According to incomplete statistics, currently,China Has Approved 37 AAV Gene Therapy Drug IND ApplicationsAmong them, at least 3 have entered Phase III clinical trials.According to its INDSummary of Approval Timeline in Chronological Order:
1. StarryGene's XMVA09
On March 1, 2024, the Investigational New Drug (IND) application for "XMVA09 Injection," a Class I innovative gene therapy drug independently developed by StarryGene (Hefei StarryGene Biotechnology Co., Ltd.), was approved by the National Medical Products Administration (NMPA). XMVA09 Injection is the first gene therapy drug that combines dual-specificity targets with an intravitreal injection capsid, indicated for wet age-related macular degeneration (wAMD).
XMVA09 Injection is a gene therapy drug independently developed by StarryGene. Compared with the 1-3 month injection cycle of traditional antibody therapy drugs, gene therapy drugs can produce sustained therapeutic effects, with the potential to achieve lifelong efficacy with a single injection, greatly benefiting patients. XMVA09 Injection uses a novel AAV capsid, and through intravitreal injection, it can infect retinal pigment epithelium (RPE) cells closely adjacent to the choroidal lesion, providing a more convenient route for subsequent clinical applications. Additionally, XMVA09 Injection employs a bispecific design that simultaneously targets vascular endothelial growth factor (VEGF) and angiopoietin-2 (ANG-2), enhancing the drug's therapeutic efficacy and expanding its coverage to include patients who are insensitive to VEGF. The IIT study previously conducted at the First Affiliated Hospital of the University of Science and Technology of China demonstrated promising clinical outcomes for XMVA09 Injection. Among the six enrolled wAMD subjects, all of whom had not received anti-VEGF prospective loading treatment prior to dosing, excellent therapeutic effects were still observed. The first patient, who has been clinically followed up for nearly a year after treatment, showed a significant increase in BCVA. The subject with the best clinical benefit (high-dose group) exhibited an impressive improvement of 17 letters in BCVA from baseline at the most recent follow-up. The interim results of the IIT indicate that XMVA09 Injection is safe, well-tolerated, and has preliminarily met the expected research goals for evaluating efficacy.On February 22, 2024, the Investigational New Drug (IND) application for "JWK001 Injection," a Class I innovative gene therapy drug independently developed by Chengdu Jinweike Biotechnology Co., Ltd. ("Jinweike") for neovascular age-related macular degeneration (nAMD), was successfully approved by the National Medical Products Administration (NMPA).
JWK001 Injection is the first AAV gene therapy new drug that adopts the "two-plasmid packaging system." It is reported that the two-plasmid suspension HEK293 cell packaging technology independently developed by Jinweike Biotechnology significantly improves the AAV packaging efficiency and reduces production costs. JWK001 Injection continuously and efficiently expresses anti-VEGF protein in retinal cells by delivering an AAV vector carrying a newly self-designed anti-VEGF protein expression cassette. This gene therapy approach avoids risks associated with traditional nAMD treatments, such as damage to ocular tissues caused by repeated intravitreal injections and poor patient compliance, achieving the goal of a one-time treatment with lifelong effectiveness.
3. CHIGENOVO's ZVS203e
On February 7, 2024, CHIGENOVO Co., Ltd.'s first ophthalmic gene-editing Class 1 innovative drug—ZVS203e Injection—received the implied permission for clinical trial (IND) from the Center for Drug Evaluation (CDE) of the National Medical Products Administration. ZVS203e Injection is a Class 1 innovative drug developed using AAV as a vector and based on gene-editing technology, intended for treating retinitis pigmentosa caused by RHO gene mutations.
ZVS203e Injection utilizes the third-generation artificial nuclease CRISPR/Cas9 to perform targeted editing on the mutated RHO gene, addressing the root cause of the disease by targeting the pathogenic gene for treatment, achieving a one-time administration with lifelong curative effects. The drug received FDA Orphan Drug Designation in July 2022 and completed the world's first injection administration in an RP patient with RHO gene mutation in an investigator-initiated clinical study (IIT) conducted at Peking University Third Hospital in September 2023, demonstrating good safety and efficacy in the first patient.
