tag:blogger.com,1999:blog-78570541496754246092012-01-28T10:45:42.136-08:00rnaitherapeutics.blogspot.com follows the development in RNAi Therapeutics from bench to clinic. Comments on basic science and clinical issues as well as investment opportunities in this rapidly evolving field. From siRNAs to Dicer, from Alnylam to Tekmira- it's here.Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.comBlogger3231100tag:blogger.com,1999:blog-7857054149675424609.post-43912040148856732522012-01-28T02:33:00.000-08:002012-01-28T10:45:42.147-08:00

Note from 28 January, 2012:On January 3, 2012, the EPO issued an 'Intention to grant'. Unlike suggested in the following blog entry, the claims in the accompanying 'Druckexemplar' are not blocking any more for blunt-ended RNAi triggers. The coverage, however, does include 19bp blunt end RNAi triggers which is certainly a useful structure. This is the main claim that is likely to be issued. As

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com20tag:blogger.com,1999:blog-7857054149675424609.post-61658054183447810742012-01-21T05:34:00.000-08:002012-01-21T13:15:14.589-08:00

Available only until the end of January, the report reviews the highlights from the ‘Keystone Nucleic Acid Therapeutics’ meeting which was held in Santa Fe, NM, from January 10-15, 2012. The focus will be on RNAi Therapeutics induced by synthetic RNAi triggers. It will, however, also cover the most interesting presentations in the related fields of microRNA Therapeutics and antisense. Please

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com15tag:blogger.com,1999:blog-7857054149675424609.post-69426966015497070462012-01-19T16:08:00.000-08:002012-01-21T01:52:33.331-08:00

When I saw the announcement a few minutes ago, I had to gasp for air: A little more than a year after laying off 25-30% of its workforce after Novartis failed to exercise the $100M IP option, Alnylam announced this evening that it will further reduce its workforce by another 33%. Even the PR person seemed shocked, as the same PR went out twice. Following recent guidance that the company would

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com6tag:blogger.com,1999:blog-7857054149675424609.post-29370152627944367052012-01-19T03:28:00.000-08:002012-01-19T09:42:35.915-08:00

Yesterday, UK-based Plant Biosciences Limited (PBL) announced that it was awarded a fundamental RNAi patent in the US. The patents can be considered necessary for commercializing RNAi in the US as long as both strands of the RNAi trigger are between 20-24 nucleotides in length. Claim 1 of US patent 8,097,710: 1. A method of silencing a gene in cells by post-transcriptional gene

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com9tag:blogger.com,1999:blog-7857054149675424609.post-41388726218451005862012-01-05T07:10:00.000-08:002012-01-05T07:18:26.045-08:00

ISIS today reported knockdown results from a phase I trial with its RNase H antisense compound ISIS-TTRRx against the transthyretin gene for the treatment of TTR amyloidosis. The results show that while high 81% knockdowns can be achieved, very large amounts of oligonucleotides (400mg weekly for 4 weeks) had to be given. When clinically more relevant doses were given (200mg as in mipomersen and

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com4tag:blogger.com,1999:blog-7857054149675424609.post-68280235255726644702012-01-04T06:16:00.000-08:002012-01-15T10:30:48.335-08:00

If the experts at Roche and other Big Pharma companies got you convinced that RNAi Therapeutics was about to disappear from the scene by pulling the plug on the technology…think again. Alnylam this morning reported tantalizing insights into the safety and efficacy of ALN-PCS02, a SNALP-formulated RNAi Therapeutic targeting PCSK9 for the treatment of hypercholesterolemia. Given that PCSK9 is

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com10tag:blogger.com,1999:blog-7857054149675424609.post-82235023872946067742011-12-30T04:23:00.000-08:002011-12-30T09:30:21.450-08:00

With the Notice of Allowance of a US patent application covering its basic Dicer-substrate RNAi trigger technology and a $5M milestone payment from Kyowa Hakko Kirin (KHK) due to the formal initiation of development work for a Dicer-substrate oncology candidate by its Japanese partner, Dicerna can look optimistically into the New Year. These events also symbolize some of the important shifts

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com0tag:blogger.com,1999:blog-7857054149675424609.post-85192942669028550552011-12-28T05:45:00.001-08:002011-12-28T06:05:05.628-08:00

I’ve been reminded a number of times by the staunch Benitec-supporters here that Texas-based biotech company Gradalis has been moving a ddRNAi-enhanced cancer vaccine candidate (‘FANG’) aggressively through clinical development. Virtually out of nowhere, Gradalis initiated clinical trials two years ago and there are now two active phase II trials, one in ovarian cancer and one for advanced

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com8tag:blogger.com,1999:blog-7857054149675424609.post-5203564147822042742011-12-13T02:58:00.000-08:002011-12-14T03:11:45.201-08:00

Silence Therapeutics reported that a number of valuable RNAi trigger patents related to the Zamore Design Rules that were issued last year in the US were upheld following a re-examination request by an anonymous 3rd party. Even stronger claims related to the same Zamore patent series have been issued in Europe, and unsurprisingly Alnylam, but also Novartis and Alcon are opposing them with the

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com18tag:blogger.com,1999:blog-7857054149675424609.post-85701940167969542522011-12-10T05:33:00.000-08:002011-12-12T09:58:33.367-08:00

According to Linkedin, Alnylam's General Counsel, Philip Chase, has just left the company to become General Counsel of Adimab [note: an earlier version incorrectly stated that P. Chase had been with the company for only a little over a year, while in fact he had been there for 4 years]. This departure comes at a critical time for Alnylam as it is involved in a fierce battle with Tekmira for

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com4tag:blogger.com,1999:blog-7857054149675424609.post-23381534292914786652011-12-09T07:05:00.000-08:002011-12-09T18:17:14.434-08:00

The Tekmira and Alnylam dispute is a classical David versus Goliath encounter. By my standards, Protiva/Tekmira clearly deserves credit for developing the industry’s most advanced systemic siRNA delivery technology, SNALP. Alnylam, however, is trying to take all of that away from a company that it knows it stands a good chance at silencing with sheer money and influence. It therefore struck a

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com10tag:blogger.com,1999:blog-7857054149675424609.post-37575603834907491602011-12-07T21:28:00.000-08:002011-12-12T08:38:00.505-08:00

Following recent phase I results from ISIS Pharmaceutical’s Factor XI (ASH abstract 12999; addendum: PR on phase I data reported on December 12) and Apo C III programs, there is little doubt left that phoshorothioate-based RNaseH antisense as developed by this company and Santaris can mediate target-specific gene knockdown in the liver in Man. These results confirm the clinical experience with

