Genetic Diseases News
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Comprehensive Gene Therapy Development Solutions to Further Boost Rare Disease Research
As several gene therapy approvals for rare disease treatment gain steam in 2023, a comprehensive set of gene therapy development solutions is released to pave richer avenues for rare disease research. This path-breaking move sees the integration of cutting-edge technology with state-of-art research facilities to amplify the possibilities of devising effective remedies for rare diseases. Rare ...
By Protheragen
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Advances in Newborn SMA Screening research by SNP Biotechnology
BSD Robotics is proud to share a recent study conducted by SNP Biotechnology R&D Ltd “A new line method; A direct test in spinal muscular atrophy screening for DBS” now published in the journal of Molecular Genetics and Genomic Medicine. Given spinal muscular atrophy (SMA) is one of the most prevalent genetic disorders affecting young children and a major cause of death in ...
By BSD Robotics
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CD Genomics Launches Advanced Whole Exome Sequencing Service, Empowering Genetic Research
CD Genomics, a renowned provider of genomics services, is proud to unveil its cutting-edge Whole Exome Sequencing (WES) service. This offering provides researchers and clinicians with a powerful tool to investigate the exome, shedding light on the intricacies of the human genome and propelling our understanding of genetic diseases. In the era of precision medicine, comprehensive and precise ...
By CD Genomics
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Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics
Clinically validated lipid nanoparticle (LNP) delivery technology will further strengthen Bayer’s gene therapy portfolio High potency ionizable lipid technology and LNP carriers allow for efficient, targeted, and transient delivery of gene editing RNA components to the liver Bayer AG is joining forces with Acuitas Therapeutics, Inc., a biotechnology company specializing in the ...
By Bayer AG
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CD Genomics Unveils Advanced Mitochondrial Diseases Panel Sequencing Service
CD Genomics, a leading biotechnology company specializing in genomics and sequencing, has announced the launch of its advanced Mitochondrial Diseases Panel Sequencing services. This new panel enables rapid and accurate detection of mutations in mitochondrial DNA (mtDNA) that are associated with a wide range of mitochondrial disorders. Mitochondrial diseases are a complex and heterogeneous group ...
By CD Genomics
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BIO Investor Forum Company Snapshot: InvivoSciences
BIO Investor Forum Company Snapshot: InvivoSciences What is your company’s lead product or technology? InvivoSciences, Inc. (IVS) is a leading developer of proprietary human 3D cell/tissue culture models for highly-predictive, multi-parameter phenotypic assays both in pharmacology safety & discovery screening and in personalized and regenerative medicine. IVS’s Functional 3D ...
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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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SeqOne Genomics and the French Thrombotic MicroAngiopathies National reference center (CNR-MAT) pioneer the use of Oxford Nanopore sequencing technology to improve patient outcomes in kidney disease while reducing turnaround times
SeqOne today announced the results of a long-standing research collaboration with Pr. Laurent Mesnard, Co-Director of the French National center for Thrombotic MicroAngiopathies (CNR-MAT), aimed at improving the diagnosis and management of patients suspected of suffering from thrombotic microangiopathies and atypical Hemolytic syndrome (aHUS). The program involved using Oxford Nanopore’s ...
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AskBio Announces Strategic Collaboration with ReCode Therapeutics to Explore Single Vector Gene Editing Platform
Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, announced today that it has signed a multi-year research collaboration and option agreement with ReCode Therapeutics. Under this agreement, the companies will work together to potentially discover precision genetic medicines through the development of a novel platform for full gene ...
By Bayer AG
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CRISPR Off-Target Analysis Now Available with CD Genomics’ Bioinformatics-Analysis Platform
The Bioinformatics-Analysis is a subdivision of CD Genomics, providing reliable next-generation and third-generation high-throughput sequencing data analysis, comprehensive technology services, database construction services, and other related data analysis services. The company is pleased to announce the launch of the CRISPR off-target analysis service to help researchers optimize the CRISPR ...
