article thumbnail

Unlocking the potential of synthetic DNA 

Drug Discovery World

Raquel Sanches-Kuiper , Vice President of Science and Applications at Evonetix, and Clare Whitewoods , Marketing Communications Manager at Evonetix, look at the benefits synthetic DNA brings to pharmaceutical development. Their speed also allows for their potential use in personalised therapy for diseases such as cancer.

DNA 173
article thumbnail

Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

Insiders

Sign Up for our Newsletter

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

article thumbnail

AI in gene delivery vector discovery and design

Drug Target Review

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union. Molecular Therapy 20 , 1831-1832 (2012). Nature 578 , 229-236 (2020).

article thumbnail

The science of ageing and restoring healthspan

Drug Target Review

Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a clinical-stage private biopharma company developing therapies for neurological and psychiatric diseases.

Science 145
article thumbnail

The power of combinations in blood cancers

Drug Target Review

2 It is this complexity that necessitates powerful, targeted combination therapies. 4, 5 More recently, new combination regimens have emerged that incorporate targeted therapies to treat a variety of blood cancers, including multiple myeloma (MM), chronic lymphocytic leukaemia (CLL) and acute myelogenous leukaemia (AML).

article thumbnail

Where is the drug discovery expertise happening in the UK?

Drug Discovery World

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. CellProthera also appointed the Newcastle Centre for Life’s GMP cell therapy centre to produce the ATMP.”

Drugs 189
article thumbnail

ViiV Healthcare Announces Analysis Showing no Antiretroviral Therapy Interruptions Due to COVID-19 Across its Clinical Development Programme for Investigational, Long-Acting Cabotegravir and Rilpivirine

The Pharma Data

In the midst of the global pandemic, the analysis found no antiretroviral therapy interruptions across the entirety of the ongoing clinical development programme for long-acting cabotegravir and rilpivirine. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days.