The U.S. FDA has cleared Tessera Therapeutics Inc.’s IND application for TSRA-196, its lead in vivo gene editing therapy for α-1 antitrypsin deficiency (AATD). The Australian Human Research Ethics Committee (HREC) has also granted approval for the company to begin the phase I/II clinical study.
Askbio Inc., a subsidiary of Bayer AG, has received IND clearance from the FDA for AB-1009, an AAV gene therapy being developed for the treatment of late-onset Pompe disease.
Genprex Inc. has released preliminary in vivo proof-of-concept data for GPX-002, the company’s diabetes gene therapy drug candidate, in a nonhuman primate (NHP) model of type 2 diabetes and in a mouse model of type 2 diabetes. GPX-002 is being developed for the potential long-term control of both type 1 and type 2 diabetes.
In a recent publication in Molecular Therapy, researchers from Drexel University College of Medicine and UMass Chan Medical School presented a silence-and-replace gene therapy strategy aiming to address both the gain-of-toxicity and loss-of-function components of the disease hereditary spastic paraplegia (HSP).
SYNGAP1-related disorders (SRDs) are rare neurodevelopmental conditions characterized by a wide range of symptoms, including intellectual disability, epilepsy, motor deficits and increased risk-taking behavior.
Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them.
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 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.
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