Drug Discovery World

JANUARY 11, 2024

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. We believe it can now do the same for rare diseases.”

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Research Gene Therapy RNA Gene 59

Pharmaceutical Technology

MAY 8, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. The company's lead asset involving this technology is isaralgagene civaparvovec (ST 920).

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Genetics DNA Genetic Engineering Antibody 130

Roots Analysis

FEBRUARY 26, 2024

Whether it was a common cold or a genetic disease, prayers and sacrifices to the gods were the only hope of a remedy at such times. It is possible that the legend of Big Foot may be based on an unfortunate person suffering from elephantiasis or gigantism or some other genetic predisposition that would have rendered him abnormal.

Genetics 52

Genetics Medicine Genetic Engineering Genetic Disease 52

Drug Discovery World

MAY 11, 2023

In its pipeline, Imophoron is working on ADDomer based vaccines for three diseases, respiratory syncytial virus (RSV), Covid-19 and Chikungunya. Its clinical pipeline includes a TRPML1 activator, Beclin activator neuro, Beclin activator peripheral, and GTPase activator, the first three of which are preparing for Phase Ia/b trials.

Life Science 52

Life Science Genetics Vaccination Vaccine 52

Delveinsight

JANUARY 12, 2021

This will have an advantage over the multiple vaccines that have been getting EUA, which are not suitable for immunocompromised patients. Bluebird bio recently announced its plans to split its genetic disease and oncology businesses. BeiGene, Novartis partner to develop cancer drug Tislelizumab, an Anti-PD-1 Antibody.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

Delveinsight

AUGUST 17, 2021

Additional 3rd dosage of Pfizer-BioNTech and Moderna COVID-19 Vaccine approved for immunocompromised populations. The Pfizer-BioNTech COVID-19 Vaccine is sanctioned for emergency use in individuals of 12 years and above whereas the Moderna COVID-19 Vaccine is approved for emergency use in individuals of 18 years and above.

FDA Approval 98

FDA Approval Medicine Vaccination Vaccine 98

Pharma Mirror

APRIL 21, 2024

With the rapid development of biotechnology and molecular medicine, the introduction of mRNA as a vaccine or therapeutic agent enables the production of almost any desired functional protein/peptide within the human body.

In-Vitro 130

In-Vitro Drugs Genetic Disease RNA 130

The Pharma Data

JANUARY 17, 2021

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. The Wall Street Journal notes that Moderna, currently one of two companies to have an authorized COVID-19 vaccine in the U.S.,

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

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. vaccine, mRNA-1273.815.

Genetics 111

Genetics Vaccination Vaccine DNA 111

pharmaphorum

JANUARY 27, 2023

“We’ve almost got an opportunity to take it to the next level now, because we saw this with the collaboration with the COVID vaccine development and roll-out of the vaccination programme. As an overall term, ‘patient centricity’ itself has undergone a transformation, as well.

Clinical Trials 111

Clinical Trials Clinical Trials Trials Development 111

Drug Discovery World

FEBRUARY 28, 2023

mRNA technology helped us to vaccinate the world against SARS-CoV-2. Approximately 7,000 rare and ultra-rare diseases have been described, and yet, 90% have no approved therapeutic options 1. Therefore, the risk for off-target insertional mutagenesis is greatly reduced.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 52

The Pharma Data

DECEMBER 20, 2020

Food and Drug Administration (FDA) is plenty busy with COVID-19 vaccine Emergency Use Authorizations (EUAs) this month, but they’re also wrapping up the year with a few PDUFA dates for other therapies. CF is a rare, life-shortening genetic disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.

In-Vitro 52

In-Vitro FDA Approval Manufacturing Licensing 52

XTalks

AUGUST 16, 2021

The US Food and Drug Administration (FDA) has amended its emergency use authorizations (EUA) for both the Pfizer and Moderna COVID-19 vaccines to incorporate a third dose for immunocompromised individuals. A small study from John Hopkins University has shown that a third dose of a COVID-19 vaccine could indeed help immunocompromised patients.

Vaccination 104

Vaccination Vaccine Antibody Immune Response 104

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. Whilst research into mRNA has been ongoing for decade, interest has sparked in recent years and research has begun to expand into areas of vaccinology, infectious diseases and other indications.

RNA 52

RNA Protein Vaccination Vaccine 52

XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

RNA 105

RNA Genetics Genetic Disease Medicine 105

pharmaphorum

JANUARY 10, 2022

The arrival of COVID-19 brought precious few positives, but one that emerged for the pharmaceutical industry was the emergence and validation of mRNA vaccine technology to prevent disease. Pfizer already has a head start on competitors in the field, having achieved the first US FDA approval for an mRNA vaccine, alongside BioNTech.

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RNA Vaccination Vaccine Pharma Companies 111

XTalks

DECEMBER 29, 2022

Given their precise targeting, gene therapies have largely been focused on oncology indications followed by rare genetic diseases. As such, most gene therapies in late-stage clinical trials are in oncology or rare disease indications.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

XTalks

DECEMBER 22, 2021

The RNA Revolution: From mRNA Vaccines to RNA Editing. The power of its real-world application was demonstrated by the resounding success of the mRNA COVID-19 vaccines. The mRNA COVID-19 vaccines have showcased the speed, versatility, scalability and flexibility that mRNA-based interventions offer.

Life Science 98

Life Science RNA Vaccination Vaccine 98

The Pharma Data

DECEMBER 27, 2020

CureVac and the University Medical Center of Mainz launched a Phase III trial of CVnCoV, their COVID-19 vaccine candidate, in healthcare workers. The study will evaluate the safety and immunogenicity of the vaccine in a two-dose schedule of 12 micrograms. Most Read Today. Source link.

Trials 52

Trials Antibody Gene Therapy Clinical Trials 52

The Pharma Data

OCTOBER 15, 2020

Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

The Pharma Data

NOVEMBER 25, 2020

The virus was also sequenced and hundreds of vaccine projects were initiated. In Russia and China, vaccines are already being distributed to front-line workers and manufacturing is ramping up for broader distribution. In the west, we are just weeks away from seeing the first coronavirus vaccine receive Emergency Use Authorization.

Vaccination 52

Vaccination Vaccine Genetic Disease Genetics 52

Drug Discovery World

FEBRUARY 22, 2024

For diseases as serious and debilitating for patients and their families as leukodystrophies, learning about such advances in knowledge carries a great deal of hope, which IRCM warmly welcomes,” adds Dr Jean-François Côté, the IRCM’s President and Scientific Director.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

Pharmaceutical Technology

APRIL 24, 2023

Since then, the field of nanomedicine has steadily progressed to reach high points such as the successful use of nanotechnology to deliver messenger RNA (mRNA)-based Covid-19 vaccines. In the case of most mRNA vaccines, a lipid nanoparticle-based approach was chosen due to its ability to protect the mRNA in the body and prevent degradation.

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Drug Delivery Vaccination Vaccine Research 147