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com8tag:blogger.com,1999:blog-7857054149675424609.post-28275264528078901792011-11-29T18:58:00.001-08:002011-11-29T20:10:19.167-08:00

On Monday, Tekmira announced that it has received the Green Light from the FDA to go ahead with clinical studies of its SNALP-enabled biodefense candidate for the treatment of Ebola infection. Tekmira is developing TKM-EBOLA under a $140M contract from the US Department of Defense following spectacular results in non-human primates reported last year in The Lancet. Depending on whether you want

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com12tag:blogger.com,1999:blog-7857054149675424609.post-85297144062414090882011-11-21T04:09:00.000-08:002011-11-23T18:43:09.845-08:00

The long wait is finally over: Systemic RNAi delivery has been proven in Man. No 'ifs' or 'buts'. Alnylam just announced that Tekmira’s SNALP-enabled ALN-TTR01 reduced target transthyretin (TTR) protein levels in ATTR patients in a dose-dependent manner- without causing an elevation of liver enzymes or serious adverse event (SAE).Following preliminary evidence in Tekmira’s TKM-ApoB study, this

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com6tag:blogger.com,1999:blog-7857054149675424609.post-10476602511348504952011-11-19T21:08:00.000-08:002011-11-21T04:49:42.720-08:00

(For a discussion of the phase I results, see here).Alnylam is about to reveal results from its phase I study with ALN-TTR01 for the treatment of transthyretin amyloidosis (ATTR) at the Nov20-22 FAP meeting in Kunamoto, Japan. This blog provides a brief overview of the rational for RNAi Therapeutics in this disease and the importance of this particular study for the field of RNAi Therapeutics.

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com4tag:blogger.com,1999:blog-7857054149675424609.post-71287245166731703222011-11-16T19:27:00.000-08:002011-11-17T01:33:40.453-08:00

5 million Euros these days is serious money in RNAi Therapeutics. Last week, Irish biotechnology company Genable Technologies announced that it succeeded in raising such funds for its pre-clinical DNA-directed RNAi Therapeutics-Gene Replacement combo for the treatment of rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO-adRP), an inherited retinal degenerative condition leading to

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com4tag:blogger.com,1999:blog-7857054149675424609.post-50215085849146392472011-11-07T20:57:00.000-08:002011-11-09T22:20:07.377-08:00

Four years after Mirus scientists published their seminal paper in polymer-mediated siRNA delivery, and 3 years after the acquisition of the Dynamic Polyconjugate (DPC) technology by Roche, Arrowhead Research, the new owner of the technology, finally lifted the veil on recent progress in bringing DPC technology to the clinic. The White Paper reveals, for the first time, that DPCs have been

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com1tag:blogger.com,1999:blog-7857054149675424609.post-60818007674623148992011-11-07T07:36:00.000-08:002011-11-08T04:55:09.238-08:00

I’ve just come back from working at the Starbucks across my street which strongly reminded me that Christmas was just around the corner. Christmas this year in RNAi Therapeutics is synonymous with data releases by Alnylam from its transthyretin amyloidosis (ALN-TTR01; data presentation November 20-22 in Japan) and hypercholesterolemia (ALN-PCS02; release of top-line results by year-end) phase I

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com20tag:blogger.com,1999:blog-7857054149675424609.post-17315152570324507002011-10-24T04:08:00.000-07:002011-10-24T05:10:27.521-07:00

The long wait is finally over. Almost a year* after infamously announcing its retreat from developing RNAi Therapeutics in-house as part of a corporate restructuring, Roche has sold its related assets to Arrowhead Research. That announcement sent shockwaves through the RNAi Therapeutics World as investors and potential licensing partners alike took this to mean that something was very wrong with

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com7tag:blogger.com,1999:blog-7857054149675424609.post-30245164251221434652011-10-24T03:15:00.000-07:002011-10-24T03:22:43.664-07:00

It’s actually very simple, and apparently it is small biotechnology companies that are first to realize and act on it: Develop a technology that can deliver small silencing RNAs to a given cell/tissue type, and only our exploding insights into the genetics of disease set the limit for the number of potential indications. This benefits both the delivery company that can re-coup some of their

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com0tag:blogger.com,1999:blog-7857054149675424609.post-62353661724393170282011-10-16T16:35:00.000-07:002011-10-16T17:02:21.508-07:00

The goal in developing a RNAi Therapeutics delivery technology is to achieve gene knockdown in a certain cell or tissue type. Once this is achieved, only our exploding genetic insights into disease limit the potential therapeutic applications. This is for example why we are seeing this SNALP-fuelled expansion in the clinical pipeline for indications in which gene knockdown is targeted in the

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com5tag:blogger.com,1999:blog-7857054149675424609.post-44736264204882018852011-10-11T16:24:00.000-07:002011-10-11T16:41:51.746-07:00

There has been some unhappiness among certain Big Pharma companies about their adventures in RNAi Therapeutics. Roche, Pfizer, and Abbott Labs were among the publicized companies that discontinued RNAi Therapeutics platform development efforts not too long ago. Unsurprisingly, you could hear criticism from these companies about alleged immaturity of the technology and having been misled by the

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com7tag:blogger.com,1999:blog-7857054149675424609.post-2975724024499960502011-10-07T02:13:00.000-07:002011-10-10T01:30:22.962-07:00

Antisense company Santaris seems hardly able to catch its breath these days. After being sued by ISIS Pharmaceuticals for patent infringement, reporting first clinical proof-of-concept for a MicroRNA Therapeutic, it has now been revealed that the company prematurely terminated a phase I trial of its PCSK9 phosphorothioate LNA RNase H antisense inhibitor SPC5001 for the treatment of

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com4tag:blogger.com,1999:blog-7857054149675424609.post-25297660590328557832011-10-04T03:46:00.001-07:002011-10-04T04:52:48.816-07:00

Santaris reported yesterday intriguing antiviral HCV efficacy results from an ongoing phase IIa study of miravirsen, Santaris’ LNA-based antisense inhibitor of microRNA-122 (miR-122), miravirsen, an important host factor in HCV replication. Full interim results will be presented in a late-breaking oral session at the upcoming AASLD, The Liver Meeting. The phase IIa study investigates 3, 5, and

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com8tag:blogger.com,1999:blog-7857054149675424609.post-73948908027961458702011-09-27T00:01:00.000-07:002011-09-27T22:13:42.523-07:00

