Pharmaceutical Technology

JUNE 16, 2023

The Abu Dhabi Department of Health (DoH) in the UAE has made a declaration of collaboration with Mass General Brigham’s (MGB) International Center for Genetic Disease (iCGD) to advance life sciences.

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Life Science Genetic Disease Genome Genomics 130

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies. How Can Study Protocols Be More Effective?

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Gene Therapy Gene Trials Clinical Trials 95

Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . Some have even been successfully harnessed as gene therapy vectors for the treatment of genetic diseases. .

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Gene Therapy Gene Genetic Disease Genetics 52

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.

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Gene Therapy Gene Genome Genomics 52

Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. Patients with MPS-IIIA have a mutation in the SGSH gene, causing them to lack an enzyme which normally breaks down large sugar molecules.

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Gene Therapy Gene Genetics Genetic Disease 52

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.

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Genome Genomics Medicine Development 105

Medical Xpress

NOVEMBER 21, 2022

In a new study published in Circulation: Genomics & Precision Medicine, Mayo Clinic researchers designed and developed the first suppression-replacement KCNH2 gene therapy for correcting both long QT syndrome (LQTS) and short QT syndrome (SQTS).

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Gene Therapy Gene Genome Genomics 67

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

pharmaphorum

JANUARY 24, 2023

Ben Hargreaves finds that the vast amount of genetic data that exists today could help provide a faster, more targeted way of developing new drug candidates. The logical extension to this kind of approach is treating individual patients, with their individual genetic makeup.

Genetics 98

Genetics Drugs Genome Project Biobanking 98

Drug Discovery World

MAY 24, 2023

Listen here: The post New podcast on animal genomics, immunology and CGTs appeared first on Drug Discovery World (DDW). You can also find The Drug Discovery World Podcast on Spotify , Google Play and Apple Podcasts.

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Genome Genomics Gene Therapy Gene 52

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. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Gene Therapy Gene Production Clinical Trials 52

Drug Discovery World

APRIL 17, 2024

Join DDW for this exclusive free event, ‘ rAAV genome sequencing with Oxford Nanopore uncovers transgene integrity and packaging impurities ’. Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The event will be supported by Oxford Nanopore Technologies.

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DNA Genome Genomics Gene Therapy 98

pharmaphorum

APRIL 27, 2021

Dosing can start once again in uniQure’s phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by the treatment. . The post FDA lifts hold on uniQure gene therapy after cancer case review appeared first on.

Gene Therapy 52

Gene Therapy Gene Genome Genomics 52

Scienmag

MAY 19, 2021

Grew over $200 billion since 2010, outlines new areas of promising application WASHINGTON, DC (May 19, 2021) – Human genetics and genomics contributed $265 billion to the U.S.

Genome 43

Genome Genomics Genetics Gene Therapy 43

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

STAT News

FEBRUARY 22, 2023

The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham.

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Gene Editing Gene Genetic Disease Gene Therapy 126

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

Gene Therapy 59

Gene Therapy Gene Trials Gene Expression 59

Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Drugs Gene Editing Genome Genomics 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

Pharmaceutical Technology

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Development In-Vivo Genome Genomics 130

pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Then over the next two or three years, gene therapy was accepted as something that companies got involved in, and several biotechs have been bought up by big pharma.”. The genomic medicine journey.

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Gene Editing Gene Gene Therapy In-Vivo 145

Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Editing Gene In-Vivo Gene Therapy 147

Drug Discovery World

JUNE 22, 2023

“This is part of our long-term business strategy of leveraging exosome-mediated delivery to create and enable genetic medicines such as gene therapy and genome editors”.

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Gene Therapy Gene Editing Drug Delivery Gene 52

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. per share, a rare 660% premium on LogicBio’s share price. .

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Gene Editing Gene Genome Genomics 64

Scienmag

FEBRUARY 21, 2021

‘Exploring one of the biggest challenges facing genomics today – understanding genetic variants’ SEATTLE (February 22, 2021) – Global experts performing leading edge research in precision medicine, functional genomics, protein science, and variant interpretation and prediction will share their expertise and insights April 5-7 (..)

Genome 40

Genome Genomics Genetics Protein 40

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.

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Genome Genomics Research Gene Therapy 71

Pharmaceutical Technology

DECEMBER 14, 2022

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics. By Cytiva Thematic.

Life Science 162

Life Science Development Gene Therapy Genetics 162

Pharmaceutical Technology

JULY 29, 2022

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. Their platform identifies which regRNAs control the specific gene of interest.

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RNA Gene Expression Gene Gene Therapy 245

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Roots Analysis

FEBRUARY 23, 2022

Over the past few years, several companies have started offering a diverse range of genome sequencing products and services using various second and third generation sequencing technologies. The post Next Generation Sequencing (NGS) Library Preparation Kits: Revolutionizing the Field of Genetic Sciences appeared first on Blog.

Genetics 52

Genetics DNA RNA Reagent 52

Drug Discovery World

OCTOBER 10, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Gene Editing 59

Gene Editing Genome Genomics Genetics 59

Drug Discovery World

DECEMBER 6, 2023

We feel this reflects the strong support for our vision and confirms the potential of Eligo to create a novel class of transformative genetic medicines.” This is a defining time for Eligo as this funding puts us in a strong position to make a significant leap in treating diseases by editing the genetic makeup of the human microbiome.”

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Gene Editing Gene In-Vivo Genetics 52

XTalks

DECEMBER 4, 2023

XTALKS WEBINAR: Cryopreservation Best Practices for Cell & Gene Therapy Source Material Live and On-Demand: Monday, January 22, 2024, at 11am EST (4pm GMT/UK) Register for this free webinar to learn about cryopreservation, a vital step in the manufacturing of today’s cell and gene therapies.

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Gene Therapy Gene Clinical Trials Clinical Trials 69

Drug Discovery World

NOVEMBER 1, 2022

Researchers say they have successfully used a cell’s natural process for making proteins to ‘slide’ genetic instructions into a cell and produce critical proteins missing from those cells. . Such disorders include neurodegenerative diseases that affect the brain, including Alzheimer’s disease, forms of blindness and some cancers. .

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Gene Expression Gene Packaging Packaging 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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Genome Genomics Medicine Gene Therapy 244

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. OVs can be attenuated natural viruses or recombinant viruses.

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Protein Gene Genetics Gene Therapy 130

pharmaphorum

JUNE 23, 2022

The deal comes a year after Intellia and partner Regeneron reported promising results from the first clinical trial of a drug used to edit the genomes of cells within the body, in patients with rare disease ATTR amyloidosis. billion in potential milestones if the project advances through development and onto the market.

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