With the PDUFA date fast approaching for Regenxbio Inc.’s gene therapy RGX-121, the U.S. FDA placed the drug on clinical hold along with another, RGX-111, after preliminary analysis of a single case of neoplasm (specifically, an intraventricular central nervous system tumor) in a participant treated in the phase I/II study with the latter treatment.

Following a clinical hold last October of Intellia Therapeutics Inc.’s Magnitude and Magnitude-2 phase III trials of CRISPR/Cas9 gene editing therapy nexiguran ziclumeran (nex-z) to treat transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN), respectively, the U.S. FDA lifted the hold on Magnitude-2, pushing the company’s shares up by 22% in early trading Jan. 27.

After a roller coaster of a year for Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec), Sarepta Therapeutics Inc. looks to focus on the efficacy narrative in 2026, starting with newly unveiled three-year data showing durable efficacy across all key motor function assessments for treated DMD patients vs. external controls.

Lexeo Therapeutics Inc.’s phase I/II data – characterized by Oppenheimer analyst Leland Gershell as “heroic” – this week with gene therapy LX-2020 in PKP2-associated arrhythmogenic cardiomyopathy pushed the New York-based firm’s shares (NASDAQ:LXEO) down 35% to a low of $6.90 Jan. 12, following a $10.54 close the previous trading day.

There was an upbeat message for cell and gene therapy companies in the 2026 industry update presented as the J.P. Morgan Healthcare Conference opened on Monday, with Tim Hunt, CEO of the Alliance for Regenerative Medicine, telling delegates that after lean years of learning, adapting and setbacks, the sector is now self-sustaining.

Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them. However, many approaches still struggle to reach target cells or tissues – either they fail to arrive, or their efficacy is low. In vivo therapies face numerous challenges, but despite these hurdles, 2025 has marked a year of remarkable progress.

Gene therapy has had its commercial struggles in the past year. The cost to patients is in the millions and fewer are stepping forward for treatment than companies would like. While development continues in this game-changing field, some have struggled with regulatory authorities during development while others have just stepped away altogether.

Rznomics Inc. continued South Korea’s year-end biotech rally with a ₩46.35 billion (US$31.35 million) IPO Dec. 18. Proceeds will fund Seongnam-si, South Korea-based Rznomic’s pipeline of gene therapies, built on the company’s trans-splicing ribozyme RNA Replacement Enzyme technology platform.