As Tekmira is struggling to regain possession over its delivery technology from mighty Alnylam, it is foremost Silence Therapeutics' business development that is benefitting from having one of the most advanced, clinically tested, and commercially uncontested systemic delivery technologies in RNAi Therapeutics. Less than a month after closing a deal with Dutch company InteRNA under which Silence’

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com2tag:blogger.com,1999:blog-7857054149675424609.post-46841291878302983952011-09-26T07:27:00.000-07:002011-09-26T07:41:07.615-07:00

If you just blinked, you may have missed it: RXi Pharmaceuticals, now re-named Galena Biopharma, is to be a cancer vaccine company. As you may remember, when RNAi was still a hot technology in 2007, the parent company of RXi, small molecule biopharma CytRx, under the helm of corporate maverick Steven Kriegsman, acquired and then spun out RNAi assets in the form of RXi to be a pure-play RNAi

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com1tag:blogger.com,1999:blog-7857054149675424609.post-73496284086141271432011-09-23T08:10:00.000-07:002011-10-08T21:08:50.848-07:00

It is not a big secret that antisense therapeutics companies ISIS and Santaris are fierce competitors. Today, ISIS filed an unusual, because rather aggressive lawsuit against Santaris Pharma that alleges the Danish company to be selling to the industry technology that is covered by at least two of ISIS’ literally thousands of patents. Because ISIS considers itself the gate-keeper of

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com8tag:blogger.com,1999:blog-7857054149675424609.post-39895654479902581432011-09-19T19:16:00.000-07:002011-09-19T21:42:07.386-07:00

2000-2001 was one of the most exciting, fast-moving periods in RNAi history. All the evidence from the various fields converged to unravel the central mechanisms of RNAi resulting, amongst others, in the discovery of how RNAi can be triggered in human cells. Plant scientists discovered that small RNAs were involved, a group in Cold Spring Harbor found that the 3’ overhang-generating enzyme

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com1tag:blogger.com,1999:blog-7857054149675424609.post-20883815169856150672011-09-12T20:28:00.000-07:002011-09-12T21:01:58.459-07:00

The OTS Meeting has just finished and it looks like it was a success in that meaningful progress, especially on delivery, but also specific development candidates was presented. As the press releases by Tekmira and Alnylam today showed, the abstracts do not necessarily reflect the full progress of the respective studies as these have been submitted months ahead of the conference and also because

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com1tag:blogger.com,1999:blog-7857054149675424609.post-28919389488507289322011-09-12T07:13:00.000-07:002011-09-12T07:26:55.628-07:00

Silence Therapeutics and InteRNA Technologies announced today the signing of a collaboration agreement following which the companies will use the endothelial cell-directed AtuPLEX gene knockdown delivery technology for the development of microRNA cancer therapeutics. The news comes as Atu027, Silence’s RNAi Therapeutics candidate in oncology which also uses AtuPLEX delivery technology, is

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com0tag:blogger.com,1999:blog-7857054149675424609.post-45207405380474273532011-09-08T06:11:00.001-07:002011-09-09T05:25:28.617-07:00

You will see this theme continue: Systemic RNAi delivery technologies that have proven themselves in non-human primate and clinical studies will continue to yield pipeline candidates and attract potential partners. Today’s announcement by Alnylam at the Annual OTS Meeting that it has chosen SNALP-enabled ALN-APC targeting the liver-expressed Protein C in hemophilia as its 5x15TM development

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com9tag:blogger.com,1999:blog-7857054149675424609.post-75054736437666424032011-09-07T02:52:00.000-07:002011-09-13T01:55:58.942-07:00

The Annual Meetings of the Oligonucleotide Therapeutics Society are among the best on the conference circus related to, well, oligonucleotide therapeutics drug development. One benefit of bringing together RNAi Therapeutics, traditional RNaseH and steric block antisense, aptamers, and a few other oligo-based approaches is that researchers can benefit from sharing lessons in safety, how

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com0tag:blogger.com,1999:blog-7857054149675424609.post-59828122918094314342011-08-26T03:33:00.000-07:002011-08-26T10:06:32.546-07:00

It must have been over either coffee, the fuel of scientific discovery and frequent source of business development inspiration, or beer, a beverage that you may enjoy but not mix with business, that Seattlelites Mark Murray (CEO of Tekmira) and Todd Hauser (CEO/inventor of Halo-Bio) initially came up with the idea of joining forces to revolutionize one of the attractions of RNAi Therapeutics:

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com8tag:blogger.com,1999:blog-7857054149675424609.post-88014714033133622562011-08-19T02:40:00.000-07:002011-08-20T07:40:12.887-07:00

A series of recent papers (e.g. Ason et al. 1; Tadin-Strapps et al.; Ason et al. 2) shows that Merck wishes to use RNAi Therapeutics for the treatment of hypercholesterolemia, a precursor of cardiovascular disease. Despite the success that widely prescribed drugs such as statins have had in lowering bad cholesterol, there are still many patients in need of additional treatment options, patients

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com2tag:blogger.com,1999:blog-7857054149675424609.post-34613071571140962652011-08-11T23:56:00.000-07:002011-08-12T23:49:11.571-07:00

This week, important developments for two of the most advanced cancer RNAi Therapeutic candidates were announced. Arrowhead reported that it has now completed enrolment of the phase I CALAA-01 study and is initiating a dose-optimizing phase Ib study, while Tekmira disclosed that it has received the green light from the FDA to go ahead with an additional phase I study of TKM-PLK1, this time using

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com6tag:blogger.com,1999:blog-7857054149675424609.post-73752033439902787852011-08-08T03:16:00.000-07:002011-08-08T22:48:55.888-07:00

Following major IP battles with Benitec and tensions high within the company, DNA-directed RNAi Therapeutics company Nucleonics became the first major RNAi Therapeutics company to go out of business in 2008. During the liquidation process, Alnylam surprised observers by scooping up Nucleonics’ IP assets. Adding to the confusion, Nucleonics had just initiated dosing in a phase I study of NucB1000

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com5tag:blogger.com,1999:blog-7857054149675424609.post-36361047149337091382011-08-02T05:58:00.000-07:002011-08-04T13:30:58.325-07:00

One apparent tactic employed by Alnylam in the ongoing litigation with Tekmira is not to win on arguments, but by coming out with 'revelations' aimed at unsettling Tekmira investors and potential collaborators. A weakened share price and financial position would supposedly make Tekmira more amenable to a light settlement or lowball takeover offer. Much of this has been discussed on this blog

