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)Posts Tagged with: TTR-Mediated Amyloidosis
December 16, 2011
Podcast: ALN-TTR Phase I Preliminary Results
We recently presented preliminary clinical results for our Phase I study of ALN-TTR01 at the International Symposium on Familial Amyloidotic Polyneuropathy. In this new podcast, Dinah Sah, Ph.D., VP of Research and lead for the ALN-TTR program, discusses the ALN-TTR01 Phase I results she presented at the Symposium, late November. (Length 00:19:32)
Listen to the podcast (22.9 MB mp3) ALN-TTR01 PhI Prelim Results (821 KB PPT)November 21, 2011
ALN-TTR01 Preliminary Phase I Clinical Results
In November 2011, we presented positive preliminary data from our ALN-TTR01 Phase I study at the VIIIth International Symposium on Familial Amyloidotic Polyneuropathy. These data show that administration of ALN-TTR01 resulted in statistically significant reductions in serum TTR protein levels in ATTR patients and demonstrate human proof of concept for RNAi therapeutics.
Read our press release ALN-TTR01 PhI Prelim Results (821 KB PPT) Listen to replay of conference call (7.1 MB mp3July 24, 2011
“Alnylam 5×15″ Product Strategy
In January 2011, we were very excited to launch our Alnylam 5×15™RNAi therapeutic product strategy. Alnylam 5×15 was established to focus on the development and commercialization of novel RNAi therapeutics addressing genetically defined diseases with major unmet medical need.
Read our press release Listen to our CEO discuss our 5×15 Strategy (4.7MB MP3) Rare Disease Day podcast (7.2MB MP3)July 23, 2011
ALN-TTR Data Presented at Peripheral Nerve Society Meeting
Our flagship effort from the Alnylam 5×15 strategy is our ALN-TTR program, which is currently enrolling patients in a Phase I study for the treatment of a disease called transthyretin-mediated amyloidosis (ATTR).
Read our press release ALNY-PNS-Poster-ALN-TTR-NT-Study-June2011 (720KB PPT) ALNY-PNS-Poster-ALN-TTR-June2011 (1.1MB PPT)ALN-TTR RNAi Roundtable, July 2010 (1.0 MB PPT) Continue reading…
February 28, 2011
Spotlight on Rare Disease Day Podcast
Disussion with the National Organization for Rare Disorders (NORD) and the Amyloidosis Foundation both fighting to raise awareness and improve health care for those living with rare diseases.
Speakers: Tia Spargo, NORD and Mary O’Donnell, Amyloidosis Foundation
Listen to the Podcast (8MB MP3)July 22, 2010
Alnylam RNAi Roundtable: ALN-TTR Program
Following the initiation of our Phase I clinical study with ALN-TTR01 in July 2010, we hosted a virtual RNAi Roundtable to provide investors with a full overview of our ALN-TTR program.
ALN-TTR RNAi Roundtable webcast, July 2010 (61.9 MB MP3) ALN-TTR RNAi Roundtable, July 2010 (1.0 MB PPT)April 20, 2010
International Symposium on Amyloidosis, 2010
Alnylam scientists and collaborators presented new pre-clinical data from our ALN-TTR program at the XII International Symposium on Amyloidosis in Rome on April 18 – 21, 2010. These data demonstrated, for the first time, that treatment with an RNAi therapeutic can result in regression of pre-existing pathogenic TTR deposits in peripheral tissues.
View our collaborator’s presentation (1.4MB PPT) View our ALN-TTR program overview (376KB PPT) Read our press release
The new pre-clinical studies, performed in collaboration with Dr. Maria Saraiva at the Institute for Molecular and Cellular Biology in Portugal, used a transgenic mouse model where the mutant human V30M TTR gene is over-expressed. Specifically, the new data demonstrated that administration of ALN-TTR01 in mature transgenic mice resulted in the regression of existing pathogenic mutant human V30M TTR deposits in tissues, including: dorsal root ganglia, sciatic nerve, stomach, and intestines. These effects in diseased tissues were mediated through silencing of the mutant human TTR expressed in the liver, resulting in regression of established peripheral TTR deposits. ALN-TTR01 administration resulted in a greater than 90% regression of measurable TTR deposits as compared with control-treated animals.
