World of DTC Marketing

JANUARY 24, 2022

The amount of money required to gain approval of a new drug has been hotly debated, but it also costs a hell of a lot of money to launch a drug in today’s market. A study in 2020 estimated that the median cost of getting a new drug into the market was $985 million, and the average price was $1.3

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Life Science Drugs Development Genetic Disease 225

BioTech 365

DECEMBER 3, 2020

RNAi Technology Market- by Application (Cancer, Infectious Diseases, Ophthalmic Diseases, Genetic Diseases, and Other Diseases) and by Region – Global Forecasts 2020-2024 | Technavio RNAi Technology Market- by Application (Cancer, Infectious Diseases, Ophthalmic Diseases, Genetic Diseases, and Other Diseases) and … Continue reading (..)

Genetic Disease 40

Genetic Disease Genetics Marketing 40

Pharmaceutical Technology

JULY 29, 2022

The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. Drugs for dialysis, amino acid supplements, and drugs which convert blood ammonia are marketed right now as the go-to urea cycle disorder treatments.

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RNA Gene Expression Gene Gene Therapy 245

Pharmaceutical Technology

AUGUST 17, 2022

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.

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RNA Vaccination Vaccine In-Vivo 147

The Pharma Data

JANUARY 26, 2021

Orthopedics Market: Overview. Strides in the orthopedics market have been primarily driven by changing demographic profile of patient populations and advances made in orthopedic surgeries. Concomitant research in biomaterials, biomechanics, electrophysiology, and molecular biology has expanded the horizon of the orthopedics market.

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Marketing Development Genetic Disease Research 52

BioTech 365

OCTOBER 5, 2020

Diamond Pharma Services has provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight, leading to the Marketing Authorisation Application for LUMEVOQ LUMEVOQ is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary … Continue reading →

Gene Therapy 40

Gene Therapy Gene Genetic Disease Marketing 40

BioPharma Reporter

JULY 21, 2022

US biotech, CAMP4 Therapeutics, has secured US$100m in a Series B financing round that it says will be used to accelerate expansion of its regulatory RNA (regRNA) platform.

Genetic Disease 52

Genetic Disease RNA Genetics Regulation 52

XTalks

MARCH 19, 2025

This drop likely reflects market concerns over the therapys safety profile. However, since the news broke, Sareptas shares fell about 26.3%, closing at $74.55 on March 18, 2025, where more than 8.2 million shares had exchanged hands in the first few hours of trading according to Reuters.

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Gene Therapy Gene Genetics Clinical Trials 80

Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases.

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Gene Editing Gene In-Vivo Gene Therapy 147

XTalks

JANUARY 27, 2023

The website brings awareness for facial angiofibroma, an aspect of the rare genetic disease tuberous sclerosis complex (TSC). This includes a greater focus on digital campaigns and diversifying marketing campaigns with race and ethnicity insights.

Life Science 98

Life Science Genetic Disease FDA Approval Marketing 98

Roots Analysis

FEBRUARY 21, 2022

The various advantages of NGS are briefly described below: Next Generation Sequencing (NGS) Library Preparation Kits Market – Roots Analysis. Next Generation Sequencing (NGS) Library Preparation Kits Market – Roots Analysis. Next Generation Sequencing (NGS) Library Preparation Kits Market – Roots Analysis.

Marketing 52

Marketing Gene Sequencing DNA Genome 52

pharmaphorum

OCTOBER 7, 2020

Axovant has said it plans to continue developing its Parkinson’s Disease gene therapy after reporting supportive data from a small cohort of patients from a phase 2 trial. Parkinson’s is not a true genetic disease, although certain genes have been linked to an increased likelihood of onset.

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Gene Therapy Gene Genetic Disease Trials 119

Outsourcing Pharma

OCTOBER 22, 2024

The European Medicines Agencyâs Committee for Medicinal Products for Human Use (CHMP) has once again snubbed Translarna (ataluren), PTC Therapeuticsâ treatment for the genetic disease Duchenne muscular dystrophy (DMD).

Genetic Disease 52

Genetic Disease Genetics Medicine Production 52

pharmaphorum

AUGUST 12, 2020

The enzyme replacement therapy (ERT) – also known as PRX-102 – has been granted a priority review by the US regulator, and is the top prospect in Chiesi’s recently formed rare diseases division. Market research firm Optima Insights has predicted that sales of Fabry disease drugs will more than double from around $1.8

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Drugs Genetic Disease Gene Therapy Trials 98

Pharmaceutical Technology

MARCH 1, 2023

The company is pioneering tRNA-based therapeutics for modulating mRNA function and curing several genetic diseases. It is exploring the use of its new Enhancer tRNA, mRNA Amplifier, and Suppressor tRNA platforms in heart disease, muscular dystrophies, and neurological disorders.

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Development Gene Therapy Genetics Genetic Disease 130

Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences president and CEO Bo Cumbo said: “This collaboration with Phlox Therapeutics is an exciting opportunity to work with a company that shares our commitment and innovative approach to bringing transformative therapies to patients with rare genetic diseases.

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RNA Gene Therapy Genetics Genetic Disease 130

Pharmaceutical Technology

JANUARY 10, 2023

“We are excited to combine these unique platforms to develop next-generation genetic therapies with the potential to transform the lives of patients suffering from a wide range of debilitating genetic diseases.”. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Editing Gene Gene Therapy Genetics 130

pharmaphorum

NOVEMBER 27, 2020

CF is a rare, life-shortening genetic disease that affects the lungs, liver, gastrointestinal tract, sinuses, sweat glands, pancreas and reproductive tract. It is caused by a defective or missing CFTR gene, and there are around 75,000 people worldwide with the disease.

