Drug Discovery World

MARCH 26, 2024

More importantly, it also highlighted that significant challenges remain in selecting the right therapies for the right patients despite the progress made through genomic testing and implementation of circulating tumour (ct) DNA analyses. Results of that Phase I trial are expected to be presented later this year.

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RNA In-Vivo In-Vitro DNA 64

Drug Discovery World

AUGUST 14, 2023

For example, the FDA’s Center for Biologics Evaluation and Research (CBER) 3 , which regulates biological products for human use, differentiates between in vivo gene therapy (new genes are inserted into the body to target cells) and ex vivo gene therapy (cells are modified outside the body and then administered back into the patient).

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Gene Therapy Marketing Gene DNA 52

Drug Discovery World

DECEMBER 15, 2022

These platforms all induce double strand cuts in the chromosomal DNA that can be sealed by the cell, leading to a specific gene disruption, or resulting in a new target site for inserting DNA segments. Early phase clinical trials for gene editing therapies. Gene editing challenges and potential solutions.

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Gene Editing Gene In-Vivo Genome 52

The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. “We

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Clinical Trials Clinical Trials Gene Therapy Trials 52

Roots Analysis

AUGUST 31, 2023

Gene switches can be regulatory proteins or specific DNA sequences that act to either switch on or off the expression of a gene. Basic Components of Gene Switch Gene switches are composed of noncoding DNA sequences and transcription factors. These genetic switches assist transcription factors in binding to the promoter region.

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Gene Gene Therapy Gene Expression Regulation 40

Roots Analysis

SEPTEMBER 18, 2023

Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry. The above figure presents a general overview of the cryopreservation process.

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Engineer Containment In-Vivo Production 52

Drug Discovery World

NOVEMBER 30, 2023

DDW’s Megan Thomas attended PEGS Europe 2023 in Lisbon, Portugal, from Tuesday 14 November to Thursday 16 November 2023. The company’s goal is to accelerate functional characterisation and shortenoptimisation time in antibody discovery and t-cell workflows. GSK has developed high-throughput mammalian and E.

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Antibody Protein Engineer In-Vivo 59