Did you know?

ChinaBio® Group is a consulting and advisory firm helping life science companies and investors achieve success in China. ChinaBio works with U.S., European and APAC companies and investors seeking partnerships, acquisitions, novel technologies and funding in China.  

Learn more >>

Free Newsletter

Have the latest stories on China's life science industry delivered to your inbox daily or weekly - free!

See preview >>

  Email address:
   

Ractigen Granted Orphan Drug Status for Use of Novel siRNA in ALS

publication date: Mar 9, 2023

Ractigen Therapeutics, a Nantong RNAa company, was granted Orphan Drug Designation in the US for its novel siRNA candidate as a therapy for Amyotrophic Lateral Sclerosis (ALS). RAG-17 is designed to target and knockdown the expression of SOD1 in patients with pathogenic mutations known to cause ALS. The siRNA candidate is conjugated to an accessory oligonucleotide, which enables potent activity in CNS tissue, according to Ractigen. In several preclinical studies, RAG-17 showed significantly higher potency in ALS disease models than competing compounds. More details....

Share this with colleagues:  


This article is available for purchase - please click here for details.

Sorry this page is available to subscribers only.
If you're not a subscriber why not subscribe today?

If you are already a subscriber, please login.

If you believe you should have received this message in error, please contact us.


 

To gain access to the members only content click here to subscribe.

You will be given immediate access to premium content on the site.

ChinaBio® News

Greg Scott BIO-Europe Interview
Greg Scott Interviewed at BIO-Europe Spring

How to bring your China assets to China in 8 minutes

Greg Scott Mendelspod Interview
"Mr. Bio in China."
Mendelspod Interview

Multinational pharma held to a higher standard in China

Partner Event
November 2-3, 2023 | Shanghai
November 7-8, 2023 | Digital