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. DS: How easy was it to transfer theoretical research into a viable business?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

Delveinsight

JULY 24, 2021

The necessity of a robust Angelman syndrome pipeline is indispensable at the current moment because of a shortage of any approved therapy option available in the market. The Angelman Syndrome current treatment landscape consists of symptomatic therapy options worth USD 330.2 million in 2017.

Gene Therapy 96

Gene Therapy Gene Expression Gene RNA 96

pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

XTalks

MARCH 13, 2024

Related: Transforming Eye Care: Innovating New Glaucoma and Dry Eye Disease Treatments — Featuring Pierre Simon, VP Marketing, Sight Sciences – Xtalks Life Science Podcast Ep. There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease.

Gene Therapy 111

Gene Therapy Gene Life Science DNA 111

Roots Analysis

JANUARY 14, 2024

Like all drugs, biologics are regulated by the FDA. Usually, the desired gene, such as human insulin gene, when inserted into the plasmid of the host cell uses transcriptional and translational machinery of the host to express itself. They are different from small molecules in terms of their size and complexity.

Contract Manufacturing 40

Contract Manufacturing Manufacturing Protein Bacteria 40

Pharmaceutical Technology

JANUARY 24, 2023

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

Gene Therapy 208

Gene Therapy Gene Gene Expression Regulation 208

Delveinsight

JANUARY 18, 2021

The present Angelman syndrome treatment market revolves around the management of symptoms rather than curing the condition. . Several pharmaceutical and biotech companies in the Angelman syndrome market are exploring the novel curative approaches. GeneTx and Ultragenyx are co-developing the therapy.

Marketing 52

Marketing Gene Drugs Protein 52