disease causing gene News
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Agilent Technologies Releases New Target-Enrichment Solutions for Disease Research
Agilent Technologies Inc. (NYSE: A) today announced new additions to its market-leading SureSelect target-enrichment product family, which has already enabled researchers to identify more than 50 Mendelian disease-causing genes. The new products-the SureSelect Human All Exon V6, V6+UTR and V6+COSMIC-are designed to address the current limitations of exome sequencing, giving researchers better ...
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CD Genomics Expands Its Sequencing Portfolio with the Launch of Whole Exome Sequencing
CD Genomics is a world-leading genomics services company that continues to innovate in sequencing solutions for the genomics industry, covering genotyping, library construction, bioinformatics, microarrays, mutagenesis analysis, and more. CD Genomics is pleased to offer whole exome sequencing services, which can help scientists discover disease-related mutation sites more accurately. Exomes are ...
By CD Genomics
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Dicerna’s GalXC-Plus™ RNAi Technology Delivers Target Knockdown Across CNS and to Specific CNS Cell Types in Preclinical Studies
Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced new data from preclinical studies of its GalXC-Plus™ RNAi technology demonstrating its potential to deliver deep and sustained messenger RNA (mRNA) knockdown against prespecified gene ...
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ViGeneron announces research collaboration with Daiichi Sankyo to evaluate vgAAV for novel ophthalmic gene therapy
ViGeneron GmbH, a gene therapy company, today announced a research collaboration to utilize its novel engineered adeno-associated virus (vgAAV) vectors with Daiichi Sankyo Company, Limited (“Daiichi Sankyo”), for delivering a novel therapeutic protein to address an undisclosed target in a highly prevalent ophthalmic disease. ViGeneron’s vgAAVs enable the efficient transduction ...
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Emerging Trends in the BioPharma Industry that you Need to Know
Emerging trends in the biopharma industry include new developments in cancer treatments, advances in gene therapy, and more data-driven decision-making. As a leader in the sector, it’s important to be aware of these developments to make informed decisions about your organization’s direction. In this blog post, we’ll explore some of the most significant emerging trends in ...
By Brio Group
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ViGeneron signs global collaboration agreement for ophthalmic gene therapy development
Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target The companies will use ViGeneron’s proprietary vgAAV technology to efficiently transduce target cells via intravitreal injections ViGeneron GmbH, a gene therapy company, today announced a global collaboration and licensing agreement with Biogen ...
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Agilent Technologies Introduces Two Innovative Products to Advance Clinical Cancer Research
Agilent Technologies Inc. (NYSE: A) today launched two new products-the HaloPlexHS target enrichment system and ClearSeq Comprehensive Cancer research panel-designed to improve the speed and accuracy of clinical cancer research. Both products will enable researchers to rapidly and accurately identify disease-associated variants in tumor samples-information that can be vital to research in cancer ...
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ViGeneron and WuXi Advanced Therapies enter Strategic Manufacturing Partnership For Next-Generation Ophthalmic Gene Therapy
ViGeneron, a gene therapy company, and WuXi Advanced Therapies Inc. (WuXi ATU), a leading Contract, Testing, Development and Manufacturing Organization (CTDMO), today announced a strategic partnership to accelerate production for the clinical development of VG901, a next-generation ophthalmic gene therapy program. ViGeneron’s innovative gene therapy pipeline addresses ophthalmic diseases ...
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ViGeneron Announces Closing of Series A Financing to Drive Development of Next Generation Gene Therapy Pipeline
ViGeneron GmbH, a gene therapy company, announced the closing of its series A financing round led by WuXi AppTec and Sequoia Capital China. The proceeds will enable ViGeneron to accelerate its proprietary viral vector-based gene therapy platforms and drive product development in its two lead ophthalmic gene therapy programs. Funds will advance development of ViGeneron’s next-generation ...
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Vertex Advances VX-147 Into Pivotal Clinical Development for People With APOL1-Mediated Kidney Disease
Single pivotal trial to initiate later this month targets the broad patient population with two APOL1 mutations and proteinuric kidney disease Pathway for accelerated approval using an interim analysis at Week 48 of eGFR slope, supported by reduction in proteinuria Final analysis evaluating eGFR slope at approximately two years serves as basis for U.S. approval Vertex Pharmaceuticals ...
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Rocket Pharmaceuticals Presents Positive Top-line Data from Severe Leukocyte Adhesion Deficiency-I Program at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)
RP-L201 well-tolerated with durable CD18 expression for all patients in Phase 2 pivotal trial 100% overall survival in patients at one year after RP-L201 infusion based on Kaplan-Meier estimate All patients showed clinical reversal of disease course, including statistically significant reduction in all-cause hospitalizations and incidence of severe infections Initiating discussions with ...
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