Charting the Trajectory of Cell and Gene Therapy from 2024 to 2025
What to Expect from the 2025 State of the Industry Briefing
This morning at Lonrú we’re looking back on 2024, and sharing our take on what to expect from today’s discussions at the Alliance for Regenerative Medicine’s (ARM) 2025 State of the Industry briefing.
The past year was marked by significant progress, including advancements in in vivo therapies, global regulatory milestones, and an ongoing push to address affordability and accessibility in cell and gene therapy (CGT). As the industry continues to mature, we anticipate that today’s briefing will highlight key inflection points and lay out the challenges and opportunities for the year ahead.
From in vivo innovations and manufacturing breakthroughs to expanded therapeutic targets and global market shifts, the CGT sector is poised for another transformative year. Here’s our take on what shaped 2024 and what may define the future.
2024: A Year of Momentum and Milestones
Breakthroughs Becoming the Norm
2024 began with optimism following a stormy 2023 in capital markets. Despite economic volatility throughout 2024, the CGT sector achieved major milestones: nine new therapies were approved in the U.S., and two in Europe. The FDA’s approval of two sickle cell gene therapies in late 2023 further underscored the field’s transformative potential. These developments heralded a “new normal” where groundbreaking therapies routinely became available to patients (ARM, 2024).
Advancing Cell Therapy
By February, the industry spotlight shifted toward engineered cell therapies. The approval of tumour-infiltrating lymphocyte (TIL) therapy for solid tumours represented a watershed moment, while investment in autoimmune applications highlighted the expanding scope of cell therapies beyond oncology (ARM, 2024).
Reframing the Value Narrative
August saw renewed focus on the affordability and accessibility of CGT. Frameworks like “Gene Therapy for Patients and Society” (GPS) and “CAR-T for Patients and Society” (CAR-PS) were developed to demonstrate the long-term value and cost-effectiveness of these therapies. Such efforts aimed to reshape public perception and healthcare decision-making (CMS, 2024).
New Gene Editing Frontiers
December capped the year with approvals for two adoptive cell therapies targeting solid tumours and the introduction of next-generation gene editing techniques like prime and epigenetic editing in clinical trials. These developments opened new possibilities for tackling previously untreatable conditions (ARM, 2024).
In Vivo Growth: A Rising Star
While much of 2024’s focus was on ex vivo therapies, in vivo approaches gained significant traction. These methods, which deliver genetic modifications directly within the patient’s body, promise to enhance precision and scalability. Key developments included:
CRISPR-Based In Vivo Therapies: Clinical trials demonstrated promising results in diseases like Duchenne muscular dystrophy and retinal disorders. These breakthroughs highlight the growing maturity of in vivo technologies (ARM, 2024).
Next-Generation Gene Editing: Techniques such as prime* and epigenetic editing, which offer higher precision and reduced off-target effects, entered early clinical stages (Tune Therapeutics, 2024) (O'Hanlon Cohrt, 2024).
Innovative Delivery Mechanisms: Advances in nanoparticle and viral vector technologies further improved the efficiency and safety of in vivo therapy delivery (ARM, 2024).
Looking ahead, in vivo growth will likely feature prominently in discussions about the next wave of CGT innovations, particularly in areas like systemic genetic disorders and regenerative medicine.
Predictions for 2025
Based on 2024’s trajectory, today’s State of the Industry briefing may shine a light on several critical themes:
1. Regulatory and Manufacturing Innovations
With the FDA’s expanded Office of Therapeutic Products, efforts to harmonise global regulatory frameworks and streamline manufacturing processes will take centre stage.
2. Expanding Therapeutic Horizons
Expect updates on the broadening range of disease targets, including autoimmune conditions and rare disorders, as clinical breakthroughs continue to multiply.
3. Addressing Affordability and Access
ARM’s ongoing advocacy for outcomes-based agreements and cost-efficiency frameworks will remain a focal point as the industry works to balance innovation with equitable patient access.
4. Continued In Vivo Innovation
Advancements in delivery systems and new data from clinical trials of in vivo therapies will likely feature heavily, positioning this approach as a cornerstone of future CGT strategies.
5. Ecosystem Collaboration
Fostering partnerships and collaboration across academia, biopharma, and regulatory bodies will underpin sustainable CGT growth.
Illuminating the Path Forward
2024 demonstrated that the CGT sector is no longer in its infancy. Breakthroughs are expected, and the focus has shifted toward refining processes, improving access, and expanding therapeutic potential. As 2025 unfolds, Lonrú Consulting will continue to illuminate the opportunities for our clients, providing data-driven insights and tailored strategies to help them navigate this complex and dynamic landscape.
*Prime Medicine’s clinical program PM359 is an ex-vivo engineered HSC product (O'Hanlon Cohrt, 2024). Prime editing technology holds promise as an in-vivo engineering platform (Newby & Liu, 2021).
References
ARM. (2024, January 8). Introduction and Industry Update - Cell & Gene State of the Industry Briefing 2024. YouTube. Retrieved January 13, 2025, from https://www.youtube.com/watch?v=8uyekVKDo8Y
ARM. (2024, April 30). SECTOR SNAPSHOT ADVANCES IN ENGINEERED CELL THERAPY. ARM - Industry updates. https://alliancerm.org/wp-content/uploads/2024/05/Sector-Snapshot-4.30.2024.pdf
ARM. (2024, December 30). Alliance for Regenerative Medicine - December 2024 Sector Snapshot. ARM Industry Updates. https://alliancerm.org/wp-content/uploads/2025/01/20250107-2024-Sector-Snapshot.pdf
CMS. (2024, March 7). Cell and Gene Therapy (CGT) Access Model. enters for Medicare & Medicaid Services. Retrieved January 13, 2025, from https://www.cms.gov/priorities/innovation/innovation-models/cgt
Newby, G., & Liu, D. (2021, November 1). In vivo somatic cell base editing and prime editing. Molecular Therapy, 29(11), 3107-3124.
O'Hanlon Cohrt, K. (2024, May 1). The FDA Has Cleared the First Clinical Trial Application for a Prime Editor. CRISPR Medicine News. https://crisprmedicinenews.com/news/the-fda-has-cleared-the-first-clinical-trial-application-for-a-prime-editor/
Tune Therapeutics. (2024, November 14). Tune Therapeutics Moves into Clinical Spotlight with TUNE-401: A First-in-Class Epigenetic Silencer for Hepatitis B. Investors and Media. https://tunetx.com/tune-therapeutics-moves-into-clinical-spotlight-with-tune-401-a-first-in-class-epigenetic-silencer-for-hepatitis-b/
