Alnylam and Tekmira Seek New Ways to Deliver RNAi Drugs Deep in the Body

Xconomy Boston — 

(Updated: 1:20 pm ET with new commentary from Alnylam’s CEO.)

Alnylam Pharmaceuticals and Tekmira Pharmaceuticals announced today that they have teamed up to develop new particles to deliver RNA-interference drugs. Their joint effort aims to identify new ways to overcome the hurdles that the gene-modulating drugs must clear to reach diseased cells deep in the body.

The two-year collaboration deal gives Cambridge, MA-based Alnylam (NASDAQ:ALNY), which is funding the effort, exclusive rights to new discoveries stemming from the research. Alnylam can also sublicense the discoveries to its partners, according the firms. As part of the agreement, Vancouver-based Tekmira (TSX:TKM) can use the discoveries for some of its own RNAi treatment programs. The research—which will take place at the University of British Columbia and at a new firm called AlCana Technologies—will focus on the discovery of new positively charged fatty molecules called cationic lipids for delivering RNAi therapeutics. No financial details of the research deal were revealed.

The problem with RNAi therapeutics, which have shown great promise in turning off disease-related genes in research studies, is that they can be chewed up by enzymes or encounter other hazards in the body before they reach the cells involved in diseases. While Alnylam has a substantial stockpile of RNAi molecules that have shown promise in treating the root causes of diseases, the company has had to turn to others such as Tekmira for the molecular vessels that can carry such therapies into cells in the body. (Luke explained the hurdles of RNAi drug delivery when he profiled Tekmira in May.) The particles that Alnylam and Tekmira hope to develop in the new research effort will allow them to deliver RNAi drugs to more parts of the body than currently available particles can reach, as well as providing other benefits.

“We are excited to participate with Tekmira to support research efforts that will focus on the discovery of novel lipids for nanoparticle-based formulations of RNAi therapeutics that we believe will have the potential to push the frontiers of systemic delivery to further improve potency and broaden biodistribution,” said Barry Greene, president and chief operating officer of Alnylam, in a statement.

Alnylam is already using Tekmira’s lipid nanoparticle technology to deliver one RNAi therapeutic, which is now undergoing an early clinical trial for blocking two key genes linked to liver cancer. And Tekmira is using the technology for delivering an RNAi drug—which is also in an initial clinical trial—that targets a protein known as ApoB that carries LDL, or “bad,” cholesterol through the bloodstream.

Alnylam also has other drug-delivery agreements underway that have not been announced yet, said Alnylam CEO John Maraganore, in an interview. He noted that his company has the capabilities to deliver short interfering RNA (siRNA) molecules to organs such as the heart and the pancreas, yet it has struck research deals with firms such as Tekmira and AlCana—which he said is a startup recently launched by former Tekmira scientists—to improve the potency and distribution of the firm’s drugs in the body. (This paragraph was added to the original story to provide further details obtained in the interview with Maraganore late this morning.)