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

Broad Institute

The first problem they had to tackle was the editor’s size, because the editor needs to be small enough to be packaged and delivered to specific cells in the body. 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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New insights into the role of viral capsids in gene therapy safety

Drug Target Review

2 However, utilisation of biological systems introduces variability and by-products, as well as a challenge unique to AAV production: the assembly of full capsids with correctly packaged DNA, partially filled capsids containing partial vector and/or host cell DNA, and empty capsids. Bio-Rad, Bulletin 3725 [cited 2024 Oct 28].

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Regulatory Submissions for Dengue Vaccine Candidate in EU and Dengue-Endemic Countries

The Pharma Data

2 Clinical Phase 2 data in children and adolescents showed that TAK-003 induced immune responses against all four dengue serotypes, in both seropositive and seronegative participants, which persisted through 48 months after vaccination, and the vaccine was found to be generally safe and well tolerated.

Vaccine 52
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Codon Digest: Seeing Colors After Gene Therapy

Codon

A new paper from Michael Elowitz’s group at Caltech reports engineered RNA exporters that “efficiently and specifically package and secrete target RNA molecules from mammalian cells within protective nanoparticles.” a lower risk of immune responses. No longer. ” And a third paper (BOOM!) mycoides cell.

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Codon Digest: Seeing Colors After Gene Therapy

Codon

A new paper from Michael Elowitz’s group at Caltech reports engineered RNA exporters that “efficiently and specifically package and secrete target RNA molecules from mammalian cells within protective nanoparticles.” a lower risk of immune responses. No longer. ” And a third paper (BOOM!) mycoides cell.

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Regeneron Announces Encouraging Initial Data from COVID-19 Antibody Cocktail Trial in Hospitalized Patients on Low-flow Oxygen

The Pharma Data

The analysis was prospectively designed to focus on patients who had not yet mounted their own immune response to SARS-CoV-2 (i.e., All patients entering the trial were hospitalized with laboratory-confirmed COVID-19 requiring low-flow oxygen, and all received other background standard-of-care as required. futility analysis).

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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. Yancopoulos , M.D., President and Chief Scientific Officer of Regeneron. Regeneron has shared these results with the U.S.

Virus 40