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com7tag:blogger.com,1999:blog-7857054149675424609.post-51991716304206827242011-07-29T23:46:00.000-07:002011-08-03T01:06:58.002-07:00

[Update: important clarification by Merck on the site closure, here]As reported by the San Francisco Business Times and Xconomy, Merck will be closing its RNAi Trigger research unit in San Francisco as part of global restructuring efforts that include cutting ~12,000 employees by 2015 as Merck seeks to drive short-term bottom-line growth. While not exactly a move that will instill confidence in

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com8tag:blogger.com,1999:blog-7857054149675424609.post-78785850915289421542011-07-27T08:32:00.000-07:002011-07-27T09:02:38.995-07:00

PF-04523655, formerly known as RTP-801i, is the clinically most advanced RNAi Therapeutic candidate and has been in two phase II studies for diabetic macular edema (DME) and the exudative form of age-related macular degeneration (wet AMD). ‘655 is a 19bp blunt-end AtuRNAi trigger targeting the RTP801/REDD1 apoptotic stress response gene in the choroid. ‘655 was originally discovered by Silence

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com2tag:blogger.com,1999:blog-7857054149675424609.post-13869578302137790232011-07-21T22:40:00.000-07:002011-07-22T09:52:29.171-07:00

The Semple-Wheeler (S-W) patents can be considered essential intellectual property (IP) for those liposomal siRNA formulations currently in development that have shown clinical promise. Because Alnylam obtained significant control over Semple-Wheeler (S-W) through a license from Tekmira, this patent estate has been important to Alnylam in controlling access to Tekmira’s SNALP delivery technology

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com11tag:blogger.com,1999:blog-7857054149675424609.post-9119292485413085172011-07-14T10:09:00.000-07:002011-07-17T08:34:08.939-07:00

With the backing of a $20M grant from the California Institute of Regenerative Medicines (CIRM), Calimmune has made progress in advancing a DNA-directed RNAi (ddRNAi) Therapeutics candidate for the treatment of HIV/AIDS towards clinical development in early 2012 (here a recent blurb in the Financial Times). Similar to an HIV candidate developed by City of Hope (CoH) and Benitec before it, the

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com24tag:blogger.com,1999:blog-7857054149675424609.post-3743850691992236772011-07-11T09:11:00.001-07:002011-07-11T21:15:37.540-07:00

Tekmira’s partner and licensee Alnylam Pharmaceuticals announced today the submission of files to a European regulatory agency in anticipation of a phase I study with a candidate targeting PCSK9 for the treatment of Severe Hypercholesterolemia. The primary aim of this early-stage study is naturally the safety and tolerability of ALN-PCS. An important secondary aim, not just for Alnylam but also

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com14tag:blogger.com,1999:blog-7857054149675424609.post-15263836849840361332011-07-06T21:23:00.000-07:002011-07-07T05:26:17.430-07:00

Last week, Alnylam responded to the Amended Complaint by Tekmira in which Tekmira disclosed the full scope of Alnylam’s alleged misappropriation and misuse of Tekmira technology and added Alnylam Canada, aka AlCana, as Co-Defendant (related blog entry here). The alleged transgressions included abusing insights into Tekmira trade secrets that Alnylam gained as a result of their collaborator status

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com11tag:blogger.com,1999:blog-7857054149675424609.post-15774690520573522852011-06-21T09:38:00.000-07:002011-06-21T23:58:43.193-07:00

The Amended Complaint against Alnylam filed in early June by Tekmira revealed that the potential damages to the company as a result of Alnylam’s alleged transgressions may be much more severe than initially feared based on the original Complaint and their public relationship over the years. In particular, Alnylam apparently used their insights into Tekmira’s technology, gained as a result of

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com18tag:blogger.com,1999:blog-7857054149675424609.post-74291066146883576852011-06-14T17:51:00.000-07:002011-06-15T05:17:25.570-07:00

‘We Make RNAi Work.’ Tekmira’s slogan may sound a bit cheesy as they all do, but when you consider that it is the only company that has managed to translate the theoretically very powerful unilamellar liposome technology into the clinic and ready for commercialization while essentially everybody else is being frustrated from following up on promising early-stage results with liposomal delivery

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com24tag:blogger.com,1999:blog-7857054149675424609.post-55925084909730339852011-06-09T07:27:00.000-07:002011-06-09T07:28:51.501-07:00

On Monday at ASCO 2011, Silence Therapeutics provided an extensive update on its ongoing phase I trial of Atu027 for the treatment of advanced solid tumors (additional background on Atu027 and the current study provided here and here). While there are a few signs already that the drug candidate may have some anti-tumor activity, the even more important message was that Atu027 has been remarkably

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com7tag:blogger.com,1999:blog-7857054149675424609.post-53960949269664136612011-06-06T07:27:00.000-07:002011-06-06T08:07:03.446-07:00

Widely expected, Alnylam presented over the weekend detailed safety and preliminary efficacy data from its multi-dose, dose-escalation phase I trial of ALN-VSP02 in 41 patients of advanced solid tumors with liver involvement. ALN-VSP02 is a SNALP formulation containing 2 siRNAs, one targeting VEGF (anti-angiogenic mechanism), the other kinesin spindle protein (KSP; anti-proliferative). The data

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com13tag:blogger.com,1999:blog-7857054149675424609.post-71270797909910826132011-06-01T22:31:00.000-07:002011-06-02T03:12:23.918-07:00

In case you have not noticed: “Big Pharma” is slowly coming back to RNAi Therapeutics. This marks a fourth phase in the delicate relationship of the small pure-play RNAi Therapeutics companies with their larger counterparts. The relationship in the first phase (2002-5) may be characterized as one of benign neglect and certain curiosity. Sure, RNAi was a hot emerging scientific area, but Big

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com19tag:blogger.com,1999:blog-7857054149675424609.post-24784849994919398312011-05-18T14:54:00.001-07:002011-05-18T23:51:07.629-07:00

It is 6 o’clock in the morning in my part of the world, have not slept all night, but am more awake now than I have been for a long time: The ASCO abstracts on Silence Therapeutics’ Atu027 (for advanced solid cancers) and Alnylam’s ALN-VSP02 (for cancers with liver involvement) have just been released and both surpassed my already optimistic expectations (copies of abstracts, see below). For

