Remove Development Remove Gene Expression Remove Gene Therapy Remove In-Vivo
article thumbnail

Gene editing: beyond the hype

pharmaphorum

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. billion in funding.

article thumbnail

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Pharma Marketing Network

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. Genetic literacy among clinicians may not be as high as you think.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Biologics – The Next Step in Revolutionary Medication

Roots Analysis

With the increased interest and gradual shift of investment from small molecule drugs to biologics and the establishment of several biologics manufacturing companies / biologics CMOs, more than 250 biologic therapies and vaccines have been developed, globally.

article thumbnail

Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder

Delveinsight

The first sign of a child suffering from Angelman syndrome is a delay in development, such as the inability to sit without support or making incoherent babbling sounds. Angelman syndrome is a result of the missing or dysfunctional gene known as UBE3A. GeneTx and Ultragenyx are co-developing the therapy.

article thumbnail

Gene Switch: A Novel Platform for Switching Genes On and Off

Roots Analysis

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

Gene 40
article thumbnail

Delivering on the promise of gene editing

Drug Discovery World

These platforms all induce double strand cuts in the chromosomal DNA that can be sealed by the cell, leading to a specific gene disruption, or resulting in a new target site for inserting DNA segments. Gene editing tools can sometimes bind to unintended sites, typically because of sequence homologies and/or mismatch tolerance.

article thumbnail

CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. Years of development and fast-paced research have continued to unlock its potential, expanding how CRISPR can be used to treat, detect, and prevent common diseases such as cancer and Covid-19.

DNA 98