pharmaphorum

JULY 13, 2021

The Danish drugmaker is paying $100 million upfront for rights to antibody drug PRX004, which is heading for phase 2 trials in cardiomyopathy associated with ATTR amyloidosis, a life-threatening disease affecting the heart and nervous system. All told, the deal could be worth up to $1.2

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Gene Silencing Sales Gene Editing Clinical Trials 98

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 transaction is expected to close in the fourth quarter of this year.

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Gene Silencing RNA Protein Gene 52

pharmaphorum

JANUARY 6, 2021

Analysts are continuing to back Inventiva’s lanifibranor as a potential “best-in-class” drug for the fatty liver disease NASH, as the company prepares for the launch of a phase 3 trial in spring. The trial will take a while however, with results not due until the second half of 2023, with sales forecast to peak at around $2.6

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Gene Silencing Regulation Pharma Companies Doctors 52

pharmaphorum

OCTOBER 19, 2020

If approved, inclisiran would become the first and only gene-silencing drug to reduce low-density lipoprotein cholesterol (LDL-C) in these patients. Novartis’ drug is also playing catch-up with its rivals on data from trials that show the reduction in LDL-C is matched by an improvement in cardiovascular outcomes.

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Drugs Gene Silencing Sales Antibody 98

pharmaphorum

OCTOBER 19, 2020

If approved, inclisiran would become the first and only gene-silencing drug to reduce low-density lipoprotein cholesterol (LDL-C) in these patients. Novartis’ drug is also playing catch-up with its rivals on data from trials that show the reduction in LDL-C is matched by an improvement in cardiovascular outcomes.

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Drugs Gene Silencing Sales Antibody 52

XTalks

FEBRUARY 14, 2022

The drug’s approval in the EU was based on analysis of clinical trial data including both pre- and early-symptomatic, and early-onset MLD patients, which showed that a single-dose treatment is effective in modulating disease progression in most early-onset MLD patients.

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Gene Therapy Drugs Gene Gene Silencing 98

pharmaphorum

SEPTEMBER 1, 2021

As well as the access agreement for high-risk patients, the Novartis deal also covers a large-scale trial of the PCSK9 inhibitor and the creation of a consortium with academic groups aimed at improving the manufacturing of oligonucleotide drugs in the UK.

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Drugs Trials Gene Silencing Genetics 98

pharmaphorum

DECEMBER 23, 2020

The American Society of Clinical Oncology (ASCO) annual conference was one of the highlights of the year in cancer research and the fact it was held virtually did not distract from some stunning clinical lung cancer trial results presented by AstraZeneca. In the 500 patient trial, patients treated with Trodelvy lived for a median of 12.1

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Marketing Gene Silencing Gene Drugs 86

Delveinsight

AUGUST 6, 2020

Gene-silencing Therapies. Silencing or modifying gene TTR aims to reduce the effect of both variant and wild-type TTR gene, thus reducing hepatic production by targeting its mRNA. The past years have witnessed a shift from off-label and symptomatic therapies to approved TTR stabilizers and gene-silencing therapies.

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Gene Silencing Protein Marketing Gene 52

Drug Discovery World

OCTOBER 4, 2022

The pandemic might have affected the number of clinical trials taking place for cell and gene therapies, with activity decreasing by around 15% compared to 2020, but major therapies such as Novartis’ gene therapy drug for spinal muscular atrophy (SMA) Zolgensma still made it to market. . billion, compared to $19.9

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Gene Therapy Drugs Gene Manufacturing 52

pharmaphorum

JUNE 28, 2021

Preliminary results from a phase 1 trial run by Regeneron and CRISPR specialist Intellia Therapeutics – co-founded by Nobel Prize winner Jennifer Doudna – showed steep reductions in a biomarker of ATTR amyloidosis disease activity with a single dose of the NTLA-2001 drug. In three more patients treated at 0.1mg/kg levels fell by 52%.

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Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. This includes Denali Therapeutics whose treatment TAK-594/DNL593 is in a Phase I/II trial. UK-based AviadoBio works on a potential gene therapy called AVB-001 that targets this form of FTD.

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Pharma Marketing Network

DECEMBER 21, 2020

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e.,

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Genetics Medicine Gene Therapy Marketing 52

Drug Discovery World

JULY 25, 2023

“In this context, the data we have published in NEJM are exciting, suggesting the potential role for zilebesiran to treat hypertension in a novel way via a novel, subcutaneously administered gene silencing approach to hypertension.”

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Gene Silencing Clinical Development Gene Medicine 97

pharmaphorum

JUNE 8, 2022

Zilebesiran (formerly ALN-AGT) is one of a new breed of gene-silencing drugs that are intended to treat common, chronic diseases with infrequent dosing to boost compliance with treatment. In a phase 1 trial, zilebesiran was found to be well-tolerated and improve systolic blood pressure over 12 weeks with a single injection.

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Gene Silencing Drugs Medicine Regulation 52

pharmaphorum

MARCH 4, 2021

Sylentis, part of PharmaMar Group, has just announced FDA approval of a phase 3 trial for eye drops containing tivanisiran, in dry eye disease associated with Sjogren’s Syndrome. The design of the protocol for the trial and its authorization are based on the scientific evidence of safety and efficacy obtained in the phase 3 HELIX trial.

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Gene Silencing FDA Approval Trials Marketing 98

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

Drug Discovery World

AUGUST 1, 2023

This Phase I trial will test the experimental vaccine, known as H1ssF-3928 mRNA-LNP, for safety and its ability to induce an immune response.

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RNA Drugs Gene Silencing Vaccination 52

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.

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Drug Discovery World

JANUARY 23, 2023

To avoid these histocompatibility challenges, early clinical trials often opt for an autologous graft-based approach 8,9 to subvert the need for prolonged immunosuppressive treatment and streamline the grafting process. The first in-person iPSC-derived regenerative therapy trial took place at Kyoto University, Japan.

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Gene Editing Gene Engineer DNA 52

The Pharma Data

NOVEMBER 24, 2020

Professor and Chief, Pediatric Nephrology and Hypertension, Jack and Lucy Clark Department of Pediatrics, Mount Sinai Kravis Children’s Hospital, New York City and Investigator on the ILLUMINATE-A trial. Saland, M.D., Until today, there were no approved pharmaceutical therapies for PH1.

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