This Day On The Street
Continue to site
ADVERTISEMENT
This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here

Alnylam Presents New Pre-clinical Data On RNAi Therapeutics Targeting Transthyretin (TTR) For The Treatment Of TTR-Mediated Amyloidosis (ATTR)

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today the presentation of new pre-clinical data on RNAi therapeutics targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR). These data were presented at the International Symposium on Amyloidosis (ISA) held April 27 – May 1, 2014 in Indianapolis, Indiana. In a poster titled “ Preclinical Evaluation of RNAi Therapeutics for the Treatment of ATTR: An Update,” Alnylam scientists presented data confirming that the degree of TTR knockdown in a mouse disease model was highly correlated with regression of TTR tissue deposits. Further, comparative studies were performed with the TTR stabilizer tafamidis and a TTR-specific antisense oligonucleotide (ASO). In these pre-clinical studies, RNAi therapeutics targeting TTR were shown to have superior pharmacologic profiles.

“We believe that these new pre-clinical data highlight the potential for RNAi therapeutics targeting TTR to emerge as the optimal approach for the treatment of ATTR. First, in a mouse model of ATTR, we’ve demonstrated that the degree of TTR suppression at steady state knockdown is highly correlated with regression of TTR deposits in multiple tissues. These data suggest that the 80% TTR knockdown target level achieved with patisiran and ALN-TTRsc could facilitate a reduced pathogenic accumulation of TTR amyloid and possibly even a regression of TTR amyloid in patients with ATTR,” said Rachel Meyers, Ph.D., Vice President, Research and RNAi Lead Development at Alnylam. “Moreover, our scientists and collaborators presented comparative studies of RNAi therapeutics with a TTR stabilizer and an antisense oligonucleotide (ASO). In a mouse model of ATTR, treatment with the TTR stabilizer tafamidis resulted in a trend for regression of TTR deposits in a limited number of tissues, while administration of an RNAi therapeutic led to a statistically significant regression in all tissues evaluated. Additional comparative studies were performed with a TTR-specific ASO and showed that RNAi therapeutics achieve a more rapid, potent, and robust knockdown of TTR than the ASO, with an over 100-fold lower drug exposure in liver and ‘bystander’ tissues such as kidney. We believe that the ability of RNAi therapeutics to achieve potent TTR knockdown with lower tissue exposure could result in a more favorable efficacy and tolerability profile.”

1 of 7

Check Out Our Best Services for Investors

Action Alerts PLUS

Portfolio Manager Jim Cramer and Director of Research Jack Mohr reveal their investment tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
Quant Ratings

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
Stocks Under $10

David Peltier uncovers low dollar stocks with serious upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
14-Days Free
Only $9.95
14-Days Free
To begin commenting right away, you can log in below using your Disqus, Facebook, Twitter, OpenID or Yahoo login credentials. Alternatively, you can post a comment as a "guest" just by entering an email address. Your use of the commenting tool is subject to multiple terms of service/use and privacy policies - see here for more details.
Submit an article to us!
SYM TRADE IT LAST %CHG

Markets

DOW 18,080.14 +21.45 0.12%
S&P 500 2,117.69 +4.76 0.23%
NASDAQ 5,092.0850 +36.0220 0.71%

Partners Compare Online Brokers

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs