pharmaphorum

JUNE 28, 2021

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105

pharmaphorum

JUNE 1, 2022

The US biotech has just reported phase 2 trial results with the small, interfering RNA (siRNA) gene-silencing drug showing that it an cause a 90% or greater reduction in Lp(a) levels – a risk factor for cardiovascular disease – that was sustained over 48 weeks of follow-up. .

Gene Silencing 59

Gene Silencing Drugs Medicine RNA 59

pharmaphorum

MARCH 4, 2021

Tivanisiran is based on gene silencing technology using RNA interference (RNAi) and selectively inhibits production of the transient receptor potential cation channel (TRPV1). ” The post Two biotechs target dry eye disease after Novartis’ Xiidra problems appeared first on.

Gene Silencing 98

Gene Silencing FDA Approval Trials Marketing 98

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. Alnylam licensed inclisiran to The Medicines Company, which was bought last year by Novartis for $9.7

FDA Approval 52

FDA Approval Gene Silencing Drugs RNA 52

pharmaphorum

JANUARY 19, 2023

Alnylam’s gene-silencing drug Amvuttra has been recommended as a treatment for hereditary transthyretin-related (ATTR) amyloidosis by NICE, paving the way for the drug to be made available on the NHS in England and Wales.

Gene Silencing 52

Gene Silencing RNA Protein Production 52

pharmaphorum

DECEMBER 23, 2020

But there was some considerable progress in other fields of medicine even though research efforts were diverted away, reports Richard Staines. It was the third approval from Alnylam’s pipeline of RNA interference therapeutics to make it to market.

Marketing 96

Marketing Gene Gene Silencing Drugs 96

The Pharma Data

NOVEMBER 24, 2020

Results from the ILLUMINATE-A and ILLUMINATE-B studies demonstrate that Oxlumo addresses the underlying pathophysiology of PH1 in adults, children and infants, and we believe this newly approved medicine has the potential to change the course of this progressive disease,” said Akshay Vaishnaw, M.D., President of R&D at Alnylam.

FDA Approval 52

FDA Approval Production RNA Medicine 52