Milestone for RNAi therapeutics?

A company developing therapeutics using RNA interference (RNAi) today (February 29) linkurl:announced;http://phoenix.corporate-ir.net/phoenix.zhtml?c=148005&p=irol-newsArticle&ID=1113937&highlight= positive results of a clinical trial in humans ? marking a first for the much-touted promise of RNAi-based therapies. Alnylam, based in Cambridge, Mass., exposed 88 male volunteers to respiratory syncytial virus, which affects mostly young children and the elderly. Half of the subjects received the

| 1 min read

Register for free to listen to this article
Listen with Speechify
0:00
1:00
Share
A company developing therapeutics using RNA interference (RNAi) today (February 29) linkurl:announced;http://phoenix.corporate-ir.net/phoenix.zhtml?c=148005&p=irol-newsArticle&ID=1113937&highlight= positive results of a clinical trial in humans ? marking a first for the much-touted promise of RNAi-based therapies. Alnylam, based in Cambridge, Mass., exposed 88 male volunteers to respiratory syncytial virus, which affects mostly young children and the elderly. Half of the subjects received the RNAi treatment, delivered as a nasal spray, which blocked a protein made by the virus, while the other half received a placebo. The upshot: 67% of the treatment group became infected with the virus, while 88% of the placebo group were infected, the linkurl:Wall Street Journal;http://online.wsj.com/article/SB120425827872802381.html?mod=health_home_stories reports; though small, the difference was statistically significant. Researchers have placed high hopes on the potential for RNAi-based therapies, but have linkurl:struggled;http://www.the-scientist.com/blog/display/54268/ with delivery strategies. "With the positive results from GEMINI, we believe that Alnylam has demonstrated the first ever human proof of concept with an RNAi therapeutic," said John Maraganore, the compan"?s CEO, in a press release. The results are not published, but further details of the study will be described this weekend at the International Symposium on Respiratory Viral Infections in Singapore.
Interested in reading more?

Become a Member of

The Scientist Logo
Receive full access to more than 35 years of archives, as well as TS Digest, digital editions of The Scientist, feature stories, and much more!
Already a member? Login Here

Meet the Author

  • Alla Katsnelson

    This person does not yet have a bio.
Share
May digest 2025 cover
May 2025, Issue 1

Study Confirms Safety of Genetically Modified T Cells

A long-term study of nearly 800 patients demonstrated a strong safety profile for T cells engineered with viral vectors.

View this Issue
Detecting Residual Cell Line-Derived DNA with Droplet Digital PCR

Detecting Residual Cell Line-Derived DNA with Droplet Digital PCR

Bio-Rad
How technology makes PCR instruments easier to use.

Making Real-Time PCR More Straightforward

Thermo Fisher Logo
Characterizing Immune Memory to COVID-19 Vaccination

Characterizing Immune Memory to COVID-19 Vaccination

10X Genomics
Optimize PCR assays with true linear temperature gradients

Applied Biosystems™ VeriFlex™ System: True Temperature Control for PCR Protocols

Thermo Fisher Logo

Products

The Scientist Placeholder Image

Biotium Launches New Phalloidin Conjugates with Extended F-actin Staining Stability for Greater Imaging Flexibility

Leica Microsystems Logo

Latest AI software simplifies image analysis and speeds up insights for scientists

BioSkryb Genomics Logo

BioSkryb Genomics and Tecan introduce a single-cell multiomics workflow for sequencing-ready libraries in under ten hours

iStock

Agilent BioTek Cytation C10 Confocal Imaging Reader

agilent technologies logo