Drug Discovery World

APRIL 16, 2024

Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The post rAAV Genome sequencing uncovers transgene integrity and packaging impurities appeared first on Drug Discovery World (DDW). A live Q&A session follows the webinar presentation.

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Packaging Packaging Genome Genomics 52

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.

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Drug Discovery World

JUNE 14, 2024

With a lead story on the restoration of congenital hearing loss following gene therapy, the news highlights selection this week focuses on cell and gene therapies (CGT) and genetics, particularly some key progress in relation to CAR-T therapies.

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Gene Therapy Drugs Gene Genetics 52

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

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

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.

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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

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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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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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.

Drugs 52

Drugs Gene Editing Genome Genomics 52

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.

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Genetics Medicine Gene Therapy Marketing 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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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

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

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

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 (..)

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Scienmag

JULY 23, 2021

Jude Children’s Research Hospital study highlights the power of comprehensive whole genome, whole exome and RNA sequencing to better understand and treat each patient’s cancer Credit: St. Jude Children’s Research Hospital St.

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Medicine Genome Genomics RNA 96

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.

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Gene Editing Genome Genomics Genetics 59

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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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

Scienmag

NOVEMBER 12, 2020

In a study that has unprecedented implications to advance both medicine and biodiversity conservation, researchers have sequenced 131 new placental mammal genomes, bringing the worldwide total to more than 250 In a study that has unprecedented implications to advance both medicine and biodiversity conservation, researchers have sequenced 131 new placental (..)

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Biobanking Medicine Genome Genomics 41

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. . Therapies that don’t precisely target diseased cells can have unintended effects in other healthy cells.” . Splicing-linked expression design.

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Gene Expression Gene Packaging Packaging 52

Pharmaceutical Technology

MAY 17, 2023

Under the deal, Prevail obtains exclusive rights to use Scribe’s CRISPR X-Editing (XE) technologies to develop the medicines. The deal will see the integration of Scribe’s new CRISPR by Design approach and Prevail’s expertise in developing genetic medicines for neurological disorders for specific genetic targets.

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XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. a biotech company specializing in the development and commercialization of genetic medicines for rare diseases, announced FDA approval for Vyjuvek for the treatment of DEB.

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Drug Discovery World

JUNE 10, 2024

Five children who were born with congenital hearing loss can hear in both ears after taking part in a gene therapy trial at Fudan University in Shanghai, China. Within weeks of receiving the therapy, the children could locate the sources of sounds, and recognised speech in noisy environments. Read the full paper.

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Gene Therapy Gene Genetics Clinical Trials 59

Cloudbyz

MARCH 6, 2024

The Advantages of Biologics: The rise of biologics has brought forth numerous advantages over traditional pharmaceutical therapies: Targeted Therapies: Biologics can be designed to target specific molecules or pathways implicated in disease pathology, leading to enhanced efficacy and reduced side effects compared to non-specific treatments.

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Cloudbyz

JUNE 26, 2023

This can aid in personalized medicine, disease management, and risk assessment, leading to improved patient care and outcomes. Generative AI has the potential to transform clinical research by leveraging large-scale data analysis, simulating complex biological systems, and facilitating personalized medicine.

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Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. billion, compared to $19.9

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Gene Therapy Drugs Gene Manufacturing 52

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. billion in funding. Government investment of £1.3

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Drugs Life Science Gene Therapy Genome 75

Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine.

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Marketing Gene Sequencing DNA Genome 52

Drug Discovery World

SEPTEMBER 14, 2023

Excluding contributions from Comirnaty and Paxlovid, company revenues grew $537 million, or 5%, operationally. AstraZeneca saw total revenue reach $22,295m, up 4% despite a decline of $2,181m from Covid-19 medicines. Excluding Covid-19 medicines, total revenue increased 16% and product sales increased 15%. Specialty care grew 11.8%

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Drugs Vaccination Vaccine Medicine 59

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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Drug Discovery World

NOVEMBER 27, 2023

In the UK, following Medicines and Healthcare products Regulatory Agency (MHRA) approval on 15 November 2023, and as a result of the existing reimbursement agreement between Vertex and the NHS, children aged two years and above in the UK have access to this expanded indication for Kaftrio in a combination regimen with ivacaftor.

Protein 52

Protein Gene Medicine Gene Therapy 52

pharmaphorum

MAY 18, 2022

At UCB, we are trying to understand the whole spectrum of triggers causing epileptic seizures, from genetic factors, brain injury to stroke, cancer or other traumas,” Charl states. Charl believes we are witnessing the ‘decade of the brain’ with significant leaps in scientific understanding of disease pathways and genetic correlations.

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Gene Therapy Gene Genetics Development 92

Drug Discovery World

DECEMBER 15, 2022

Vor Bio, a clinical-stage cell and genome engineering company, has announced initial clinical data from VBP101, its Phase I/IIa multicentre, open-label, first-in-human study of tremtelectogene empogeditemcel or “trem-cel” (formerly VOR33) in patients with acute myeloid leukemia (AML). . Official comments .

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Drugs Genome Genomics Engineer 52

pharmaphorum

SEPTEMBER 12, 2022

Alongside drugs and therapeutics, data collection and technology enhancements have redefined the traditional healthcare experience, especially within oncology, and genomic profiling has become a significant factor in allowing for personalised care. In other words, every person’s cancer has its own genetic construct. Tailored oncology.

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Genome Genomics Clinical Trials Clinical Trials 106

XTalks

MARCH 8, 2022

From leading COVID-19 vaccine developments to being at the helm of the latest gene therapies and inventing ground-breaking DNA technologies like CRISPR, women have been at the forefront of some of the most leading-edge scientific discoveries in recent years, both as innovators and leaders.

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