Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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Pharmaceutical Technology

APRIL 28, 2023

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022.

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pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. billion in funding.

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Drug Discovery World

MAY 17, 2024

From a weight loss drug that prevents heart attacks and a gene therapy that restores hearing, to a vaccine that can treat viruses that don’t exist yet, our chosen news stories this week all represent potential breakthroughs in their respective fields.

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Drug Discovery World

DECEMBER 6, 2023

Gene editing company Eligo Bioscience has announced a successful $30 million Series B funding round, led by Sanofi Ventures. “We By focusing on the in-vivo delivery of genetic cargoes to the microbiome, Eligo’s hopes to go beyond traditional gene therapy and gene editing.

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Drug Discovery World

MARCH 22, 2024

This has been an interesting week for cell and gene therapies, with two landmark FDA approvals, two significant fundraising efforts and potentially ground-breaking study results in glioblastoma. The post This week in drug discovery (18-22 March) appeared first on Drug Discovery World (DDW).

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Drug Discovery World

FEBRUARY 23, 2024

In a breakthrough for advanced therapies, this week saw the FDA approve the first ever cell therapy for solid tumour cancers, but there were other significant developments in the cell and gene therapy space. The post This week in drug discovery (19-23 February) appeared first on Drug Discovery World (DDW).

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Drug Discovery World

AUGUST 7, 2023

The collaboration will work on developing exosome-encapsulated AAV (exoAAV) vectors as a novel gene delivery technology aimed at improving treatments for heart disease. Susmita Sahoo, Associate Professor of Medicine, Cardiology at Icahn Mount Sinai has been exploring the use of exosomes in gene therapy for several years.

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XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

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Roots Analysis

FEBRUARY 27, 2024

The biopharmaceutical pipeline includes close to 500 gene therapies , several of which are being developed based on CRISPR technology. Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. The global genome editing market is anticipated to grow at a CAGR of 12.6%

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pharmaphorum

JUNE 12, 2022

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has continued to show impressive results in clinical trial, with an update at the EHA congress showing long-term effects on the symptoms of both beta thalassaemia and sickle cell disease. billion product if it gets approved for both indications.

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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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pharmaphorum

JUNE 23, 2022

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). Other companies – notably Vertex Pharma/CRISPR Therapeutics and bluebird bio – are taking an ex vivo approach to SCD and thalassaemia and are much closer to market.

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pharmaphorum

NOVEMBER 18, 2022

Gene-testing specialist Editas Medicine has halted development of its lead clinical programme for congenital eye disorders after it generated lacklustre results in a phase 1/2 trial. LCA10 is caused by mutations in the CEP290 gene and accounts for approximately 20?30% 30% of all LCA cases.

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Drug Discovery World

MARCH 10, 2023

Perhaps most interestingly this week, after previous caution over in vivo gene therapy trials, the FDA has given the go ahead to Intellia Therapeutics to study NTLA-2002 in hereditary angioedema (HAE). The post This week in drug discovery (6-10 March) appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JANUARY 27, 2023

The post $5 million raised towards gene therapies for muscular diseases appeared first on Drug Discovery World (DDW). As proof of principle for the platform, success in treating Duchenne would open up opportunities for Myosana to target a large range of neuromuscular and cardiac diseases.”

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pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

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Gene Editing Gene In-Vivo Clinical Trials 59

Drug Discovery World

MARCH 13, 2023

Perhaps most interestingly this week, after previous caution over in vivo gene therapy trials, the FDA has given the go ahead to Intellia Therapeutics to study NTLA-2002 in hereditary angioedema (HAE). You can listen below, or find The Drug Discovery World Podcast on Spotify , Google Play and Apple Podcasts.

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Gene Therapy Drugs In-Vivo Clinical Trials 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

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., LNPs are spheric drug delivery bodies which can be equipped with therapeutic payloads for intracellular delivery.

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Drug Discovery World

JANUARY 5, 2024

In non-human primate studies, the therapy has demonstrated effective, durable, and well tolerated in vivo CAR-T cell generation. In non-human primate studies, the therapy has demonstrated effective, durable, and well tolerated in vivo CAR-T cell generation.

