pharmaphorum

OCTOBER 12, 2022

David Del Bourgo (CEO and co-founder, Whitelab Genomics) has always been passionate about introducing disruptive, innovative technologies to markets. We founded Whitelab Genomics after realising the potential to use data, data science, and AI in a more systematic way to develop genomic therapies,” Del Bourgo says.

Genome 105

Genome Genomics Medicine Development 105

Drug Discovery World

DECEMBER 29, 2023

Known as ‘CReATiNG’ (Cloning Reprogramming and Assembling Tiled Natural Genomic DNA), the method offers a simpler and more cost-effective approach to constructing synthetic chromosomes. The thing about most synthetic genomics research is that it involves building chromosomes or genomes from scratch using chemically synthesised DNA pieces.

Genome 52

Genome Genomics DNA Scientist 52

Scienmag

OCTOBER 27, 2021

The rapid scientific advancements that followed the mapping of the human genome have revealed just how staggeringly complex the world of genetics is. We now know that proteins are not just the products of genes, but that they also interact with genes, influencing and regulating the rhythm of their expression.

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Gene Genome Genomics Genetics 72

Drug Discovery World

OCTOBER 10, 2022

By Dr Dina Finan, PhD, Product Manager at 10x Genomics and Dr Nick Downey, PhD, NGS Collaborations Lead at Integrated DNA Technologies. The post Is single-cell gene expression the next trend in next generation sequencing? To read this content in full, you need to login. Not yet a DDW member? Username or E-mail. Remember Me.

Gene Expression 98

Gene Expression Gene RNA DNA 98

Scienmag

DECEMBER 20, 2021

Department of Energy (DOE) announced a plan to provide up to $30 million for basic research that will lead to transformative approaches to determine and validate gene function in plant species relevant to the sustainable production of bioenergy and bioproducts.

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Gene Genome Genomics Research 83

Drug Discovery World

NOVEMBER 27, 2023

A complex journey Biotherapeutics discovery and development upstream encounters challenges in cell line development (CLD), clone screening and characterisation, scale up, and eventually production. Next-generation engineering tools, such as gene editing have enabled knock-out cell lines with enhanced characteristics.

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Production Engineer Gene Editing Gene 59

Drug Discovery World

FEBRUARY 8, 2024

In 2023, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers. How is the titer determined?

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Gene Therapy Gene Bioinformatics Life Science 59

Drug Discovery World

MAY 16, 2023

New South African company NewBiologix has launched with the aim of developing a proprietary and breakthrough platform for the advanced engineering of cell lines used to manufacture gene and cell therapies. NewBiologix is currently focused on recombinant adeno-associated virus (rAAV) vectors, the preferred delivery vehicle for gene therapies.

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Production Gene Therapy Gene Bioinformatics 52

BioTech 365

APRIL 1, 2021

10x Genomics Puts Single Cell Analysis Within Reach of All With Low Throughput Gene Expression Kit 10x Genomics Puts Single Cell Analysis Within Reach of All With Low Throughput Gene Expression Kit Along with recent release of CellPlex, product increases … Continue reading →

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Gene Expression Genome Genomics Gene 40

Drug Discovery World

AUGUST 16, 2023

Decoding ‘junk DNA’ The Human Genome Project and subsequent studies discovered that most of our DNA (approximately 98%) does not actually code for proteins, with humans having approximately 20,000 tox 25,000 protein-coding genes. This leads to potentially severe, debilitating and unbearable toxicities for patients.

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RNA Genome Genomics DNA 52

Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.

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Gene Therapy FDA Approval Gene Protein 97

Worldwide Clinical Trials

JUNE 15, 2022

At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Clinical holds are becoming more common, especially in gene therapy programs.

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Gene Therapy Gene Clinical Trials Clinical Trials 130

Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. General AI tools such as ChatGPT have acted as a catalyst, igniting a wave of AI innovation akin to the early days of the internet era.

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Gene Therapy Gene In-Vitro Development 64

Drug Discovery World

JULY 18, 2023

Global health science solutions company Revvity, PerkinElmer’s Life Science and Diagnostics business’ new name and branding , has announced the establishment of a Scientific Centre of Excellence in Cambridge, UK. Focused on driving genomic insights to accelerate therapeutic discovery, Revvity’s Scientific Centre of Excellence supports the company’s (..)

Genome 52

Genome Genomics Life Science Gene Editing 52

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Drug Discovery World

SEPTEMBER 20, 2022

Breakthroughs in gene therapy are only possible with an exact understanding of the genetic underpinnings of disease. To develop safe and effective gene therapies, researchers need confidence that genomic data is both complete and accurate. Could you explain the differences between short-read and long-read sequencing? .

Gene Therapy 52

Gene Therapy Gene Genome Genomics 52

pharmaphorum

APRIL 19, 2021

French genomic medicines firm SparingVision has agreed to buy GAMUT Therapeutics, a biotech specialising in gene therapies for inherited eye diseases such as retinitis pigmentosa (RP) that could compete against Roche/Spark’s Luxturna in a wider patient group. . million ($52.7 million) fundraiser in October to develop SPVN06.

Gene Therapy 106

Gene Therapy Gene Production Genome 106

Drug Discovery World

OCTOBER 3, 2023

Earlier this year, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

Gene Therapy 52

Gene Therapy Gene Life Science Bioinformatics 52

Drug Discovery World

MARCH 16, 2023

The cell and gene therapy (CGT) market is rapidly expanding, and projections suggest it will reach over $90B by 2023. Challenges of cell and gene therapy manufacturing Compared to traditional biologics, CGTs are living drugs, making their development and manufacturing substantially more complex.