4. R&B Bio's ZS805
On January 31, 2024, ZS805 Injection, a gene therapy product for Fabry disease independently developed by Sichuan Real&Best Biotech Co., Ltd., has received clinical trial implied permission and is about to enter Phase I/II clinical trials. R&B Bio has become the first pharmaceutical company in China to receive clinical approval for an original gene therapy drug for Fabry disease.
S805 is a novel Class 1 rAAV gene therapy drug developed for the treatment of Fabry disease. The gene expression cassette framework of ZS805 incorporates the world's smallest liver-specific promoter independently developed by R&B Bio, along with an optimized signal peptide sequence for the α-galactosidase A gene. This ensures specific expression and efficient secretion of the α-galactosidase A protein in liver cells, enhancing the drug’s safety and efficacy. To ensure that more patients can receive effective treatment, R&B Bio selected an AAV vector serotype suitable for the Chinese population. Evaluation in animal models has shown that the distribution of α-galactosidase A protein expressed by ZS805 in disease-related organs such as the heart and kidneys surpasses that of foreign competitors. In the IIT study conducted at West China Hospital, two patients have been enrolled to receive ZS805 gene therapy. Currently, the treated patients are in good overall condition, with improvements in various indicators. The galactosidase activity in the patients' bodies has reached normal levels, preliminarily confirming the safety and efficacy of ZS805 in humans.
5. Lingyi Biotech's LY-M001
On January 16, 2024, Lingyi Biotech Co., Ltd. (hereinafter referred to as "Lingyi Biotech") independently developed a Class I therapeutic biological product, LY-M001 Injection, and its Investigational New Drug (IND) application was successfully granted implied approval by the National Medical Products Administration (NMPA) (Acceptance No.: CXSL2300730).
LY-M001 Injection is China's first self-developed AAV gene therapy drug targeting Type I or Type III Gaucher disease. This product uses recombinant adeno-associated virus (rAAV) as a vector, and after a single intravenous infusion, it expresses the glucocerebrosidase required by patients. Lingyi Biotech has developed a proprietary improved glucocerebrosidase gene therapy vector with full independent intellectual property rights, enabling long-term stable expression in vivo and degradation of harmful glycolipid metabolites, thereby achieving the goal of long-term treatment for Gaucher disease. The IIT clinical research project led by Dean Huang He of the First Affiliated Hospital of Zhejiang University School of Medicine has officially started and completed the first patient dosing. Preliminary IIT data shows that LY-M001 demonstrates good performance in terms of efficacy and safety, with no adverse events reported.
6. Skyline Therapeutics' SKG0201
In December 2023, the IND for SKG0201 Injection, an AAV gene therapy drug under research by Skyline Therapeutics, was approved by the National Medical Products Administration (NMPA) to initiate a Phase I clinical trial for the treatment of Type I Spinal Muscular Atrophy (SMA).
SKG0201 Injection is a next-generation SMN1 gene replacement therapy designed for single-dose intravenous administration to treat Type I SMA. Its innovative vector design incorporates a human SMN1 cDNA sequence optimized with a unique CNS-specific promoter and fully optimized codons. This cutting-edge design achieves better tissue targeting, enabling the normal SMN1 gene to exert maximum therapeutic effects in the central nervous system (CNS) region after being introduced into the body. At low doses, it rapidly restores the expression of normal SMN protein in motor neurons, thereby improving the function of affected cells, such as motor neurons, more safely and effectively. Addressing the clear pathogenic mechanism of SMA and the limitations of existing drugs in terms of safety, SKG0201, this comprehensively innovative SMN1 gene replacement therapy, has demonstrated superior efficacy and safety in preclinical studies. In an investigator-initiated clinical study conducted by Xinhua Hospital affiliated with Shanghai Jiao Tong University School of Medicine for the treatment of Type I SMA, SKG0201 Injection has completed subject enrollment across multiple dose groups and shown favorable tolerability and therapeutic effects in continuous follow-up assessments.
7、Frontera Therapeutics' FT-002
On November 6, 2023, FT-002 Injection, an innovative gene therapy drug independently developed by Frontera Therapeutics, Inc. (referred to as "Frontera Therapeutics"), received clinical trial approval from the CDE, intended for the treatment of X-linked Retinitis Pigmentosa (XLRP) patients caused by RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutations.
