Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What diseases are you currently targeting? Can you give some examples?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

Camargo

JULY 27, 2021

Research and development in the area is currently growing at a fast rate, and the National Institute of Health reports hundreds of clinical trials to test gene therapies for different genetic diseases, immune system disorders, oncology treatments, neurogenerative diseases, infectious diseases, and more.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

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. Epigenetic Editing with CRISPR. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

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. Epigenome Editing with CRISPR. pyogenes dCas9.

DNA 52

DNA Gene Gene Silencing Genome 52

The Pharma Data

DECEMBER 27, 2020

The drug is a topical ointment applied to the lower lid to address the build-up and shedding of proteins at the opening of the Meibomian gland. GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder and a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases.

Trials 52

Trials Antibody Gene Therapy Clinical Trials 52