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Gene Therapy Gene Manufacturing Life Science 52

pharmaphorum

SEPTEMBER 26, 2022

The past twelve months have culminated in an unprecedented level of excitement, investment, and clinical progress within the gene therapy field. This year’s agenda includes a novel track designed for quality control and process development groups working in gene therapy. Download the full event guide to find out more.

Gene Therapy 52

Gene Therapy Gene Development Bioassay 52

Drug Discovery World

MAY 25, 2023

Several new products for the life sciences sector have been launched over the last month, from miniature semiconductor chip (MSC) technology to a new inhalation testing service. Astera Software: Astera API Management The new product is a no-code, automated, unified platform for complete API lifecycle management and integrations.

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Production Reagent DNA Drugs 52

Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

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Gene Editing DNA Gene Gene Therapy 235

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. Recent Problems in Cell and Gene Therapy Development. Dealing with confusing regulatory guidelines is one of the top obstacles researchers face as they develop CGT products 1.

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Gene Therapy Gene Production Clinical Trials 52

Drug Discovery World

APRIL 28, 2023

UK company Plasticell and Singapore-based LambdaGen have entered into a strategic collaboration to exploit genome editing technologies based on synthetic lambda integrases that allow specific insertion of large gene cassettes into the human genome.

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Engineer Genome Genomics Gene 98

Drug Discovery World

NOVEMBER 1, 2022

If further studies verify the results, this could be a new method for targeting specific cell types for a variety of disorders that could be treated with gene therapies. Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

Gene Expression 52

Gene Expression Gene Packaging Packaging 52

XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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Gene Therapy Gene Gene Editing In-Vivo 59

Scienmag

JULY 12, 2021

Research team from the Universities of Göttingen and British Columbia investigates evaluations of breeding technology A research team from the University of Göttingen and the University of British Columbia (Canada) has investigated how people in five different countries react to various usages of genome editing in agriculture.

Genome 71

Genome Genomics Research Gene Therapy 71

Scienmag

NOVEMBER 25, 2020

Credit: Photo: IPK Leibniz Institute/ Andreas Bähring In order to record all genetic information of an individual, its genome must be completely decoded. IPK scientists and international partners for barley already succeeded in doing this three years ago (Mascher et al.

Genome 88

Genome Genomics Scientist Genetics 88

WCG Clinical

NOVEMBER 17, 2023

Although the NIH Guidelines were originally written with non-clinical laboratory research in mind, they also apply to human gene transfer (HGT) research, wherein rsNA or rsNA-containing products are administered to research participants. Since then, however, certain genetic engineering technologies (e.g.,

Genome 52

Genome Genomics Gene Editing Genetic Engineering 52

Pharmaceutical Technology

MARCH 8, 2023

The new technologies will also overcome the manufacturing bottlenecks in delivering advanced engineered cell products. Its focus areas include human systems, precision diagnostics, data sciences, next-generation biomanufacturing, and genomic medicines. The University of Pennsylvania Immunotherapy Richard W.

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Gene Therapy Manufacturing Gene Production 244

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. million price point per dose.

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Gene Therapy Gene Manufacturing Medicine 52

Scienmag

JULY 12, 2021

2021 Agricultural Greater Good Grant awarded to Göttingen University molecular biologist The international Illumina Agricultural Greater Good Grant recognizes research proposals that will increase the sustainability, productivity, and nutritional density of agriculturally important crop and livestock species.

Genome 75

Genome Genomics Production Medicine 75

Pharmaceutical Technology

SEPTEMBER 29, 2022

The alliance will leverage the CRISPR genome editing technologies of Scribe to facilitate in genetic modification of new natural killer (NK) cell therapeutics for cancer. Sanofi will also make tiered royalty payments on net sales of products resulting from the collaboration in the future.

Development 130

Development In-Vivo Genome Genomics 130

Drug Discovery World

FEBRUARY 12, 2024

Leslie Orne, President and CEO, Trinity Life Sciences “AI and ML will play an increasingly important role in the clinical development and commercialisation of cell and gene therapies, which are typically targeted to small patient populations. Looking even further into the future, Industry 4.0

Genome 139

Genome Genomics Life Science Gene Therapy 139

Drug Discovery World

SEPTEMBER 4, 2023

AviadoBio, a gene therapy company developing medicines for neurodegenerative disorders, will establish laboratory and office presence at the Innovation Centre. Kadans’ London Innovation Centre at Canary Wharf, London has launched its wet lab innovation and flexible workspace centre and welcomed a new tenant.

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Life Science Gene Therapy Genome Genomics 59

Drug Discovery World

SEPTEMBER 6, 2023

VTA-100 utilises this CRISPR genome editing technology to insert a functional copy of the Calpain 3 (CAPN3) gene, which is mutated in patients with LGMD2A, into Human Satellite Cells, generated using Vita’s proprietary production technology, for use as a cell therapy.

Genome 52

Genome Genomics Gene Life Science 52

Drug Discovery World

MARCH 25, 2024

Allogene Therapeutics and Arbor Biotechnologies Biotechnology companies Allogene Therapeutics and Arbor Biotechnologies entered a non-exclusive, global gene editing licensing agreement for use of Arbor’s proprietary CRISPR gene-editing technology in Allogene’s AlloCAR T platform for the treatment of autoimmune disease (AID).

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Gene Editing Drugs Genome Genomics 52