Capella

Posts Tagged with: TTR-Mediated Amyloidosis

January 2, 2013

RNAi Roundtable: Conjugate Delivery

In December 2012, we hosted a roundtable discussion focused on our activities and progress with our conjugate platform to deliver RNAi therapeutics subcutaneously.  The roundtable  included a review of our two leading conjugate programs: ALN-TTRsc for the treatment of transthyretin mediated amyloidosis, and ALN-AT3 for the treatment of hemophilia and other bleeding disorders.

Listen to the replay View the presentation (254 KB PDF)

 

November 1, 2012

8th Annual Meeting of the Oligonucleotide Therapeutics Society

At the 8th Annual Meeting of the Oligonucleotide Therapeutics Society, held in Boston October 28-31, 2012, we presented exciting data covering several of the company’s therapeutic programs and delivery platforms.

THERAPEUTIC PROGRAMS

Therapeutic research highlighted recent clinical and pre-clinical work from our programs in transthyretin-mediated amyloidosis (TTR), hemophilia, hypercholesterolemia, and liver cancer. Click here for presentations.

Continue reading…

August 6, 2012

New Data from Conjugate Delivery Platform

New data from our conjugate delivery platform was presented at the XX International Roundtable on Nucleosides, Nucleotides and Nucleic Acids held in August 2012.  This delivery platform enables subcutaneous dose administration of RNAi therapeutics with a very wide therapeutic index.  We are integrating this approach in our ‘Alnylam 5×15’ efforts with ALN-TTRsc for the treatment of ATTR, ALN-AT3 for the treatment of hemophilia, and potentially future programs.

View our presentation (1.6 MB PDF)

Read our press release (PDF)

July 16, 2012

Positive ALN-TTR02 Clinical Data

At the Boston University School of Medicine, our scientists presented positive clinical results from our Phase I trial with ALN-TTR02 for the treatment of transthyretin-mediated amyloidosis (ATTR).  These new data show that we have achieved very robust knockdown of TTR, the disease causing protein, including up to 94% reduction of serum TTR, and a nearly 80% level of suppression sustained at one month with just a single dose.  ALN-TTR02 was found to be generally safe and well tolerated.

View our presentation (758 KB PDF) Read our press release

May 10, 2012

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)

Continue reading…

May 10, 2012

ALN-TTR Historical Press Releases

Below is a list of historical press releases related to our ALN-TTR program for the treatment of transthyretin-mediated amyloidsis:

2012

July 16: Alnylam Reports Positive ALN-TTR02 Clinical Data, with Robust Knockdown of Serum Transthyretin (TTR) of up to 94% After Single Dose June 19: Alnylam Receives Orphan Drug Designation from U.S. Food & Drug Administration for ALN-TTR02, an RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis (ATTR) June 7: Alnylam Completes Enrollment in Phase I Clinical Trial and Initiates Phase II Clinical Trial of ALN-TTR02 May 10: Alnylam Presents Final Results from a Phase I Clinical Trial of ALN-TTR01 March 19:  Alnylam Initiates Phase I Clinical Study of ALN-TTR02 Jan 6:  Alnylam Files Clinical Trial Application for ALN-TTR02

Continue reading…

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 mp3

Continue reading…

July 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)

Continue reading…

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…

This part of our website contains archived information which should not be considered current and may no longer be accurate.