Pharmaceutical Technology

JUNE 29, 2022

This year has already been eventful when it comes to the development of therapies for rare diseases. Additionally, pricing and access for rare disease therapies continue to be scrutinized closely. Most, if not all, of these therapies used the FDA’s Orphan Drug Designation to aid their development plans.

Drugs 246

Drugs FDA Approval Marketing Development 246

The Pharma Data

MARCH 7, 2022

“The beauty of this approach is we can safely upregulate specific genes to affect cell activity without permanently changing the genome and cause unintended mistakes,” Levy said. PRC2 can be blocked with chemicals, but they are imprecise, affecting PRC2 function throughout the genome.

Protein 52

Protein Gene Genome Genomics 52

The Pharma Data

MARCH 23, 2022

Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. In fact, antiviral therapies designed to treat viruses such as hepatitis C (Sofosbuvir/SOVALDI) work by blocking viral polymerase. Drugs that target polymerase could potentially treat Ebola virus infections and save lives.

Protein 52

Protein Genome Genomics Research 52

The Pharma Data

MARCH 16, 2022

using SNAP-tag) involves labeling cell proteins with a fluorescent dye, thereby enabling researchers to observe how proteins interact in the living cell and where in the cell they are located. With Spirochrome, he commercialized fluorescent probes for cell biology, while with Covalys, he marketed SNAP-tag and other protein labeling methods.

Protein 52

Protein Scientist Gene Development 52

Delveinsight

FEBRUARY 25, 2021

Instead of snipping the genome, base editing lets for edits of individual letters in a genetic sequence. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. If they eliminated the T cells, the therapy stopped working. Beam employs three approaches to deliver its genetic medicines to cells.

DNA 52

DNA In-Vivo Genetics Medicine 52

XTalks

FEBRUARY 27, 2024

The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies. Casgevy (exa-cel) and Lyfgenia (lovo-cel) were the first gene therapies to be approved by the FDA for the treatment of sickle cell disease (SCD).

Research 59

Research Drugs Gene Therapy Clinical Trials 59

Roots Analysis

AUGUST 28, 2022

Recent advances in multi-omics approaches, including genomics, transcriptomics, proteomics, metabolomics, cytometry and imaging, in combination with bioinformatics and biostatistics, have been translated into several popular bioanalytical platforms, such as next-generation sequencing (NGS), single-cell analysis, flow cytometry and mass spectrometry.

Genome 52

Genome Genomics RNA Genome Project 52