligence could lead to a transformative era in gene therapy, addressing a broader range of conditions and improving patient care.
The recent panel discussion on the "2025 Cell and Gene Therapy Market Outlook" brought together industry leaders to explore the current state and future prospects of the biotechnology sector. Moderated by Vincent Xiang, Founding and Managing Partner at 7G Bioventures, the session featured prominent figures from various investment firms and biotech companies, who shared their insights on the evolving landscape of cell and gene therapies.
The panel opened with a reflection on the tumultuous market conditions of 2024. Vincent emphasized the need for innovation and adaptability in a market that has seen both highs and lows, particularly in the wake of the COVID pandemic.
Bing Li, Founder and CIO at Lake Blue Capital, provided a perspective on the Chinese biotech landscape. He remarked, "China is currently a quick follower in the biotech space, primarily producing best-in-class rather than first-in-class innovations. However, the rapid translation of academic research into viable products is promising." Li highlighted the importance of cross-border collaborations, suggesting that the future of innovation lies in global partnerships.
Josh Resnick, a Senior Managing Director at RA Capital, echoed the sentiment of cautious optimism. "While 2024 was challenging, the market continues to reward genuine innovation that addresses real needs," he stated. Resnick emphasized the necessity of maintaining strong fundamentals in the face of unpredictable capital markets, asserting, "The world population continues to need what we're making."
Caroline Stout, a Partner at EcoR1 Capital, shared her excitement for the innovative projects underway despite the hurdles faced in 2024. "Thanks to regulatory advancements, particularly from the FDA, we now have pathways that were previously unavailable. This sets the stage for an exciting year ahead," she noted, highlighting the importance of patient-oriented missions in the biotech community.
Alan Bash, Head of the CARVYKTI franchise at Legend Biotech, provided a unique perspective from the therapeutic side. He celebrated the recent successes of CAR-T therapies, particularly CARVYKTI's demonstration of overall survival in multiple myeloma patients who had relapsed to previous therapies. "This is a transformative moment for the treatment of multiple myeloma, showcasing the potential of CAR-T therapies to change lives," Bash remarked.
Yan Zhu, the VP and Head of Global Open Innovation, at Mitsubishi Tanabe Pharma America, Inc., pointed out the challenges faced by pharmaceutical companies in Japan, particularly regarding regulatory conservatism and cost pressures. "Japan's market has been declining due to stringent government policies on drug pricing, which poses a significant challenge for the introduction of high-cost therapies," he explained.
The discussion also delved into the challenges of scaling cell and gene therapies beyond academic centers. Bash highlighted the need for community oncology practices to adopt CAR-T therapies, stating, "The opportunity ahead is to bring CAR-T to community settings, making it more accessible for patients."
As the panel concluded, the key lessons learned emerged clearly:
- Innovation is Crucial: The biotechnology sector must continue to prioritize genuine innovation that addresses real-world medical needs.
- Global Collaboration is Essential: Cross-border partnerships are vital for fostering innovation and overcoming regional challenges, particularly in markets like China and Japan.
- Regulatory Pathways are Evolving: Recent advancements in regulatory frameworks, particularly in the U.S., are paving the way for more efficient development and approval processes.
- Accessibility is Key: Expanding the reach of therapies beyond academic centers to community practices is essential for improving patient access to cutting-edge treatments.
- Focus on Fundamentals: Despite market volatility, maintaining strong fundamentals and a clear mission will be crucial for success in the biotechnology landscape.
Industry leaders gathered for a pivotal discussion on the theme "Conquering GCT Challenges in Biomanufacturing and Supply Chain Management," moderated by Matthew Pillar, Chief Editor of Bioprocess Online at Life Science Connect. The event brought together experts from various sectors of the biomanufacturing field, including Jason Bock, CEO of CTMC; Jonathan Esensten, Senior Advisor at Multiple Labs; and Rey Mali, CBO of Accelix, among others. The panelists shared insights on the current landscape of cell and gene therapy manufacturing, addressing the challenges and opportunities that lie ahead.
The discussion opened with a focus on the rapid advancements in cell and gene therapies, which have undoubtedly outperformed the approval timeline of monoclonal antibody drugs. Pillar highlighted the promise of these therapies, stating, "There are great aspirations to expand indications into common diseases like osteoarthritis and cardiac disease, but significant challenges remain."
Rey Mali emphasized the need for improved scalability and distribution in the industry. "The technology is there, but now it's about scale and distribution," she noted, projecting that these areas will be a primary focus for 2025. She underscored the importance of making therapies more accessible and affordable for patients.
Hing Wong, Founder & Chief Executive Officer at HCW Biologics Inc., echoed this sentiment, stating, "Cost is a major issue. We need to lower manufacturing costs while ensuring the effectiveness of the therapies." He pointed out that managing clinical development costs is equally crucial to achieving this goal.
Jonathan Esensten highlighted the role of automation and technology in overcoming current challenges. "The history of monoclonal antibodies shows us that innovation takes time, but we must push for advancements in automation and closed systems to improve access and efficiency," he said. He also mentioned the potential of distributed manufacturing technologies to enhance access to therapies globally.
