pharmaphorum

JUNE 27, 2022

The safety of Astellas’ gene therapy portfolio has been thrust into the spotlight once again, after the FDA placed a clinical hold on a trial of its Pompe disease candidate AT845. The delivery vector – an AAV8 capsid – is designed to allow gene expression directly in target tissues like skeletal muscle.

Gene Therapy 52

Gene Therapy Gene Gene Expression Trials 52

XTalks

MAY 4, 2021

These modifications regulate gene expression without changing the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

Roots Analysis

AUGUST 31, 2023

Type of Gene Regulation Based on the type of transcription factor ( activators / repressors ), gene regulation has been categorized into positive and negative gene regulation. When activator binds to the operon, it either speeds up or permits gene expression.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

XTalks

MAY 4, 2021

These modifications regulate gene expression without altering the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. pyogenes dCas9.

DNA 52

DNA Gene Gene Silencing Genome 52

The Pharma Data

OCTOBER 15, 2020

“For cell therapy products to mature into ‘real’ products that deliver on the promises of 10 years ago, they must be scalable – which drives affordability – and they must solve their purity issues.”. Real-time regulatory feedback isn’t available, so you proceed, presuming that what you’re doing will be acceptable to regulators.”.

Manufacturing 52

Manufacturing Gene Therapy Gene Gene Expression 52

Roots Analysis

JANUARY 14, 2024

Like all drugs, biologics are regulated by the FDA. Usually, the desired gene, such as human insulin gene, when inserted into the plasmid of the host cell uses transcriptional and translational machinery of the host to express itself. They are different from small molecules in terms of their size and complexity.

Contract Manufacturing 40

Contract Manufacturing Protein Manufacturing Production 40

The Pharma Data

MAY 15, 2021

I was recently reminded of my journey out of the HCT nightmare as I watched a documentary about gene therapy. Gene therapy is a huge deal – some gene therapies are already achieving jaw-dropping results for people with previously untreatable illnesses. It’s fascinating stuff.

Bacteria 52

Bacteria Gene Gene Therapy Gene Expression 52

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. Vaccine Considerations. CRISPR Therapeutics.

Life Science 111

Life Science Vaccination Vaccine Gene 111