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

Gene Silencing 98

Gene Silencing Sales Gene Editing Clinical Trials 98

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 52

FDA Approval Gene Silencing Drugs RNA 52

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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Gene Silencing Sales Marketing Drugs 110

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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Gene Therapy Drugs Gene Gene Silencing 98

Delveinsight

AUGUST 6, 2020

The liver is the major production house of the protein TTR; therefore, orthotopic liver transplantation is an attractive and disease-modifying treatment available to the patients, which can reduce the quantity of the mutant protein significantly, thus halting the progression of the disease. Gene-silencing Therapies.

Gene Silencing 52

Gene Silencing Protein Marketing Gene 52

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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Gene Therapy Licensing Licensing Gene 277

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. 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. Rare disease progress.

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Marketing Gene Silencing Gene Drugs 78

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. Knockout’ mice are bred in a laboratory with specific genes silenced or ‘knocked out’.

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52

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.

Gene Silencing 52

Gene Silencing Drugs Medicine Regulation 52

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 52

Biobanking Gene Gene Silencing Drugs 52

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. In three more patients treated at 0.1mg/kg levels fell by 52%.

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In-Vivo Gene Editing Trials Drugs 100

Drug Discovery World

AUGUST 1, 2023

As per a definition provided by AstraZeneca: “Once inside, ASOs bind with high specificity to target mRNA or pre-mRNA, inducing its degradation – effectively silencing it – to prevent its translation into a detrimental protein product.

RNA 52

RNA Drugs Gene Silencing Vaccination 52

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 98

Gene Silencing FDA Approval Trials Marketing 98

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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Gene Silencing Protein Gene Engineer 40

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 52

FDA Approval Production RNA Medicine 52

Roots Analysis

OCTOBER 22, 2023

ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein. RNA interference (RNAi) Therapeutics : Fundamentally, RNAi is a natural process of post-transcriptional gene silencing, involving short strands of nucleic acids.

RNA 40

RNA Protein Genetics Gene Expression 40

Drug Discovery World

JANUARY 23, 2023

Many trials have successfully modified iPSCs with the CAR transgene, providing a highly scalable bank of cells that can easily be expanded and differentiated into therapeutic products (22). About the author: Kevin Hemphill is an R&D Manager at PerkinElmer’s Horizon Discovery.

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Gene Editing Gene Engineer DNA 52