pharmaphorum

OCTOBER 6, 2020

The vaccine is based on different technology from AZ’s rival: while AZ’s uses a weakened and genetically modified cold virus to prime the immune system, BNT162b2 is based on a string of RNA. According to latest figures from the World Health Organization, there are 42 potential COVID-19 vaccines in clinical development.

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Vaccine Vaccination RNA Immune Response 59

pharmaphorum

NOVEMBER 18, 2021

The two companies have been working together since 2019 to develop multiple RNA interference (RNAi) candidates for disorders such as non-alcoholic steatohepatitis (NASH), type 2 diabetes, obesity and rare diseases. billion in cash. If approved, it could become a $500 million-a-year product, according to the company.

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Gene Silencing RNA Protein Gene 52

pharmaphorum

MARCH 29, 2022

“C9orf72-associated ALS is a complex genetic form of ALS and there are multiple mechanisms by which the scientific community believes the C9orf72 gene causes disease. Based on the results from this study, Biogen and Ionis have confirmed that the BIIB078 clinical development programme will be discontinued.

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Trials RNA Containment Genetics 49

The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.

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Protein RNA Production Genetics 52

The Pharma Data

MARCH 28, 2022

Based on these results, the BIIB078 clinical development program will be discontinued, including its ongoing open-label extension study. “We Vice President and Head of the Neuromuscular Development Unit at Biogen. About Ionis Pharmaceuticals, Inc.

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Drugs Genetics RNA Development 52

The Pharma Data

DECEMBER 8, 2020

Now poised to advance a robust therapeutics pipeline to clinical development, Nuance will use the funds for ongoing R&D of existing products and business development of potential new assets. Laying down a new track for RNA processing, Remix launched with $81 million in financing. Sigilon Therapeutics .

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RNA Antibody Life Science Protein 52

pharmaphorum

NOVEMBER 23, 2021

The bolt-on deal comes – which includes an upfront payment of $120 million and up to $910 million in milestones – covers rights to ARO-HSD, Arrowhead’s RNA interference drug targeting an enzyme called HSD17B13 that seems to contribute to the pathology of NASH and other liver diseases.

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Sales Drugs Trials RNA 52

The Pharma Data

DECEMBER 13, 2020

Dr. Kehua Fan has over 15 years of Business Development, Clinical Development of innovative drugs and other healthcare industry experience with MNCs and biotech companies. He received his B.S. degree from Henan Normal University, M.S. degree from Chinese Academy of Sciences and Ph.D. from University of New Hampshire.

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The Pharma Data

OCTOBER 19, 2020

“Molecular profiling is a powerful tool that Winship’s physicians can utilize to optimize cancer treatments by accessing relevant genetic information that can have a meaningful impact on patient care at a personalized level.” Brille , Vice Chairman of Caris Life Sciences.

Life Science 52

Life Science Clinical Trials Clinical Trials RNA 52

Camargo

JULY 27, 2021

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.

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Gene Therapy Gene Gene Editing Genetics 238

XTalks

FEBRUARY 24, 2025

Carlsbad, California-based Ionis Pharmaceuticals announced it will be sharing promising new trial data for its RNA-targeted prophylactic treatment donidalorsen for hereditary angioedema (HAE) at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress in San Diego, California.

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Trials RNA Allergies Drugs 59

The Pharma Data

SEPTEMBER 22, 2020

Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. About Spinal Muscular Atrophy SMA is the leading genetic cause of infant death.

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Clinical Development Gene Therapy Development Gene 52

pharmaphorum

NOVEMBER 9, 2020

The vaccine is based on a strand of messenger RNA – a short segment of genetical material that codes for the “Spike” protein found on the surface of the coronavirus. Around 42% of global participants and 30% of U.S. participants have racially and ethnically diverse backgrounds.

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Vaccine Vaccination Clinical Trials Clinical Trials 98

XTalks

OCTOBER 26, 2023

She has 23 years of experience working with clients to move vaccine candidates through the clinical development pipeline, including regulatory submission. The Flu versus COVID-19: Virology and Vaccines SARS-CoV-2 versus Influenza Virology Viruses contain genetic material that can either be RNA or DNA.

