Jinlan Gene Submits IPO Prospectus Highlighting AAV Gene Therapy Pipeline for Rare Diseases
Genecradle
Gene Therapy Drug Developer
Genecradle's Wilson's Disease AAV Gene Therapy GC310 Injection Receives IND Approval
On February 6, 2025, the gene therapy drug GC310 Adeno-Associated Virus Injection ("GC310 Injection") independently developed by Beijing Genecradle Therapeutics Co., Ltd. received implied permission for registration clinical trials from the National Medical Products Administration, with an indication for Wilson's disease. GC310 Adeno-Associated Virus Injection is the fourth Class 1 new gene therapy product to receive IND approval from Genecradle.
GC310 Injection is an AAV gene therapy drug independently developed by Genecradle for the treatment of Wilson's disease. Non-clinical studies have demonstrated good drug safety and significant efficacy. A single treatment enables the target tissue to express the miniATP7B copper transporter with biological function, restoring copper metabolism capacity and increasing ceruloplasmin levels, which is expected to improve the condition of the Wilson's disease community over the long term.
Article link:Genecradle's Wilson's Disease AAV Gene Therapy GC310 Injection Receives IND Approval
Belief BioMed's AAV Gene Therapy BBM-D101 Injection for Duchenne Muscular Dystrophy IND Accepted by NMPA
On February 5, 2025, Belief BioMed, an innovative enterprise focusing on cutting-edge gene therapy, announced that the clinical trial application (IND) for BBM-D101 injection, a gene therapy drug for Duchenne Muscular Dystrophy (DMD), has been accepted by the National Medical Products Administration (NMPA). Previously, the IND for BBM-D101 injection in the United States had already been approved by the FDA.
This clinical trial is an open-label, single-dose study designed to evaluate the safety and efficacy of intravenous infusion of BBM-D101 injection in 4-9-year-old boys with DMD. BBM-D101 utilizes an engineered capsid with higher muscle tissue delivery efficiency, and the proposed clinical dose is lower than that of similar adeno-associated virus (AAV) gene therapy products already marketed abroad, with expectations of good safety.
Sanfilippo Syndrome AAV Gene Therapy UX111 Significantly Improves Developmental Function in Children
According to Global News Agency, Ultragenyx Pharmaceutical Inc. announced new data on February 5, 2025, showing that treatment with UX111 (ABO-102) AAV gene therapy resulted in statistically significant improvements in Bayley-III raw scores in cognitive, receptive communication, and expressive communication subdomains compared to the natural history data of untreated patients with Sanfilippo Syndrome Type A (MPS IIIA). These clinical endpoints were associated with a substantial and sustained reduction in heparan sulfate (HS) levels in cerebrospinal fluid (CSF).
UX111 is an innovative in vivo gene therapy currently in Phase 1/2/3 development for the treatment of MPS IIIA, a rare and fatal lysosomal storage disorder with no approved treatments available, primarily affecting the brain. UX111 is designed to be administered via a single intravenous infusion, utilizing a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells. The therapy aims to address the underlying SGSH enzyme deficiency that causes abnormal accumulation of HS (a glycosaminoglycan) in the brain, leading to progressive cellular damage and neurodegeneration. The UX111 program has received Regenerative Medicine Advanced Therapy, Fast Track, Pediatric Rare Disease, and Orphan Drug designations in the United States, as well as PRIME and Orphan Drug designations in the European Union.
Viralgen Collaborates with Axovia to Develop AAV Gene Therapy for Treating Bardet-Biedl Syndrome
Recently, Viralgen and Axovia Therapeutics announced that they will join hands to advance the development and manufacturing of an investigational gene therapy based on AAV9, aiming to treat retinal atrophy in patients with Bardet-Biedl Syndrome (BBS) caused by biallelic mutations in the BBS1 gene. This collaboration will provide patients with an innovative investigational treatment that has the potential to halt photoreceptor cell death and retinal degeneration, thereby preventing vision loss.
