Remove Antibody Remove Gene Expression Remove RNA
article thumbnail

US FDA grants Fast Track status for Avidity’s AOC 1020 to treat FSHD

Pharmaceutical Technology

This DUX4 protein abnormal expression leads to modifications in gene expression in muscle cells which are associated with progressive muscle function loss in FSHD patients. Avidity Biosciences’ AOC 1020 is intended to reduce the DUX4 mRNA and DUX4 protein expression in muscles in these patients.

article thumbnail

Sirnaomics Announces First Patient Dosed In Phase 2a Study of STP705 for Treatment of Cutaneous Basal Cell Carcinoma

The Pharma Data

About STP705 Sirnaomics’ leading product candidate, STP705, is a siRNA (small interfering RNA) therapeutic that takes advantage of a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to directly knock down both TGF-?1 1 and COX-2 gene expression. About Sirnaomics, Inc.

Insiders

Sign Up for our Newsletter

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

article thumbnail

RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

RNA 40
article thumbnail

Leading innovators in transcription factors for genetically modified cells for the pharmaceutical industry

Pharmaceutical Technology

Adeno-associated virus vectors, alcohol dehydrogenase compositions, and antibody serum stabilisers are some of the accelerating innovation areas, where adoption has been steadily increasing. Among maturing innovation areas are anti-influenza antibody compositions and anti-interleukin-1, which are now well established in the industry.

Genetics 130
article thumbnail

Cash round sets up trial of InteRNA’s microRNA for cancer

pharmaphorum

InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells.

Trials 59
article thumbnail

Clinical Catch-Up: December 21-25 | BioSpace

The Pharma Data

Vir Biotechnology and GlaxoSmithKline dosed the first patient in a new sub-trial of a Phase III study of monoclonal antibody VIR-7831 for hospitalized adults with COVID-19. Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. Here’s a look. COVID-19-Related. Non-COVID-19-Related.

Trials 52
article thumbnail

2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences

XTalks

Most antibody drugs and vaccines have been developed to target parts of the spike protein. These include remdesivir, baricitinib, dexamethasone and three antibody drugs. Antibody drugs were also developed as targeted COVID-19 treatments. Drug Approvals.