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Epigenetic editing: the next generation of genetic medicine

Drug Target Review

The reasons for this are multifaceted, including concerns over the safety of directly altering DNA sequences and subsequent regulatory restrictions that have arisen as a result. The epigenome (meaning ‘above the genome’) is a system of reversible marks regulating how the DNA is read, translated and used.

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DNA Analysis Finds New Target for Diabetes Drugs

NIH Director's Blog: Drug Development

Credit: Jane Ades, National Human Genome Research Institute, NIH Type 2 diabetes (T2D) tends to run in families, and over the last five years the application of genomic technologies has led to discovery of more than 60 specific DNA variants that contribute to risk. Nat Rev Drug Discov. 2013 Aug;12(8):581-94. [3]

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Navigating the challenges and opportunities of AI in drug development and personalised medicine

Drug Target Review

I think it is astonishing that The Human Genome Project was completed exactly 20 years ago and DNA was discovered by Franklin and published by Watson and Crick exactly 70 years ago, yet that 10 percent success rate has still not improved. This means that we are not doing something right.

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

Drug Target Review

2 Structure and function of AAV capsids in gene therapy Wild-type AAV is composed of a protein shell (capsid) that contains a single-stranded DNA genome encoding proteins involved in viral replication, structure and assembly. Therefore, vectors based on these serotypes may enable targeted gene delivery and optimal transduction efficiency.

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Tonix Pharmaceuticals Enrolls First Participant in the PRECISION Study, an Observational Study to Facilitate Development of Precision Medicine Techniques for COVID-19 Vaccines and Therapeutics

The Pharma Data

Assistant Professor of Medical Sciences at Columbia University Vagelos College of Physicians and Surgeons, is designed to generate DNA aptamer-based anti-idiotypes to selected monoclonal antibodies identified in Dr. Ilya Trakht’s study. The study led by Dr. Sergei Rudchenko, Ph.D.,

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Analysis Life Sciences Thank You FDA lays out new assessment framework for certain nitrosamines

Agency IQ

EMA’s investigation ended up revealing even more causes of nitrosamine impurities in drugs, discovering, for example , that formation happened during the heat-sealing process of in certain products using blister-packaging. Since this discovery, it’s been unclear how regulators or sponsors might assess or address these potential risks.

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Article FDA Thank You Draft guidance on potency assays for CGT products garners extensive stakeholder input

Agency IQ

The regulations also explain that this trait can be demonstrated via appropriate laboratory tests or adequately controlled clinical data. Additionally, six of these comments came from the same organization (Integrated DNA Technologies). However, only 40 of these comments have been posted to the docket for public review.