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

PLOS: DNA Science

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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AI in gene delivery vector discovery and design

Drug Target Review

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Molecular Therapy 20 , 1831-1832 (2012).

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2021 Biopharma Update on the Novel Coronavirus: January 12

The Pharma Data

Testing Therapies, Antivirals and Vaccines. Pfizer and BioNTech reported that their COVID-19 vaccine appears to be effective against a key mutation in new variants of the SARS-CoV-2 virus that causes COVID-19. SARS-CoV-2, the virus that causes COVID-19, has mutated into several strains. A study published by the U.S.

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Regeneron’s COVID-19 Outpatient Trial Prospectively Demonstrates that REGN-COV2 Antibody Cocktail Significantly Reduced Virus Levels and Need for Further Medical Attention

The Pharma Data

“We continue to see the strongest effects in patients who are most at risk for poor outcomes due to high viral load, ineffective antibody immune response at baseline, or pre-existing risk factors. In the overall patient group with detectable virus at baseline, the average daily reduction in viral load through day 7 was a 0.36

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Clover and Dynavax Announce Planned Global Phase 2/3 Efficacy Trial of Adjuvanted COVID-19 Vaccine Candidate

The Pharma Data

Clover’s S-Trimer antigen adjuvanted with CpG 1018 plus alum demonstrated low reactogenicity while providing high levels of neutralizing antibodies and a strong Th1-biased cell-mediated immune response. Dynavax developed CpG 1018 to provide an increased vaccine immune response, which has been demonstrated in HEPLISAV-B.

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Roche launches new quantitative antibody test to measure SARS-CoV-2 antibodies, to support the evaluation of vaccines

The Pharma Data

The new Elecsys Anti-SARS-CoV-2 S test can quantitatively measure the level of antibodies against SARS-CoV-2 in patients who have been exposed to the virus. This is the focus of vaccines in development and convalescent plasma therapy. The test targets antibodies against the spike protein. said Thomas Schinecker, CEO Roche Diagnostics.

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FIRST AUTHORIZATION IN EUROPEAN UNION FOR COVID-19 VACCINE IN ADOLESCENTS

The Pharma Data

This is a meaningful moment for adolescents, parents and entire families who are seeking protection from this virus and a return to normalcy,” said Albert Bourla, Chairman and Chief Executive Officer, Pfizer. “We “Making vaccines available to adolescents will help re-open schools, and support the return to a normal day-to-day life.”.

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