Bardet-Biedl Syndrome (BBS) is a genetic disorder that causes multi-systemic dysfunction, where obesity and progressive retinal dystrophy are among the most devastating symptoms. The condition occurs in individuals with biallelic mutations in the BBS1 gene, leading to progressive vision loss and complete blindness, along with other manifestations.
Article link:Viralgen Collaborates with Axovia to Develop AAV Gene Therapy for Treating Bardet-Biedl Syndrome
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First-in-class gene therapy ABO-101 for Type 1 Primary Hyperoxaluria receives FDA Orphan Drug and RPDD designations
According to Global News Agency, Arbor Biotechnologies announced on February 5, 2025, that the U.S. FDA has granted its ABO-101 Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for the treatment of Primary Hyperoxaluria Type 1 (PH1). These designations follow the FDA's approval in December 2024 of the Investigational New Drug (IND) application for this innovative gene-editing therapy.
ABO-101 is an innovative experimental gene-editing drug designed to serve as a one-time liver-targeted gene-editing therapy. It aims to permanently inactivate the HAO1 gene in the liver to reduce oxalate production associated with PH1. PH1 is a rare genetic disorder where the lack of a liver enzyme leads to excessive oxalate production and accumulation, ultimately causing kidney stones, which can progress to end-stage renal disease and systemic oxalosis. ABO-101 is intended to knock down the expression of the HAO1 gene in the liver, thereby achieving a sustained reduction in oxalate production.
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Limb-Girdle Muscular Dystrophy Gene Therapy SRP-9003 Phase III Clinical Trial Completes Patient Enrollment and Dosing
Sarepta Therapeutics announced that the Phase 3 clinical trial EMERGENE (Study SRP-9003-301) for its gene therapy drug SRP-9003 (bidridistrogene xeboparvovec), targeting Limb-Girdle Muscular Dystrophy Type 2E/R4 (LGMD2E/R4), has completed patient enrollment and dosing.LGMD2E/R4 is a rare hereditary neuromuscular disorder caused by the absence of β-sarcoglycan, and SRP-9003 aims to restore the expression of this protein through gene therapy.
EMERGENE (Study SRP-9003-301) is a Phase 3, multinational, open-label study designed to evaluate the efficacy and safety of SRP-9003 in treating LGMD2E/R4 patients aged 4 years and older.The primary endpoint of the study is the expression level of β-sarcoglycan protein 60 days after administration.Secondary endpoints include functional assessments and safety monitoring within 60 months.The research data is expected to be released in the first half of 2025.
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Familial Chylomicronemia Syndrome siRNA New Drug VSA001 Accepted for Marketing by NMPA
On January 26, 2025, Viazen Biotechnology (Suzhou) Co., Ltd. announced that the marketing authorization application for its APOC3-targeted siRNA Class 1 new drug – VSA001 Injection (Pulesiran Sodium) has been officially accepted by the NMPA for the treatment of Familial Chylomicronemia Syndrome (FCS). Currently, there are no approved drugs for this disease available in China. Pulesiran Sodium was previously included in the NMPA’s Breakthrough Therapy Designation list and Priority Review and Approval process to expedite the product’s approval and market launch in China.
VSA001 is a liver-targeted small interfering RNA (siRNA) drug that effectively and persistently silences the mRNA level of apolipoprotein C-III (APOC3) to reduce the expression of APOC3 protein. Through both lipoprotein lipase-dependent and -independent pathways, it significantly lowers serum TG levels, as well as triglyceride-rich lipoproteins and their degradation remnants.
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Microglow Gene Completes New Round of Tens of Millions of Financing, Developing VLP/LNP Delivery for In Vivo Epigenetic Editing
Recently, Firefly Gene (Suzhou) Co., Ltd. announced the successful completion of its Pre-A round of financing, with a total funding amount reaching tens of millions of RMB. This round of financing was jointly invested by Guangzhou Industrial Investment Group and Guangzhou Youjiu Investment. The funding will help Firefly Gene rapidly advance its epigenetic editing pipeline research for common diseases with unmet clinical needs.