Clinical Research Informer

Can gene therapies for haemophilia defend their high price tags?

Pharmaceutical Technology

NOVEMBER 9, 2022

million price tag. The post Can gene therapies for haemophilia defend their high price tags? After its approval, bluebird had set Zynteglo’s price at roughly EUR1.6 million ($1.8 million at the time). Since its FDA approval in August 2022, the beta-thalassemia therapy was launched in the US with a $2.8

Gene Therapy

Gene Therapy Gene FDA Approval Medicine

Biogen’s Alzheimer’s drug: What’s the price tag on hope?

World of DTC Marketing

JUNE 1, 2021

If the drug is approved you can bet that it’s going to carry a huge price tag. It could carry a price tag as high as $50,000-a-year per patient, according to drug analysts, adding billions of dollars to the nation’s health tab. . Will they follow the science or will they put a price tag on hope?

Drugs

Drugs Trials Doctors Doctor

Lutris wins FDA orphan drug tag for EGFR inhibitor-induced rash candidate

Pharmaceutical Technology

FEBRUARY 29, 2024

LUT014 is being investigated in an ongoing Phase II trial as a first-line treatment for EGFR inhibitor-induced acneiform rash, for which there is no approved drug.

Drugs

Drugs Trials

Disc wins orphan drug tag for rare blood cancer

Pharmaceutical Technology

FEBRUARY 12, 2024

The humanised monoclonal antibody DISC-3405 is under investigation in a Phase I clinical trial, with data expected this year.

Clinical Trials

Clinical Trials Clinical Trials Antibody Drugs

Moderna now in the crosshairs of Sens. Warren, Welch over price tag for COVID vaccine

Fierce Pharma

JANUARY 25, 2023

Warren, Welch over price tag for COVID vaccine kdunleavy Wed, 01/25/2023 - 13:16 Moderna now in the crosshairs of Sens.

Vaccination

Vaccination Vaccine

Sporting a $3.5M price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA finish line

Fierce Pharma

NOVEMBER 23, 2022

price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA finish line. Sporting a $3.5M Wed, 11/23/2022 - 10:10.

Gene Therapy

Gene Therapy Gene

Vicore’s lung disease digital therapy gets breakthrough tag

pharmaphorum

MARCH 20, 2024

Vicore gets FDA breakthrough status for Almee, a cognitive behavioural therapy (CBT) digital health therapy used to support people with pulmonary fibrosis

FDA puts breakthrough tag on Roche, Lilly Alzheimer’s test

pharmaphorum

APRIL 11, 2024

Roche and Eli Lilly have been granted breakthrough status by the FDA for their pTau217 blood test for Alzheimer's disease.

Cybin bags breakthrough tag for psychedelic depression drug

pharmaphorum

MARCH 14, 2024

Cybin files a $150m placement on the back of new clinical data with its psychedelic drug CYB003 for depression and an FDA breakthrough designation.

Drugs

‘Click’ chemistry used to tag proteins made by cancer cells

Drug Discovery World

OCTOBER 26, 2022

The new method involves adding chemical tags to sugar molecules which are added to cells. When the cells make these proteins, they remain marked with the chemical tag, meaning that researchers can identify them. . The post ‘Click’ chemistry used to tag proteins made by cancer cells appeared first on Drug Discovery World (DDW).

Protein

Protein Genetics Containment Research

Lilly wades deeper into copycat insulin market, scoring interchangeability tag for Lantus biosim

Fierce Pharma

NOVEMBER 18, 2022

Lilly wades deeper into copycat insulin market, scoring interchangeability tag for Lantus biosim. fkansteiner. Fri, 11/18/2022 - 11:01.

Insulin

Insulin Marketing

MindMed gets FDA breakthrough tag for LSD in anxiety

pharmaphorum

MARCH 8, 2024

In another inflection point for the psychedelic medicines category, Mind Medicine (MindMed) has claimed breakthrough status from the FDA for its LSD-based treatment candidate MM120 as a treatment for anxiety.

Medicine

Ultimovacs bags orphan drug tag for mesothelioma vaccine

Pharmaceutical Technology

OCTOBER 9, 2023

The FDA decision follows a similar designation for the vaccine UV1 against malignant melanoma in December 2021.

Vaccination

Vaccination Vaccine Drugs

Click bags FDA breakthrough tag for schizophrenia DTx

pharmaphorum

JANUARY 4, 2024

Digital health company Click Therapeutics has won an FDA breakthrough device designation for CT-155, a digital therapeutic (DTx) for the negative symptoms of schizophrenia, like social withdrawal and apathy, that tend not to respond well to antipsychotic drugs.

