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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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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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

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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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

Drug Discovery World

SEPTEMBER 5, 2022

Knockout’ mice are bred in a laboratory with specific genes silenced or ‘knocked out’. The goal of this final phase is to produce and phenotype hundreds of knockout mouse models for genes with little to no known function, to better understand the genetic bases for diseases in humans and animals. .

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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

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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

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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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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Pharmaceutical Technology

FEBRUARY 20, 2023

Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions. This is also one of the major challenges that slow the development of effective treatments for FTD, he adds. Do we develop a single treatment for all three?

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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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Gene Therapy Gene Gene Silencing Protein 40

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

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 Silencing Gene Drugs 80

Drug Discovery World

OCTOBER 4, 2022

As it stands, there are currently 19 gene therapies approved for clinical use, alongside 18 RNA therapies and 59 non-genetically modified cell therapies, according to the Q2 report by the American Society of Cell & Gene Therapy. million by Horizon Europe which will go towards its goal of developing bioprocessing technologies. .

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Pharmaceutical Technology

JANUARY 31, 2023

Under the terms of the deal, uniQure will obtain the worldwide rights to develop and market the clinical stage gene therapy, APB-102. uniQure stated that the license of APB-102 further strengthens its gene therapies pipeline developed for the treatment of neurological disorders as well as miRNA-based gene silencing programmes.

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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

Drug Discovery World

JULY 25, 2023

Alnylam Pharmaceuticals has revealed results from its Phase I study of zilebesiran, an investigational RNAi therapeutic targeting liver-expressed angiotensinogen (AGT) in development for the treatment of hypertension. The results are published in the New England Journal of Medicine (NEJM).

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

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DNA Gene Gene Silencing Genome 98

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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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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Drug Discovery World

AUGUST 1, 2023

In terms of partnerships and development , Orbit Discovery and start-up SanegeneBio have entered into a Master Service Agreement to identify tissue-specific delivery of a wide range of RNA therapeutics, as reported by DDW in January 2023. Over at UK biotech firm SynaptixBio, a company which secured £13.2

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RNA Drugs Gene Silencing Vaccination 52

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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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

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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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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Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. In addition, iPSC lines, despite their pluripotency, have skewed suitability towards certain cell types 12,13.

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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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