Alnylam Gene-Silencing Therapy To Treat Kidney Disorder Succeeds In Late Stage Study

61
915

Alnylam Gene-Silencing Therapy To Treat Kidney Disorder Succeeds In Late Stage Study

(Reuters) – Alnylam Pharmaceuticals Inc’s gene-silencing therapy for a rare kidney disorder met the main goal of a late-stage study on Tuesday, bringing the company a step closer to marketing the first approved treatment for the condition.

The study tested Alnylam’s experimental drug, lumasiran, against placebo in patients aged six and above with primary hyperoxyluria type 1 (PH1), a life-threatening condition that is estimated to affect one in 58,000 people globally.

Alnylam plans to file for the drug’s approval in the United States and Europe early next year and hopes to launch the drug before the end of 2020.

The company’s president, Barry Greene, estimates the market opportunity for PH1 treatments to be over $500 million.

Lumasiran works using a mechanism called RNA interference (RNAi) to target and “silence” the genetic material involved in making excess amounts of a chemical called oxalate.

Jared Leto on movies, tech and the Met Gala

Excess oxalate builds up in the kidneys of patients with PH1, eventually leading to kidney and bladder stones. In severe cases, they may have to undergo dialysis, kidney or liver transplants.

https://www.reuters.com

In the trial, lumasiran was found to significantly reduce the production of oxalate in patients taking a monthly dose for three months, followed by maintenance doses, compared to those administered with a placebo.

Needham analyst Alan Carr had forecast global peak sales of between $450 million and $500 million for lumasiran in 2032, before the trial results were announced.

Alnylam already has two gene-silencing treatments in the market for rare hereditary disorders.

In 2018, Onpattro, Alnylam’s treatment targeting a symptom of a potentially fatal condition called hereditary ATTR amyloidosis, became the first RNAi treatment to be approved in the United States. (reut.rs/2sEXmHR)

In November, the company received approval for Givlaari to treat acute hepatic porphyria, a rare disorder that can lead to severe pain and paralysis, respiratory failure and seizures.

Reporting by Ruhi Soni and Tamara Mathias; Editing by Vinay Dwivedi

Alnylam Gene-Silencing Therapy To Treat Kidney Disorder Succeeds In Late Stage Study

61 COMMENTS

  1. Keep up the great work , I read few content on this website and I conceive that your website is very interesting and has got circles of superb info .

  2. I discovered your blog site on google as well as check a few of your early messages. Remain to keep up the excellent operate. I just additional up your RSS feed to my MSN News Visitor. Seeking ahead to finding out more from you in the future!?

  3. I’ve been exploring for a bit for any high-quality articles or blog posts on this sort of area . Exploring in Yahoo I at last stumbled upon this website. Reading this info So i’m happy to convey that I have an incredibly good uncanny feeling I discovered exactly what I needed. I most certainly will make certain to don’t forget this site and give it a glance on a constant basis.

  4. Thanks for sharing your ideas in this article. The other matter is that each time a problem develops with a laptop or computer motherboard, people today should not have some risk involving repairing the item themselves because if it is not done right it can lead to irreparable damage to the complete laptop. Most commonly it is safe to approach a dealer of that laptop for the repair of motherboard. They’ve already technicians with an know-how in dealing with laptop motherboard troubles and can have the right analysis and undertake repairs.

  5. Neat blog! Is your theme custom made or did you download it from somewhere? A design like yours with a few simple tweeks would really make my blog shine. Please let me know where you got your design. Cheers

  6. I don’t even know how I ended up here, but I thought this post was good. I do not know who you are but certainly you’re going to a famous blogger if you are not already 😉 Cheers!

  7. Great ¡V I should definitely pronounce, impressed with your web site. I had no trouble navigating through all tabs and related info ended up being truly simple to do to access. I recently found what I hoped for before you know it in the least. Reasonably unusual. Is likely to appreciate it for those who add forums or anything, web site theme . a tones way for your client to communicate. Excellent task..