FT-002 Injection is aRecombinant Adeno-Associated Virus (rAAV)Gene therapy drugs using rAAV as a vector to carry the target gene are administered via intravitreal injection, enabling retinal cells to express active RPGR protein. This protein participates in the ciliary transport function of photoreceptors, rescuing photoreceptor cell loss caused by RPGR gene mutations and improving patients' visual function or slowing the progression of vision loss. The preliminary results of the IIT study on FT-002 injection are encouraging. Objective indicators such as retinal thickness in the macular area, visual field, and visual acuity showed significant improvement after administration. In some patients, a marked increase in best-corrected visual acuity (BCVA) from baseline was observed two months post-surgery (measured by the ETDRS chart).
8、RRGENER's RRG001
On November 6, 2023, Shanghai RRGENER Biotechnology Co., Ltd. ("RRGENER") announced that the clinical trial application (IND) for its self-developed RRG001 intravitreal injection has been approved by the CDE.RRG001 Intravitreal InjectionThe injection is RRGENER's firstApplicable toAge-related Wet Macular Degeneration (nAMD)TheGene therapy drugs.
RRG001 Intravitreal Injection is a recombinant adeno-associated virus (rAAV) gene therapy drug independently developed by RRGENER. It delivers the epidermal growth factor receptor (VEGFR) fusion protein gene into the subretinal space of patients with neovascular age-related macular degeneration (nAMD), enabling retinal cells to act as protein factories that continuously express the VEGFR fusion protein required by the patient. This avoids the drawbacks of frequent dosing associated with traditional antibody therapies, aiming for a "one-time administration, long-term effectiveness" treatment for macular degeneration. In February 2023, this drug entered an investigator-initiated clinical trial (IIT study) at Jiangsu Provincial People's Hospital. Preliminary clinical research data showed significant clinical benefits for participants, with no serious adverse events reported.
9. Skyline Therapeutics' SKG0106
In October 2023, the AAV ophthalmic gene therapy SKG0106 developed by Skyline Therapeutics was approved for clinical trials by the National Medical Products Administration (NMPA) in China, with an indication for neovascular age-related macular degeneration (nAMD). Additionally, SKG0106 received FDA approval in June this year to initiate a global Phase I/IIa clinical trial for the treatment of nAMD.
SKG0106 is an innovative ophthalmic gene therapy drug under research. This therapy uses recombinant AAV as a vector to express a unique anti-vascular endothelial growth factor (VEGF) protein. The route of administration is intravitreal injection, offering long-lasting effects with a single dose.Potential for effective term.
10. Suzhou NoJieBei Biotechnology Co., Ltd.
In September 2023, Suzhou NoJieBei Biotechnology Co., Ltd.'s AAV therapy NGGT001 was approved for clinical trials by the National Medical Products Administration. NGGT001 is an innovative gene therapy drug developed by NoJieBei Biotechnology for the treatment of Bietti's Crystalline Dystrophy (BCD).
NGGT001 is an investigational gene therapy based on the rAAV2 vector, which treats BCD by expressing a codon-optimized CYP4V2. In wild-type mice and Cyp4v3 knockout mouse models, a single subretinal injection of NGGT001 achieved stable vector transduction and long-term expression of the CYP4V2 protein in the eye chamber. In adult cynomolgus monkeys, a single subretinal injection of NGGT001 showed robust vector distribution and sustained CYP4V2 mRNA expression in retinal and choroidal tissues. Moreover, in a 13-week toxicology study, this therapy demonstrated good tolerability through subretinal administration.。
11、Janssen's JNJ-81201887
In early August 2023, Janssen Pharmaceuticals, a subsidiary of Johnson & Johnson, received the CDE's implied permission for clinical trials of its AAV gene therapy JNJ-81201887 (AAVCAGsCD59). The therapy is being developed to treat adult patients with geographic atrophy secondary to age-related macular degeneration.
JNJ-81201887 (JNJ-1887, AAVCAGsCD59) is an investigational gene therapy based on the AAV vector, designed to increase the expression of soluble CD59 (sCD59), protecting retinal cells and thereby slowing and preventing disease progression.
