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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. Affected children rarely live past infancy.

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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

” Dr. Stanley Plotkin , Professor Emeritus at The Wistar Institute, said, “INOVIO’s DNA vaccine appeared to be quite safe with few significant reactions but yet induced both antibody and T cell responses to SARS-CoV-2.” About INOVIO’s DNA Medicines Platform. mg and 2.0

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Revolutionary nanoparticles enable gene-editing in lungs

Drug Target Review

Gene-editing methods allow researchers to modify DNA, resulting in alterations in physical characteristics such as eye colour and susceptibility to diseases. To achieve this, scientists utilise various technologies that function similarly to scissors, precisely cutting the DNA at a particular location.

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Unveiling neoantigen-directed cancer treatment

Drug Target Review

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

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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. 9 In addition, host immune responses further add to the complexity of developing cell-specific AAV capsids for clinical applications. Nature reviews Drug discovery 18 , 358-378 (2019).

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Accelerating biotherapy and personalised medicine with long DNA

Drug Discovery World

Matt Hill , Founder and CEO of Elegen writes about the importance of innovation in DNA synthesis to address critical bottlenecks in biotherapy development. Regardless of the type of disease targeted, at the core of biotherapy development is synthetic biology and the use of synthetic DNA to programme specific behaviours within living cells.

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AstraZeneca takes COVID-19 vaccine to China with BioKangtai deal for 200M-dose capacity by 2021

The Pharma Data

Early clinical results showed AstraZeneca’s candidate, AZD1222, triggered both antibody and T-cell immune responses. In addition, Inovio previously teamed up with Beijing Advaccine Biotechnology for work on its DNA vaccine candidate, INO-4800. The vaccine was moved into phase 2/3 in May.

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