From the Editors—Beyond the Frontier: Q1 2026 and the Shift to Scalable CGT Reality

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Dr. Adaeze Ekwe, PhD
Junior Associate Editor, ISCT Telegraft
Australia

The first quarter of 2026 has been a pivotal period for the global cell and gene therapy (CGT) sector, defined by clinical milestones, regulatory reforms and the launch of next-generation manufacturing hubs. These advancements signal an industry transitioning from a promising frontier toward commercial scalability and expanded patient access. At the centre of this momentum, ISCT has spearheaded global actions to bridge the gap between innovation and global care. Here are the key highlights of Q1 2026.

In a historic first for rare diseases, market authorisation was issued by the European Medicines Agency (EMA) for Waskyra (etuvetidigene autotemcel), the first gene therapy for Wiskott-Aldrich Syndrome (WAS) [1]. Waskyra is an ex vivo therapy that modifies a patient's own CD34+ hematopoietic stem cells using a lentiviral vector to deliver a functional copy of the WAS gene. Similarly, the United States Food and Drug Administration (FDA) granted approval for Kresladi (marnetegragene autotemcel), a genetically modified autologous hematopoietic stem cell therapy, marking the first-ever gene therapy for pediatric and adult patients with severe Leukocyte Adhesion Deficiency Type I (LAD-I) [2]. Kresladi has navigated a complex regulatory journey, including two previous Chemistry Manufacturing and Controls (CMC)-related Complete Response Letters. Continued approval is dependent on the clinical benefit of Kresladi confirmed in patients with severe LAD-I through post-approval studies [2].

The regulatory landscape has been defined by a concerted effort to align oversight with the unique biological and manufacturing characteristics of advanced therapies. Both the FDA and the EMA have introduced measures designed to expedite access while maintaining rigorous standards for safety and potency [3, 4]. A pivotal change involves the FDA's stance on manufacturing modifications during the transition from early-phase trials to pivotal studies [3, 5]. The EMA Committee for Advanced Therapies (CAT) 2026 workplan is centered on three pillars: scientific guidance, assessor training, and the integration of Advanced Therapy Medicinal Product (ATMP) expertise into the broader EMA framework [4].

As the science of CGT matures, manufacturing reliability and scalability have become the primary drivers of commercial success. This shift has triggered substantial capital investments in infrastructure, the adoption of advanced automation technologies and a move towards decentralized and point-of-care manufacturing models [6 – 9].

At the society front, ISCT has driven several high-impact initiatives focusing on clinical translation, global partnerships and commercialization. Key among them include:

The Inaugural Industry Virtual Town Hall (March 11th 2026), serving as a direct dialogue between the Society’s leadership and global industry stakeholders to address translation and commercialization hurdles.
A five-year strategic collaboration between ISCT and the Japanese Society for Regenerative Medicine (JSRM) launched on March 21, 2026, in Kobe, Japan. The collaboration aims at advancing clinical-grade induced pluripotent stem cell (iPSC) therapies. This partnership seeks to harmonize international regulatory structures for iPSC-based products and identify manufacturing bottlenecks to accelerate global patient access.

These initiatives are unfolding alongside critical organizational milestones. Voting for the 2026 ISCT Elections is ongoing, with a mandate for new leadership to manage the society’s exponential growth and regional expansion. Concurrently, preparations are underway for the ISCT 2026 Annual Meeting in Dublin (May 6th to 9th), which is slated to host over 3,000 delegates and feature 250+ speakers focused on the "Golden Age of Cell and Gene Therapy Translation”. The momentum continues later in the year with two key regional meetings: the ISCT ANZ 2026 meeting will take place from July 20th to 22nd, at the Melbourne Convention & Exhibition Centre, Australia and the inaugural ISCT Asia 2026 Regional Meeting will be held from September 2nd to 5th at the Suntec Singapore Convention & Exhibition Centre.

Ultimately, these milestones, society-led efforts and regional expansions are a microcosm of a larger evolution. As the CGT sector navigates this transformative first quarter, the convergence of regulatory clarity, clinical breakthroughs, and manufacturing innovation marks a definitive transition from a promising frontier to a robust, scalable reality. Moving deeper into this commercial era, the strategic focus has bifurcated: balancing the necessity for industrial-scale manufacturing with the rise of highly individualised pathways for ultra-rare conditions. Supported by flexible CMC guidelines, the maturation of in vivo platforms, and the emergence of decentralized manufacturing models, the industry is no longer just proving its potential, it is delivering on the promise of a new standard of global care.