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SineuGene Therapeutics’ SNUG01 Receives FDA Clearance for ALS Clinical Study
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Beijing-based SineuGene Therapeutics, a developer of gene therapies for brain disorders, announced last week that it has received clearance from the US Food and Drug Administration (FDA) to conduct a clinical study for its lead candidate SNUG01. This best-in-class (BIC) TRIM72-targeted gene therapy will be evaluated for the treatment of…
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AccurEdit Therapeutics Receives FDA Orphan Drug Designation for ATTR Treatment
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China-based AccurEdit Therapeutics (Suzhou) Co., Ltd announced receiving Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its investigational drug ART001, targeting transthyretin amyloidosis (ATTR). This marks a significant milestone in the development of this innovative gene-editing therapy. Drug Profile and MechanismART001 is the first non-viral…
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Frontera Therapeutics Receives NMPA Approval for FT-003 in Diabetic Retinopathy Study
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Sino-US firm Frontera Therapeutics, Inc. announced receiving approval from China’s National Medical Products Administration (NMPA) to initiate a clinical study for its FT-003, a recombinant adeno-associated virus (rAAV) gene therapy for diabetic retinopathy (DR). This follows previous approvals for studies in neovascular age-related macular degeneration (nAMD) and diabetic macular edema…
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Cell Store and Cygenta Forge Strategic Alliance in Cell and Gene Therapy Field
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Beijing-based biotech firms Cell Store, a biological sample cryopreservation technology platform, and Cygenta, a Contract Development and Manufacturing Organization (CDMO) specializing in cell and gene therapy (CGT), have entered into a long-term strategic alliance. Financial terms of the partnership were not disclosed. Partnership DetailsThe alliance aims to leverage Cell Store’s…
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YolTech Therapeutics’ YOLT-203 Shows Promise in Primary Hyperoxaluria Type 1 Trial
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YolTech Therapeutics, a Shanghai-based startup specializing in lipid nanoparticle delivery and gene editing, announced positive results from an investigator-initiated trial (ITT) of YOLT-203 in patients with primary hyperoxaluria type 1 (PH1). The therapy marks the first in vivo gene-editing treatment to demonstrate positive clinical data for PH1, showing excellent safety…
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Amoytop Biotech to Acquire Skyline Therapeutics’ Gene Therapy Assets for $15M Plus Milestones
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Xiamen-based Amoytop Biotech Co. Ltd (SHA: 688278) has announced plans to acquire partial assets of Skyline Therapeutics Limited, a clinical-stage gene therapy developer, excluding its U.S. subsidiary Skyline Therapeutics (US) Inc. The transaction includes a $15 million merger consideration plus up to $43 million in development, sales milestones, sub-license commissions,…
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Gene Cradle and Byongen Therapeutics Partner on Next-Generation DMD Gene Therapies
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Gene Cradle and Byongen Therapeutics, both Beijing-based gene therapy specialists, have formed a strategic alliance to develop next-generation gene therapies for Duchenne muscular dystrophy (DMD). Collaboration FocusGene Cradle, which initiated its DMD gene therapy project in 2021, has focused on optimizing Dystrophin proteins and regulatory elements. The company has designed…
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Boehringer Ingelheim Launches LENTICLAIR Study for CF Gene Therapy BI 3720931
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German pharmaceutical company Boehringer Ingelheim announced the initiation of the Phase I/II LENTICLAIR study for its gene therapy candidate BI 3720931 in cystic fibrosis (CF). The study is being conducted in collaboration with IP Group, the UK Respiratory Gene Therapy Consortium (GTC), and OXB. Study DetailsBI 3720931 is an inhaled…
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Bayer’s AskBio Receives RMAT Designation for AB-1005 in Parkinson’s Disease
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German pharmaceutical giant Bayer (ETR: BAYN) announced that its gene therapy subsidiary AskBio Inc. has received Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA) for its investigational gene therapy AB-1005 as a treatment for Parkinson’s disease (PD). Drug ProfileAB-1005 is an investigational gene therapy…
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Boehringer Ingelheim Partners with ExpressionEdits on Gene Therapy Tech
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German pharmaceutical giant Boehringer Ingelheim has entered into a collaboration and licensing agreement with UK-based ExpressionEdits. The deal leverages ExpressionEdits’ Genetic Syntax Engine technology to advance gene therapy development. Technology DetailsThe Genetic Syntax Engine is a proprietary AI-powered platform. It uses optimized introns to enhance gene expression without altering the…
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VectorBuilder and Eureka Bio Form Alliance to Advance CGT Solutions Globally
