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RNAi Therapeutics

Tuesday, June 23, 2015. The RNAi Therapeutics Blog is Taking a Break. When it feels that everything has been said, it may be time to be silent for a while. This point has come for me and the RNAi Therapeutics blog and I look forward to take part in the conversation with renewed energy and ideas. Until then, you can follow the 'light' version of this blog on Twitter @RNAiAnalyst. Tuesday, June 23, 2015. Thursday, June 11, 2015. There is No Doubt: Splice Modulator Drug for Spinal Muscular Atrophy Works.

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RNAi Therapeutics | rnaitherapeutics.blogspot.com Reviews
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Tuesday, June 23, 2015. The RNAi Therapeutics Blog is Taking a Break. When it feels that everything has been said, it may be time to be silent for a while. This point has come for me and the RNAi Therapeutics blog and I look forward to take part in the conversation with renewed energy and ideas. Until then, you can follow the 'light' version of this blog on Twitter @RNAiAnalyst. Tuesday, June 23, 2015. Thursday, June 11, 2015. There is No Doubt: Splice Modulator Drug for Spinal Muscular Atrophy Works.
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1 pages
2 about me
3 posted by
4 dirk haussecker
5 20 comments
6 provided an update
7 11 comments
8 labels infant
9 isis pharmaceuticals
10 isis smnrx
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RNAi Therapeutics | rnaitherapeutics.blogspot.com Reviews

https://rnaitherapeutics.blogspot.com

Tuesday, June 23, 2015. The RNAi Therapeutics Blog is Taking a Break. When it feels that everything has been said, it may be time to be silent for a while. This point has come for me and the RNAi Therapeutics blog and I look forward to take part in the conversation with renewed energy and ideas. Until then, you can follow the 'light' version of this blog on Twitter @RNAiAnalyst. Tuesday, June 23, 2015. Thursday, June 11, 2015. There is No Doubt: Splice Modulator Drug for Spinal Muscular Atrophy Works.

INTERNAL PAGES

rnaitherapeutics.blogspot.com rnaitherapeutics.blogspot.com
1

RNAi Therapeutics: What to Watch for in RG-101 Post Regulus Therapeutics Executive Departures

http://rnaitherapeutics.blogspot.com/2015/06/what-to-watch-for-in-rg-101-post.html

Thursday, June 4, 2015. What to Watch for in RG-101 Post Regulus Therapeutics Executive Departures. As you will probably know already, Regulus Therapeutics surprised us this week with the sudden departures of both. Of getting rid of the virus. In more positive news, Regulus Therapeutics today reported that a first-in-man study with their second most advanced microRNA Therapeutic, RG-012 for the kidney-related orphan disease Alport Syndrome, has begun dosing. Thursday, June 04, 2015. Mr X has left RGLS to...

2

RNAi Therapeutics: June 2015

http://rnaitherapeutics.blogspot.com/2015_06_01_archive.html

Tuesday, June 23, 2015. The RNAi Therapeutics Blog is Taking a Break. When it feels that everything has been said, it may be time to be silent for a while. This point has come for me and the RNAi Therapeutics blog and I look forward to take part in the conversation with renewed energy and ideas. Until then, you can follow the 'light' version of this blog on Twitter @RNAiAnalyst. Tuesday, June 23, 2015. Thursday, June 11, 2015. There is No Doubt: Splice Modulator Drug for Spinal Muscular Atrophy Works.

3

RNAi Therapeutics: June 2014

http://rnaitherapeutics.blogspot.com/2014_06_01_archive.html

Friday, June 27, 2014. ISIS Pharmaceutical Reveals Dynamic PolyConjugate Efforts. When Arrowhead Research made a splash 1 ½ years ago. At the Boston OTS meeting in late October 2012 with impressive subQ DPC knockdown efficacy in monkeys, there was a large number of meeting participants crowding the speaker after his presentation. Although I sat a few rows away, the most eager questioner was an employee from ISIS Pharmaceuticals who managed to beat the Merck representative to the podium! Of December 4, 20...

4

RNAi Therapeutics: May 2015

http://rnaitherapeutics.blogspot.com/2015_05_01_archive.html

Wednesday, May 27, 2015. RXi Pharmaceuticals Could Be Much More Than Skin Wound Healing. RXi Pharmaceuticals today commenced. A secondary offering setting it on course to raise $10M, enough to finance the company for another year while expanding its pipeline and technology. It could thus mark a new chapter in the life of this company which had shoe-boxed itself into a single-product (RXI-109 for dermal wound healing) company following a toxic 2012 financing that gave Tang Capital Partners de facto. The e...

5

RNAi Therapeutics: October 2014

http://rnaitherapeutics.blogspot.com/2014_10_01_archive.html

Wednesday, October 22, 2014. Anti-MiR122 Therapeutic Stuns HCV World with Single-Dose Efficacy Results. This morning, Regulus Therapeutics greeted us. With amazing results from a phase I study of RG-101, an anti-microRNA 122 oligo for the treatment of HCV infection. The results show that in the exploratory HCV-infected patient subgroup, a single dose. Of 2mg/kg of RG-101 resulted in a mean viral load reduction of 4.1log. These results even exceed my own wildest imaginations (as discussed here. Interestin...

