Remove Gene Expression Remove Protein Remove RNA
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US FDA grants Fast Track status for Avidity’s AOC 1020 to treat FSHD

Pharmaceutical Technology

AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4. Avidity Biosciences’ AOC 1020 is intended to reduce the DUX4 mRNA and DUX4 protein expression in muscles in these patients.

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Gene Therapy and Pharmacokinetics

Camargo

Administration routes for gene therapies can differ depending on the indication target’s delivery needs. For example, it is beneficial to administer RNA treatments for some lung diseases directly to the disease site through inhalation. Gene Expression Considerations. Route of Administration Considerations.

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Combining the Powers of Single Cell Sequencing and AI in Understanding Disease Biology and Drug Development

XTalks

In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.

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Key early steps in gene expression captured in real time by CSU researchers

Scienmag

Capturing how RNA polymerase enzymes kick off transcription On scales too small for our eyes to see, the business of life happens through the making of proteins, which impart to our cells both structure and function.

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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

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AI-designed protein awakens silenced genes, one by one

The Pharma Data

By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. These methyl groups must be refreshed so if PRC2 is blocked the genes it has silenced. Cas9 binds and uses RNA as an address-tag.

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Leading innovators in transcription factors for genetically modified cells for the pharmaceutical industry

Pharmaceutical Technology

Innovation S-curve for the pharmaceutical industry Transcription factors for genetically modified cells is a key innovation area in the pharmaceutical industry Transcription factors are proteins which control gene expression by controlling the rate of transcription of genetic information from DNA to RNA.

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