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China-based gene delivery technology developer VectorBuilder has formed a strategic alliance with compatriot firm Shenzhen Eureka Biotechnology Co., Ltd., a specialist in cell and gene therapy (CGT). The partnership, which leverages the companies’ respective expertise and successful track records in virus vector production, aims to deliver innovative solutions in the…
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Gene Cradle’s GC310 Receives Tacit Approval from NMPA for Wilson’s Disease
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Beijing-based gene therapy specialist Gene Cradle announced that it has received tacit clinical approval from the National Medical Products Administration (NMPA) for its GC310, an adeno-associated virus (AAV) gene therapy for hepatolenticular degeneration, also known as Wilson’s disease. This approval marks a significant step forward in the development of a…
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YolTech Initiates Study for YOLT-204 in Transfusion-Dependent Beta-Thalassemia
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Shanghai-based YolTech Therapeutics, a biotech startup specializing in lipid nanoparticle delivery and gene editing, has announced the initiation of a dosage escalation study to preliminarily assess the safety and efficacy of a single-dose regimen with YOLT-204 in transfusion-dependent beta-thalassemia (TDT). This marks a significant step forward in the development of…
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Belief BioMed’s BBM-D101 Receives FDA Approval for DMD Clinical Trial
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China-based gene therapy specialist Belief BioMed Inc. (BBM) has announced receiving clinical trial approval from the US Food and Drug Administration (FDA) for its BBM-D101. An open, single-dose clinical study is scheduled to commence, assessing the safety and efficacy of the gene therapy in boys aged 4 to 9 with…
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Reforgene Medicine Secures Hundreds of Millions in Financing for Gene Therapy Expansion
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Reforgene Medicine, a prominent gene editing therapy company headquartered in Guangzhou, has reportedly secured hundreds of millions of renminbi in a new financing round. This funding round was led by Guangzhou Industry Investment, with additional contributions from Guangzhou Financial Holdings, Technology Financial Holdings, and returning investor IFSC. The capital raised…
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Astellas Pharma Inks Licensing Deal with Sangamo Therapeutics for Neurological AV Capsid STAC-BBB
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Japan-based Astellas Pharma Inc. (TYO: 4503) has revealed a strategic licensing agreement with US-based Sangamo Therapeutics, Inc. (NASDAQ: SGMO). This collaboration will enable Astellas to utilize Sangamo’s novel proprietary neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, targeting an undisclosed neurological condition. Financial Terms and Potential Impact on Astellas’ PipelineThe agreement allows…
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HuidaGene Therapeutics Initiates HERO Trial for RNA-Editing Therapy HG204 in MECP2 Duplication Syndrome
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Shanghai-based HuidaGene Therapeutics has announced the first subject dosing of the HERO clinical trial, which is evaluating HG204, an RNA-editing therapy, for the treatment of MECP2 duplication syndrome (MDS). CRISPR RNA-Editing Therapy and Its MechanismThe CRISPR RNA-editing therapy utilizes a single adeno-associated virus vector to deliver HuidaGene’s proprietary high-fidelity RNA…
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GRIT Biotechnology and Vitalgen BioPharma Partner on CRISPR-AaCas12bMAX Cell Therapies
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China-based cell therapy specialists GRIT Biotechnology and Shanghai Vitalgen BioPharma Co., Ltd have entered into a collaboration agreement to leverage GRIT’s CRISPR-AaCas12bMAX gene editing tool and related gene editing techniques for the development, manufacturing, and commercialization of certain cell therapies. The agreement, for which no financial details were disclosed, highlights…
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AccurEdit Therapeutics’ ART001 Shows Over 90% TTR Reduction in ATTR Amyloidosis Trial
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Suzhou-based gene editing specialist AccurEdit Therapeutics has announced positive results from an ongoing investigator-initiated trial (ITT) for its in vivo gene editing product, ART001. This lipid nanoparticle (LNP) delivered treatment is designed for transthyretin amyloidosis (ATTR), a rare, progressive, and fatal disease. In subjects who completed at least 48 weeks…
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Lingyi Biotechnology and Jolly Pharmaceutical Ink Comprehensive Collaboration Agreement
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China-based gene therapy specialist Lingyi Biotechnology Co., Ltd has entered into a comprehensive collaboration agreement with Zhejiang Jolly Pharmaceutical Co., Ltd. The partnership aims to leverage the respective strengths of both companies in equity, research and development (R&D), and sales. Enhancing Drug Development and CommercializationUnder the terms of the agreement,…
