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Final results from our Phase I trial of ALN-TTR01
Today, scientists presented final results from our Phase I clinical trial of ALN-TTRo1 at the XIII International Symposium on Amyloidosis held in Groningen. Data from this study show that administration of ALN-TTR01 resulted in statistically significant reductions in serum TTR protein levels, including both wild-type and mutant TTR protein, in ATTR patients. Knockdown of TTR, the disease-causing protein, was found to be dose dependent, rapid, and durable after just a single dose.
View our presentation (842 KB PDF) View our release Listen to KOL, Dr. Hawkins discuss the ALN-TTR program (recorded Jan. 2012) (27.7MB MP3) -
Positive Clinical Results for ALN-PCS, Reported at ATVB 2012
Yesterday, scientists presented positive results from our Phase I clinical trial of ALN-PCS at the American Heart Association’s Arteriosclerosis, Thrombosis and Vascular Biology 2012 Scientific Sessions held in Chicago. Results showed that administration of a single dose of ALN-PCS, in the absence of concomitant lipid-lowering agents such as statins, resulted in statistically significant and durable reductions of PCSK9 plasma levels of up to 84% and lowering of LDL-C of up to 50%.
View our presentation (572 KB PDF) View our release (572 KB PDF) -
Testimony provided at FDA User Fees 2012 hearing
Our CEO John Maraganore provided testimony at the Subcommittee on Health hearing titled “FDA User Fees 2012: Hearing on Issues Related to Accelerated Approval, Medical Gas, Antibiotic Development and Downstream Pharmaceutical Supply Chain.” In his testimony, John highlighted critical FDA reforms that could accelerate the availability of medicines to patients afflicted with serious disease. These reforms offer the promise of delivering the next generation of medical breakthroughs to improve the lives of patients, and provide potential solutions to our nation’s most critical health care needs.
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Delivery Strategies for RNAi Therapeutics
Our scientists presented an update on our RNAi delivery strategies at the AsiaTIDES conference this week. The presentation focuses on our recent progress utilizing conjugates and lipid nanoparticles for systemic adminstration of RNAi therapeutics.
View our presentation (2.4MB PDF)
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