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 Today

OCTOBER 27, 2022

27, Vilnius will host the EFIB’2022 event which will bring the European life science sector together. In the field of life sciences, Lithuania is best known for the discovery of gene scissors by Prof. Lab, focused on research and development of novel genome editing tools. Virginijus Šikšnys.

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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

XTalks

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

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Gene Therapy Gene Genome Genomics 105

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

XTalks

MAY 12, 2021

In this episode, Ayesha discusses a new tool that uses CRISPR to modulate gene expression without editing DNA sequences. Dubbed “CRISPRoff,” the technology targets the epigenome to silence genes involved in diseases, with applications in cancer, AML and other conditions with a heritable component.

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Life Science Gene Expression Gene DNA 52

XTalks

JULY 14, 2022

Every gene in a cell is expressed at exact levels due to complex gene regulatory networks. The connections between regulatory genes and their downstream targets have been mapped to some extent — they can be thought of as a subway or metro map, with connections existing between major “hubs.”

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BioSpace

NOVEMBER 15, 2021

This year’s top 30 large and small companies operate in some of the Life Sciences’ hottest spaces – precision medicine, vaccines, gene editing, genomics and oncology.

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Gene Editing Life Science Genome Genomics 75

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.

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Genome Genomics Life Science Gene Therapy 139

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

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.

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Gene Therapy Gene Trials Clinical Trials 95

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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Drug Discovery World

MAY 6, 2024

Thanks to a prestigious academic legacy, Europe has long been a dominant region for life sciences. Europe is traditionally a life sciences powerhouse, with a strong tradition in pharmaceutical discovery. The oldest, still active pharmaceutical company worldwide, Merck, started life in 1668 in Darmstadt, Germany.

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Drugs Life Science Gene Editing Gene 59

Drug Discovery World

SEPTEMBER 6, 2023

A new licensing agreement between Vita Therapeutics and MilliporeSigma (the US and Canada Life Science business of Merck) will see Vita leverage MilliporeSigma’s foundational CRISPR patents to advance its preclinical asset VTA-100 for limb-girdle muscular dystrophy (LGMD2A).

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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

Drug Discovery World

MAY 5, 2023

Eterna Therapeutics, a life science company focused on mRNA cell engineering, has acquired Exacis Biotherapeutics’ immuno-oncology platform. In contrast to DNA-based reprogramming and gene editing, the mRNA-based approach does not expose cells to expensive and potentially harmful viruses or DNA vectors.

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Gene Editing Engineer DNA Life Science 52

XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. These companies are at various stages of research and clinical trials.

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Life Science Gene Editing Development Clinical Trials 119

Drug Discovery World

NOVEMBER 27, 2023

By Anis H Khimani , PhD, Senior Strategy Leader, Life Sciences strategy group, Revvity, Inc. Upstream cell line development has seen notable progress with recent advances in cell line engineering, particularly in enhancing gene of interest integration specificity, clone selection, and production efficiency.

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Drug Discovery World

OCTOBER 16, 2023

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. CellProthera also appointed the Newcastle Centre for Life’s GMP cell therapy centre to produce the ATMP.”

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Drug Discovery World

MARCH 13, 2024

With government support and strong academic institutions, does Singapore have what it takes to become a global centre for life sciences? DDW’s Diana Spencer explores how the country is turning pioneering science into a successful biotech industry. We are now working at ways to target TOX2 including use of PROTAC to degrade TOX2.

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Life Science Genome Genomics Research 69

The Pharma Data

JANUARY 12, 2021

In December, life sciences giant Bayer launched a cell and gene therapy platform within its pharmaceutical division in order to become a leading company within a rapidly emerging and evolving field that offers the potential of life-saving therapies. Photo courtesy of Bayer. The platform is already bearing fruit.

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XTalks

SEPTEMBER 12, 2023

Life science podcasts have emerged as an invaluable tool for building connections with audiences in the digital era. Furthermore, we’ll explore the unique advertising opportunities that this platform offers, positioning brands at the forefront of the life science industry. The result?