12、Shanghai Belief-Delivery BioMed Co.,Ltd.TheBBM-H803
On July 24, 2023, the clinical trial application for "BBM-H803 Injection" submitted by Shanghai Belief-Delivery BioMed Co., Ltd. and Shanghai Mianyi Biotechnology Co., Ltd., wholly-owned subsidiaries of Belief BioMed (BBM), received tacit approval for clinical trials. The indication is Hemophilia A.
BBM-H803 is an AAV gene therapy drug independently developed by Belief BioMed. It delivers the human coagulation factor VIII gene into patients with hemophilia A through intravenous administration, thereby increasing and maintaining the level of coagulation factor in patients over the long term.
13. VGM-R02b by Shanghai Tianze Yuntai Biomedical Co., Ltd.
On July 13, 2023, Shanghai Tianze Yuntai Biomedical Co., Ltd. (referred to as "Skyline TherapeuticsVGM-R02b, independently developed by R&B Bio, has been approved by the National Medical Products Administration to conduct clinical trials for the treatment of Glutaric Acidemia Type I.
VGM-R02b is a potential treatment to prevent the progression of severe or life-threatening diseases caused by glutaric acidemia in infants and children. It is also the world's first gene therapy product for Glutaric Acidemia Type I (GA-I). This therapy is based on the principle of gene replacement.Recombinant Adeno-Associated Virus (rAAV) as a vector, the therapy wasOn May 25, 2022, it was granted Rare Pediatric Disease Designation (RPDD) by the FDA for the treatment of GA-I.
14、 Frontera Therapeutics' FT-004
On July 12, 2023, Frontera Therapeutics, developed by Frontera Therapeutics, Inc. (Suzhou), targetedHemophilia B(Endogenous FIX activity ≤2%)) ofAAV Gene Therapy Drug FT-004 Receives CDE Clinical Trial Approval
FT-004 is a gene therapy drug based on the AAV vector. Preclinical data shows that FT-004 can efficiently enter liver cells, sustainably and stably express and secrete functional hFIX protein into the blood, effectively enhance the coagulation ability of model animals over the long term, and has demonstrated good safety.On June 28, 2023, Sichuan Real&Best Biotech Co., Ltd. (hereinafter referred to as "R&B Bio") received CDE approval for its AAV gene therapy drug "ZS802 Injection" for Hemophilia A, and will soon commence clinical Phase I/II trials.
ZS802 is an AAV gene therapy drug independently developed by R&B Bio. It utilizes the company's self-developed smallest liver-specific promoter globally, solving the challenge of packaging large gene capacity and significantly improving product quality. Additionally, ZS802 incorporates an optimized and modified FVIII gene sequence developed by R&B Bio, effectively enhancing its efficacy. Currently, the ZS802 project has initiated IIT research at the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, with preliminary results demonstrating the drug’s safety and efficacy.16. EXG102-031 by Jiayin Biotech
On June 1, 2023, the AAV gene therapy drug EXG102-031 ophthalmic injection developed by Hangzhou Jiayin Biotechnology Co., Ltd. ("Jiayin Bio") was approved by the CDE for the indication of wet age-related macular degeneration (wAMD).
This therapy uses AAV as a vector to overexpress a therapeutic fusion protein, which can bind/neutralize all known vascular endothelial growth factor (VEGF) and angiopoietin-2 (ANG2) subtypes, theoretically providing long-term efficacy with a single administration.April 27, 2023, NorthAnlong Biopharmaceuticals Co., Ltd. (hereinafter referred to as "Anlong Bio") announced that the IND for its ophthalmic gene therapy product "AL-001 Ophthalmic Injection" has been approved by the CDE, with the indication for: wet age-related macular degeneration (wAMD). On May 29, 2023, Anlong Bio reported that the project had received approval from the Institutional Project Initiation and Ethics Committee of Peking Union Medical College Hospital, the leading institution for this research, marking the official entry of this project into the clinical trial phase.
The therapy is in China forwAMDThe first approved gene therapy administered via suprachoroidal space (SCS) injection,This therapyOverexpression of anti-VEGF protein using AAV as a vector is theoretically effective for an extended period with a single administration. This product utilizes an advanced, self-developed rAAV production process employing Sf9 suspension cells free of helper viruses.18、Frontera Therapeutics' FT-003
On April 26, 2023, Frontera Therapeutics announced that the Investigational New Drug (IND) application for FT-003 injection was approved by the CDE.LinBed Test LicenseThe product is indicated for neovascular age-related macular degeneration (nAMD).