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AUPH | Open Reading Frame

http://www.orf-blog.com/tag/auph

Skip to primary content. Skip to secondary content. Could gene therapy become biotech’s growth driver in 2017? July 4, 2016. Exelixis Renal cancer data are too good to ignore. August 2, 2015. As followers of this blog know, I have been a long term Exelixis ( EXEL. On Could gene therapy become biotech’s growth driver in 2017? On Could gene therapy become biotech’s growth driver in 2017? On Could gene therapy become biotech’s growth driver in 2017? Proudly powered by WordPress.

orf-blog.com orf-blog.com

TRVN | Open Reading Frame

http://www.orf-blog.com/tag/trvn

Skip to primary content. Skip to secondary content. Biotech selloff leaves Wall Street disillusioned. January 18, 2016. Biotech portfolio updates Thoughts on ongoing correction and upcoming catalysts. August 22, 2015. Trevena Late stage innovation flying under the radar. July 19, 2015. Modest market cap ($264M) despite a positive clinical signal, an ongoing phase 2b and a potential FDA approval within three years. Continue reading →. Biotech portfolio update Sage, Marinus and Immunogen. June 14, 2015.

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ESPR | Open Reading Frame

http://www.orf-blog.com/tag/espr

Skip to primary content. Skip to secondary content. Biotech catalysts for 2016. April 4, 2016. After last week’s pessimistic post, this week I am focusing on potential catalysts in 2016 that could improve sentiment towards biotech as a sector. As if to remind us late stage trials don’t always fail, last week saw positive news from three different programs, all of which are antibodies in non-oncology indications. Regeneron ( REGN. And its partner Sanofi ( SNY. Had positive P3 data for its PCSK9 program.

orf-blog.com orf-blog.com

PCRX | Open Reading Frame

http://www.orf-blog.com/tag/pcrx

Skip to primary content. Skip to secondary content. Trevena Late stage innovation flying under the radar. July 19, 2015. Modest market cap ($264M) despite a positive clinical signal, an ongoing phase 2b and a potential FDA approval within three years. Continue reading →. On Could gene therapy become biotech’s growth driver in 2017? On Could gene therapy become biotech’s growth driver in 2017? On Could gene therapy become biotech’s growth driver in 2017? Proudly powered by WordPress.

orf-blog.com orf-blog.com

Uncategorized | Open Reading Frame

http://www.orf-blog.com/category/uncategorized

Skip to primary content. Skip to secondary content. Could gene therapy become biotech’s growth driver in 2017? July 4, 2016. Notes from ASCO 2016. June 9, 2016. Although this year’s ASCO contained a limited amount of groundbreaking data, it provided some interesting take-aways and signaled important trends in oncology drug development. Below is my take on a quiet but important meeting. Immuno-oncology PD-1 combinations at their infancy. Biotech catalysts for 2016. April 4, 2016. After last week’s pessimi...

orf-blog.com orf-blog.com

AERI | Open Reading Frame

http://www.orf-blog.com/tag/aeri

Skip to primary content. Skip to secondary content. Are biotech valuations sustainable? June 21, 2015. The biotech sector is currently in the most successful period in its history based on valuation metrics, amount of money raised and number of IPOs. The perception around the current situation ranges from very bearish (it’s a bubble) to very bullish (there’s enough innovation to fuel future growth). Esperion and Aerie Countdown to M&A. February 22, 2015. At first glance Esperion ( ESPR. And Aerie ( AERI.

orf-blog.com orf-blog.com

Open Reading Frame | Analyzing biotech stocks | Page 2

http://www.orf-blog.com/page/2

Skip to primary content. Skip to secondary content. Newer posts →. ECC 2015 Renal cancer showdown between BMS and Exelixis. September 20, 2015. Biotech portfolio updates Thoughts on ongoing correction and upcoming catalysts. August 22, 2015. Exelixis Renal cancer data are too good to ignore. August 2, 2015. As followers of this blog know, I have been a long term Exelixis ( EXEL. Trevena Late stage innovation flying under the radar. July 19, 2015. Modest market cap ($264M) despite a positive clinical sign...

orf-blog.com orf-blog.com

GEVA | Open Reading Frame

http://www.orf-blog.com/tag/geva

Skip to primary content. Skip to secondary content. Are biotech valuations sustainable? June 21, 2015. The biotech sector is currently in the most successful period in its history based on valuation metrics, amount of money raised and number of IPOs. The perception around the current situation ranges from very bearish (it’s a bubble) to very bullish (there’s enough innovation to fuel future growth). On Could gene therapy become biotech’s growth driver in 2017? On Could gene therapy become biotech’s growt...

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RNAi Therapeutics

Tuesday, June 23, 2015. The RNAi Therapeutics Blog is Taking a Break. When it feels that everything has been said, it may be time to be silent for a while. This point has come for me and the RNAi Therapeutics blog and I look forward to take part in the conversation with renewed energy and ideas. Until then, you can follow the 'light' version of this blog on Twitter @RNAiAnalyst. Tuesday, June 23, 2015. Thursday, June 11, 2015. There is No Doubt: Splice Modulator Drug for Spinal Muscular Atrophy Works.

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