Five CGT Therapies Receive CDE IND Approval or Acceptance
Novabio Therapeutics
Developer of Multi-Modal Treg Cell Therapy
December 2, 2025
eMedClub News
According to the Center for Drug Evaluation of the National Medical Products Administration of China（CDE）According to incomplete statistics from the official website and publicly available information, last week(November 24 - November 30), Over 30 Class 1 innovative drugs are proposed to be included in the breakthrough therapy category, with INDs approved for clinical trials or IND applications accepted, covering fields such as immune cell therapy, gene therapy, mRNA vaccines, XDC, and bi-/tri-specific antibodies.
The following will introduce representative Class 1 CGT therapies.
NOVABIOTX:
Autologous Treg Cells
NOVABIOTX Autologous Polyclonal Regulatory T Cell Injection（NP001）NOVABIOTX's IND Application Accepted by CDE for the Treatment of Spinal Bulbar Muscular Atrophy（SBMA）NP001 IIT Study for SBMA Has Also Been Submitted, with Clinical Trials Planned to Start in February Next Year.
SBMA is an X-linked recessive neurodegenerative disease that primarily damages motor neurons in the brainstem and spinal cord, and can also affect sensory nerves, skeletal muscles, and the endocrine system. Currently, there are no treatments available that can effectively slow or halt the progression of the disease.
NP001 Aims to Utilize Treg Therapy IntegrationRegulating Neuroinflammation and Delaying Disease Progression, andPromote Skeletal Muscle Repair and Limit Muscle FibrosisProvides new insights for cell therapy in neurodegenerative diseases such as SBMA.
In terms of immune regulation, Treg promotes the transformation of macrophages from pro-inflammatory M1 type to reparative M2 type by secreting anti-inflammatory factors such as IL-10 and TGF-β, thereby suppressing excessive inflammatory responses. Meanwhile, Treg regulates dendritic cell function and limits effector T cell activation by expressing inhibitory receptors like CTLA-4 and LAG-3. Additionally, Treg can comprehensively suppress abnormal immune responses through producing immunosuppressive adenosine and inducing apoptosis in inflammatory cells.
In terms of muscle tissue repair, Treg cells are recruited to the injury site via the IL-33/ST2 signaling axis. They then promote the polarization of macrophages from M1 to M2 type by secreting factors such as IL-4, IL-10, and IL-13, thereby inhibiting the fibrosis process. Notably, amphiregulin secreted by Treg cells can directly activate muscle stem cells through the EGFR pathway, promoting their proliferation and differentiation, and enhancing tissue regeneration capacity.
NP001 Other indications are also proceeding smoothly. This November, NP001 for the treatment of Amyotrophic Lateral Sclerosis (ALS)（ALS）The first patient was dosed in the Phase I/IIa registrational clinical trial. The study involves a single intrathecal injection of 1×10^7 Treg cells. After treatment, the patient underwent 4 hours of close medical observation, with stable vital signs and no adverse events such as fever, allergic reactions, or neurological deterioration.（AE）, was discharged the next day. The indication has been approved for clinical use by the CDE in April.The World's First Innovative Therapy for ALS Treatment via Intrathecal Injection of Treg CellsIn September, NP001 for the treatment of multiple system atrophy（MSA）The IND application was accepted by the CDE.
Gene Enlightenment:
iNKT Cells
Gene Illumination GKL-006(iNKT Cells)The IND application for the injection has been accepted by the CDE. GKL-006 is the world's first iNKT cell injection independently developed, produced, and fully owned by Gene Illumination, which has obtained three clinical approvals in China. The proposed indications are: advanced pancreatic cancer, prevention of high-risk recurrence after radical resection in patients with primary hepatocellular carcinoma, and unresectable primary hepatocellular carcinoma.
iNKT cells, the main subtype of human NKT cells, possess the functions of both T cells and NK cells. Activated iNKT cells can not only directly kill tumors but also activate other immune cells, such as NK cells and CTL cells, to collaboratively attack tumors, and are considered a bridge between the innate immune system and the adaptive immune system.
As the world's first company to develop a series of iNKT cell pipeline products, Gene Illumination has obtained 5 clinical approvals from the CDE.
