Science News

[edafan]

This is what my son, Eric, is working on.

Judgment day nears for Alnylam and a new technology

Soon-to-be released clinicial trial data will reveal much about the Cambridge biotech’s future — as well as RNAi drug development
A $7 billion market valuation and the credibility of the entire field of RNA interference drug development is riding on the results of Alnylam Pharmaceuticals’ [[ALNY1) next big clinical trial. No pressure, folks.

edafan

[marv2]

Edafan, here's a link to the story:

https://www.statnews.com/2017/09/15/alnylam-rnai-rare-disease/

[marv2]

Here is another related article : https://pharmaphorum.com/news/alnylam-haemophilia-death/

[edafan]

Latest News
Alnylam’s rare disease drug shines in trial, paving way for a brand-new class of medicines

The age of RNA interference drugs has arrived. Alnylam Pharmaceuticals announced positive results Wednesday 9/20/2017 from a closely watched phase 3 clinical trial of patisiran, the company’s lead drug designed to treat a rare nerve disorder known as familial amyloid polyneuropathy [[FAP.)
Most importantly, the safety profile of patisiran in the FAP study looks clean, assuaging concerns raised by toxicity reported with some of Alnylam’s other pipeline drugs.
Sept 20 [[Reuters) - Alnylam Pharmaceuticals Inc's ALNY.O drug to treat a rare genetic disease cleared a key study, moving the company closer to filing a marketing application for the drug, which is part of a new class of medicines that block disease-causing proteins. The company's shares soared as much as 38.4 percent to hit a 21-month high in morning trading on Wednesday.
Alnylam's drug, patisiran, belongs to a class of drugs that use RNA interference [[RNAi) technology based on manipulating ribonucleic acid to interfere with or "silence" targeted genes in order to prevent the formation of proteins that can cause diseases.
Patisiran was being evaluated in a late-stage study against a placebo for the treatment of patients with hereditary ATTR [[hATTR) amyloidosis with polyneuropathy, which affects about 50,000 people worldwide, Alnylam said. a symptom of the disease, is the simultaneous malfunction of many peripheral nerves in the body resulting in tingling, numbness and kidney dysfunction.
The study evaluated nerve impairment at 18 months and showed significant overall disease improvement.
Leerink analyst Paul Matteis said the results were a "big win" for Alnylam and RNAi as they validated the platform, noting that the safety data looked clean and sufficient to quell any concerns.
Alnylam, which said it would file a marketing application for patisiran in late 2017, has been weighed down in the past by safety concerns of its drugs.
The company in early September stopped giving doses of its fitusiran drug for a rare bleeding disorder to patients enrolled in clinical studies after the death of a patient. year, Alnylam stopped developing an RNAi drug to treat hereditary amyloidosis with cardiomyopathy, due to patient deaths in the study. noted patisiran's safety results, a major focus for investors, appear to trump those of a rival drug being developed by Ionis Pharmaceuticals Inc IONS.O .
Ionis' treatment cleared a study for the same disease earlier this year, but reported side effects that sent its shares down. only approved treatments for hATTR are liver transplantation for early-stage disease and tafamidis, a drug approved in Europe, Japan and certain countries in Latin America, the drugmaker said.
Under an agreement, Alnylam said it would advance patisiran in the United States, Canada and Western Europe while French drugmaker Sanofi SASY.PA would commercialize it in the rest of the world.

[edafan]

This is what my son just emailed me:
"it could change medicine - basically RNAi therapeutics could essentially treat any disease because its further up regulated than any current DNA/host targetted therapeutics.



You gave me all the skills and drive to be successful in life and my work...........




love you"

[edafan]

Here's an instruction video on how it works


https://www.youtube.com/watch?v=cK-OGB1_ELE

[marv2]

This is what my son just emailed me:
"it could change medicine - basically RNAi therapeutics could essentially treat any disease because its further up regulated than any current DNA/host targetted therapeutics.



You gave me all the skills and drive to be successful in life and my work...........




love you"

That is great.

[TomatoTom123]

Wow, this sounds pretty big. And good.

[edafan]

Wow, this sounds pretty big. And good.


thanks

TomatoTom123

I am very proud of his work As my late wife, his step mom, was dying he and I held her hands. I said to her "Eric will work to save the lives of those children in your honor" She nodded her head, and she passed away.

edafan

[TomatoTom123]

https://www.youtube.com/watch?v=cK-OGB1_ELE

Wow, I'm gonna have to say that I didn't quite get all of that [[lol) but it sounds very impressive.

My limited knowledge of biology tells me that treating things at a bacteria level is good, but actually changing things at a DNA level is even better.

[TomatoTom123]

thanks

TomatoTom123

I am very proud of his work As my late wife, his step mom, was dying he and I held her hands. I said to her "Eric will work to save the lives of those children in your honor" She nodded her head, and she passed away.

edafan

Oh, edafan, I'm sorry to hear of your loss. But your son seems to be involved in some really incredible things. And your wife knew that before she passed away. :)