FT-003 Injection is a novel recombinant adeno-associated virus gene therapy drug. Preclinical study data show that after the injection of FT-003, it can efficiently infect multi-layer retinal cells in animals, enabling sustained expression and secretion of anti-angiogenic factors, reducing vascular endothelial permeability, and inhibiting the formation of new blood vessels. A single injection in patients is expected to achieve long-term efficacy.
Moreover,FT-003 for the treatment of Diabetic Macular Edema (DME) IND application has also beenObtainCDELinClinical Trial Approval.
On May 19, 2023, the team led by Professor Li Xiaorong from the Ophthalmology Hospital of Tianjin Medical University successfully completed the first patient dosing in China for FT-003 in the treatment of DME.
19. Hui-Gene TherapeuticsTheHG004
On April 18, 2023, Hui-Gene Therapeutics (Shanghai) Biotechnology Co., Ltd. ("Hui-Gene Therapeutics") announced thatThe IND for HG004, the company's first self-developed ophthalmic AAV gene therapy drug, has been approved by the CDE for the indication of Leber's Congenital Amaurosis Type 2 (LCA2). Additionally, in January this year, HG004 received IND approval from the FDA.
HG004 is an AAV-mediated gene replacement therapy designed to treat RPE65 mutation-associated retinopathy. Mutations in the RPE65 gene may lead to severe early-onset childhood retinal dystrophy, early-onset severe retinal dystrophy, Leber congenital amaurosis (LCA), or retinitis pigmentosa (RP). The AAV vector used in HG004 achieves a transduction efficiency in retinal pigment epithelium that is at least 10 times higher than that of AAV2, with an effective starting dose much lower than the marketed AAV2-hRPE65 (LUXTURNA). In the IIT clinical study conducted at Xinhua Hospital in Shanghai, China, HG004 demonstrated significant clinical efficacy, with patients experiencing substantial recovery in vision.April 14, 2023Neurophth Biotechnology Co., Ltd.The IND application for AAV gene therapy drug NFS-02 ophthalmic injection has received tacit approval from the CDE, with the indication being Leber's hereditary optic neuropathy (G3460A). Additionally, last year, NFS-02 received FDA's new drug clinical trial (IND) approval.NFS-02 is a novel intravitreal gene therapy drug based on AAV2. The therapeutic gene can be delivered to the patient's damaged retinal ganglion cells through a single intravitreal injection of the drug, repairing the mitochondrial bio-respiratory chain damaged by genetic mutations, thereby restoring visual function to the retinal ganglion cells.On January 16, 2023, the clinical trial application for GS1191-0445 injection, an AAV gene therapy drug for the treatment of Hemophilia A developed by Gritgen Therapeutics Co., Ltd, was approved by the CDE.Gritgen officially submitted the IND application for this therapy to the CDE in November 2022 and received acceptance, becoming the hemophilia A treatment drug in China to enter the IND stage. In addition, the drug has initiated IIT research at the Tianjin Blood Research Institute., 9 patients have been enrolled, and after medication, good safety and exciting efficacy data were obtained.22、Innostellar BiotherapeuticsLX102On December 23, 2022, Innostellar Biotherapeutics announced that its subsidiary, Innostellar Biotherapeutics Co., Ltd. (Suzhou)(Innostellar Biotherapeutics)The IND application for the AAV gene therapy product LX102 injection has been approved by the CDE for the treatment ofWet Age-related Macular Degeneration (wAMD).This therapy is based on AAV vector-mediated in vivo gene therapy.Method, can express anti-VEGF fusion proteinThe target gene is introduced into the patient's retinal cells, and theoretically, a single administration can have long-term efficacy. In previously conducted IIT clinical studies, this therapy demonstrated good safety and effectiveness.。