pharmaphorum

DECEMBER 13, 2020

Ultomiris is already approved for PNH and aHUS and in late-stage development for NMOSD, which could lend further momentum. Along with new indications for its existing drugs, the Swiss biopharma has four more drugs in phase 3 development that could benefit from the increased financial and development muscle that AZ will bring to the table.

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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. 70 mg/dL) when given on top of standard treatment.

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XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. million (approximately $3.8 million USD).

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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 billion in cash. The $38.25-per-share per-share deal represents a premium of around 80% on Dicerna’s closing price yesterday.

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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.

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Biobanking Gene Gene Silencing Drugs 52

pharmaphorum

DECEMBER 23, 2020

Originally developed to treat late-stage EGFR-mutated disease, the ADAURA study tested Tagrisso as an adjuvant treatment in patients with early disease following surgery. A molecule codenamed DNL151, currently in phase 1 development, has been selected to progress into late-stage clinical studies that are expected to begin in 2021.

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XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. Epigenome Editing with CRISPR.

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pharmaphorum

OCTOBER 29, 2020

Already a major player in gene therapy, Novartis has swooped on US startup Vedere Bio in a $280 million deal that builds its position in inherited eye diseases that can lead to blindness. It also gets two preclinical-stage development projects. IRDs affect around 2 million people worldwide.

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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. These therapeutics are broadly classified into two categories, namely coding RNAs and non-coding RNAs.

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The Pharma Data

APRIL 27, 2023

(Nasdaq: ALNY ) announced today positive interim results from the ongoing single ascending dose part of the Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy (CAA).

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The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. Together, the ILLUMINATE studies comprise a comprehensive clinical development program intended to demonstrate the safety and efficacy of Oxlumo across the full spectrum of patients diagnosed with PH1. For more information about Oxlumo, visit OXLUMO.com.

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