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

Broad Institute

To deliver CHARM throughout the brain, the team collaborated with scientists in the lab of Ben Deverman , senior director of vector engineering at the Broad and a co-author of the study. They used an engineered adeno-associated virus (AAV) that crosses the blood-brain barrier after intravenous administration. Online June 27, 2024.

Disease 142
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Unveiling neoantigen-directed cancer treatment

Drug Target Review

As part of the TCR discovery process and preclinical data package, Adaptive performs rigorous functional characterisation and safety assessments of potent, therapeutic grade antigen-specific TCRs. Genentech is responsible for engineering adaptive-discovered TCRs into T cells and manufacturing the T cell therapy product candidate.

Treatment 105
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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. An immune response is two-tiered.

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Scaling Phage Therapy

Codon

Such contaminants could have the same toxic effects as the bacterial infection, or provoke a severe immune response in the patient, and therefore the phage mixtures must go through extensive purification and testing procedures. The resulting data are fed into an analytical engine that runs millions of simulations. aeruginosa, S.

Therapies 117
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New insights into the role of viral capsids in gene therapy safety

Drug Target Review

2,3 Engineering both the capsid and DNA cargo improves the safety and efficacy of rAAV vectors and expands their clinical applications. Therefore, vectors based on these serotypes may enable targeted gene delivery and optimal transduction efficiency.

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Novavax COVID-19 vaccine demonstrates 90% overall efficacy and 100% protection against moderate and severe disease in PREVENT-19 Phase 3 trial

The Pharma Data

NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of the first strain of SARS-CoV-2, the virus that causes COVID-19 disease. It is packaged in a ready-to-use liquid formulation in 10-dose vials. BARDA is providing up to $1.75 billion under a Department of Defense agreement. About NVX-CoV2373.

Vaccine 40
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Codon Digest: Seeing Colors After Gene Therapy

Codon

But for a recent paper out of Nanjing, China, researchers engineered another enzyme and showed that, when mixed with FAST-PETase, the two worked together to break down plastic twice as fast as the Texas enzyme alone. a lower risk of immune responses. They don’t always break down PET into a form that is easily recycled.