Cell and Gene Therapies: A Surge in Clinical and Regulatory Momentum in 2025–2026

The cell and gene therapy (CGT) landscape is undergoing a transformative phase, marked by accelerated clinical progress and robust regulatory momentum. As we approach the end of 2025, the sector is witnessing a confluence of scientific innovation, strategic partnerships, and regulatory advancements that position CGTs as a cornerstone of modern medicine. For investors, this dynamic environment presents both opportunities and challenges, demanding a nuanced understanding of the evolving pipeline and approval landscape.

Accelerating Clinical Progress: Neurology and Hematology Lead the Charge

The acceleration of clinical trials in CGTs is most evident in neurology and hematology, where novel therapies are addressing unmet medical needs with unprecedented precision. In neurology, the U.S. clinical trials market is prioritizing disease-modifying and gene-based interventions for conditions such as Alzheimer's, Parkinson's, and rare neurological syndromes. A 2025–2033 analysis highlights the role of decentralized and hybrid trial models in expediting data collection and broadening patient access, while increased healthcare spending and industry-academia collaborations are streamlining trial execution.

Hematology, meanwhile, is seeing groundbreaking advancements in targeted therapies. At the ASH 2025 Annual Meeting, Merck showcased its pipeline for hematologic malignancies, including MK-1045 (a CD19xCD3 T-cell engager for B-cell acute lymphoblastic leukemia), bomedemstat (an LSD1 inhibitor for myeloproliferative neoplasms), and nemtabrutinib (a non-covalent BTK inhibitor for chronic lymphocytic leukemia). These therapies underscore the industry's shift toward precision oncology and the potential to redefine treatment paradigms.

Regulatory Momentum: Breakthrough Designations and Strategic Approvals

Regulatory agencies are playing a pivotal role in fast-tracking CGTs to market, with the FDA and EMA granting critical designations to therapies demonstrating transformative potential. In 2025–2026, tovorafenib (OJEMDA™) emerged as a standout candidate, receiving Breakthrough Therapy and Priority Review designations from the FDA for pediatric low-grade glioma (pLGG) with BRAF gene alterations. The drug also earned Orphan Drug status in the U.S. and EU for glioma treatment, following a Phase 2 trial (FIREFLY-1) that reported a 53% overall response rate and a median time to next treatment exceeding 3.5 years.

The FDA's approval pipeline for CGTs remains robust, driven by the commercialization of therapies like Casgevy (for sickle cell disease) and Elevidys (for Duchenne muscular dystrophy), which entered the market in 2023–2024. These approvals reflect a broader trend: over 2,000 ongoing CGT trials are advancing through development stages, creating a surge in demand for contract development and manufacturing organizations (CDMOs) to support specialized production needs.

However, regulatory pathways are not without hurdles. Agios Pharmaceuticals, for instance, faces a mixed outlook for its sickle cell therapy, mitapivat (Pyrukynd). While the drug demonstrated hemoglobin improvement in the RISE UP Phase 3 trial, it fell short of reducing pain crises-a key endpoint. Despite this, the company plans to engage with the FDA in early 2026 to seek approval, highlighting the delicate balance between scientific rigor and commercial strategy in the CGT space.

Investment Implications: Navigating a High-Stakes Landscape

For investors, the CGT sector in 2025–2026 represents a high-reward, high-risk proposition. The convergence of clinical innovation and regulatory support is creating a fertile ground for growth, particularly in neurology and hematologic oncology. However, the complexity of manufacturing, variable trial outcomes, and pricing pressures remain critical risks.

Key opportunities lie in companies with strong partnerships, scalable manufacturing capabilities, and therapies targeting well-defined patient populations. Merck's hematology pipeline and Day One's tovorafenib exemplify how Breakthrough Therapy designations can catalyze market entry and investor confidence. Conversely, firms like Agios illustrate the importance of adaptive strategies in the face of mixed clinical data.

As the CGT ecosystem matures, stakeholders must remain agile, leveraging real-world evidence and decentralized trial models to de-risk development and accelerate approvals. For now, the sector's trajectory is clear: innovation is accelerating, and regulatory momentum is building, making CGTs an indispensable focus for forward-looking investors.