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Gene Genetic Disease FDA Approval Genetics 134

The Pharma Data

JANUARY 17, 2021

But that number doesn’t quite reflect the company’s likely market valuation , which is expected to be between $9 billion and $12 billion. Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases.

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In-Vivo Marketing Engineer Genetics 52

pharmaphorum

DECEMBER 8, 2021

The post Acadia closer to bringing first Rett syndrome drug to market appeared first on. Another late-stage candidate – Newron Pharma’s sarizotan – failed a late-stage trial and was axed by its developer last year.

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Marketing Drugs Life Science Trials 59

XTalks

FEBRUARY 25, 2025

XTALKS WEBINAR: Key Strategies for Successful Orphan Drug Launch in Europe Live and On-Demand: Friday, March 28, 2025, at 1pm EDT (5pm GMT) Register for this free webinar to learn how to optimize pricing and reimbursement strategies and leverage patient advocacy for a successful market launch of orphan drugs.

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FDA Approval Gene Therapy Gene Genetics 59

pharmaphorum

OCTOBER 4, 2020

“CALD is a devastating disease, often marked by rapid neurodegeneration, the development of major functional disabilities, and eventual death,” said Gary Fortin, head of severe genetic disease programmes at Bluebird. “If

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Gene Therapy Gene Genetic Disease Protein 98

pharmaphorum

NOVEMBER 19, 2021

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company.

Drugs 98

Drugs Gene Therapy Genetic Disease Gene 98

pharmaphorum

SEPTEMBER 17, 2020

There are two antibodies already on the market that target PCSK9 – Amgen’s Repatha (evolocumab) and Sanofi/Regeneron’s Repatha (alirocumab) – but these require subcutaneous injection every two to four weeks.

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Drugs Antibody Sales Branding 65

pharmaphorum

JANUARY 10, 2022

The first mRNA-based vaccines were brought onto the market in 2020, with Pfizer/BioNtech and Moderna receiving emergency use authorisation from the US Food and Drug Administration (FDA) for the prevention of COVID-19. The breakthrough was hailed as a revolution for disease treatment and particularly a major success against the pandemic.

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RNA Vaccine Vaccination Pharma Companies 111

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

pharmaphorum

JANUARY 9, 2023

Along with Zolgensma – which made its debut in 2019 – Biogen has had approval to market its antisense-based therapy Spinraza (nusinersen) since 2016, while Roche got a green light for its orally-administered therapy Evrysdi (risdiplam) in 2020. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1

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Gene Therapy Genetics Gene Clinical Trials 119

pharmaphorum

FEBRUARY 17, 2022

The drug – which costs $125,000 at US list prices – can be used either alone or in combination with hydroxyurea, a well-established therapy for the disease.

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Drugs Gene Therapy Gene Editing Gene 59

XTalks

FEBRUARY 24, 2025

Despite limited markets for the rare disease drugs, analysts predict that Wainua could reach $700 million in peak sales in just the ATTRv-PN indication. In 2024, Qalsody brought in $32 million, and Wainua generated $85 million in global sales , with $4 million and $20 million in royalties for Ionis, respectively.

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Trials RNA Allergies Drugs 59

XTalks

MAY 17, 2023

In the past several years, gene therapies have finally been hitting the market after decades of setbacks and uncertainties. In 2022, the FDA approved four new gene therapies, and more than a handful are looking to get approval this year for diseases including Duchenne muscular dystrophy (DMD), sickle cell disease (SCD) and hemophilia A.

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Gene Therapy Clinical Trials Clinical Trials Gene 98

The Pharma Data

NOVEMBER 27, 2020

Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity. BOSTON, Nov.

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FDA Approval Genetic Disease Genetics Clinical Trials 52

pharmaphorum

DECEMBER 9, 2021

An FDA advisory committee has delivered a blow to Reata Pharma, after voting unanimously that the drugmaker’s data on bardoxolone – a drug for kidney disease – did not show it is effective. ” Trading in Reata’s shares was halted ahead of the meeting, but the stock was down 38% pre-market this morning. .”

Drugs 52

Drugs Clinical Trials Clinical Trials Genetic Disease 52

pharmaphorum

JANUARY 11, 2021

US biotech bluebird bio has announced plans to split into two this year, with a separate oncology business spinning off as the company prepares to bring its products to market. Meanwhile the as-yet unnamed oncology company will spin off under the leadership of bluebird’s current chief executive Nick Leschly.

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Gene Therapy Gene Genetic Disease Manufacturing 65

XTalks

FEBRUARY 28, 2022

In the US, 25 to 30 million Americans, or one in ten, are living with a rare disease. Are Most Rare Diseases Genetic? While 72 percent of rare diseases are genetic, not all are. Rare genetic diseases can be caused by either somatic or germline genetic changes.

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Clinical Trials Clinical Trials Genetics Trials 98

Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years.

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Gene Therapy Gene Packaging Packaging 130

pharmaphorum

OCTOBER 4, 2022

Through Alexion , which AstraZeneca acquired in 2021, the company will gain access to LogicBio’s technology platforms for the delivery and insertion of genes to address genetic diseases, as well as a platform designed to improve viral vector manufacturing processes. per share, a rare 660% premium on LogicBio’s share price.

Gene Editing 64

Gene Editing Gene Genome Genomics 64

pharmaphorum

AUGUST 1, 2022

The US biotech – which already has three antisense drugs for DMD on the market – had said earlier it was planning to delay the application into 2023, so the new announcement marks an acceleration of its plans.

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Gene Therapy Gene Genetics Trials 105