pharmaphorum

OCTOBER 5, 2022

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. billion deal in 2018.

RNA 98

RNA Genetics Dermatology Antibody 98

pharmaphorum

DECEMBER 23, 2021

At its second attempt, Novartis has won FDA approval for its cholesterol lowering drug inclisiran, which can reduce levels with just two injections a year. Novartis added inclisiran to its pipeline after buying The Medicines Company – which had licensed the drug from Alnylam – for $9.7 It has a long way to go.

Drugs 101

Drugs Sales Antibody FDA Approval 101

Drug Discovery World

MARCH 3, 2023

Under the terms of the agreement SiSaf has an exclusive option to acquire a worldwide license to a patent by the University. miRNAs are involved in the regulation of various physiological and pathological processes.

Development 52

Development RNA Drug Delivery Regulation 52

pharmaphorum

OCTOBER 27, 2020

US biotech BioSig Technologies has abandoned a phase 2 trial of its antiviral drug merimepodib with Gilead’s Veklury in severe COVID-19 patients, after concluding the safety of the combination was in doubt. Moreover, as an orally-active drug merimepodib could also have found a role outside the hospital setting.

Drug Trials 98

Drug Trials Trials Drugs Contract Manufacturing 98

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.

Gene Silencing 57

Gene Silencing Drugs Medicine RNA 57

Drug Discovery World

OCTOBER 16, 2023

The United Kingdom has established itself as a hub for drug discovery expertise worldwide. A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases.

Drugs 75

Drugs Life Science Gene Therapy Genome 75

pharmaphorum

DECEMBER 21, 2020

Novartis’ cholesterol drug inclisiran has hit a speed bump with the FDA, which has rejected the potential blockbuster because a manufacturing facility has yet to be inspected. The European Commission has already approved the drug under the brand name Leqvio earlier this month following backing from the CHMP regulatory committee.

Drugs 52

Drugs RNA Manufacturing Licensing 52

pharmaphorum

JANUARY 7, 2022

AstraZeneca has clearly made the rare disease transthyretin amyloidosis (ATTR) a key component of its rare disease pipeline, licensing a second drug candidate in the space of a couple of months. The new agreement comes just days after AZ closed a $3.1 billion on the first nine months of 2021, well ahead of Pfizer’s own forecasts.

Antibody 113

Antibody Licensing Licensing Protein 113

pharmaphorum

APRIL 5, 2022

AstraZeneca’s take on the PCSK9 inhibitor – an antisense drug licensed from Ionis six years ago – has cleared a proof-of-concept trial in high-risk patients with high blood cholesterol, but it remains to be seen where it will fit into the increasingly busy category. billion in sales by 2027, ahead of Repatha at $2.2

Drugs 52

Drugs Antibody Sales FDA Approval 52

pharmaphorum

JULY 6, 2021

Novartis has tried to get its marketing application for high cholesterol therapy inclisiran in the US back on track, after the FDA rejected it last year, by changing the factory that makes the drug. Novartis added inclisiran to its pipeline after buying The Medicines Company, which had licensed the drug from Alnylam, for $9.7

RNA 102

RNA Manufacturing Sales Marketing 102

pharmaphorum

AUGUST 28, 2020

An antisense drug developed by GlaxoSmithKline has been shown to suppress the hepatitis B virus (HBV) in a phase 2 trial, raising hopes of a “functional cure” for the widespread disease. That is because viral DNA fragments can lurk in cells, hiding from current drug therapy and evading the immune system.

Drugs 52

Drugs DNA Licensing Licensing 52

The Pharma Data

JUNE 7, 2023

Acuitas’ LNP technology will support Bayer’s in vivo gene editing and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver. LNPs are spheric drug delivery bodies which can be equipped with therapeutic payloads for intracellular delivery.

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

pharmaphorum

JANUARY 6, 2021

Two years after starting to work together, Novo Nordisk and Dicerna have selected the first candidate from a joint project to find new, gene-silencing drugs for liver-related cardiometabolic diseases. Several others are in late-stage development, including fitusiran for haemophilia, which Sanofi is developing under license from Alnylam.

Gene Silencing 58

Gene Silencing Gene RNA Contract Manufacturing 58

Drug Discovery World

SEPTEMBER 14, 2023

Moreover, in July 2023, AstraZeneca and Vaxess Technologies commenced a collaboration for the evaluation of a novel RNA-based pandemic influenza prototype vaccine in patch format.

