Pharmaceutical Technology

JULY 29, 2022

Last week, CAMP4 Therapeutics announced the close of a $100 million Series B round , which will be used to advance their regulatory RNA (regRNA)-focused programs. CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

Pharmaceutical Technology

JANUARY 19, 2023

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. “We

Gene Expression 246

Gene Expression Gene Protein RNA 246

Camargo

JULY 27, 2021

Additionally, gene editing allows us either to remove or to modify harmful genes. While most gene therapy clinical studies are ongoing, a number of products are in advanced clinical development, and several are approved by FDA. Gene Expression Considerations. Route of Administration Considerations.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

pharmaphorum

FEBRUARY 9, 2021

million ($22 million) in second-round financing as it prepares to start clinical trials of its lead drug candidate INT-1B3 for solid tumours. The start of dosing in the phase 1/1b trial of the drug has been delayed by the COVID-19 pandemic, but recruitment of the target 80 participants with advanced solid tumours is reported to be ongoing.

Trials 59

Trials Gene Silencing RNA Gene Expression 59

Delveinsight

JULY 24, 2021

Ovid Therapeutics is running Phase III clinical trials for its drug, Gaboxadol ( OV101 ). Another therapy by the same company named GTX-102 is also an antisense therapy; however, it silences gene expression by binding to the RNA molecule that regulates its expression.

Gene Therapy 96

Gene Therapy Gene Gene Expression RNA 96

XTalks

JUNE 18, 2024

The company is currently testing its lead candidate, EXG-34217 — an autologous cell therapy in Phase I/II trials for treating telomere biology disorders (TBDs), conditions known to cause bone marrow failure with treatments mainly limited to complicated bone marrow transplants. This metaphor captures the essence of their therapeutic approach.

Protein 52

Protein RNA Genetics Gene Expression 52

Roots Analysis

AUGUST 31, 2023

Mechanism of Gene Switch During the transcription process, the promoter region, which is located near the upstream end of each gene, binds to transcription factor, which is a specific type of protein. An additional level of genetic control is provided by gene switches that are located upstream of the promoter region.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40