Drugs

Revolo advances plans to tackle eosinophilic esophagitis with orphan drug tag

Pharmaceutical Technology

JANUARY 31, 2024

The FDA has granted an orphan drug designation to Revolo’s ‘1104, with plans for a Phase IIb study later this year.

Drugs

Sanders takes aim at US drug prices of Novo’s Ozempic and Wegovy

Pharmaceutical Technology

APRIL 25, 2024

A Senate Committee is investigating the “outrageously high” US price tags of the blockbuster diabetes and obesity drugs.

Drugs

Grünenthal’s non-opioid pain drug gets breakthrough tag

pharmaphorum

MAY 23, 2023

Grünenthal’s non-opioid pain drug gets breakthrough tag Phil.Taylor Tue, 05/23/2023 - 08:20 Bookmark this

Drugs

Harmony wins orphan drug tag for pitolisant to treat Prader-Willi syndrome

Pharmaceutical Technology

FEBRUARY 22, 2024

Harmony is advancing pitolisant into a Phase III clinical trial that is expected to start in Q1 2024.

Clinical Trials

Clinical Trials Clinical Trials Drugs Trials

With European approval secured, BioMarin puts roughly $1.5M price tag on hemophilia gene therapy

Bio Pharma Dive

AUGUST 25, 2022

On a conference call, BioMarin executives revealed the anticipated price for Roctavian and the company's initial launch plans, which include pay-for-performance deals customized to different markets.

Gene Therapy

Gene Therapy Gene Marketing

Novel tag provides first detailed look into goliath grouper behavior

Scienmag

NOVEMBER 4, 2021

Persistent observations of large underwater animals are difficult to achieve without the help of electronic, multi-sensor tags.

Scientist

Scientist Engineer

NorthSea Therapeutics bags FDA rare paediatric disease tag for NASH drug

Pharmaceutical Technology

OCTOBER 17, 2023

The Netherlands-based company’s treatment has been awarded the designation by the US FDA following a successful Phase I trial.

Drugs

Drugs Trials

NeoImmuneTech bags orphan drug tag for IL-7 acute radiation syndrome treatment

Pharmaceutical Technology

NOVEMBER 20, 2023

NeoImmuneTech is developing NT-17, an IL-7 treatment, to promote rapid T-cell recovery in patients with acute radiation syndrome.

Drugs

Drugs Development

#news #biotech Bluefin tuna tagged for the first time in UK waters with acoustic ‘residency’ tags

BioTech 365

JANUARY 18, 2022

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Bluefin tuna tagged for the first time in UK waters with acoustic ‘residency’ tags.Bluefin tuna have been tagged with state-of-the-art acoustic tracking tags for the first time … Continue reading →

Research

Novartis-linked startup launches with technology designed to remove ‘destroy’ tags on helpful proteins

Bio Pharma Dive

JULY 28, 2022

Vicinitas and its protein-stabilizing research have attracted $65 million from a group of investors that includes a16z, Deerfield and GV.

Protein

Protein Research

FDA approves Duchenne gene therapy with hefty $3.2m price tag

Pharmaceutical Technology

JUNE 23, 2023

Sarepta will now need to complete a confirmatory trial to prove the therapy improves physical function and mobility.

Gene Therapy

Gene Therapy FDA Approval Gene Trials

FDA gives AbbVie’s c-Met lung cancer ADC a breakthrough tag

pharmaphorum

JANUARY 5, 2022

The post FDA gives AbbVie’s c-Met lung cancer ADC a breakthrough tag appeared first on. Hutchmed’s drug is also being paired with Tagrisso and AZ’s PD-L1 blocker Imfinzi (durvalumab) in global registrational trials.

Antibody

Antibody Drugs Protein Gene

BioMarin's hemophilia gene therapy could have warranted a record price tag, ICER finds

Bio Pharma Dive

AUGUST 28, 2020

A draft report from the drug pricing watchdog concluded that, under the right conditions, Roctavian may be cost-effective at a price of $2.5

Gene Therapy

Gene Therapy Gene Drugs

Genprex’s Reqorsa gene therapy picks up orphan drug designation from FDA for SCLC

Pharmaceutical Technology

AUGUST 11, 2023

The latest tag adds to three fast track designations for Reqorsa, with the company initiating a Phase I/II trial in Q4 2023.

Gene Therapy

Gene Therapy Gene Trials Drugs

Pfizer and Arvinas win UK’s fast track tag for breast cancer drug

Pharmaceutical Technology

AUGUST 1, 2023

Vepdegestrant was awarded the Innovation Passport designation for treating advanced or metastatic breast cancer.

