XTalks

JANUARY 5, 2022

In the first episode of 2022, Ayesha provided updates on Omicron and COVID-19 vaccine boosters. These include the continued promise of RNA-based vaccines as well as therapeutics based on RNA editing approaches for the treatment of various conditions including rare genetic diseases.

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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.

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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.

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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. They are the smallest type of programmable nuclease.

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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.

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Roots Analysis

FEBRUARY 26, 2024

Literary and archaeological evidences suggest that diseases have plagued living organisms since the beginning of time. 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. The science of vaccinology has evolved significantly since Jenner’s discovery.

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Medicine Genetics Genetic Engineering Genetic Disease 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.

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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.

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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.

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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.

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XTalks

FEBRUARY 24, 2025

As an RNA ligand-conjugated antisense (LICA) prophylactic, donidalorsen aims to bring long-term control over the frequency and severity of HAE attacks.

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Trials RNA Allergies Drugs 59

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.

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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.

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Life Science Gene Therapy Gene Manufacturing 52

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.,

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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.

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Trials Antibody Gene Therapy Gene 52

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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XTalks

JANUARY 6, 2025

Additionally, CRISPR genome-wide screening holds great potential for identifying key disease-associated genes and uncovering novel therapeutic targets. Furthermore, CRISPR/Cas9 presents a promising avenue for overcoming genetic diseases in the near future.

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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.

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