  8. Thanks for your fascinating article. One other problem is that mesothelioma cancer is generally a result of the breathing of dust from asbestos, which is a carcinogenic material. It can be commonly witnessed among employees in the engineering industry who definitely have long exposure to asbestos. It can also be caused by living in asbestos insulated buildings for a long period of time, Inherited genes plays an important role, and some consumers are more vulnerable on the risk than others.

  9. We are Daylight Studio, a boutique photography studio in East London available for hire. We are a creative natural light studio space with decorated French walls, lots of props and furniture and massive windows for great natural light. He have a unique selection of chairs, fabric backgrounds and a sofa for your next natural light photography project. The studio is great for fashion, portrait and commercial photography and if natural light photography studio is not your thing we have a lot of lighting equipment available for you as well and that includes studio strobe lights with various modifiers and LED constant lights for video production. We also have facilities for e-commerce photography. Please see the studio’s gallery here.

  10. I have to convey my appreciation for your kind-heartedness in support of visitors who absolutely need assistance with in this topic. Your special dedication to passing the message all through became incredibly beneficial and have usually enabled guys and women much like me to arrive at their dreams. Your personal invaluable hints and tips signifies a lot a person like me and still more to my office colleagues. Many thanks; from all of us.

  11. Hi there very cool web site!! Man .. Excellent .. Wonderful .. I will bookmark your site and take the feeds additionally…I’m satisfied to find so many helpful information here within the submit, we want work out extra techniques on this regard, thanks for sharing.

  12. I as well as my buddies have been examining the good solutions located on your website and so then came up with a horrible feeling I had not thanked the web site owner for those techniques. My people had been certainly stimulated to read through them and have in effect very much been enjoying them. I appreciate you for actually being so helpful as well as for picking this form of marvelous ideas millions of individuals are really eager to learn about. Our own sincere apologies for not expressing gratitude to you earlier.

  13. I cling on to listening to the news update lecture about getting boundless online grant applications so I have been looking around for the best site to get one. Could you advise me please, where could i acquire some?

  14. Attractive section of content. I just stumbled upon your web site and in accession capital to claim that I acquire in fact enjoyed account your weblog posts. Anyway I will be subscribing in your augment and even I fulfillment you get entry to constantly rapidly.|

  15. An excellent share, I simply offered this onto an associate who was doing a little evaluation on this. And he actually purchased me breakfast due to the fact that I found it for him. smile. So let me rephrase that: Thnx for the treat! However yeah Thnkx for investing the time to discuss this, I really feel highly regarding it and like reading more on this topic. When possible, as you end up being know-how, would certainly you mind upgrading your blog site with even more information? It is very useful for me. Large thumb up for this post!

  16. Hi! Someone in my Myspace group shared this site with us so I came to give it a look. I’m definitely enjoying the information. I’m book-marking and will be tweeting this to my followers! Wonderful blog and excellent style and design.

  17. Get help to stop drinking. Helping women quit drinking alcohol. Coach Mary Wagstaff. Feel better, mind, body and soul. Mindfulness based coaching and alcohol recovery. Yoga for recovery.

  18. Thanks for any other informative blog. The place else could I am getting that type of information written in such a perfect manner? I have a challenge that I am simply now operating on, and I’ve been at the look out for such information.

  19. Excellent read, I just passed this onto a colleague who was doing a little research on that. And he just bought me lunch as I found it for him smile Thus let me rephrase that: Thank you for lunch!

  20. Great goods from you, man. I have understand your stuff previous to and you’re just too great. I really like what you have acquired here, really like what you are stating and the way in which you say it. You make it entertaining and you still take care of to keep it sensible. I can’t wait to read far more from you. This is actually a great web site.

  21. Hello, i believe that i noticed you visited my weblog so i got here to “return the favor”.I’m trying to in finding things to enhance my website!I suppose its ok to make use of a few of your ideas!!

