Pharmaceutical Technology

JULY 29, 2022

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. GlobalData is the parent company of Pharmaceutical Technology.

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RNA Gene Expression Gene Gene Therapy 245

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

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

Pharmaceutical Technology

JANUARY 10, 2023

The multi-year collaboration will focus on discovering precision genetic medicines by developing a new platform for complete gene insertion by delivering a single vector of gene editing and DNA cargo. Beyond the liver, ReCode’s SORT LNP genetic medicines technology enables the delivery to target cells and organs.

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Gene Editing Gene Gene Therapy Genetics 130

XTalks

SEPTEMBER 27, 2024

In a news release published yesterday, Pfizer announced it will voluntarily withdraw its sickle cell disease (SCD) therapy Oxbryta (voxelotor) from all markets worldwide. Pfizer said it has notified regulatory authorities of the market withdrawal. This is a significant turn of events as Pfizer acquired the drug in the $5.4

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Marketing Gene Therapy Sales Drugs 105

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 Vaccine Vaccination In-Vivo 147

Pharmaceutical Technology

JANUARY 12, 2023

Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM). Our pipeline of programmes focused on neuromuscular and cardiac rare disease gives us deep insight into the diverse and complex nature of dilated cardiomyopathy.”.

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RNA Gene Therapy Genetics Genetic Disease 130

XTalks

APRIL 1, 2025

Hemophilia A and B are rare genetic bleeding disorders caused by a deficiency or dysfunction of clotting factors VIII (FVIII) and IX (FIX), respectively. Qfitlia will be entering a small but competitive market. Roches hemophilia A treatment Hemlibra (emicizumab-kxwh) is currently the market leader in the indication.

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FDA Approval RNA Clinical Trials Clinical Trials 52

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

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

XTalks

FEBRUARY 25, 2025

CTX is a rare, progressive genetic disorder caused by mutations in the CYP27A1 gene, which disrupts the livers ability to produce chenodeoxycholic acid, a bile acid. In addition to its clinical promise, Mirums FDA approval of Ctexli has significantly bolstered its market position.

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FDA Approval Gene Therapy Gene Genetics 59

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

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

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 →

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Gene Therapy Gene Genetic Disease Marketing 40

The Pharma Data

NOVEMBER 19, 2020

MD, FAAP, Cincinnati Children’s Hospital, who will discuss genetic forms of hearing loss in children. Dr. John Greinwald is a tenured professor of Otolaryngology and Pediatrics with over 20 years of experience with a focus on the genetic causes and treatment of deafness. To register for the call, please click here.

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Genetics Containment Genetic Disease Gene 52

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

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

pharmaphorum

JANUARY 9, 2023

First introduced in 2020, the global Managed Access Programme (gMAP) has provided Zolgensma (onasemnogene abeparvovec) free of charge to nearly 300 children with the genetic disorder across 36 countries where the therapy has not yet received approval or in which no formal access pathway exists.

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Gene Therapy Genetics Gene Clinical Trials 119

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

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

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

XTalks

FEBRUARY 24, 2025

Specifically, Ionis said donidalorsen was demonstrated to result in sustained reductions in HAE attack rates while providing enhanced disease control. HAE is a rare genetic disorder characterized by unpredictable, recurrent episodes of severe swelling that can affect various parts of the body, including the hand, feet and airways.

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Trials RNA Allergies Drugs 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

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

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

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

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.

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Drugs Gene Therapy Genetic Disease 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

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

XTalks

DECEMBER 13, 2023

Moreover, it has submitted global marketing authorization applications for mRNA-1345, a vaccine designed to prevent RSV-associated lower respiratory tract disease (RSV-LRTD) and acute respiratory disease (ARD), supported by favorable data from a predefined interim analysis. vaccine, mRNA-1273.815.

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Genetics Vaccination Vaccine DNA 111

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. The confounder for both GBT and Novartis’ hopes for their drug could be genetic therapies for SCD, which offer a one-shot treatment for the disease.

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Drugs Gene Therapy Gene Editing Gene 59

Roots Analysis

AUGUST 12, 2024

These diagnostic solutions are essential for detecting and monitoring diseases, identifying genetic abnormalities, and guiding personalized treatment plans. Molecular diagnostic solutions are pivotal across various medical fields, including oncological disorders, infectious diseases, genetic testing, and personalized medicine.

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Genetics Gene Expression RNA DNA 40

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. As per the agreement, Alexion will initiate a cash tender offer through a subsidiary to acquire all outstanding shares of LogicBio for $2.07

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Gene Editing Gene Genome Genomics 64

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

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

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 66

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.

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Gene Editing Gene In-Vivo Clinical Trials 59