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Vaccine Vaccination RNA Manufacturing 105

pharmaphorum

DECEMBER 23, 2020

In summer, Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder Oxlumo was approved in the European Union and the US. It was the third approval from Alnylam’s pipeline of RNA interference therapeutics to make it to market.

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Marketing Gene Gene Silencing Drugs 96

Pharmaceutical Technology

MARCH 1, 2023

Further, the acceptance of new mRNA vaccines has rejuvenated activity within previously established categories of RNA therapeutics including lifesaving antisense technologies. These include antisense oligonucleotides (ASO), RNA interference (RNAi), and RNA aptamers.

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RNA Vaccine Vaccination Genetics 147

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. The safety and efficacy of Oxlumo are also being evaluated in the ongoing ILLUMINATE-C Phase 3 clinical trial in patients of all ages with advanced PH1, including patients on dialysis. Oxlumo should be administered by a healthcare professional.

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FDA Approval Production RNA Medicine 52

Roots Analysis

AUGUST 13, 2024

Messenger ribonucleic acid (mRNA) is a type of single-stranded ribonucleic acid (RNA), which helps in transferring genetic information in order to produce proteins. The success of mRNA-based therapeutics and vaccines has increased the research activity in novel / next generation RNA therapeutics market.

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Vaccine Vaccination Manufacturing Protein 40

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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RNA Genetics Genetic Disease Medicine 105

Delveinsight

AUGUST 5, 2021

Sanofi is betting the genetic technology behind the fast development of two highly effective coronavirus shots last year will result in vaccines for other viruses as well as drugs for diseases of the lung and liver, announcing a deal to acquire a research partner Translate Bio for USD 3.2 Sanofi bets USD 3.2

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Vaccine Vaccination Gene Therapy Development 98

Worldwide Clinical Trials

MAY 30, 2024

Differences in regulatory sentiments and industry sponsors’ subsequent clinical development strategies historically restricted access based on geography. An area of interest has been deciphering ALS’s genetic underpinnings and delivering functional copies of dysfunctional genes to the patient.

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Gene Clinical Trials Clinical Trials Gene Therapy 204

The Pharma Data

SEPTEMBER 8, 2020

Together, these nine companies have collectively developed more than 70 novel vaccines that have helped to eradicate some of the world’s most complex and deadly public health threats, underscoring their experience in clinical development and regulatory rigor, as well as their longstanding commitments to patient safety and public health.

Vaccine 52

Vaccine Vaccination Containment Clinical Trials 52

The Pharma Data

SEPTEMBER 8, 2020

Together, these nine companies have collectively developed more than 70 novel vaccines that have helped to eradicate some of the world’s most complex and deadly public health threats, underscoring their experience in clinical development and regulatory rigor, as well as their longstanding commitments to patient safety and public health.

Vaccine 52

Vaccine Vaccination Containment Clinical Trials 52

XTalks

JANUARY 6, 2025

From leveraging artificial intelligence (AI) to streamline diagnostics and treatments to exploring the untapped potential of RNA-based therapeutics, biotechnology is shaping the future of healthcare and beyond. As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas.

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Gene Therapy RNA Gene Editing Gene 111

XTalks

DECEMBER 24, 2020

However, for virologists like Lori Frappier, PhD, professor in the department of molecular genetics at the University of Toronto, the outbreak of SARS-CoV-2 was less of a surprise and more of an inevitability. Omer maintains that the solution to this challenge is something that’s already in practice by developers of the mRNA vaccines.

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Life Science Vaccine Vaccination Gene 111

XTalks

SEPTEMBER 14, 2020

The company’s candidate vaccine, mRNA-1273, is a synthetic messenger RNA that encodes the stabilized SARS-CoV-2 spike protein. Unlike other candidate vaccines, CoronaVac is an inactivated whole virus vaccine developed through the same process as has long been used to make influenza vaccines, among many others.

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