By CD Genomics
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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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Jubilant Therapeutics Inc. to Present at Credit Suisse and Jefferies Investor Conferences
Jubilant Therapeutics Inc., a clinical stage biopharmaceutical company developing precision oral medicines with enhanced therapeutic index to serve genetically defined patients suffering from cancer and autoimmune diseases, today announced that Syed Kazmi, Chief Executive Officer, will be making a business update presentation and meeting with institutional investors at the following conferences ...
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Longenesis and Dante Genomics Partner to Make Personalised Medicine More Accessible Across Europe
Biotech and genomics companies Longenesis and Dante Genomics launch more personalised genomic services to more than 10,000 people across the Baltics and Italy, thus expanding and bridging the reach of valuable health insight delivery to patients across Europe. Latvian Biotechnology startup Longenesis has partnered with Dante Genomics, a global company of genomics and precision medicine, to offer ...
By Longenesis
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Ray Therapeutics and Forge Biologics Expand Their Viral Vector cGMP Partnership to Encompass Plasmid DNA Manufacturing
Ray Therapeutics, a biotechnology company developing optogenetic gene therapies for patients with retinal degenerative conditions, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization, announced further collaboration for their manufacturing partnership to include clinical stage plasmid DNA production to support Ray Therapeutics’ lead optogenetics ...
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Bayer and Mammoth Biosciences to Collaborate on Novel Gene Editing Technology
Agreement strengthens Bayer’s new cell and gene therapy platform further Supports Mammoth’s vision of unlocking the full potential of novel CRISPR systems Mammoth to receive upfront payment of USD 40 million, and potential future milestone payments of more than one billion USD upon successful achievement Bayer AG and Mammoth Biosciences, Inc., which is harnessing the diversity ...
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Alnylam Announces FDA Approval of Supplemental New Drug Application for OXLUMO® (lumasiran) in Advanced Primary Hyperoxaluria Type 1
- OXLUMO Now Indicated for the Treatment of Primary Hyperoxaluria Type 1 (PH1) to Lower Urinary and Plasma Oxalate Levels in Pediatric and Adult Patients - - Approval is Based on Positive Efficacy and Safety Results of the ILLUMINATE-C Phase 3 Study of OXLUMO in PH1 Patients with Severe Renal Impairment, Including Those on Hemodialysis - CAMBRIDGE, Mass.--(BUSINESS WIRE)--Oct. 6, 2022-- Alnylam ...
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Alnylam Receives Approval in Europe for AMVUTTRA® (vutrisiran) for the Treatment of Hereditary Transthyretin-mediated (hATTR) Amyloidosis in Adult Patients with Stage 1 or Stage 2 Polyneuropathy
– AMVUTTRA Demonstrated Halting or Reversal in Neuropathy Impairment with Subcutaneous Administration Once Every Three Months – – Decision Follows Positive Opinion from the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) in July 2022 – CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 20, 2022-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), ...
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Alnylam to Host Ninth Annual “RNAi Roundtable” Webcast Series
– Series Will Culminate in a Virtual R&D Day on December 15, 2022 – CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 19, 2022-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it plans to host its 9th annual series of “RNAi Roundtable” webinars over the coming weeks. The series will offer presentations from Alnylam ...
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Alnylam to Present Additional Data from the APOLLO-B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy at the Heart Failure Society of America Annual Scientific Meeting 2022
– Company to Webcast Investor Event on September 30th at 8:00 p.m. ET – CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 13, 2022-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the Company will present additional data from the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment ...
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OncoDNA Announces the Operational Launch of its OncoDEEP Kit for Comprehensive Genomic Profiling of Solid Tumors
A UNIQUE WORKFLOW SOLUTION INTEGRATING A PAN-CANCER NEXT-GENERATION SEQUENCING (NGS) PANEL POWERED BY A BIOINFORMATICS SOFTWARE SUITE FOR CUSTOMIZABLE BIOLOGICAL AND CLINICAL INTERPRETATION ONCODNA, A GENOMIC AND THERANOSTIC COMPANY SPECIALIZING IN PRECISION MEDICINE FOR THE TREATMENT OF CANCER AND GENETIC DISEASES, TODAY ANNOUNCED THE OFFICIAL LAUNCH OF THE ONCODEEP KIT DURING THE ESMO ...
By OncoDNA S.A.
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