Scienmag

JULY 2, 2021

A newly developed CRISPR-Cas9-based tool carries out efficient and long-term gene silencing by epigenetic editing Credit: BioDesign Research Originally discovered as a bacterial mode of defense against invading viruses, the remarkable ability of CRISPR-Cas9 to modify specific locations of DNA has made it a researcher favorite among gene editing tools. (..)

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Gene Silencing Gene Editing Gene DNA 75

Scienmag

OCTOBER 16, 2020

Credit: TMIMS RNA interference is a gene regulatory mechanism in which the expression of specific genes is downregulated by endogenous microRNAs or by small interfering RNAs (siRNAs). Although siRNAs have broad potential for gene-silencing therapy, their instability is one of the difficulties to develop siRNA-based agents.

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Gene Silencing RNA Gene Development 57

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. This is a huge development for patients, more info at: [link] !

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Gene Silencing Gene RNA Drugs 98

XTalks

JUNE 14, 2023

Sheppard, MD, MMSc, FACS, professor of ophthalmology at Eastern Virginia Medical School, and mid-atlantic medical director for Eye Care Partners and investigator in the development program. Attendees will understand best practices for novel drug delivery design and development. What Is Dry Eye Disease?

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Gene Silencing Drug Delivery FDA Approval RNA 97

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

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pharmaphorum

MARCH 4, 2021

The pipeline for dry eye disease drugs is beginning to heat up, with two biotechs announcing key developments, aiming to disrupt a market led by Allergan and Novartis. 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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Gene Silencing FDA Approval Trials Marketing 98

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 through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.

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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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Gene Silencing Sales Marketing Drugs 111

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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Gene Silencing Drugs Medicine RNA 59

pharmaphorum

APRIL 13, 2021

Antios has raised $96 million in Series B financing to support the ongoing phase 2 development of its hepatitis B drug candidate, ATI-2173. There are several competitors developing treatments for hepatitis B in what could turn out to be a competitive market.

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Gene Silencing Trials Drugs DNA 64

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

FEBRUARY 18, 2022

With 28 pioneering speakers from large pharma, innovative biotech and KOLs of academia who are ready to discuss the full and comprehensive range of RNAi drugs from discovery to development and beyond, join us in Boston to hear how they address the major challenges facing the industry. Key Benefits of Attending.

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XTalks

FEBRUARY 14, 2022

Developed by Orchard Therapeutics, Libmeldy received approval from the European Commission (EC) in December 2020 and about a month later, received a regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) to expedite its path to approval in the US. million (approximately $3.8 million USD).

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Gene Therapy Gene Drugs Gene Silencing 98

pharmaphorum

FEBRUARY 9, 2021

InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells.

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Trials Gene Silencing RNA Gene Expression 59

pharmaphorum

JANUARY 6, 2021

The Jefferies team also noted that the French biotech is also partnered with AbbVie to develop an oral successor to its inflammatory diseases blockbuster Humira (adalimumab). Proof-of-concept phase 1b data is due from the drug codenamed ABBV-157 is due this quarter, which will help AbbVie decide on whether to continue development.

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Gene Silencing Regulation Pharma Companies Trials 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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Marketing Gene Gene Silencing Drugs 97

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

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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Biobanking Gene Gene Silencing Drugs 52

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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DNA Gene Gene Silencing Genome 52

The Pharma Data

JANUARY 26, 2021

Scopus is a biopharmaceutical company developing transformational therapeutics based on groundbreaking scientific and medical discoveries. The company’s lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. Scopus BioPharma Inc.

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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.

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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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Pharma Marketing Network

DECEMBER 21, 2020

Here are 5 considerations when developing a communications strategy for a novel genetic medicine. #1. Early development of a simple narrative is important to provide a shared understanding of and early scientific differentiation for a novel genetic therapy. Genetic literacy among clinicians may not be as high as you think.

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Genetics Medicine Gene Therapy Marketing 52

Roots Analysis

OCTOBER 22, 2023

Furthermore, these drug candidates can be used to develop precise and individualized therapies that allow patients to produce therapeutic proteins in their own bodies. RNA interference (RNAi) Therapeutics : Fundamentally, RNAi is a natural process of post-transcriptional gene silencing, involving short strands of nucleic acids.

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The Pharma Data

NOVEMBER 24, 2020

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. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development.

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