Drugs 59

Drugs Vaccination Vaccine Medicine 59

pharmaphorum

JANUARY 22, 2021

Veklury is currently the only antiviral drug licensed for the treatment of COVID-19 and works by interfering with a polymerase enzyme central to the production of viral RNA particles.

In-Vitro 140

In-Vitro RNA Licensing Licensing 140

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. Oxlumo (lumasiran) was also approved in the European Union last week, making it the third from Alnylam’s pipeline of RNA interference therapeutics to make it to market.

FDA Approval 52

FDA Approval Gene Silencing Drugs RNA 52

XTalks

JUNE 20, 2022

Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.

FDA Approval 98

FDA Approval Protein Genetics RNA 98

Pfizer

DECEMBER 20, 2022

FDA Extends Review of Pfizer’s New Drug Application for PAXLOVID™. FDA Extends Review of Pfizer’s New Drug Application for PAXLOVID™. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for its COVID-19 oral treatment, PAXLOVID™ (nirmatrelvir tablets and ritonavir tablets).

Drugs 69

Drugs Licensing Licensing Containment 69

pharmaphorum

FEBRUARY 9, 2021

million ($22 million) in second-round financing as it prepares to start clinical trials of its lead drug candidate INT-1B3 for solid tumours. The start of dosing in the phase 1/1b trial of the drug has been delayed by the COVID-19 pandemic, but recruitment of the target 80 participants with advanced solid tumours is reported to be ongoing.

Trials 57

Trials Gene Silencing RNA Gene Expression 57

pharmaphorum

MAY 11, 2022

The pharma group will provide funding for the research in return for exclusive license options on the programmes. billion takeover of gene-silencing specialist Dicerna Pharma last year, another deal aimed at extending its R&D capabilities by bolting on a new platform technology – in this case RNA interference (RNAi) drugs.

Gene Silencing 120

Gene Silencing Medicine RNA Licensing 120

Delveinsight

JANUARY 7, 2021

Ikena secures USD 120 Million to advance a growing pipeline of cancer drugs. Ikena Oncology has bagged USD 120 million to develop its expanding pipeline of cancer drugs. The newer side of the pipeline points targeted oncology drugs. Terns raises USD 87 Million for NASH drugs. IND-enabling studies are underway.

RNA 52

RNA Clinical Trials Clinical Trials Bioavailability 52

Pharmaceutical Technology

FEBRUARY 15, 2023

The swift development and deployment of messenger-RNA (mRNA) vaccines against the SARS-CoV-2 virus during the COVID-19 crisis has catapulted the pharmaceuticals industry into a new paradigm. RNA has the potential to underpin breakthrough treatments for a wide variety of diseases, including many cancers, and transform medicine as we know it.

Bioassay 130

Bioassay Development RNA Drugs 130

XTalks

DECEMBER 13, 2023

Related: Top 30 Pharma Companies in 2023: Statistics and Trends 1) Moderna Compound annual growth rate: 415 percent Moderna, headquartered in Massachusetts, is a prominent biotechnology company specializing in RNA therapeutics, particularly mRNA vaccines. This substantial investment is accelerating the growth of their new drug pipeline.

Genetics 111

Genetics Vaccination Vaccine DNA 111

XTalks

JULY 14, 2022

Rare and rheumatic disease-focused biotech Horizon Therapeutics has received expanded approval from the US Food and Drug Administration (FDA) for its gout injection Krystexxa (pegloticase) to include its co-administration with the immunomodulator methotrexate. The drug has proven to be quite profitable for Horizon as it hit a record of $565.5

FDA Approval 105

FDA Approval Antibody Trials Drugs 105

pharmaphorum

JANUARY 12, 2021

US biotech Atalanta Therapeutics has come out of stealth mode backed with $110m from Biogen and Roche, who have also signed separate partnerships to develop new therapies for neurological diseases using RNA interference (RNAi) technology. It also reflects the contribution from Khorova as the founder of the company.