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com17tag:blogger.com,1999:blog-7857054149675424609.post-55844869920754742172011-05-13T12:12:00.000-07:002011-05-15T23:17:15.784-07:00

This year’s ASCO meeting will feature two important RNAi Therapeutics clinical studies: Silence Therapeutics’ Atu027 for advanced solid cancers, and Alnylam’s ALN-VSP02 for cancers with liver involvement. The data presentations kick off the most important period of clinical newsflow in the history of RNAi Therapeutics. Over the next couple of months, first clinical data from the best crop yet of

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com9tag:blogger.com,1999:blog-7857054149675424609.post-40661610613054142832011-05-08T02:02:00.000-07:002011-05-10T00:33:27.814-07:00

Shareholders of Alnylam Pharmaceuticals may be forgiven for heaving a sigh of relief when news broke of a Settlement between Alnylam and Max Planck on the one hand and the Whitehead Institute and the University of Massachusetts (UMass) on the other hand which seemed to resolve the Tuschl II ownership issue and finally put an end to the lengthy and costly Tuschl Litigation (follow filings and

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com6tag:blogger.com,1999:blog-7857054149675424609.post-46447193850829601912011-05-03T10:56:00.000-07:002011-05-03T11:35:38.938-07:00

Alnylam Pharmaceuticals disclosed in their Q1 2011 results that it will expand the dose escalation of the phase I study of ALN-TTR01 in TTR amyloidosis, increasing the target dose in that study from 0.4mg/kg to now 1mg/kg. This puts the company in a good position to demonstrate, unambiguously, that systemically administered SNALPs can knock down genes in the human liver. This dose expansion is

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com1tag:blogger.com,1999:blog-7857054149675424609.post-500426742250981042011-04-30T03:26:00.001-07:002011-05-03T00:42:41.382-07:00

Besides the cloudy economic picture in the established pharmaceutical markets, much of the current cost-cutting in pharmaceutical R&D can be explained by a loss of confidence of the industry in their own ability to efficiently develop drugs. This assessment is largely made by MBAs without a biomedical background who will measure current industry productivity in terms of how many drugs get

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com0tag:blogger.com,1999:blog-7857054149675424609.post-35197282429486545272011-04-25T07:22:00.000-07:002011-04-25T18:06:23.121-07:00

As consolidation in RNAi Therapeutics in the Old Economies continues, the balance of power is rapidly shifting towards the resource-rich and growing economies of the Far East where national RNAi champions are set to make their mark on the international RNAi Therapeutics scene. After some notable mergers between private and public RNAi companies (Intradigm-Silence, Cequent-Marina Biotech), and

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com14tag:blogger.com,1999:blog-7857054149675424609.post-56188014353160563602011-04-20T00:59:00.000-07:002011-04-20T04:36:53.543-07:00

Hardly a day goes by without news of delivery deals, or universities issuing a press release that their scientists have just published on a revolutionary and ‘novel’ delivery technology that, by the way, is available for licensing. To be fair, public relations is a necessary evil of drug development and even academia these days, and don’t expect any company to blink and admit to their weaknesses.

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com4tag:blogger.com,1999:blog-7857054149675424609.post-81270489737547127152011-04-09T03:57:00.000-07:002011-04-09T10:42:12.923-07:00

Alnylam this week responded to Tekmira’s Complaint a month ago in which it alleges Alnylam to have repeatedly misappropriated and misused Tekmira property (see also blog entry 'Enough is Enough'). In its Response, Alnylam points to the various legal arrangements between the two companies which the company feels entitles it to broad use of Tekmira’s property. In the good tradition of David vs

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com22tag:blogger.com,1999:blog-7857054149675424609.post-14543755827782089132011-03-31T04:25:00.000-07:002011-03-31T04:26:37.782-07:00

Times have never been tougher if you are a preclinical-stage biotechnology company with a high demand for cash to support operations. At least this must be the conclusion that RXi Pharmaceuticals drew after it struggled to attract investor interest in their self-delivering RNAi trigger technology, but a technology that has yet to enter the clinic. As a result, it announced today that it will

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com3tag:blogger.com,1999:blog-7857054149675424609.post-39624887262066367922011-03-29T04:33:00.000-07:002011-03-29T05:19:51.091-07:00

(Notice: The following account is a largely hypothetical explanation of what is underlying Alnylam's current troubles)The last time Alnylam expressed surprise and regret was in September 2010 when Novartis declined to exercise an option for wide, non-exclusive rights to Alnylam’s RNAi Therapeutics IP estate ('Adoption License'). Since then Alnylam has been hit with a string of other partnership

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com8tag:blogger.com,1999:blog-7857054149675424609.post-22781949670425900352011-03-23T10:58:00.000-07:002011-03-24T11:01:36.814-07:00

Not a day goes by without a lawsuit involving Alnylam. This time it would seem unjustly so. The company disclosed in a regulatory filing today that the University of Utah (‘Utah’) is suing the owners of the valuable Tuschl II (T-II) patent estate (Max Planck, the Whitehead Institute, MIT, and UMass) along with licensee Alnylam (but not Merck) for wrongfully omitting Utah scientist Brenda Bass

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com4tag:blogger.com,1999:blog-7857054149675424609.post-36618736216635486092011-03-21T06:37:00.000-07:002011-03-24T13:16:34.550-07:00

A lot has changed in the treatment of diabetic macular edema (DME) since Quark sub-licensed its AtuRNAi compound to Pfizer in 2006. Most notably, the monoclonal antibodies against VEGF, Lucentis and the cheaper derivative Avastin, are replacing laser photocoagulation as the standard of care in this condition that adversely impacts the vision of up to 10% of those living with diabetes. Quark last

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com4tag:blogger.com,1999:blog-7857054149675424609.post-72364094353653255692011-03-17T03:48:00.000-07:002011-03-17T05:39:41.753-07:00

The reason why I have been a strong supporter of Tekmira is because I give them the scientific credit for developing the most valuable systemic RNAi Therapeutics delivery technology without which the field would be in much worse shape. The gate-keeping position of Tekmira being the real enabler and innovator of LNP delivery, the result of having persisted through many years of dogged, high-class

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com22tag:blogger.com,1999:blog-7857054149675424609.post-69850988215197883062011-03-11T11:53:00.000-08:002011-03-12T07:30:40.511-08:00

Recent entries to the ocular clinical trials by Pfizer listed in ClinicalTrials.gov (see here and here) suggest that its first-generation RNAi Therapeutics candidate for wet age-related macular degeneration and diabetic macular edema, PF-04523655, is about to meet the same fate as did those for similar indications by Opko and Sirna Therapeutics (Merck)/Allergan before. The phase II study in

