Drug Discovery World

NOVEMBER 27, 2023

Across the biotherapeutics workflow The landscape of large molecule drug development has evolved significantly in recent decades. Prioritising critical attributes in the upstream workflow is crucial for predicting and preventing challenges downstream, ultimately avoiding high development costs and delays in drug approvals.

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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. Financial details were not disclosed. Source link: [link]

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Drug Discovery World

OCTOBER 16, 2023

The United Kingdom has established itself as a hub for drug discovery expertise worldwide. There is a range of reasons why this area has become focal point for drug discovery, one of which is the fact that it encompasses the University of Oxford and the University of Cambridge, two of the oldest and most prestigious universities in the world.

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Drug Discovery World

FEBRUARY 16, 2023

The post Start-up aims to advance transformative genetic medicines appeared first on Drug Discovery World (DDW). “Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings.

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Drug Discovery World

JANUARY 12, 2023

Oksana Sirenko looks at the advantages and limitations of using organoids from human cell sources in drug development and drug screening. . The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. They also present ethical, monetary, and time-related challenges. .

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XTalks

JULY 14, 2022

For example, when T cells (a type of white blood cells that fight off infections and cancer) are activated in our immune systems, thousands of proteins in these cells change. The proteins are interconnected and changes in one protein level can impact that of another.

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XTalks

NOVEMBER 30, 2023

In 2022, the US Food and Drug Administration (FDA) granted approval to the medication Sunlenca (lenacapavir) for adults living with multi-drug resistant HIV-1 infection. Developed by Gilead, Sunlenca is the first drug in a novel class known as capsid inhibitors to receive FDA approval for treating HIV-1.

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XTalks

MARCH 28, 2024

Gene Editing and CAR T-Cell Therapy Genetic engineering technologies, such as CRISPR-Cas9, are providing newer potential avenues for cancer treatment. For example, gene editing can possibly be used to correct cancer-causing mutations or enhance the effectiveness of chimeric antigen receptor (CAR) T-cell therapy.

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Cloudbyz

MARCH 6, 2024

Traditionally, pharmaceutical therapies have primarily consisted of small molecule drugs, commonly known as pills. This shift signifies not just a change in the way drugs are manufactured and administered, but also a fundamental transformation in the approach to treating diseases at their root cause.

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The Pharma Data

JANUARY 24, 2021

“We are excited to welcome Chris, a highly regarded drug developer and team builder who brings to Notch great depth of experience and expertise in development of cell therapies, gene editing, and cell engineering spanning discovery through IND,” said David Main , President and Chief Executive Officer of Notch.

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Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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Delveinsight

AUGUST 27, 2020

Freenome’s diagnostic searches for direct indication of tumors and signs of the immune system’s response, integrating genomics, transcriptomics, methylomics, and proteomics technologies. Targeted drug combo attacks breast cancer brain metastases in mice. White fat, in contrast, can build up and cause obesity.

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Drug Discovery World

DECEMBER 13, 2022

One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. The genetic anomaly blocks the chemical glycosylation of a biologically important protein. The post RNA interference method could treat muscular dystrophy appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MARCH 9, 2023

The US Food and Drug Administration (FDA) has cleared Intellia Therapeutics’ Investigational New Drug (IND) application for NTLA-2002 for the treatment of hereditary angioedema (HAE). This includes the measurement of plasma kallikrein protein levels and clinical activity as determined by HAE attack rate measures.

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Pharmaceutical Technology

FEBRUARY 13, 2023

Although recently approved drugs can treat seizures more safely, they do not treat the comorbidities that patients experience. Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome.

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pharmaphorum

FEBRUARY 3, 2021

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

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pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. . — Eric Topol (@EricTopol) June 26, 2021.

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Drug Discovery World

MAY 2, 2023

Steve Wowk , VP, Business Unit and General Management of Integrated DNA Technologies, shared with DDW the value of CRISPR-Cas9 in drug discovery. The CRISPR-Cas9 system, awarded the Nobel Prize in Chemistry in 2020 for its applications for gene editing, was initially encountered in the bacterial immune system.

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Drug Discovery World

MAY 30, 2023

From antibody-drug conjugates and RNA, to CRISPR and CAR-T therapy, DDW asked the drug discovery industry what the most important development in oncology over the last five years has been. Immunopheresis is a ‘subtractive therapy,’ in contrast to alternative drugs that constitute ‘additive’ therapies.

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Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

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Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. While type II (Cas9) is mainly used for gene-editing applications, CRISPR detecting tools primarily rely on type V (Cas12) and type VI (Cas13) collateral activity.

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Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. The success of base editing has led to rapid advances toward commercialisation and clinical application.

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The Pharma Data

JANUARY 10, 2021

Nasdaq: EDIT), a leading genome editing company, today announced the U.S. Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. Enrolled patients will receive a single administration of EDIT-301.

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The Pharma Data

JANUARY 24, 2021

The drug appears to inhibit IL-6, IL-1beta, TNF-alpha and other pro-inflammatory cytokines and chemokines. It includes adults with confirmed COVID-19 on supplemental oxygen or mechanical ventilation with abnormal levels of at least two serum biomarkers, C reactive protein, D dimer, lactate dehydrogenase, or ferritin.

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XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. Drugs, Testing and Vaccines. This makes the latter potentially easier to target with drugs and vaccines.

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