RNA 81

RNA Production Development Pharma Companies 81

The Pharma Data

MARCH 8, 2021

Week 96 findings reinforce the primary (proportion of participants with plasma HIV-1 RNA ?50 50 c/mL at Week 48 (Snapshot, ITT-E)), and secondary endpoints (proportion of participants with plasma HIV-1 RNA ?50 Week 48 primary endpoint (proportion of participants with plasma HIV-1 RNA ?50

RNA 52

RNA Branding Development Licensing 52

pharmaphorum

SEPTEMBER 2, 2022

Novo Nordisk licensed rights to EPI01 from EpiDestiny in 2018, paying $400 million for rights to the drug in haemaglobinopathies, while the biotech retained rights in oncology. billion acquisition of RNA interference specialist Dicerna, which closed earlier this year. It follows Novo Nordisk’s $3.3

RNA 52

RNA Licensing Licensing Trials 52

pharmaphorum

MAY 24, 2022

Monkeypox is considered less likely to mutate quickly because it is a DNA virus rather than an RNA virus like influenza or COVID-19. ” Vaccines and drugs are available. There’s also a licensed antiviral drug for monkeypox. The US FDA also approved a new intravenous form of the drug last week.

Vaccination 80

Vaccination Vaccine RNA DNA 80

pharmaphorum

NOVEMBER 2, 2022

CSL’s vaccines unit CSL Seqirus division is paying Arcturus $200 million upfront to gain access to its self-amplifying mRNA technology platform and exclusive licenses to several vaccine candidates, with another $4.3 billion on offer in milestones if the programmes meet development and commercial objectives.

Vaccination 52

Vaccination Vaccine Immune Response Gene Therapy 52

Pfizer

DECEMBER 13, 2022

In June of 2022, Pfizer submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for approval of PAXLOVID for patients who are at high risk of progression to severe disease from COVID-19. The target Prescription Drug User Fee Act (PDUFA) action date for a decision by the FDA is in February 2023.

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Drugs Licensing Licensing Production 110

pharmaphorum

NOVEMBER 23, 2021

GlaxoSmithKline has joined the ranks of drugmakers trying to develop new drugs for non-alcoholic steatohepatitis (NASH) – a condition with no approved therapies and a lengthy list of failed drug candidates – via a deal with Arrowhead Pharmaceuticals.

Sales 52

Sales Drugs Trials RNA 52

The Pharma Data

DECEMBER 8, 2020

AbCellera’s AI-powered antibody discovery platform speeds the otherwise lengthy and grueling process by analyzing the database of natural immune systems to find antibodies that can be developed into drugs. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Remix Therapeutics .

RNA 52

RNA Antibody Life Science Protein 52

Drug Discovery World

DECEMBER 12, 2023

KRRO-110 is designed to co-opt an endogenous enzyme Adenosine Deaminase Acting on RNA (ADAR) to repair a pathogenic single nucleotide variant (SNV) on RNA and restore production of normal A1AT.

RNA 52

RNA In-Vivo Genotype Genetics 52

The Pharma Data

MARCH 8, 2021

Of 202 treated participants, no safety signals have been identified and of the 4 serious adverse events reported, none were considered to be study drug related. This program included assays such as Big Blue and PIG-a designed to provide a robust measure of a drug or chemical’s ability to induce mutations in vivo.

Trials 69

Trials In-Vivo Genotoxicity RNA 69

The Pharma Data

NOVEMBER 29, 2020

Food and Drug Administration (FDA). Y-mAbs Therapeutics has a target action date of November 30 for its Biologics License Application (BLA) for Danyelza (naxitamab) for patients with relapsed/refractory high-risk neuroblastoma. The FDA approved the drug on November 24. It is an RNA interference (RNAi) therapeutic.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

Pfizer

JANUARY 27, 2023

Nirmatrelvir is designed to inhibit viral replication at a stage known as proteolysis, which occurs before viral RNA replication. Sufficient information is not available to assess for a potential drug interaction. Modification of other medications is needed due to a potential drug interaction. AUTHORIZED USE The U.S.

Marketing 75

Marketing Production Drugs Licensing 75

The Pharma Data

AUGUST 19, 2021

The application will be reviewed by the EMA’s Committee for Medicinal Products for Human Use (CHMP) under the centralized licensing procedure for all 27 Member States of the European Union, as well as Norway, Iceland and Liechtenstein. In June 2021, Gilead submitted a New Drug Application (NDA) for lenacapavir seeking U.S.

Medicine 52

Medicine Marketing Drugs FDA Approval 52