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Drug Discovery World

NOVEMBER 1, 2023

DDW’s Diana Spencer meets Dr Stephen Jones, Research Professor and Group Leader at the EMBL Partnership Institute for Genome Editing Technologies at Vilnius University’s Life Sciences Center. DS: Can you tell us what you do and what you’re working on at the moment?

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Pharmaceutical Technology

MAY 15, 2023

A major strategy for OV development is genetic manipulation to weaken virus pathogenicity, enhance target selectivity, reduce adverse reactions, and/or insert exogenous therapeutic genes into the virus genome.

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Protein Gene Genetics Gene Therapy 130

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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The Pharma Data

JANUARY 5, 2021

Over the past several weeks, Helix has analyzed recent positive samples and identified those with ‘S gene dropout’ on their diagnostic PCR assay, indicating the potential presence of the emerging B1.1.7 Illumina then sequenced a subset of these ‘S gene dropout’ samples using Illumina’s COVIDSeq Test, which identified the B.1.1.7

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Genome Genomics Life Science Development 52

Pharmaceutical Technology

FEBRUARY 3, 2023

The field of genomic medicine has reached a true turning point. With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more.

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XTalks

DECEMBER 1, 2021

To ensure the new tests can identify Omicron based on its mutations, the companies assessed their tests against sequences in public databases of genomic data, such as GISAID and GenBank. Cue is also currently working with Google Cloud and genomics company Helix to study variants and share mutation sequencing data.

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Gene Genome Genomics Genetics 105

pharmaphorum

JANUARY 19, 2022

The process enables researchers to examine the transcriptome – the sum total of all the messenger RNA molecules expressed from the genes of an organism – of individual cells, which gives them information that might otherwise escape scrutiny. Developments in the sector have already led to significant scientific breakthroughs.

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RNA Research Life Science Bioinformatics 72

XTalks

AUGUST 5, 2020

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. 10x Genomics’ chromium single cell solutions are designed to accommodate a wide range of single cell or nuclei suspensions as a starting sample.

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Gene Expression Development RNA Drugs 112

XTalks

MAY 10, 2022

CancerVar will help researchers standardize and automate clinical interpretations for 13 million somatic mutations from 1,911 cancer census genes. The gene mutations that cause sporadic cancer occur only in the tumor cells and are acquired during an individual’s life, not inherited.

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Bioinformatics Development Gene Genome 98

XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. Vyjuvek is a gene therapy that employs a herpes simplex virus type 1 (HSV-1) vector. Upon Vyjuvek’s entry into cells, the vector genome is integrated into the nucleus, initiating the transcription of the encoded human COL7A1.

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FDA Approval Gene Therapy Gene Trials 111

XTalks

JUNE 16, 2023

. “Companion diagnostics are high-quality, well-validated genomic tests that provide critical information to help oncologists make informed treatment decisions for their patients,” said Mia Levy, MD, PhD, chief medical officer at Foundation Medicine. ” What is FoundationOne Liquid CDx? .

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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. DDW provides an update.

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XTalks

MARCH 28, 2024

Building Cancer Awareness and Empowering Métis Youth: Featuring Jordyn Playne, President, Métis Nation of Ontario Youth Council – Xtalks Life Science Podcast Ep. Gene Editing and CAR T-Cell Therapy Genetic engineering technologies, such as CRISPR-Cas9, are providing newer potential avenues for cancer treatment.

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Gene Editing Life Science Clinical Trials Clinical Trials 59

XTalks

NOVEMBER 30, 2023

More Investigational Therapies for HIV Are on the Way American Gene Technologies, a pioneering biotech company headquartered in Rockville, Maryland, has released promising results from its Phase I trial of AGT103-T, a gene therapy designed for individuals with chronic HIV disease.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

pharmaphorum

AUGUST 11, 2022

Pharmacogenomics, the understanding of how genes impact an individual’s response to medications, provides a potential outlet to better optimised therapies. There have been great strides made in genomics to refine tools that determine the safest, most effective course of treatments for patients across a number of disease states.

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