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Drug Discovery World

DECEMBER 7, 2023

Inipharm has initiated Phase I dosing of its lead candidate, INI-822, which is a small-molecule inhibitor for HSD17B13, a gene implicated in nonalcoholic steatohepatitis (NASH). This is the first example of an OOC provider’s data supporting clinical progression of a drug for metabolic, fibrotic liver disease.

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Drugs In-Vivo Gene Development 59

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. There is another $1 billion in downstream payments on offer, assuming the drug candidates all make it through to regulatory approval and hit sales targets.

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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. billion, compared to $19.9

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pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. Its main platform – called X-editing – can generate CRISPR drugs with improved activity, specificity and deliverability compared to rivals, according to the start-up’s website.

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Drug Discovery World

MAY 1, 2023

Download Emulate’s Introduction to Organ-on-a-Chip Technology eBook to learn about: What Organ-Chips are, how they work, and why they are transforming the way drugs are discovered and developed How Organ-Chips overcome the main challenges of conventional in vivo and in vitro models The wide variety of insights and endpoint analyses Organ-on-a-Chip (..)

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Drug Discovery World

MAY 29, 2023

Gene delivery technologies company VectorBuilder has opened a new office in Edinburgh, hoping to bolster its presence in the United Kingdom. The post Viral vector provider opens new UK headquarters in Edinburgh appeared first on Drug Discovery World (DDW).

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Drug Discovery World

APRIL 15, 2024

DDW’s Megan Thomas speaks to experts from the drug discovery industry about their predictions on the future of stem cells in drug discovery. Gene therapy will move to ex vivo gene modification and the delivery of gene-modified cell types.

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Drug Discovery World

MARCH 7, 2024

Ginkgo Bioworks has acquired Patch Biosciences, with the intention to strengthen its gene therapy services, cell therapy services, and RNA therapeutics services. The collaboration achieved its goals of enhancing the AAV production titres of Biogen’s gene therapy manufacturing processes.

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Drug Discovery World

JUNE 30, 2023

Research tools, safety testing and regenerative medicines – these endless stem cell applications are powering precision, speed, and new modalities in drug development. Stem cells for use in drug discovery Stem cells are fast becoming an invaluable tool in the drug discovery process.

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Drug Discovery World

AUGUST 15, 2023

The post New whitepaper explores digital PCR for lentiviral vector characterisation appeared first on Drug Discovery World (DDW). Reliable analytical methods such as digital PCR to characterise lentiviral vectors and the cells transduced with them are critical to helping ensure the safety and efficacy of these biologics.

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Drug Discovery World

AUGUST 15, 2023

Recombinant lentiviral vectors are used for ex vivo transgene delivery in several gene-modified cell therapies, such as chimeric antigen receptor (CAR) T cell therapy.

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The Pharma Data

JANUARY 4, 2021

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc. MUNICH, Germany, Jan.

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Pharmaceutical Technology

MAY 18, 2023

Takeda oncology drug discovery unit head Kathy Seidl stated: “We are encouraged by KSQ’s CRISPRomics platform and its ability to perform in vivo genetic screens for the discovery and validation of tumour targets, which have the potential to modulate the innate and adaptive immune system. “We

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Pharmaceutical Technology

MAY 24, 2023

to investigational new drug-enabling trials, expected in 2024, to treat aggressive diffuse large B-cell lymphomas (DLBCLs). Morphogenesis is targeting a newly identified delta receptor on myeloid-derived suppressor cells (MDSCs) using its new bi-functional antibody-drug conjugates (ADCs) class.

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Drug Discovery World

APRIL 10, 2024

iECURE has been given the go-ahead to begin US trials of its investigational gene editing-based therapy in newborn males with neonatal onset Ornithine Transcarbamylase (OTC) deficiency. The post FDA approves first in vivo gene insertion programme in infants appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MAY 1, 2023

The post New eBook: An introduction to organ-on-a-chip technology appeared first on Drug Discovery World (DDW).

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