Drugs

Amylyx ALS drug draws criticism over $158,000 price tag

pharmaphorum

OCTOBER 3, 2022

Following news of Relyvrio’s $158,000 annual price tag, both ALS advocates and outside observers have voiced outrage at the cost. The post Amylyx ALS drug draws criticism over $158,000 price tag appeared first on. ALS affects over 30,000 people in the US. It can lead to progressive paralysis and death.

Drugs

Drugs Generic Drugs FDA Approval Trials

Sanders Launches Probe of ‘Outrageously High Prices’ of Novo’s Ozempic, Wegovy

BioSpace

APRIL 24, 2024

on Wednesday launched an investigation into the exorbitant prices of Novo Nordisk’s Ozempic and Wegovy blockbusters, and has asked the Danish drugmaker to justify their price tags. Bernie Sanders (I-Vt.)

Orchard sets new gene therapy price tag at $4.25M—the steepest of any drug in the US

Fierce Pharma

MARCH 20, 2024

After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy (MLD) in the U.S., Kyowa Kirin and its subsidiary Orchard Therapeutics have | After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy in the U.S.,

Gene Therapy

Gene Therapy Gene Genetic Disease Genetics

Acorn woodpeckers wage days-long battles over vacant territories, radio tag data show

Scienmag

SEPTEMBER 7, 2020

Researchers used radio tags to understand the immense effort woodpecker warriors expend traveling to and fighting in these dangerous battles. Credit: Neil Losin When acorn woodpeckers inhabiting high-quality territories die, nearby birds begin a battle royal to win the vacant spot.

Research

Blue Note leukaemia DTx gets FDA breakthrough tag

pharmaphorum

JANUARY 11, 2022

The post Blue Note leukaemia DTx gets FDA breakthrough tag appeared first on. In 2020, it formed an alliance with Memorial Sloan Kettering Cancer Centre in the US to develop an app that can be used by patients who are living in fear of their cancer, for example. Other DTxs are aimed at people being treated for breast and lung cancers.

Development

Development Production Drugs

FDA gives Daiichi Sankyo’s HER3 drug a breakthrough tag

pharmaphorum

DECEMBER 23, 2021

The post FDA gives Daiichi Sankyo’s HER3 drug a breakthrough tag appeared first on. The Japanese drugmaker was originally developing patritumab as an antibody for NSCLC, but abandoned the drug after it showed little efficacy in mid-stage testing, pivoting to the ADC version.

Drugs

Drugs Antibody Clinical Trials Clinical Trials

Applied DNA Receives Purchase Orders Totaling $1.6 Million to Tag Textile Fibers

BioTech 365

SEPTEMBER 27, 2021

Million to Tag Textile Fibers Applied DNA Receives Purchase Orders Totaling $1.6 Million to Tag Textile Fibers – Company Progressing Textile Projects Representing New Commercial Opportunities Catalyzed by Brands and Suppliers Prioritizing Their … Continue reading → Applied DNA Receives Purchase Orders Totaling $1.6

DNA

DNA Branding

CRISPR tagging improves accuracy of model cells grown from stem cells

Scienmag

DECEMBER 1, 2020

Tagging produces detailed catalog of transcription factors key to making each cell type Credit: Gersbach Lab, Duke University DURHAM, N.C. – A team of biomedical engineers at Duke University has created a new way to turn stem cells into a desired cell type by mastering the language of gene regulatory networks.

Engineer

Engineer Gene Bioinformatics

Enhertu gets breakthrough tag in HER2-low breast cancer

pharmaphorum

APRIL 27, 2022

The post Enhertu gets breakthrough tag in HER2-low breast cancer appeared first on. .” AZ and Daiichi Sankyo have previously been awarded breakthrough designations for Enhertu as a treatment for second- and later-line therapy of HER2-positive breast cancer, as well as HER2-positive non-small cell lung cancer (NSCLC) and gastric cancer.

Sales

Sales Drugs Antibody Licensing

Orchard’s MLD gene therapy becomes costliest US medicine

pharmaphorum

MARCH 20, 2024

Orchard Therapeutics has revealed the US price of Lenmeldy, its gene therapy for rare disease MLD, placing a $4.25m price tag on the one-shot treatment

Gene Therapy

Gene Therapy Gene Medicine

NSF tags FAU researcher for post-quantum cryptography in NextG networks

Scienmag

MAY 26, 2022

The National Science Foundation (NSF) recently announced a new investment of more than $37 million aimed to develop intelligent, resilient and reliable next generation – of NextG – Networks.

Research

Research Development

Pear claims breakthrough tag for alcohol use disorder DTx

pharmaphorum

NOVEMBER 24, 2021

The post Pear claims breakthrough tag for alcohol use disorder DTx appeared first on. The company – which is in the process of going public via a merger with special purpose acquisition company (SPAC) Thimble Point – has predicted it will make around $4 million from its DTx portfolio in full-year 2021. Photo by thom masat on Unsplash.

