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.

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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. .

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Gene Expression Gene Protein RNA 246

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.

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Gene Therapy Gene Gene Expression RNA 96

The Pharma Data

MARCH 3, 2022

In 2014, Shalev’s UAB research lab reported that verapamil completely reversed diabetes in animal models, and she announced plans to test the effects of the drug in a human clinical trial. The United States Food and Drug Administration approved verapamil for the treatment of high blood pressure in 1981.

Insulin 52

Insulin Gene Expression Gene Protein 52

The Pharma Data

JANUARY 17, 2021

” The Company expects to report initial clinical data from the trial in 2021. Additional information about this clinical trial is available at clinicaltrials.gov using the identifier: NCT04669808. 1 and COX-2 gene expression.

Gene Expression 52

Gene Expression Trials Development Production 52

Delveinsight

JANUARY 18, 2021

For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation. Although, it can be relieving that other players turned to different approaches for finding a cure for Angelman syndrome.

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Marketing Gene Drugs Protein 52

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.

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Gene Therapy Gene Gene Editing Genetics 238