On December 20, 2022, Beijing Genecradle Therapeutics Co., Ltd. (Genecradle)'s AAV gene therapy drug GC301The IND application for the adeno-associated virus injection has received tacit approval from the CDE, with the indication being early-onset Pompe disease (IOPD). On June 2, 2023, the Phase I/II clinical trial kick-off meeting for GC301 was successfully held at Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, marking the official launch of the clinical trial for GC301 injection in treating infantile Pompe disease at the main research center.Its treatment strategy is: after a single intravenous infusion, the therapeutic gene can be widely expressed throughout the body, with the aim of compensating for GAA enzyme gene defects in tissues such as the liver, muscles, and central nervous system. GC301 has demonstrated good safety and efficacy in an investigator-initiated clinical study (IIT).2022December 6Recently, the IND application for "ZVS101e Injection" by CHIGENOVO Co., Ltd. (referred to as "CHIGENOVO") received tacit approval from the CDE, with the indication being crystalline retinal degeneration (carrying CYP4V2 biallelic mutations). On February 20, 2023, the Phase I/II clinical trial of ZVS101e Injection was successfully completed with the first subject dosed at Tianjin Medical University Eye Hospital.ZVS101e is a gene replacement therapy using AAV8 as the vector.The therapy, which targets the same indications and treatment principles as VGR-R01 developed by Shanghai Tianze Yuntai Biomedical Co., Ltd. that was approved for clinical trials in November 2022.November 2022, Beijing GenecradleGenecradle's AAV gene therapy drug GC304 Adeno-Associated Virus Injection ("GC304 Injection") IND application received implied permission from CDE for the treatment of hypertriglyceridemia with recurrent acute pancreatitis.GC304 Gene Drug-Loaded Therapeutic BaseBecause of the lipoprotein lipase (LPL) gene, LPL is a key enzyme in hydrolyzing triglycerides (TG) in plasma lipoproteins.26. KH631 by Chengdu Hongji Biotechnology Co., Ltd.November 15, 2022HealthChengdu Hongji Biotechnology Co., Ltd. (Hongji Bio), a subsidiary of Staidson, has received the National Medical Products Administration's (NMPA) tacit approval for five Investigational New Drug (IND) applications for its AAV gene therapy drug "KH631 Ophthalmic Injection," intended for treating neovascular (wet) age-related macular degeneration (wetAMD). In early May 2023, Professor Wenbin Wei's team from Beijing Tongren Hospital, Capital Medical University, completed the first patient dosing in the Phase I clinical trial of KH631 for AMD treatment.27. VGR-R01 by Shanghai Tianze Yuntai Biomedical Co., Ltd.On November 1, 2022, the IND application for VGR-R01 injection, an AAV gene therapy drug developed by Shanghai Tianze Yuntai Biomedical Co., Ltd. (Tianze Yuntai), received tacit approval from the CDE. Its indication is crystalline retinal dystrophy (Bietti crystalline dystrophy, BCD) caused by CYP4V2 gene mutations.(Full text link:Shanghai Tianze Yuntai Biomedical Co., Ltd.'s BCD Gene Therapy Drug IND Approved）。In October 2022, the AAV gene therapy drug GC101 Adeno-Associated Virus Injection ("GC101 Injection") developed by Beijing Genecradle Therapeutics Co., Ltd. received implied permission for clinical trials from the CDE, with the clinical indication being Type 1 Spinal Muscular Atrophy (Type 1 SMA). On November 15 of the same year, the IND application for "GC101 Injection" for Type 2 SMA indication also received implied permission from the CDE.29. Frontera Therapeutics' FT-001In September 2022, Frontera Therapeutics' IND application for FT-001 injection, a gene therapy drug targeting RPE65 biallelic variant hereditary retinal degeneration, received tacit approval from the CDE. On January 9, 2023, Frontera Therapeutics announced that the first patient dosing of FT-001 injection was completed at Peking Union Medical College Hospital (full text link:Frontera Therapeutics' FT-001 Injection Completes First Patient Dosing）。On September 1, 2022, the clinical application of ZS801 Injection from Sichuan Real&Best Biotech Co., Ltd. was approved by the CDE for the control and prevention of bleeding in male patients aged 18 years and above with severe or moderately severe Hemophilia B (congenital Factor IX deficiency, Factor IX <2%).This is an AAV gene therapy. The serotype of the AAV vector used in this therapy has very low pre-existing neutralizing antibodies in patients, allowing it to cover more patients in China.。31. EXG001-307 by Jiayin BiotechOn June 21, 2022, Jiayin Bio announced that the clinical trial application for its self-developed AAV gene therapy EXG001-307 injection was approved by the CDE for the treatment of Type 1 Spinal Muscular Atrophy (Type 1 SMA) with bi-allelic mutations (deletions) in the Survival Motor Neuron 1 (SMN1) gene.This is the first gene therapy product for the treatment of Type 1 SMA via intravenous injection approved for registration clinical trial in China (Full text link:Another AAV Gene Therapy IND Clinical Trial Approved by CDE in China）。