  22. I do consider all the ideas you have introduced in your post. They are really convincing and will definitely work. Nonetheless, the posts are very brief for novices. May just you please prolong them a little from subsequent time? Thanks for the post.

  23. Thanks a lot for sharing this with all of us you actually know what you are talking about! Bookmarked. Kindly also visit my site =). We could have a link exchange contract between us!

  24. I recently found your blog site yahoo and checked out several of your earlier posts. Keep up the great work. I also added your Rss to my MSN News Reader. Looking forward to reading more of your stuff at a later point!…

  25. Hi there are using WordPress for your site platform? I’m new to the blog world but I’m trying to get started and set up my own. Do you require any html coding expertise to make your own blog? Any help would be really appreciated!|

  26. There are very plenty of details like that to think about. This is a fantastic point out retrieve. I provide thoughts above as general inspiration but clearly you will find questions including the one you talk about the place that the most essential factor will likely be doing work in honest good faith. I don?t know if guidelines have emerged about such thinggs as that, but I am sure that the job is clearly defined as a reasonable game. Both youngsters glance at the impact of simply a moment’s pleasure, for the rest of their lives.

  27. Thanks for a marvelous posting! I seriously enjoyed reading it, you could be a great author.I will be sure to bookmark your blog and definitely will come back at some point. I want to encourage one to continue your great job, have a nice afternoon!|

  28. Please let me know if you’re looking for a author for your site. You have some really good articles and I feel I would be a good asset. If you ever want to take some of the load off, I’d love to write some articles for your blog in exchange for a link back to mine. Please blast me an email if interested. Regards!

  29. Good ¡V I should certainly pronounce, impressed with your web site. I had no trouble navigating through all the tabs as well as related information ended up being truly simple to do to access. I recently found what I hoped for before you know it at all. Reasonably unusual. Is likely to appreciate it for those who add forums or something, website theme . a tones way for your customer to communicate. Nice task..

  30. Howdy! I simply would like to give an enormous thumbs up for the great data you could have right here on this post. I might be coming back to your blog for more soon.

  31. Woah! I’m really enjoying the template/theme of this website. It’s simple, yet effective. A lot of times it’s very difficult to get that “perfect balance” between usability and appearance. I must say you’ve done a amazing job with this. In addition, the blog loads extremely fast for me on Chrome. Outstanding Blog!

  32. I think that is among the so much important information for me. And i am glad reading your article. However want to observation on some general things, The web site style is perfect, the articles is truly nice : D. Excellent process, cheers

  33. That is the fitting blog for anybody who wants to search out out about this topic. You notice so much its almost laborious to argue with you (not that I truly would want…HaHa). You definitely put a brand new spin on a subject thats been written about for years. Nice stuff, just great!

  34. Excellent blog! Do you have any helpful hints for aspiring writers? I’m planning to start my own blog soon but I’m a little lost on everything. Would you recommend starting with a free platform like WordPress or go for a paid option? There are so many options out there that I’m completely overwhelmed .. Any ideas? Bless you!

  35. An outstanding share, I just offered this onto a colleague that was doing a little evaluation on this. As well as he as a matter of fact acquired me breakfast due to the fact that I located it for him. smile. So let me rephrase that: Thnx for the reward! But yeah Thnkx for investing the moment to review this, I feel highly regarding it as well as enjoy reading more on this subject. Preferably, as you become know-how, would certainly you mind updating your blog with more information? It is very valuable for me. Large thumb up for this post!

  36. I am also commenting to let you be aware of what a fabulous encounter my princess developed going through your webblog. She figured out a wide variety of things, which include what it’s like to possess a marvelous coaching mindset to let many people clearly know precisely some specialized subject areas. You actually exceeded visitors’ desires. Thank you for supplying these essential, trustworthy, educational and also easy tips about that topic to Evelyn.

LEAVE A REPLY

Please enter your comment!
Please enter your name here