Recently, the authoritative international journal in the field of neurotherapy, *Neurology and Therapy*, published online the 2-year follow-up results of the pivotal Phase III clinical trial EMBARK for delandistrogene moxeparvovec (brand name Elevidys), the world’s first gene therapy for Duchenne Muscular Dystrophy (DMD). The data showed that Elevidys demonstrated statistically significant and clinically meaningful benefits in several key functional outcomes when treating ambulatory DMD boys aged 4 to 8, compared with a matched external control group. These findings confirm that the therapy can consistently stabilize or slow disease progression.

2026On January 6, Taysha Gene Therapies announced the latest progress of its key program for TSHA-102, a gene therapy under investigation for Rett Syndrome. This update clarified important milestones in the ongoing REVEAL pivotal trial and unveiled a new study plan (ASPIRE study) agreed upon with the U.S. Food and Drug Administration (FDA), targeting younger patients (ages 2-4). The move lays out a clear regulatory path for TSHA-102 to potentially benefit a broader patient population across various age groups in the future.

Recently, Kelonia Therapeutics, a clinical-stage biotechnology company focused on in vivo gene delivery technology, announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for KLN-1010, its treatment targeting patients with relapsed or refractory multiple myeloma. KLN-1010 is an innovative in vivo CAR-T therapy designed to generate anti-BCMA CAR-T cells directly within the patient through a single infusion, without the need for preconditioning chemotherapy or customized CAR-T cell production. The FDA's approval of the IND application allows Kelonia to expand its ongoing inMMyCAR™ Phase I clinical trial, which is currently enrolling patients in Australia, to multiple clinical research centers across the United States.

Recommended Reading:FDA Approves IND Application for in vivo BCMA CAR-T Therapy

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CK0804, a first-in-class, allogeneic, high CXCR4-expressing regulatory T cell (Treg) therapy for the treatment of myelofibrosis. CK0804 is an investigational, allogeneic, off-the-shelf therapy composed of Tregs with high CXCR4 expression, which selectively migrate to areas with a concentration gradient of their ligand, CXCL12. In patients with myelofibrosis, this ligand is overexpressed in the bone marrow and extramedullary hematopoietic sites such as the spleen. Preclinical studies have shown that CXCR4-rich Tregs preferentially migrate to the bone marrow and suppress pro-inflammatory cytokine signaling; preliminary efficacy signals in myelofibrosis were reported previously.

Recommended Reading:CK0804 Receives FDA Orphan Drug Designation for the Treatment of Myelofibrosis

In recent years, hundreds of gene and cell therapies effective for patients have been shelved by pharmaceutical companies due to insufficient commercial returns. In January 2026, the American Society of Gene and Cell Therapy (ASGCT) partnered with the Orphan Therapies Accelerator (OTXL) to launch the CGTxchange platform, utilizing AI technology to create a dedicated marketplace. This platform aims to match these "abandoned" therapies with new partners and funding, restoring hope for treatment to patients with ultra-rare diseases.

Recommended Reading:AI + Gene Therapy! Hundreds of Rare Disease Treatments Brought Back to Life Through This Collaboration

On January 10, 2026, Mengchao Hepatobiliary Hospital of Fujian Medical University (Wu Mengchao Memorial Hospital of Fujian Medical University) officially inaugurated the "Cell and Gene Therapy Research Center" and simultaneously signed a major scientific and technological achievement transformation agreement with Fujian Haixi Cell Bioengineering Co., Ltd., transferring two original cell therapy technologies targeting advanced liver cancer for 20 million yuan. This move marks a key breakthrough in the frontier field of biomedicine in Fujian Province and injects strong momentum into the high-quality development of the regional industry.

Recommended Reading:Today, the official inauguration of a center at this hospital in Fujian Province! Patients will welcome new hope...

The team led by Gregory Corder at the University of Pennsylvania published a research paper titled “Mimicking opioid analgesia in cortical pain circuits” in *Nature*. This study reveals a groundbreaking strategy for treating chronic pain without the use of opioids. The researchers found that the anterior cingulate cortex (ACC) in the brain is responsible for encoding the "unpleasant" emotional aspect of pain, and opioids achieve pain relief by acting on specific neurons in this region. Based on this, the team developed a gene therapy combining adeno-associated virus (AAV) with chemogenetics, which acts like "precision guidance" to selectively install an inhibitory "switch" on opioid-sensitive neurons in the ACC region. Experimental results show that this strategy can accurately replicate the potent analgesic effects of opioids without using any opioid drugs, significantly alleviating chronic neuropathic pain while perfectly avoiding side effects such as addiction, tolerance, and sensory impairment caused by traditional drugs. This indicates that precise regulation of brain circuits using AAV technology holds promise as a powerful new alternative to systemic opioid treatment.

Recommended Reading:Nature |告别成瘾风险！靶向ACC环路实现阿片样精准镇痛

On January 8, 2026, the team led by Hai Feng Ye from the School of Life Sciences at East China Normal University, the Shanghai Key Laboratory of Regulatory Biology, and the Center for Medical Synthetic Biology published a research article titled “A compact and inducible dCas12f-based CRISPRa platform for programmable in vivo gene activation” in Nature Communications. The study successfully constructed a more compact, stronger-activating, and flexibly regulatable dCas12f-derived transcription activation platform—HEAL. Compatible with AAV delivery, this platform demonstrates endogenous gene transcription activation capability of up to hundreds of thousands-fold and combines red light and small-molecule induction switches to achieve controllable activation of disease-critical genes in mice, providing a new paradigm for the development of miniaturized, high-performance CRISPRa systems.

Recommended Reading:Research Progress | Nature Sub-Journal: Ye Haifeng's Team from Our Hospital Launches Miniature Controllable CRISPRa Platform HEAL, Enabling Powerful and Controllable Gene Activation In Vivo

On January 5, 2026, the Pagliari research team published a groundbreaking study in the *Journal of Biomedical Science*, setting a new therapeutic benchmark for two rare genetic disorders: spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot-Marie-Tooth disease type 2S (CMT2S). Both diseases are caused by mutations in the IGHMBP2 gene, leading to the gradual loss of motor neurons and resulting in respiratory failure. To address this challenge, the research team comprehensively upgraded and screened an adeno-associated virus serotype 9 (AAV9) vector capable of crossing the blood-brain barrier. In mouse model experiments, researchers compared the therapeutic effects of different gene "promoter switches" (CBA and P546 promoters) via intracerebroventricular injection. The results were highly encouraging: gene therapy not only significantly extended the lifespan of the affected mice, improved motor function, and protected neurons but also, for the first time, revealed and alleviated spinal cord neuroinflammation associated with the disease. After rigorous comparison, the team found that the modified vector using the P546 promoter demonstrated superior long-term efficacy and safety. This study not only highlights the critical role of precise screening of vector components in ensuring treatment durability but also lays a solid foundation for overcoming complex regulatory hurdles and advancing this life-saving therapy into human clinical trials.

Recently, Professor Zheng Tongsen's team from Harbin Medical University Cancer Hospital made significant progress in the field of tumor immunotherapy. The research paper "Engineered outer membrane vesicles enhance solid tumour CAR-T cell therapy" was published online in *Nature Biomedical Engineering* on January 7. The research team successfully developed a novel CAR-T enabling platform based on bacterial outer membrane vesicles (OMV)—"BROAD-CAR". This platform integrates immune suppression reversal and antigen modification functions, significantly enhancing the therapeutic effects of CAR-T cells in antigen-heterogeneous solid tumors, providing a universal solution strategy to broaden the clinical application of CAR-T therapy.