Remove DNA Remove Immune Response Remove Trials
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A research team searches for every gene that helps tumors evade immunotherapy

Broad Institute

By then, a few cancer immunotherapies had entered clinical trials and only a handful had been approved by the FDA. As a first step, Manguso set his sights on treatments called PD-1 checkpoint inhibitors, which at the time had shown tremendous promise in clinical trials and would receive their first FDA approval in 2014.

Research 137
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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

Unlike gene editing, this “epigenetic” editing does not modify the underlying DNA sequence, but it should switch the gene off permanently, which means that this could be a one-time treatment. ZFPs are also common in human cells, meaning they are less likely to trigger an immune response against themselves than the bacterial Cas9.

Disease 142
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UK Pharma Company Scancell Selects COVID-19 Vaccine for Clinical Trials | 2020-12-21

The Pharma Data

UK drug developer Scancell said it has chosen a COVID-19 vaccine candidate, SN14, from more than a dozen potential products to advance to a clinical trial. . SN14 works by targeting the coronavirus’ nucleocapsid and spike proteins to prevent viral replication using the company’s ImmunoBody DNA vaccine technology. Source link.

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FDA Returns Disappointing News for ALS Stem Cell Therapy

PLOS: DNA Science

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). The two conditions and the therapeutic approaches differ, but their clinical trials illustrate the importance of selecting patients whose characteristics suggest that they are the most likely to respond.

Therapies 111
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Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

Can they deliver healing genes without triggering an overactive immune response? The details are disturbingly reminiscent of the famous case of Jesse Gelsinger , who died from a ferocious immune response to experimental gene therapy in September 1999. million DNA bases. Muscles stop working.

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INOVIO Announces Publication of Phase 1 Data from its COVID-19 DNA Vaccine Candidate, INO-4800 in The Lancet’s EClinicalMedicine

The Pharma Data

Joseph Kim , President and CEO of INOVIO, said, “We are very pleased to share peer-reviewed Phase 1 clinical data for INO-4800 published in The Lancet’s EClinicalMedicine , and are grateful for the support of all participants and investigator staff involved in the clinical trial.” . PLYMOUTH MEETING, Pa. ,

DNA 40
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Scaling Phage Therapy

Codon

1 Yet even after more than two decades of research, media hype, and dozens of clinical trials and biotech start-ups that have come and gone, phage therapy has not scaled. No phage-based therapeutic has reached the latter stages of the clinical trial pipeline (where promising results lead to an expanded trial involving thousands of patients).

Therapies 117