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. Transgenic mice in which the gene has been deleted have improved control of blood glucose and insulin sensitivity.

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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).

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pharmaphorum

DECEMBER 13, 2020

It marks something a departure from AZ’s relentless focus on deal-making in oncology, its top product category, and also comes as Alexion has been locked in a battle with activist shareholders pushing for a sale. In the past, Novartis, Roche, Pfizer and Amgen have all been mentioned as potential suitors.

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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.

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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.

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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.

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pharmaphorum

JUNE 8, 2022

The passport means that LentiGlobin will be reviewed by the Medicines and Healthcare products Regulatory Agency (MHRA) via the new innovative licensing and access pathway (ILAP) introduced in 2020, which reduces the review time for a new medicine to 150 days.

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The Pharma Data

APRIL 27, 2023

These early results establish the first human translation of Alnylam’s proprietary C16-siRNA conjugate platform for central nervous system (CNS) delivery and are the first clinical demonstration of gene silencing in the human brain using an RNAi therapeutic. Yancopoulos, M.D., President and Chief Scientific Officer of Regeneron.

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XTalks

FEBRUARY 14, 2022

MLD is a lipid storage disease caused by defects in the production of the fatty myelin coating on nerve fibers in the central and peripheral nervous systems (the “white matter”), leading to severe neurological and organ damage that can be fatal. The incidence of MLD is approximately one in 100,000 worldwide.

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pharmaphorum

JULY 13, 2021

The bulk of Novo Nordisk’s revenues come from its diabetes products, although it also active in the development and sale of drugs for growth disorders, haemophilia and sickle cell disease. All told, the deal could be worth up to $1.2 The post Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal appeared first on.

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pharmaphorum

DECEMBER 23, 2020

2020 also saw some of the first “tumour agnostic” cancer drugs get to market, with Bayer’s Vitravki (larotrectinib) getting funding in the UK for tumours with confirmed neurotrophic tyrosine receptor kinase (NTRK) gene fusions.”. Rare disease progress.

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Delveinsight

AUGUST 6, 2020

Hereditary ATTR amyloidosis , also known as Familial ATTR, is an autosomal dominant disease and a result of mutations in the TTR gene, thus it is passed down from one generation to the next one. They help in preventing confrontational changes and dissociation of protein, and not halt the production of the protein.

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Roots Analysis

OCTOBER 22, 2023

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein.

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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.

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