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.

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Marketing Gene Sequencing DNA Genome 52

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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Drug Discovery World

APRIL 17, 2024

JMB: There’s no doubt that it’s been a tough environment to raise funds, and the market has only worsened in 2023. DS: You say on your website that your approach is applicable to any disease where increasing protein expression is beneficial. What diseases are you currently targeting? Can you give some examples?

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Gene Expression Gene Genetic Disease RNA 59

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 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

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

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

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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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

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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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

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

Drug Discovery World

FEBRUARY 15, 2024

Whilst the currently published work focuses on the design of mRNA LNPs for more effective vaccines, we also have designed LNP platforms based on similar chemistries for the immune-silent delivery of mRNA to hematopoietic stem cells, macrophages and hepatocytes, thereby extending the utility of our LNP platforms to haematological disorders, rare genetic (..)

Immune Response 52

Immune Response In-Vivo Vaccination Vaccine 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

Drugs 98

Drugs Genetic Disease Gene Therapy Trials 98

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

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

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

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

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

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.

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Gene Editing Gene Genome Genomics 64

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

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

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

Drug Discovery World

JANUARY 30, 2024

Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Lentivirus strategies are one of the most widely used tools in biomedical research and clinical testing, says Stella Vnook, CEO, Likarda. “Dr

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Gene Therapy Manufacturing Gene Clinical Trials 71

Drug Discovery World

AUGUST 28, 2023

PH : This is a relatively new area of biology and it takes many years to get a new drug to the market. Could it be possible to prevent genetic diseases? But we also want to develop autophagy agents for other diseases where the process is implicated, such as fibrotic diseases or even cardiovascular disease.

Genetics 52

Genetics Protein Genetic Disease Clinical Trials 52

Delveinsight

JANUARY 12, 2021

Already in markets in China, BeiGene’s Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to Fc?R Bluebird bio recently announced its plans to split its genetic disease and oncology businesses. R on macrophages. and Europe.

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Licensing Licensing Antibody Gene Therapy 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 66

pharmaphorum

AUGUST 24, 2021

According to the terms of the agreement, Arbor is in line for an undisclosed upfront payment and equity investment, plus milestone payments tied to a maximum of seven programmes and royalties on sales if any reach the market.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

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

XTalks

DECEMBER 29, 2022

This highlights potentially new marketing trends that life science companies may have to start looking into if they haven’t already. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. 4. Social Media Marketing.

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Life Science Gene Therapy Gene Manufacturing 52

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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The Pharma Data

NOVEMBER 29, 2020

PH1 is an ultra-rare genetic disease marked by overproduction of oxalate, which causes deposits of calcium oxalate crystals in the kidneys and urinary tract and can lead to painful and recurrent kidney stones, nephrocalcinosis, progression to kidney failure, and system organ dysfunction. The FDA approved the drug on November 24.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 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

The Pharma Data

NOVEMBER 19, 2020

He has pioneered the establishment of diagnostic evaluation algorithms for children with sensorineural hearing loss and developed a next generation sequencing platform to determine the genetic causes of hearing loss in children. He has 92 peer review articles published with the majority related to hearing loss.

Genetics 52

Genetics Containment Genetic Disease Gene Therapy 52

pharmaphorum

JUNE 28, 2022

“The ASMD community has waited many years for a treatment for this rare and debilitating genetic disease,” said Sanofi’s head of R&D John Reed. The enzyme replacement therapy has also been approved in Japan, and is currently under regulatory review in the US, with a decision by the FDA expected in October.

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Genetic Disease Clinical Trials Clinical Trials Genetics 52

XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.”

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Gene Therapy FDA Approval Gene In-Vivo 98

The Pharma Data

DECEMBER 9, 2020

Rocket intends to use the net proceeds from this offering to further fund the development of its pipeline of gene therapies for rare diseases, including filing for marketing authorization for RP-L201 in the United States and Europe, accelerating the buildout of in-house manufacturing capabilities, and for general corporate purposes.

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Gene Therapy Genetics Genetic Disease Sales 40