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com5tag:blogger.com,1999:blog-7857054149675424609.post-14656365937030034712011-03-06T01:40:00.000-08:002011-03-06T01:46:35.661-08:00

Marina Biotech this week reported pre-clinical data supporting the use of local, intravesicular siRNA delivery for the treatment of nonmuscle invasive bladder cancer. The publication in Molecular Therapy comes weeks after this early-stage program was partnered with Swiss pharmaceutical Debiopharm which will be responsible for funding the rest of the development. According to the National Cancer

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com3tag:blogger.com,1999:blog-7857054149675424609.post-82822072181600987622011-02-27T12:09:00.000-08:002011-02-27T12:10:16.332-08:00

One of the biggest concerns in developing an entirely new class of drugs is whether they will be well tolerated. At the height of the TLR scare, it almost seemed that just looking at an siRNA would cause you anaphylactic shock. Nevertheless, after several hundreds of patients have been dosed with probably around 1000 siRNA administrations for diseases affecting the eye, lung, skin, blood cells,

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com2tag:blogger.com,1999:blog-7857054149675424609.post-19013876164163035282011-02-21T06:58:00.000-08:002011-02-21T07:03:18.352-08:00

You have all seen the obituaries on RNAi Therapeutics. Following the terminations of in-house RNAi Therapeutics efforts by Roche, Pfizer, and Novartis (oh wait, Novartis actually hasn’t stopped developing RNAi Therapeutics, but with 31 targets has more than enough on its plate- minor detail), major news outlets have finally pronounced: RNAi Therapeutics is Dead! Meanwhile, in the 3-4 years since

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com13tag:blogger.com,1999:blog-7857054149675424609.post-34551429244691826602011-02-14T15:02:00.000-08:002011-02-15T12:28:45.627-08:00

After I tried to focus attention on the Silence Therapeutics presentation this afternoon at the BIO CEO & Investor Conference, as soon as I had published my earlier blog I half regretted my decision to do so because more often than not positive news does not materialize when you expect it. This blog in particular is littered with such speculations. Fortunately, this time was different as Silence

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com6tag:blogger.com,1999:blog-7857054149675424609.post-76105216090850333732011-02-14T04:57:00.000-08:002011-02-14T05:16:09.728-08:00

2011 promises to be a year of rich clinical data flow for RNAi Therapeutics. Results from clinical trials of ALN-VSP02, ALN-TTR01 (both Alnylam), Atu-027 (Silence Therapeutics), and TKM-PLK1 (Tekmira) in particular will be important in shaping the perception of RNAi Therapeutics as an investable drug modality. Following disappointment that Silence Therapeutics was not able to receive an

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com3tag:blogger.com,1999:blog-7857054149675424609.post-48527069554253823292011-02-04T05:05:00.000-08:002011-02-04T05:07:29.824-08:00

As if to add insult to injury, a few month after Roche exiting RNAi Therapeutics, plans emerged that Pfizer is about to shut down internal oligonucleotide therapeutics development efforts. This is depressing news for sure to me, and I would assume the wider RNAi Therapeutics community. But then again maybe not that surprising, and it would shock me no more to see Last-Man-Standing Merck shut down

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com11tag:blogger.com,1999:blog-7857054149675424609.post-66486427925724070882010-11-30T08:41:00.000-08:002010-11-30T09:05:41.163-08:00

The media coverage on Roche’s decision to ‘abandon’ all things RNAi Therapeutics almost makes one believe that we have just witnessed the demise of RNAi as a therapeutic modality. The naysayers of the technology had their heyday, apparently delighted by the that Roche's decision is absolute proof that RNAi has failed in its quest to improve human health. Nevermind that the Roche news has to be

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com10tag:blogger.com,1999:blog-7857054149675424609.post-89922929075770167792010-11-19T06:44:00.000-08:002010-11-19T07:21:00.510-08:00

Roche’s decision this week to terminate in-house RNAi Therapeutics development is widely reported to be a vote of no confidence in the platform. The alternative interpretation, however, namely that it might reflect a vote of no confidence in the ability of Big Pharma to innovate from within receives little or no consideration in an online media world that, at best, gets compensated according to

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com9tag:blogger.com,1999:blog-7857054149675424609.post-3336460107993614732010-11-17T05:55:00.000-08:002010-11-17T22:59:51.867-08:00

The writing has certainly been on the wall. First, in an August 2010 interview, Roche’s Head of Pharma Research and Early Development, Jean-Jaques Garaud, was a bit lukewarm about the maturity of RNAi Therapeutics. While acknowledging that ‘siRNA' was a 'hot field’, this Roche executive who assumed his current role in 2009 following the retirement of Jonathan Knowles, stated that RNAi

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com21tag:blogger.com,1999:blog-7857054149675424609.post-47163500614982820932010-11-12T05:29:00.000-08:002010-11-12T06:07:05.015-08:00

It is not the first time in RNAi Therapeutics history that we hear of promises that cash-generating deals will be closed by the end of the year. This year is no different: RXi Pharmaceuticals, Marina Biotech, and to some degree Silence Therapeutics by disclosing an approach 2 months ago, all have the markets expect lucrative Big Pharma partnerships or even acquisitions as the year winds down.

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com13tag:blogger.com,1999:blog-7857054149675424609.post-70238975427807513892010-11-11T02:39:00.000-08:002010-11-11T03:17:20.341-08:00

Alnylam provided an update on the ongoing phase I clinical studies for their RNAi Therapeutics candidate ALN-VSP02 for liver cancer. Clinical data such as these are widely anticipated by investors and potential partners alike, and Alnylam with recent comments have left themselves little choice but to be measured against the quality of the data. The presentation provided at the Chemotherapy

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com2tag:blogger.com,1999:blog-7857054149675424609.post-33604996326584434572010-11-04T09:05:00.000-07:002010-11-04T23:50:48.330-07:00

The latest quarter must have been the best quarterly performance by Alnylam in the last 3 years. After going through a rough patch that culminated in Novartis not adopting a widely expected $100M platform license with the attendant lay-off of a third of its staff, the results and also tone of the conference call struck me as if the company had re-discovered its old enthusiasm for RNAi

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com5tag:blogger.com,1999:blog-7857054149675424609.post-44429842175634698992010-10-31T21:01:00.000-07:002010-11-02T00:44:01.366-07:00

