On December 9, 2025, the U.S. FDA approved Waskyra (etuvetidigene autotemcel) — the world's first cell gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS). This breakthrough not only provides a new treatment option for WAS patients but also injects new confidence into the gene therapy industry, which has been in a slump.

On December 12, 2025, BRL Medicine Inc. (hereinafter referred to as "BRL Medicine"), a company focused on gene and cell therapy, announced that the Investigational New Drug (IND) application for its next-generation allogeneic UCAR-T product "Allogeneic Chimeric Antigen Receptor T-Cell Injection Targeting CD19 Gene Modification" (Pipeline Code: BRL-303), developed based on its proprietary universal cell platform (TyUCell®), has officially received tacit approval from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) on December 11. This is another milestone in the field of autoimmune disease treatment (Pipeline Code: BRL-303) after the dual-indication breakthrough achieved by the allogeneic UCAR-T product (Pipeline Code: BRL-301) in the field of hematological tumors.

On December 9, Kelonia Therapeutics, a pioneer in in vivo gene delivery, presented oral reports at the 2025 ASH Annual Meeting on Phase I clinical trial data (inMMyCARTM, first-in-human) for in vivo BCMA CAR-T KLN-1010 in treating patients with relapsed/refractory multiple myeloma. Kelonia's core technology is the in vivo Gene Placement System (iGPS), which uses advanced lentiviral vector particle envelope modifications to improve in vivo gene transfer efficiency and employs tropic molecules to facilitate tissue-specific delivery. KLN-1010 is Kelonia’s leading core pipeline, currently in Phase I clinical trials.

Also at the 2025 ASH Annual Meeting, the China-based in vivo CAR-T company VionBio presented, in an oral report, the first clinical data of its GT801 CD19-targeted in vivo CAR-T pipeline based on LNP-mRNA technology for the treatment of non-Hodgkin lymphoma. In the investigator-initiated Phase I clinical trial, the safety, tolerability, and preliminary efficacy of GT801 were evaluated under repeated dosing conditions in patients with relapsed/refractory CD19-positive B-cell lymphoma.

Recommended Reading:2025 ASH | In Vivo CAR-T "Heats Up"! Latest Phase I Clinical Data from Two Major Technical Routes Released

Recently, CREATE Medicines (formerly Myeloid Therapeutics) announced that the first patient has been dosed in its clinical trial (NCT06478693) for the first-line treatment of metastatic hepatocellular carcinoma (HCC). The study aims to evaluate the efficacy of MT-303, an in vivo CAR therapy targeting GPC3, in combination with atezolizumab and bevacizumab, the latter being the current global standard first-line treatment regimen for HCC.

Recently, Sana Biotechnology and Luca Biasco's team from University College London (UCL) published a paper in Nature Biotechnology titled “In vivo gene editing of human hematopoietic stem and progenitor cells using envelope-engineered virus-like particles,” which systematically demonstrated for the first time two types of "envelope-engineered" virus-like particles (VLPs) capable of editing human hematopoietic stem and progenitor cells (HSPCs) in vivo while avoiding critical filtration organs such as the liver.

Recommended Reading:NBT| "Empty-Shell Virus" Delivers Gene Editing to Hematopoietic Stem Cells

Recently, Genome Biology published online an important study by Li Mo's team from King Abdullah University of Science and Technology (KAUST), titled "CRISPR-Cas9-induced double-strand breaks disrupt maintenance of epigenetic information." This research systematically reveals that Cas9-induced double-strand breaks (DSBs) lead to the failure of DNA methylation maintenance, resulting in stable and heritable epigenetic remodeling. This discovery fills a long-standing gap in the field of gene editing and is crucial for the safety assessment of future clinical gene therapies.

Recommended Reading:Genome Biol | CAS9 Cutting Modifies Not Only Sequences but Also "Memory": Li Mo's Team Discovers DNA Breaks Remodel Epigenetic States

Columbia University Irving Medical Center's Cellular Immunotherapy and Cell Engineering Initiative (CICET) and Memorial Sloan Kettering Cancer Center research team published a groundbreaking study in the journal *Cell*, titled "pTα Enhances mRNA Translation and Augments CAR T Cell Activity to Eliminate Solid Tumors." The study significantly enhanced the persistence and anti-tumor capacity of CAR-T cells by integrating a fragment of pTα, a key protein in thymocyte development, into the CAR (chimeric antigen receptor) structure.