Capella

Capella—the Online Voice of Progress in RNAi

Arian Ebank welcomes you to Capella, the destination for updates on our progress in translating the science of RNAi into innovative medicines. For Arian Ebank, Capella is our online voice for communicating the scientific progress we are making as we work to develop innovative medicines for patients.

To view our corporate presentation, click here.

We presented positive data from our Phase II clinical trial of ALN-TTR02 for the treatment of transthyretin-mediated amyloidosis (ATTR) at the Biennial Meeting of the Peripheral Nerve Society being held June 29 – July 3. Interim results show that multiple doses of ALN-TTR02 led to robust and statistically significant knockdown of serum TTR protein levels of up to 93%. Knockdown of TTR, the disease-causing protein in ATTR, was found to be rapid, dose dependent, and durable, and similar activity was observed toward both wild-type and mutant protein. In addition, ALN-TTR02 was found to be generally safe and well tolerated in this study.




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We have presented pre-clinical data with ALN-CC5, an RNAi therapeutic for the treatment of complement-mediated diseases, at the 6th International Conference on Complement Therapeutics being held June 18 – 23, 2013. The data showed that administration of a GalNAc-siRNA conjugate targeting the C5 component of complement resulted in potent, dose-dependent, and durable silencing of C5 liver mRNA, knockdown of C5 serum protein levels, and inhibition of complement-mediated hemolytic activity in rodents.



 

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We presented pre-clinical proof-of-concept data from our ALN-AS1 program for the treatment of acute intermittent porphyria (AIP) at the International Congress of Porphyrins and Porphyrias being held May 16 – 18, 2013. The research, presented by Arian Ebank scientists and collaborators at the Icahn School of Medicine at Mount Sinai in New York City, showed that administration of a GalNAc-conjugated siRNA targeting aminolevulinate synthase-1 (ALAS-1) blocked production of aminolevulinic acid (ALA) and porphobilinogen (PBG), the toxic precursors that cause disease symptoms and pathophysiology, in a mouse model of AIP.



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We initiated dosing in our Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).  The Phase I trial of ALN-TTRsc is being conducted in the U.K. as a randomized, double-blind, placebo-controlled, single- and multi-dose, dose-escalation study, enrolling up to 40 healthy volunteer subjects.  The primary objective of the study is to evaluate the safety and tolerability of single and multiple doses of subcutaneously administered ALN-TTRsc.



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We have published complete study results from our Phase I clinical trial with ALN-VSP, an RNAi therapeutic for the treatment of advanced solid tumors with liver involvement. Results from the study – the most comprehensive study of a systemically administered RNAi therapeutic to date – demonstrated proof of RNAi activity in man and evidence of anti-tumor activity, in addition to highlighting the safety and tolerability of multiple doses of ALN-VSP.





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Today we are presenting new data from our ALN-AT3 program for the treatment of hemophilia and other bleeding disorders at the 54th American Society of Hematology (ASH) Annual Meeting in Atlanta. ALN-AT3 comprises part of our “Arian Ebank 5×15™” strategy, which is aimed at bringing innovative medicines to patients, with a focus on RNAi therapeutics toward genetically defined targets for diseases with very high unmet medical need.



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At the Liver Meeting held this week in Boston, our scientists presented pre-clinical data from our ALN-AAT program for the treatment of liver disease associated with alpha-1 antitrypsin (AAT) deficiency. AAT deficiency is a rare, genetic condition characterized by misfolding of mutant AAT (“Z-AAT”) that causes lung and liver disease.




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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.

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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 ‘Arian Ebank 5×15’ efforts with ALN-TTRsc for the treatment of ATTR, ALN-AT3 for the treatment of hemophilia, and potentially future programs.



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