The current perception is that large pharmaceutical companies have become quite a bit more conservative in their approach towards RNAi Therapeutics. This stands in stark contrast with only 3 years ago when some of the same companies topped each other in their efforts to securing a piece of the RNAi Therapeutics action. Clearly, given that the development of any new class of drugs is a gradual

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com14tag:blogger.com,1999:blog-7857054149675424609.post-85434225732249124712010-10-26T02:00:00.000-07:002010-10-26T02:28:01.765-07:00

Last week, Silence Therapeutics announced that the European Patent Office has granted a patent from the Zamore RNAi trigger design IP estate (EP 1633890 B1). This follows the issuance of related patents over the summer in the US. This IP is assigned to the University of Massachusetts and exclusively licensed to Silence Therapeutics. What is newsworthy in this latest patent issuance is that

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com9tag:blogger.com,1999:blog-7857054149675424609.post-72351603362234139032010-10-19T03:46:00.000-07:002010-10-22T22:57:15.811-07:00

The most enjoyable part in following RNAi Therapeutics is to look at the rich stream of scientific data and determine the absolute maturity and competitive position of the technologies and companies involved, as well as getting a glimpse at relationship dynamics. I therefore thought to share today two examples of this that I picked up recently. One is a paper by Sirna Therapeutics/Merck shedding

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com6tag:blogger.com,1999:blog-7857054149675424609.post-19667646098264872072010-10-14T21:16:00.000-07:002010-10-14T21:39:29.358-07:00

As often in life, involve money, and you will soon see who your true friends are. This rule also seems to apply to the fate of the Tuschl patent applications which more and more seem to go in Max Planck/Alnylam’s favor, and against the interests of UMass and Sirna Therapeutics/Merck, according to the latest coverage on the Tuschl Litigation Blog. It has always been a mystery to me why Whitehead

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com3tag:blogger.com,1999:blog-7857054149675424609.post-81688908098925351532010-10-13T06:19:00.000-07:002010-10-14T01:47:50.558-07:00

Less than a month after Napoleone Ferrara from Genentech was recognized with a 2010 Lasker Prize for identifying VEGF as the central actor in blood vessel formation, a prize widely regarded as the stepping stone towards the Nobel Prize in Physiology or Medicine, a press release by Alnylam seems to suggest that RNAi delivery to the vascular endothelium is appropriately reaching critical mass.

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com2tag:blogger.com,1999:blog-7857054149675424609.post-17026617478748566192010-10-09T02:09:00.000-07:002010-10-09T02:46:28.788-07:00

Judging from the body language of RNAi Therapeutics companies, it seems that they are still fighting a wide-spread perception, also in Big Pharma, that systemic siRNA delivery is limited to targeting genes in the liver and that as a result the current commercial opportunities are relatively small. To quote from Alnylam’s press release yesterday, on research presented at the 8th International M.

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com9tag:blogger.com,1999:blog-7857054149675424609.post-48039246795151523682010-10-04T13:29:00.002-07:002010-10-05T14:47:20.060-07:00

A week or so ago, I had the opportunity to talk by phone to the CEO and CSO of RXi Pharmaceuticals, Noah Beerman and Anastasia Khvorova respectively, about the company's scientific and business development strategy. Overall, they were quite upbeat about the prospect of turning around the perception of a company aimlessly wandering around in early technology development and increase shareholder

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com6tag:blogger.com,1999:blog-7857054149675424609.post-55324217162520241862010-09-30T08:36:00.000-07:002010-09-30T09:05:19.044-07:00

One of the sadder stories in RNAi Therapeutics history is the many years lost in the commercial development of drugs based on DNA-directed RNAi (ddRNAi) as the space had become embroiled in litigation instead of investing in the science. In a sign that this chapter may be behind us and that ddRNAi Therapeutics can now look forward to a time where the significant medical potential of the

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com29tag:blogger.com,1999:blog-7857054149675424609.post-64351603437071496812010-09-24T01:46:00.000-07:002010-09-24T09:26:21.963-07:00

It is official now: Novartis will not exercise its $100M option to broadly license Alnylam’s RNAi trigger IP. This must come as a shock to Alnylam, about to lay off 25-30% of its workforce, which seemed to have taken the option exercise for granted. The news comes after failing to deliver on its promise last year to reach one or more platform deals. With all due respect for the management of

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com9tag:blogger.com,1999:blog-7857054149675424609.post-17340687123021083052010-09-23T08:01:00.000-07:002010-09-24T14:01:36.443-07:00

After deciding to focus on the use of so-called ‘self-delivering’ siRNAs (‘sd-rxRNA’) for dermatology and ophthalmology applications, RXi Pharmaceuticals has since entered into a number of relationships with companies to facilitate the uptake of sd-rxRNA in various organs.The latest such technology partnership with EyeGate Pharmaceuticals for ocular direct RNAi applications follows another direct

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com0tag:blogger.com,1999:blog-7857054149675424609.post-74711191441170910892010-09-10T08:21:00.000-07:002010-09-10T12:20:16.315-07:00

Tekmira’s ambition to grow a substantial clinical pipeline suffered a setback when it announced today that the IND for its candidate for the treatment of hypercholesterolemia, TKM-ApoB, has been delayed following a review of pre-clinical data. These data indicated that the ‘performance characteristics of the specific lipid nanoparticle formulation [...] have not met the Company’s expectations for

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com22tag:blogger.com,1999:blog-7857054149675424609.post-37197049769992938462010-09-06T08:47:00.000-07:002010-09-06T09:15:05.027-07:00

My guess: Pfizer or Novartis. I have just returned from a vacation on the beautiful island of Taiwan that also allowed me, finally away from my laptop, to reflect on RNAi Therapeutics and the financial markets. At the end of it (I just returned four hours ago), I was quite positive that the markets for high-risk/hi-reward investments such as RNAi Therapeutics have just hit rock bottom and that

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com20tag:blogger.com,1999:blog-7857054149675424609.post-41818934901727435162010-08-26T05:46:00.000-07:002010-08-27T06:19:47.827-07:00

As I am about to do some traveling over the next couple of weeks and won’t be able to monitor the markets closely, part of my preparations included a review and slight adjustment of my portfolio so as to position it well for what are likely to be turbulent weeks in RNAi Therapeutics. With the $100M Novartis decision coming up, a lot of the dust in the RNAi Trigger landscape about to settle, and

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com8tag:blogger.com,1999:blog-7857054149675424609.post-11512232626553059582010-08-23T09:24:00.000-07:002011-05-06T05:10:51.747-07:00

