Gene Silencing, Production and RNA - Clinical Research Informer

NICE recommends gene silencing therapy for porphyria patients on NHS

Pharma Times

OCTOBER 21, 2021

Givlaari uses ‘gene silencing’ RNA interference technology, to target the production of pathogenic compounds in people AHP

Gene Silencing

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Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data

XTalks

OCTOBER 26, 2021

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

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Alnylam gets NICE backing for porphyria therapy Givlaari

pharmaphorum

OCTOBER 21, 2021

UK cost-effectiveness watchdog NICE is set to recommend NHS use of Alnylam’s gene-silencing therapy Givlaari in England and Wales for the rare disease acute hepatic porphyria (AHP), after its advisors issued a positive verdict on the drug. It was approved by the European Medicines Agency (EMA) last year. .

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Vevye: A New Cyclosporine Solution for Dry Eye Disease

XTalks

JUNE 14, 2023

Novaliq, a biopharmaceutical company specializing in ocular therapeutics, recently announced that their product Vevye (cyclosporine ophthalmic solution) 0.1 As a water-free product, it does not have an associated pH value or osmolarity. This mechanism reduces eye swelling and promotes tear production.

Gene Silencing

Gene Silencing Drug Delivery FDA Approval RNA

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

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

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Novo Nordisk snaps up RNAi partner Dicerna in $3.3bn takeover deal

pharmaphorum

NOVEMBER 18, 2021

Danish drugmaker Novo Nordisk must like what it has seen in its two-year-old alliance with gene-silencing specialist Dicerna Pharma – it has just agreed to acquire the biotech for $3.3 Once in the blood, the GalXC molecules travel to the liver, where they enter hepatocyte cells and can switch protein production on or off.

Gene Silencing

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Two biotechs target dry eye disease after Novartis’ Xiidra problems

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).

Gene Silencing

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NICE backs Alnylam’s Amvuttra for rare form of amyloidosis

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

Gene Silencing RNA Protein Production

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

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 is hoping that revenue will stream from its new products and payments from Novartis, which owns the rights to cholesterol lowering drug inclisiran.

FDA Approval

FDA Approval Gene Silencing Drugs RNA

Alnylam finds belly fat gene and drug target in UK Biobank study

pharmaphorum

JULY 28, 2022

Alnylam Pharma has made a name for itself, developing gene-silencing therapies for rare disorders, but its latest discovery could take it into a much larger category – metabolic and cardiovascular disease.

Biobanking

Biobanking Gene Gene Silencing Drugs

RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

OCTOBER 22, 2023

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

RNA

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2020 review – Pharma’s progress outside of COVID-19

pharmaphorum

DECEMBER 23, 2020

Biogen and Denali will co-market the lead LRRK2 product in the US and China, and Biogen will commercialise in all other markets. It was the third approval from Alnylam’s pipeline of RNA interference therapeutics to make it to market.

Marketing

Marketing Gene Gene Silencing Drugs

Unfolding The Folds Of Transthyretin

Delveinsight

AUGUST 6, 2020

The present hATTR treatment market constitutes compounds that stabilize the TTR tetramers, a TTR-directed small interfering RNA, and a TTR-directed antisense oligonucleotide, along with supportive therapies. They help in preventing confrontational changes and dissociation of protein, and not halt the production of the protein.

Protein

Protein Gene Silencing Marketing Gene

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

NOVEMBER 24, 2020

The excess production of oxalate results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones, nephrocalcinosis, progression to kidney failure, and systemic organ dysfunction. President of R&D at Alnylam.

FDA Approval

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Clinical Research Informer

NICE recommends gene silencing therapy for porphyria patients on NHS

Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data

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Alnylam gets NICE backing for porphyria therapy Givlaari

Webinars

Vevye: A New Cyclosporine Solution for Dry Eye Disease

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

Novo Nordisk snaps up RNAi partner Dicerna in $3.3bn takeover deal

Two biotechs target dry eye disease after Novartis’ Xiidra problems

NICE backs Alnylam’s Amvuttra for rare form of amyloidosis

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

Alnylam finds belly fat gene and drug target in UK Biobank study

RNA Therapeutics: A Novel Approach to Treating Diseases

2020 review – Pharma’s progress outside of COVID-19

Unfolding The Folds Of Transthyretin

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

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