Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. The company’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

Scienmag

NOVEMBER 16, 2020

Singapore scientists uncover potential role of long non-coding RNAs in pancreatic cancer Credit: From Figure 4 in Liu, S., Wnt-regulated lncRNA discovery enhanced by in vivo identification and CRISPRi functional validation. Harmston, N., Glaser, T.L. Genome Med 12, 89 (2020).

In-Vivo 53

In-Vivo RNA DNA Genomics 53

pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. Through base editing, drugs replace single nucleotide in the DNA strand with another, without making double-strand breaks (DSBs) in the gene.

Gene Editing 98

Gene Editing Trials Drugs In-Vivo 98

The Pharma Data

NOVEMBER 22, 2020

Back in September, Vertex Pharmaceuticals and CRISPR Therapeutics – the company behind the CRISPR Cas-9 platform – announced that the European Medicines Agency (EMA) had granted Priority Medicines (PRIME) designation to CTX001, an investigational ex vivo CRISPR Cas-9 gene-edited therapy for the treatment of severe sickle cell disease.

Gene Editing 52

Gene Editing DNA Genomics Genome 52

Pharmaceutical Technology

FEBRUARY 14, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. These systems include human and mouse cell lines, and even in vivo in live animals.

In-Vivo 130

In-Vivo DNA Antibody Gene 130

XTalks

AUGUST 12, 2020

Studies have identified the presence of two types of HIV-infected CD4+ T cells: ‘Transcriptionally inactive’ cells that do not typically produce viral RNA or viral proteins. Transcriptionally active’ cells in which HIV RNA is actively transcribed to make copies of the virus (despite long-term ART).

RNA 52

RNA Protein DNA In-Vivo 52

Delveinsight

FEBRUARY 25, 2021

The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. Beam employs three approaches to deliver its genetic medicines to cells.

DNA 52

DNA In-Vivo Genetics Medicine 52