The third quarter of 2026 reflected both the continued progress and increasing maturity of the gene, cell, and RNA landscape. Seven new approvals across the US and Japan expanded treatment options for patients with rare diseases, cancer, infectious diseases, and transplant-related conditions. Notable approvals included Fayuvi for mucopolysaccharidosis IIIA (Sanfilippo A syndrome), Genglycos for glycogen storage disease type Ia, Tregzi for hematopoietic and immunologic reconstitution, and Zanvastro for Alexander disease. Together, these milestones underscore the growing breadth of advanced therapeutic modalities reaching the market.

Clinical development remained active, with four gene therapies advancing into Phase III trials and the overall pipeline expanding to 4,594 programs. At the same time, mixed late-stage results among several Angelman syndrome programs highlighted both the promise and uncertainty that accompany the development of first-in-class approvals. Regulatory momentum remained strong, with 11 gene therapies currently awaiting approval decisions.

In contrast to Q2’s surge in investment activity, dealmaking declined 33% quarter over quarter, driven by fewer financings and alliances. Despite this pullback, acquisition activity remained steady and early-stage funding continued to outpace levels seen in the same period last year. Together, these trends point to a sector entering a more disciplined phase of growth, supported by a deep development pipeline, sustained innovation, and continued progress toward delivering transformative therapies to patients.

For the full report, please download the PDF version at the top of this page in collaboration with ASGCT.