Production

Production Development Trials

#news #biotech Ardelyx stock craters as FDA slaps biotech with dreaded ‘deficiencies’ tag on kidney drug

BioTech 365

JULY 20, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Ardelyx stock craters as FDA slaps biotech with dreaded ‘deficiencies’ tag on kidney drug.Ardelyx stock craters as FDA slaps biotech with dreaded ‘deficiencies’ tag on kidney drug … Continue reading →

Drugs

Drugs Research

Michigan State University’s Axia Institute Announces the Launch of Axia Lab™ as its RFID Tag Validation Program

BioTech 365

OCTOBER 7, 2021

Michigan State University’s Axia Institute Announces the Launch of Axia Lab™ as its RFID Tag Validation Program Michigan State University’s Axia Institute Announces the Launch of Axia Lab™ as its RFID Tag Validation Program Global pharmaceutical leader Fresenius Kabi is … Continue reading →

#news #biotech RAP tag: A new protein purification approach

BioTech 365

SEPTEMBER 25, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: RAP tag: A new protein purification approach.Whether it’s our diets, building strength, or as part of medical advancements, it is no secret that proteins form an important … Continue reading →

Protein

Protein Research

Can gene therapies for haemophilia defend their high price tags?

Biogen’s Alzheimer’s drug: What’s the price tag on hope?

Trending Sources

Lutris wins FDA orphan drug tag for EGFR inhibitor-induced rash candidate

Disc wins orphan drug tag for rare blood cancer

Moderna now in the crosshairs of Sens. Warren, Welch over price tag for COVID vaccine

Sporting a $3.5M price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA finish line

Vicore’s lung disease digital therapy gets breakthrough tag

FDA puts breakthrough tag on Roche, Lilly Alzheimer’s test

Cybin bags breakthrough tag for psychedelic depression drug

‘Click’ chemistry used to tag proteins made by cancer cells

Lilly wades deeper into copycat insulin market, scoring interchangeability tag for Lantus biosim

MindMed gets FDA breakthrough tag for LSD in anxiety

Ultimovacs bags orphan drug tag for mesothelioma vaccine

Click bags FDA breakthrough tag for schizophrenia DTx

Revolo advances plans to tackle eosinophilic esophagitis with orphan drug tag

Sanders takes aim at US drug prices of Novo’s Ozempic and Wegovy

Grünenthal’s non-opioid pain drug gets breakthrough tag

Harmony wins orphan drug tag for pitolisant to treat Prader-Willi syndrome

With European approval secured, BioMarin puts roughly $1.5M price tag on hemophilia gene therapy

Novel tag provides first detailed look into goliath grouper behavior

NorthSea Therapeutics bags FDA rare paediatric disease tag for NASH drug

NeoImmuneTech bags orphan drug tag for IL-7 acute radiation syndrome treatment

#news #biotech Bluefin tuna tagged for the first time in UK waters with acoustic ‘residency’ tags

Novartis-linked startup launches with technology designed to remove ‘destroy’ tags on helpful proteins

FDA approves Duchenne gene therapy with hefty $3.2m price tag

FDA gives AbbVie’s c-Met lung cancer ADC a breakthrough tag

BioMarin's hemophilia gene therapy could have warranted a record price tag, ICER finds

Genprex’s Reqorsa gene therapy picks up orphan drug designation from FDA for SCLC

Pfizer and Arvinas win UK’s fast track tag for breast cancer drug

Amylyx ALS drug draws criticism over $158,000 price tag

Sanders Launches Probe of ‘Outrageously High Prices’ of Novo’s Ozempic, Wegovy

Orchard sets new gene therapy price tag at $4.25M—the steepest of any drug in the US

Acorn woodpeckers wage days-long battles over vacant territories, radio tag data show

Blue Note leukaemia DTx gets FDA breakthrough tag

FDA gives Daiichi Sankyo’s HER3 drug a breakthrough tag

Applied DNA Receives Purchase Orders Totaling $1.6 Million to Tag Textile Fibers

CRISPR tagging improves accuracy of model cells grown from stem cells

Enhertu gets breakthrough tag in HER2-low breast cancer

Orchard’s MLD gene therapy becomes costliest US medicine

NSF tags FAU researcher for post-quantum cryptography in NextG networks

Pear claims breakthrough tag for alcohol use disorder DTx

#news #biotech Ardelyx stock craters as FDA slaps biotech with dreaded ‘deficiencies’ tag on kidney drug

Michigan State University’s Axia Institute Announces the Launch of Axia Lab™ as its RFID Tag Validation Program

#news #biotech RAP tag: A new protein purification approach

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