Ten Gene Therapy IND Applications Approved or Accepted in One Week

According to incomplete statistics from the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration and publicly available information, from March 2 to March 8,Over 50 Class 1 Innovative Drug IND Applications Approved/Accepted, covering CAR-T/iNKT, siRNA/ASO, AAV gene therapy, multi-specific antibodies, ADC, innovative degraders and other fields,About 10 of them are CGT therapies.

The following are representative CGT therapies that have received IND progress

Pregene: BCMA CAR-T

Pregene's BCMA-Targeted Chimeric Antigen Receptor T-Cell Injection (CAR-T) Approved for New Indication, Intended for the Treatment of Refractory Lupus Nephritis – An Important Advance in Expanding into the Field of Autoimmune Diseases Following Approval for Relapsed and Refractory Multiple Myeloma.

This CAR-T construct incorporates a chimeric heavy chain variable domain with high affinity and high specificity. The relevant research data, production processes, and quality control systems have been developed according to international standards, offering high production efficiency and significant cost advantages. Moreover, the processes and quality control systems demonstrate excellent repeatability and stability.

Bo sheng ji medicine science and technology (suzhou) co., LTD: CD7 CAR-T

Bo Sheng Ji's CD7-Targeted Autologous CAR-T Cell PA3-17 Injection Receives CDE Clinical Tacit Approval for Development in Treating Recurrent/Refractory T Lymphoblastic Leukemia/Lymphoma (R/R T-ALL/LBL) in Children and Adolescents. This move marks the official expansion of PA3-17 Injection to high-risk pediatric and adolescent patient populations, building on its previous application in adults.

PA3-17 adopts a non-gene-edited autologous CD7-CAR-T approach, successfully solving the industry challenge of "self-targeting" in T-cell therapy. For adult R/R T-ALL/LBL, this therapy has enteredPivotal Phase II Clinical Trial, and was included by the CDE as a breakthrough therapy. Data from the Phase I pivotal clinical trial showed an objective response rate (ORR) of85%`, MRD-negative complete remission rate as high as`95%, with excellent efficacy and safety performance.

Gene Key: iNKT Cells

GKL-006Allo, an off-the-shelf allogeneic iNKT cell injection developed by Beijing Gene Key Life Technology Co., Ltd., has received clinical trial approval from the CDE and is intended for the treatment of ankylosing spondylitis in patients who have failed or are intolerant to standard therapy.

This approval marks a further exploration of GKL-006Allo in non-oncology fields. Previously, another iNKT cell product, GKL-006 Injection, developed by the company, had already been approved for multiple solid tumor indications (including advanced pancreatic cancer, hepatocellular carcinoma, and postoperative prevention of high-risk recurrence), and has now expanded to moderate-to-severe psoriasis.

HemaCell: Umbilical Cord Blood Megakaryocyte Injection

HemaCell's Second IND Application for Its Self-Developed Megakaryocyte Injection Approved, with the Proposed Indication for Thrombocytopenia Caused by Aplastic Anemia (AA); Its First Indication (Thrombocytopenia Caused by Cancer Treatment) Is Currently in Phase I Clinical Trials.

As the world's first allogeneic megakaryocyte injection, it can quickly restore platelet counts and achieve a longer-lasting platelet increase. Additionally, it features low cost, high safety, no gene editing, no significant side effects, and no risk of thrombosis.

DeCom Mingji: Dual AAV Gene Therapy

DeCom Mingji VG801 Approved for Clinical Trials, Intended for Treatment DevelopmentABCA4Biallelic Mutation-RelatedRetinal dystrophy.

Public information shows that VG801 adopts the AAV vector REVeRT (Recombination through mRNA trans-splicing) technology, solving the problem of the DNA sequence encoding human ABCA4 (approximately 6.7 kb) being too large to fit into a single AAV vector.

REVeRT consists of two AAV vectors, each carrying half of the ABCA4 coding sequence, producing two independent pre-mRNAs. These pre-mRNAs are trans-spliced to form a complete mRNA, which is then translated into a functional protein. VG801 also employs a novel vgAAV capsid designed to achieve widespread retinal transduction.

Jianda Jiuzhou: AAV Gene Therapy

IND Application for JianDa JiuZhou GA002 Injection Accepted. GA002 is a gene therapy drug using AAV as the delivery vector, targeting focal refractory epilepsy. By selectively expressing inhibitory receptors in the lesion area target cells, it aims to transform refractory epilepsy into a drug-controllable state.

Cosmic Biotech: INHBE Targeting siRNA

CMS-D008 Injection from Kangzhe Biotech Approved for Clinical Trials, Aiming to Treat Overweight or Obesity. CMS-D008 is a subcutaneously injectable siRNA targeting INHBE inhibition, which can function by downregulating

INHBE

The level of the gene and its encoded protein Activin E reduces the activation of the fat metabolism pathway Activin E-ALK7, effectively decreasing lipid accumulation.

In the future, it can be developed to treat overweight/obesity, abdominal obesity, and related metabolic diseases. Preclinical studies suggest that CMS-D008 efficiently and continuously inhibits INHBE expression. In obese animal models, it achieves significant weight loss and fat reduction without affecting muscle, demonstrating good safety. It shows promising prospects for high-quality, long-term weight loss with fat reduction but no muscle loss.

HaiChang Biotech: ASO AKT-1 Inhibitor

HaiChang Bio's WGI-0301 Receives CDE Clinical Tacit Approval, with Proposed Indication for Use in Combination with Nivolumab to Treat Advanced Solid Tumors (Including Liver and Kidney Cancer).

WGI-0301 is a next-generation AKT-1 inhibitor developed based on QTsome™ nucleic acid delivery technology. By encapsulating antisense oligonucleotides (ASO) targeting AKT-1 within lipid nanoparticles (LNP), it can specifically bind to AKT-1 mRNA and downregulate its protein expression, demonstrating unique mechanistic advantages and application potential in anti-tumor therapy.

Source: eMedClub News

Join the reader communication group:

Contact Us:wbfsh@staff.weibo.com