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61-90 of 833 results

  • luxturnahcp.com
LUXTURNA® is the first gene therapy to help improve functional vision in patients with an inherited retinal disease (IRD) due to biallelic RPE65 gene mutations. 1..
Relevance: 39.90812
  • parentsforthecureofsicklecelldieaseandbetathalassemia.com
Together, Sickle Cell Disease and Beta-Thalassemia represent the most common genetic blood disorders. With the current high cost of gene therapy, a cure is unfathomable and unattainable to many. This leaves patients affected by Sickle Cell Disease..
Relevance: 39.895042
  • www.lokonpharma.com
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Lokon Pharma is a Swedish biotech company in the forefront of developing immunostimulatory gene therapy for cancer based on oncolytic viruses. Innovative gene engineering, solid expertise in cancer biology and immunology, as well as a genuine..
Relevance: 39.87861
  • sparingvision.com
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Our goal is to harness the potential of gene therapy and genome editing to bring cutting-edge innovation into ocular disease drug discovery and development... SparingVision's lead product, SPVN06, is a breakthrough gene therapy approach targeting..
Relevance: 39.667152
  • encoded.com
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Pediatric central nervous system disorders, such as Dravet syndrome, contribute both to premature mortality as well as to substantial, lifelong disabilities. Encoded is developing transformative precision gene therapies to provide potentially long-..
Relevance: 39.46555
  • ct.catapult.org.uk
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At the Manufacturing Innovation Centre at CGT Catapult Braintree, we work in collaboration with the cell and gene therapy industry to accelerate manufacturing innovation for therapy developers, technology companies and service providers. We support..
Relevance: 39.29516
  • akronbiotech.com
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Akron Bio is a leading manufacturer of ancillary materials for the cell and gene therapy industry... At Akron, we manufacture high-quality compliant cGMP plasmid DNA for pre-clinical, clinical, and commercial gene therapy and vaccine companies.....
Relevance: 39.230244
  • armatusbio.com
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Armatus Bio is raising expectations for FSHD with precision gene therapy...
Relevance: 39.088425
  • www.affiniatx.com
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Affinia Therapeutics is committed to transforming gene therapy with our platform and pipeline - to push science forward, to impact human medicine, and to give patients the options they deserve... Affinia Therapeutics is setting a new standard in..
Relevance: 38.872475
  • www.jeffnelsonconsulting.com
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Quality systems, analytical and instrument validation, deviation investigations, adenovirus gene therapy...
Relevance: 38.73365
  • agtchcp.com
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AGTC is a biotechnology company seeking to transform the lives of patients with rare and debilitating eye diseases through its proprietary gene therapy platform... Our team includes industry-leading experts and innovative thinkers working together..
Relevance: 38.559017
  • alcyonetx.com
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Alcyone is pioneering next-generation precision gene-based therapies for complicated disorders of the central nervous system (CNS). We integrate innovations in neuroscience, gene therapy, precision dosing and delivery, and manufacturing to deliver..
Relevance: 38.52562
  • beacontxhcp.com
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Beacon Therapeutics is a biotechnology company seeking to transform the lives of patients with lifelong and debilitating eye diseases through its proprietary gene therapy platform... Our team includes industry-leading experts and innovative thinkers..
Relevance: 38.35133
  • www.leanbiologix.com
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Lean Biologix is a unique service provider for the Biotech, Gene Therapy and Pharmaceutical manufacturers. We exceed engagement deliverables of on-time and on-budget execution by leveraging cutting edge technologies and best practices to deliver..
Relevance: 38.303154
  • www.sangamo.com
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Sangamo is a genomic medicine company that seeks to help millions of people who suffer from severe diseases for which today's medicine can only offer symptom management at best. Our mission is to translate ground-breaking science into medicines that..
Relevance: 38.21103
  • sparktx.com
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Our mission at Spark Therapeutics is to unlock the power of gene therapy to accelerate healthcare transformation. We have built a fully integrated company, combining our proprietary adeno-associated viral (AAV) gene therapy platform with excellence..
Relevance: 38.19268
  • www.nanliulab.com
Liu lab employs multidisciplinary cutting-edge techniques to dissect genetic and epigenetic control of developmental gene expression using embryonic/adult stem cells as model systems. We interrogate the mechanisms of disease-causing mutations in gene..
Relevance: 38.182888
  • www.cascadebiotechnology.com
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Cascade Biotechnology is developing therapeutics that use the body's complement system to attack diseases and disorders such as Alzheimer's disease (AD), schizophrenia (SZ), myasthenia gravis (MG), Neuromyelitis Optica (NMO), cancer, adult macular..
Relevance: 38.176277
  • www.sigenetic.com
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Beijing Sinogenetic Biotechnology Co., Ltd. was established in 2021. Relying on the parent company's animal cloning and gene editing technology platform, the company has developed and constructed multi-species (dog/pig/cat/rabbit/mouse/rat) gene..
Relevance: 38.1669
  • www.smartgene.si
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Gene therapy is a therapeutic strategy using genes as therapeutic agents. During the therapy therapeutic gene is inserted into targeted cells with the goal to (i) Introduce a new gene into the cell to fight a disease, (ii) replace a mutated gene..
Relevance: 38.157562
  • www.inspirebiotx.com
Inspire Biotherapeutics Inc. ("Inspire"), an emerging biotech company with a mission to create life-saving gene therapies for monogenic and acquired diseases of the lung, is poised to move its novel lung tropic AAV gene therapy into clinical..
Relevance: 38.152184
  • www.vdb-lab.org
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The Grousbeck Gene Therapy Center is an academic research center devoted to the broadening of therapeutic gene transfer in modern medicine. Our focus is on internal and collaborative preclinical and clinical research studies, technology development,..
Relevance: 38.023605
  • restore-vis.com
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Restore Vision is a startup based in Japan aiming to develop a visual restoration gene therapy...
Relevance: 37.940178
  • www.regenxbio.com
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REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy... Our gene therapy product candidates are designed to deliver genes to cells to address genetic defects or to enable..
Relevance: 37.894417
  • www.intimabioscience.com
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Intima Bioscience is conducting a clinical trial initially at the University of Minnesota Masonic Cancer Center with its Molecular and Cell Therapy (MCT) GMP gene and cell therapy manufacturing facility...
Relevance: 37.8038
  • www.drthon.com
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Dr. Thon is a Serial Biotech Entrepreneur, accomplished CEO, and experienced Board Member with technical expertise in cell & gene therapy, hematology, and cell culture/manufacturing who specializes in founding and leading startups through company..
Relevance: 37.8033
  • www.orphina.com
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Orphina is a biotechnology company at seed stage, focused on preserving and prolonging muscle health. Our initial clinical gene therapy program is for a rare disease indication of unmet medical need. Our technology allows re-dosing while limiting..
Relevance: 37.764023
  • www.centralpharma.com
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Central Pharma joins CSafe's global partner network of pharma service providers to expand supply chain solutions for the cell and gene therapy market. ​Introduction:Established in 2006, Central Pharma are GMP and GDP compliant and offer scalable..
Relevance: 37.743748
  • www.anewmeds.com
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Our vision is to build a leading gene therapy company for the treatment of neurodegenerative diseases and cancer by progressing our á-Klotho gene therapy research programs and identifying, developing, and commercializing other novel gene therapy..
Relevance: 37.735725
  • www.gene-factory.com
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cancer research, genetic constructs for cancer gene therapy, translational control, gene expression enhancement, recombinant protein biotechnology, forensic and medical diagnostics, biochemistry of polyphenols (anthocyanins, resveratrol) in cancer..
Relevance: 37.725906