I was quite amused today, while driving a car on Borneo, the local radio report the headline ‘US scientists said that they are ready to start clinical trials for Ebola’. It was yet another reminder that oligonucleotide therapeutics, including RNAi is firmly emerging from the research stage, entering the medical arena and gaining more widespread attention. As you may also have heard by now, 3

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com4tag:blogger.com,1999:blog-7857054149675424609.post-60997845417373331342010-08-19T10:03:00.000-07:002010-09-28T07:59:57.672-07:00

In a reminder that the $100M adoption license option decision is coming close, Quark just announced that it has granted Novartis, for $10M in upfront and another $600M or so in potential milestones, the option to fully license Quark’s drug candidate QPI-1002, an siRNA targeting the famous 'guardian of the genome', p53. Delivered in its naked form intravenously, QPI-1002 is currently in phase I/

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com4tag:blogger.com,1999:blog-7857054149675424609.post-51822843456635576462010-08-16T07:44:00.000-07:002010-08-16T08:05:29.368-07:00

As Silence Therapeutics is being issued one patent after another from the Zamore siRNA design rule patent families in the US, and possibly also soon in Europe, there has been some confusion about their value, both scientific and strategic. Silence claims that they are highly valuable additions to the RNAi Therapeutics toolbox; Alnylam dismisses them as something they chose to pass on as

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com5tag:blogger.com,1999:blog-7857054149675424609.post-47166573985195856542010-08-10T08:41:00.000-07:002010-08-12T01:21:56.725-07:00

Just like SNALP delivery has become a staple of this blog, Alnylam has been investing aggressively into its development as exemplified by the one dozen or so presentations by the company and a whole range of collaborators, most notably the University of British Columbia and Old Tekmira spin-off Alcana, at the recent International Liposome Research Days at the world capital of liposomal nucleic

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com8tag:blogger.com,1999:blog-7857054149675424609.post-88798080993457130842010-08-04T12:26:00.001-07:002010-08-04T23:02:00.686-07:00

ISIS Pharmaceuticals and co-development partner Genzyme reported today top-line data from the final 2 out of 4 phase III studies with the ApoB-targeting antisense compound mipomersen for the treatment of hypercholesterolemia. The efficacy results came in above my lowered expectations and support mipomersen to be an attractive treatment option for the many patients with severe

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com7tag:blogger.com,1999:blog-7857054149675424609.post-73139244819894535922010-08-03T00:43:00.000-07:002010-08-03T01:06:09.250-07:00

A study by the Zamore group published in Science (Ameres et al.: Target RNA-directed Trimming and Tailing of Small Silencing RNAs) provides intriguing insights into an important mechanism impacting siRNA stability by showing that guide strands that are perfectly complementary to their target mRNAs are subject to tailing and subsequent degradation. By better understanding siRNA stability, such

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com2tag:blogger.com,1999:blog-7857054149675424609.post-82365840925664992652010-07-29T07:14:00.000-07:002010-07-29T11:41:10.735-07:00

Marina Biotech, formerly known as mdRNA, continues to snap up assets in the oligonucleotide therapeutics and diagnostics space, this time acquiring the liposomal delivery IP from Novosom, a privately held drug delivery company based in Germany. This comes only a week after shareholders approved mdRNA’s merger with tkRNAi company Cequent Pharmaceuticals to form Marina Biotech. It will be curious

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com9tag:blogger.com,1999:blog-7857054149675424609.post-5979332879750168462010-07-26T07:18:00.001-07:002010-07-26T07:48:44.550-07:00

Orphan disease drug developer Genzyme had emerged as the drug development of the future by developing innovative drugs which have a high impact for diseases of major unmet medical need and then being rewarded for it with exceptional pricing power. Recent manufacturing problems, however, has led to the business model being forgotten over regulatory angst and activist shareholder actions.

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com4tag:blogger.com,1999:blog-7857054149675424609.post-78166954150462332562010-07-22T04:30:00.000-07:002010-07-22T05:42:29.730-07:00

There is little question that Alnylam enjoys considerable control over arguably the most desirable type of synthetic RNAi triggers: 19-21 base-pair dsRNAs with 3’ overhangs- some of this control, of course, being somewhat subject to the outcome of the Tuschl Tussle and the review of the Tuschl II patent in Europe. In any case, this had caused a number of competitors to try and avoid this IP

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com3tag:blogger.com,1999:blog-7857054149675424609.post-3570382873943518102010-07-16T00:34:00.000-07:002010-07-25T08:43:54.041-07:00

The mystery surrounding the abrupt halt to the trading of Tekmira shares on July 14 was solved when Tekmira announced that it was awarded a significant US government contract for the development of an Ebola drug worth up to US$140M, $35M of which could be earned over the next 3 years. This essentially provides Tekmira with a free shot on goal for early product revenues, and should also prove

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com2tag:blogger.com,1999:blog-7857054149675424609.post-54884864711239252962010-07-14T22:52:00.000-07:002010-07-15T00:08:33.028-07:00

As many of you will be aware by now, trading of Tekmira shares has been abruptly halted just 15 minutes before market close on Wednesday July 14, 2010. Certainly, this strongly indicates that an unexpected event has occurred, but will it be good or bad news? As a biotech investor, I am prepared for more negative than positive surprises. Adverse events from clinical trials can be a source of

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com6tag:blogger.com,1999:blog-7857054149675424609.post-40918871725549527162010-07-14T03:55:00.001-07:002010-07-14T19:00:27.155-07:00

One week after announcing the grant of a US patent covering RNAi triggers with enhanced guide strand loading, Silence Therapeutics today announced the one-year extension of its R&D collaboration with AstraZeneca for the ‘identification and optimization’ of five siRNAs targeting genes involved in cancer and respiratory disease. This relationship dates back to 2007 when AstraZeneca paid Silence a

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com3tag:blogger.com,1999:blog-7857054149675424609.post-45713354129654160732010-07-08T01:28:00.001-07:002010-07-08T02:33:39.756-07:00

Silence Therapeutics announced yesterday the issuance of a United States patent related to the design of gene silencing siRNAs. This is the second such patent issuance in little more than a month for which Intradigm, Silence’s merger partner earlier this year, had exclusively licensed from the University of Massachusetts and that is based on fundamental work by the Zamore lab on the enzymology

Dirk Hausseckerhttp://www.blogger.com/profile/18320439857875629714noreply@blogger.com8