Among them, the first domestically produced allogeneic off-the-shelf iNKT cell injection in China（GKL-006Allo）Approved for clinical use, intended to treat locally advanced or metastatic solid tumors that have failed standard treatment; another allogeneic off-the-shelf iNKT cell（GKL-006RTU）Approved for clinical trials, intended for the development of treatment for moderate to severe acute respiratory distress syndrome caused by infections.
NOVABIOTX:
Neoantigen mRNA Vaccine
NOVABIOTX R01 Injection（iNeo-Vac-R01）The IND application was accepted by the CDE. This therapy is a personalized tumor mRNA vaccine based on neoantigens, which precisely delivers mRNA encoding the patient's tumor neoantigens to antigen-presenting cells in the body through AI and LNP delivery technology.（APC）, thereby activating antigen-specific T-cell immune responses to achieve specific killing of tumor cells.
As of the end of November this year, R01 has initiated 7 IIT studies, with over 60 patients having received treatment. Relevant studies have confirmed its ability to effectively stimulate antigen-specific T-cell responses.
The clinical study of its combination with standard therapy for first-line treatment of advanced pancreatic cancer showed a median follow-up time of 15.5 months.One patient achieved complete remission after combined treatment.（CR），3 cases achieved partial remission（PR）, the remaining patients were all stable.（SD）`, Median Progression-Free Survival`（mPFS）Dá14.9 months`, Median Overall Survival`（mOS）Reach19.7 months, The 12-month progression-free survival rate and overall survival rate were respectively51.6%、88.9%，PFS and OS were extended compared to the treatment with gemcitabine plus nab-paclitaxel in the MPACT clinical study and the FOLFIRINOX regimen in the PRODIGE 4 clinical study.
The single-arm clinical study for the treatment of patients with advanced gastrointestinal tumors who failed standard therapy showed a median follow-up time of 9.3 months, with an mPFS of3.6 months, mOS is9.3 months, 12-month survival rate44.4%, All of the above indicatorsExceeding the historical data of traditional second-line and third-line standard treatments。
Belief Medicine:
rAd Gene Therapy
NOVABIOTX's BBM-C101 Injection Approved by CDE for Clinical Trial, Intended for Treating Human Papillomavirus（HPV）High-Grade Squamous Intraepithelial Lesion of the Cervix Associated with HPV16 and/or HPV18 Infection（HSIL）。
HSIL is an abnormal lesion of the cervical basement membrane caused by HPV infection, with >30% of HSIL patients at risk of developing invasive cancer. Among these, HPV16 and HPV18 are the main high-risk HPV subtypes leading to cervical cancer.
BBM-C101 is a non-replicating rAd gene therapy candidate drug with independent intellectual property rights owned by Belief BioMed. After injection, it co-expresses HPV antigens and immune-activating factors within local host cells.Through「Antigen Targeting + Immune Activation"Dual Mechanism for Effective Clearance of HPV Infection-Related Lesions."
As early as February-March 2024, two IIT studies of BBM-C101（ChiCTR2400086181/ChiCTR2400081919）Has been initiated.
Viya Zhen:
CFB Targeting siRNA
NOVABIOTX's VSA012 Injection Receives CDE Approval for Clinical Trials, Intended for the Treatment of Complement-Related Kidney Diseases, Including but Not Limited to Lupus Nephritis, IgA Nephropathy, C3 Glomerulopathy, Immune Complex-Mediated Membranoproliferative Glomerulonephritis, and Atypical Hemolytic Uremic Syndrome（aHUS）etc.
VSA012 is NOVABIOTX's first double-stranded small interfering RNA developed based on its proprietary siRNA technology platform.（siRNA）, through GalNAc conjugation, specifically targets complement factor B in the liver（CFB）mRNA, inhibiting the expression of CFB protein in blood circulation. Preclinical data show that VSA012 can achieve long-term inhibition of CFB mRNA expression in the liver, significantly suppressing the CFB protein in the blood and the activity of the alternative complement pathway, while demonstrating good safety.
In January this year, VSA012 treated paroxysmal nocturnal hemoglobinuria（PNH）Phase Ⅰ clinical trial has completed the first patient dosing.
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