EXG001-307 is an AAV-based gene therapy that has the potential to be effective for a long term with a single administration. Its mechanism of action and usage are similar to Novartis' Zolgensma. Hangzhou Jiayin has adopted an innovative design for EXG001-307, aiming to reduce side effects on the heart and liver, thereby better exerting its therapeutic effect.32. Shanghai Tianze Yuntai Biomedical Co., Ltd.VGB-R04In April 2022, VGB-R04 Injection, the first AAV gene therapy candidate independently developed by Shanghai Tianze Yuntai Biomedical Co., Ltd., received clinical trial approval for the treatment of Hemophilia B caused by congenital Factor IX deficiency.VGB-R04 is administered intravenously, delivering the target gene (therapeutic gene) to the liver cell nucleus mediated by the AAV capsid. It expresses a variant of coagulation factor IX (hFIX Padua) protein in liver cells. FIX-Padua is a naturally occurring, highly active FIX variant (R338L) with activity approximately eight times that of normal wild-type FIX. This means it can achieve normal blood clotting function at lower expression levels, thereby reducing the dosing of the viral vector and enhancing the safety and efficacy of the viral vector administration.33、Innostellar BiotherapeuticsLX101In April 2022, LX101 Ophthalmic Injection, an AAV2-RPE65 gene therapy formulation independently developed by Shanghai Innostellar Biotherapeutics Co., Ltd. (under Innostellar Biotherapeutics), received clinical trial approval for the treatment of patients with inherited retinal degeneration (IRD) associated with biallelic RPE65 mutations.The gene therapy LX101, using AAV2 as a vector, delivers the functionally normal and sequence-optimized RPE65 gene into retinal cells in the body to compensate for the protein dysfunction caused by mutations in this gene, thereby restoring vision. In previously conducted investigator-initiated clinical studies, this gene therapy demonstrated good safety and efficacy.34. Novartis' OAV101 (Already entered Phase III clinical trials)In January 2022, Novartis (NYSE: NVS) received clinical trial approval in China for its SMA gene therapy drug OAV101 injection (Zolgensma). On April 29, 2022, relevant databases showed that this therapy had initiated clinical trials in China for the first time, with plans to enroll 20 participants in China as part of the global Phase 3 clinical STEER study. On the afternoon of June 20, 2022, Novartis' gene therapy (OAV101) clinical trial commenced in China.The kick-off meeting of the research center was successfully held at Peking University First Hospital, the leading unit. This clinical trial is the China segment of the global Phase III clinical STEER study, led by Professor Xiong Hui from the Department of Pediatrics at Peking University First Hospital. It targets treatment-naïve patients aged 2 to 18 with Type 2 Spinal Muscular Atrophy (SMA).。
Zolgensma is a gene replacement therapy for the treatment of Type 1 SMA, which theoretically offers long-term or even lifelong efficacy with a single dose, serving as a one-time treatment option that addresses the root cause of the disease. This gene therapy uses an scAAV9 vector administered intravenously to deliver the normal SMN1 gene into the patient’s body, producing normal SMN1 protein and thereby improving the function of affected cells such as motor neurons. In contrast, the drugs Spinraza and Evrysdi, used for treating SMA, require repeated administration over the long term. Spinraza is administered via spinal injection every four months, while Evrysdi is an orally administered drug taken daily.35. Shanghai Belief-Delivery BioMed Co.,Ltd.BBM-H901 (Phase III Clinical Trial Initiated)In August 2021, BBM-H901 Injection, a gene therapy drug for the treatment of Hemophilia B independently developed by Shanghai Belief-Delivery BioMed Co., Ltd., a wholly-owned subsidiary of Belief BioMed, received clinical trial approval. By the end of December 2021, the clinical trial had successfully completed the first subject.Administration, which has currently entered Phase III clinical trials, is expected to be launched for sale in the second half of 2024 to the first half of 2025.