The conversation shifted towards the future of manufacturing, with panelists discussing the exciting possibility of in vivo therapies. Hari Pujar, Operating Partner at Flagship Pioneering proposed, "What if we could create CAR T-cells inside the body?" This could leapfrog many of the challenges we currently face. This sentiment was supported by Hing, who spoke about the need for innovation in the field, particularly in understanding T-cell quality and functionality.
As the panel progressed, the discussion turned to the importance of supply chain resilience. Michael, a panelist involved in establishing a new manufacturing site, emphasized the need for robust internal supply chains. "Ensuring backup suppliers and maintaining acceptable inventory levels are key to managing our supply chain effectively," he said.
The panelists also addressed the complexities of autologous cell therapies, where the patient’s cells are part of the supply chain. Jason Bock highlighted the need for a more integrated approach, suggesting that manufacturers should be closer to hospitals to streamline the process. "We need to simplify the supply chain from the hospital's perspective," he stated.
In conclusion, the panelists shared several key lessons:
- Focus on Cost Reduction: Lowering manufacturing and clinical development costs is essential to make therapies more accessible to patients.
- Embrace Automation: Investing in automation and technology can significantly enhance efficiency and reduce the risk of errors in the manufacturing process.
- Innovate for the Future: Exploring in vivo therapies could potentially address many current challenges in cell and gene therapy manufacturing.
- Strengthen Supply Chains: Building resilient supply chains with reliable backup suppliers and streamlined processes is crucial for effective therapy delivery.
- Collaboration is Key: Leveraging the expertise of various stakeholders in the industry can lead to innovative solutions and improved outcomes for patients.
The event concluded with a call to action for the industry to continue pushing the boundaries of innovation while remaining patient and focused on the long-term goals of improving patient access to life-saving therapies.
The future of gene and cell therapy was the focal point of the last panel discussion featuring prominent leaders from the biotechnology industry. Moderated by Lori Ellis, head of insights for Biospace, the panel included executives from various companies, each bringing unique perspectives on the evolving landscape of therapeutic innovations.
The discussion opened with a critical examination of the financial viability of cell and gene therapies, particularly in light of recent market fluctuations. Massimiliano Paganelli, co-founder and CEO of MORPHOCELL Technologies, highlighted the industry's past overhyped valuations, stating, "Five or six years ago, we saw excitement around CAR-T therapies leading to billion-dollar valuations for preclinical companies. However, the reality of costs and time to market has led to a slowdown in investment."
Madhusudan Peshwa, CEO of MKC Biotherapeutics, echoed Paganelli's sentiments, emphasizing the importance of defining a therapeutic product profile. "We need to work backwards from a clear therapeutic target to create an effective execution plan," he said. Peshwa, who has extensive experience in the field, noted that the excitement surrounding clinical efficacy must be balanced with realistic expectations about the complexities of developing living drugs.
Guowei Fang, CSO and head of business development at Legend Biotech, added that innovation is key to addressing the challenges in the field. "The best way to tackle the variation challenge is through continuous innovation," he asserted. Fang pointed to advancements in immune cell engineering as a promising avenue for future therapies.
The conversation also touched on the need for a shift in mindset regarding the development of therapies. Louis Breton, CEO of Rampart Bioscience, remarked on the cyclical nature of the industry, stating, "We've seen excitement, disillusionment, and now a search for realistic applications. It's crucial to focus on where these therapies can truly make an impact."
Reagan Jarvis, co-founder and CEO of Anocca AB, highlighted the importance of identifying appropriate targets and receptors for therapies, particularly in the context of solid tumors. "The challenge lies in mapping targets and generating quality receptors that drive product potency," he explained. Jarvis emphasized that personalized therapies must be developed with a clear understanding of the diverse target space.
Daniel Shelly, CBO of PoLTREG, raised concerns about the regulatory landscape, noting that the ease of regulatory pathways for certain therapies can lead to a lack of diversity in targets. "We need to marry the right tools with the right indications," he said, advocating for a more nuanced approach to therapy development.
As the discussion progressed, panelists addressed the potential for bedside manufacturing of therapies. Reagan Jarvis expressed skepticism about this model, stating, "While manufacturing on-site may be feasible in the same facility or region, the complexities involved make true bedside manufacturing unlikely." However, other panelists countered that advancements in technology could pave the way for more localized manufacturing solutions.
The panel concluded with a consensus on the importance of education within the industry. Paganelli emphasized the need for collaboration among stakeholders, stating, "We must work together to educate investors, regulators, and the public about the unique nature of these therapies." Fang added that building communication channels and outreach efforts would facilitate clinical development and patient enrollment.
Key Lessons Learned:
- Realistic Valuations: The industry must move towards more realistic expectations and valuations, learning from past overhyped scenarios.
- Innovation is Essential: Continuous innovation in therapeutic approaches and manufacturing processes is crucial for overcoming existing challenges.
- Target Identification: A clear understanding of target receptors and patient needs is vital for developing effective therapies.
- Education and Collaboration: Engaging with regulators, investors, and the public is essential to foster a supportive environment for the advancement of gene and cell therapies.
As the field of gene and cell therapy continues to evolve, the insights shared by these industry leaders underscore the importance of collaboration, innovation, and a patient-centered approach in shaping the future of healthcare.