BBM-H901 is an intravenously administered AAV gene therapy. The target gene (therapeutic gene) carried by the AAV can overexpress human coagulation factor IX (hFIX), thereby increasing and maintaining the patient's coagulation factor levels over the long term.For the prevention of bleeding in adult male patients with hemophilia BBBM-H901 is one of the earliest AAV gene therapy drugs to enter clinical trials in China, having started IIT clinical research (NCT04135300) in 2019. The results of the IIT clinical research showed that this therapy has good safety and efficacy. After administration, the level of coagulation factor IX (hFIX) in the subjects significantly increased, with long-term stability of hFIX levels in the blood. The annualized bleeding rate of patients was significantly reduced, with no significant adverse reactions observed.36. NeurophthNR082 (Entered Phase III Clinical Trial)In March 2021, Neurophth's NR082 ophthalmic injection received Investigational New Drug (IND) approval from the National Medical Products Administration. On June 28, 2021, Neurophth announced that the first patient was enrolled and dosed in the Phase I/II/III first-stage clinical trial of NR082, China’s first in vivo AAV gene therapy for ocular indications. On February 22, 2023, Neurophth announced that all patients had been enrolled and dosed in the Phase III clinical trial of NR082 ophthalmic injection for the treatment of Leber’s Hereditary Optic Neuropathy (LHON) caused by ND4 mutations in China.。
NR082 Gene Therapy Drug Uses AAV as a Vector, used for Leber's Hereditary Optic Neuropathy (LHON) caused by ND4 mitochondrial gene mutation. The administration method is a single intravitreal injection, delivering the therapeutic gene through intravitreal cavity injection to the patient's damaged retinal ganglion cells, repairing the mitochondrial bio-respiratory chain, and restoring vitality and visual function to the retinal ganglion cells.37. StaidsonSTSG-0002 (Trial and subsequent development have been terminated)In mid-June 2019, the hepatitis B AAV gene therapy STSG-0002 was applied for clinical trial by Staidson(Beijing)Biopharmaceuticals Co.,Ltd. In mid-September 2019, it was approved by the National Medical Products Administration for clinical trials targeting chronic hepatitis B treatment. In August 2020, the first subject was dosed. On December 11, 2023, Staidson announced that based on preliminary research results from the clinical trial and a comprehensive evaluation of various factors including subsequent development investment, it was decided to terminate the clinical trial and subsequent development of the STSG-0002 injection, which was at the Ib/II phase at the time.STSG-0002 is an AAV vector-based DNA gene therapy drug utilizing RNA interference technology, developed by Staidson. Compared to non-viral vector small nucleic acid RNA drugs, this drug demonstrates significantly longer efficacy and is expected to provide long-term effectiveness with a single administration.Source: Official Account "Cell and Gene Therapy Field", official reports from listed companies
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Xiong'an Officially Issues! Support for the Development of Modern Life Sciences and Biotechnology Industries such as Cell Therapy, Genetic Engineering, and Tissue Engineering
Government Invests 6 Billion Yuan to Fund the Establishment of a Life Health Research Institute, Focusing on Cutting-edge Topics such as Cell and Gene Therapy
NVIDIA CEO Jensen Huang: The era of having to learn computer science and programming is over; the next golden track is life sciences and engineering.
First in China: Universal CAR-NK for the Treatment of Systemic Lupus Erythematosus Approved for Clinical Trials
Beijing to Prioritize Cell and Gene Therapy, Synthetic Biology Manufacturing, and Other Cutting-Edge Emerging Fields as Key Tasks for 2024
FDA Releases Guidelines for the Development of Gene-Edited Therapies and CAR-T Therapies (Full Text of Guidelines Attached)
CDE Releases "Technical Guidelines for Non-Clinical Research of Human Stem Cell Products"
CRISPR/Cas9 Gene Editing Therapy Receives FDA Approval for Treating Beta Thalassemia
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