The Pharma Data

NOVEMBER 30, 2020

(Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today provided an update on its COVID-19 vaccine program. About NVX-CoV2373 NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of SARS-CoV-2, the virus that causes COVID-19 disease.

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Clinical Development Vaccination Vaccine Development 52

pharmaphorum

JULY 5, 2022

It can be caused by exposure to loud noises – including prolonged use of earbuds at high volumes – as well as cancer treatment, illnesses, genetic mutations, or aging. SNHL is the most common form of hearing loss, account for more than 90% of cases and affecting millions of people worldwide. An estimated 1.57 An estimated 1.57

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In-Vivo Bioinformatics Genetics Licensing 110

pharmaphorum

SEPTEMBER 14, 2020

It uses a weakened, non-replicating, version of a common cold virus (adenovirus) that causes infections in chimpanzees and contains the genetic material of the SARS-CoV-2 coronavirus spike protein.

Vaccination 104

Vaccination Vaccine Trials Protein 104

pharmaphorum

OCTOBER 5, 2022

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. billion deal in 2018. The aim will be to identify promising candidates in areas of “unmet patient need.”

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RNA Genetics Dermatology Antibody 105

pharmaphorum

SEPTEMBER 12, 2022

Everyone’s genetic makeup differs, and each person’s cancer experience is unique to them – how cancer develops, how fast it spreads, which drugs it responds to, and more. Furthermore, each cancer has various gene changes – also referred to as the genetic makeup of cancer. Tailored oncology.

Genome 105

Genome Genomics Clinical Trials Clinical Trials 105

XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. Related: Red Meat Allergy Test Gets FDA Clearance. “We

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Clinical Trials Clinical Trials Vaccination Vaccine 75

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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The Pharma Data

DECEMBER 13, 2020

As part of the collaboration, Hummingbird will be leveraging Tempus’ AI-enabled platform and proprietary data, as well as joining its TIME Trial® Network, for rapid identification, site activation and efficient enrollment of cancer patients who have NRG1 fusions and meet eligibility criteria for HMBD-001 clinical trials.

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pharmaphorum

SEPTEMBER 1, 2020

The vaccine is still the frontrunner in a list of 30 COVID-19 vaccines in clinical development maintained by the World Health Organization. The virus contains genetic material of the SARS-CoV-2 spike protein, priming the immune system to attack the SARS-CoV-2 coronavirus if it later infects the body.

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Vaccination Vaccine Trials Immune Response 69

The Pharma Data

OCTOBER 14, 2020

Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases.

Protein 52

Protein RNA Production Genetics 52

The Pharma Data

JULY 6, 2021

PGRN is a key regulator of immune activity in the brain with genetic links to multiple neurodegenerative disorders, making it one of the most attractive genetically validated targets for the development of new immuno-neurology treatments. ” Arnon Rosenthal, Ph.D.,

Genetics 52

Genetics Gene Antibody Development 52

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.

Vaccination 59

Vaccination Vaccine RNA Immune Response 59

pharmaphorum

NOVEMBER 18, 2021

RNAi uses small RNA molecules to bind to messenger RNA, which is responsible for synthesising proteins, and either destroy it or recruit other cellular factors that regulate its activity. Once in the blood, the GalXC molecules travel to the liver, where they enter hepatocyte cells and can switch protein production on or off.

Gene Silencing 52

Gene Silencing RNA Protein Gene 52

The Pharma Data

NOVEMBER 9, 2020

Currently in late-phase clinical development, NVXCoV2373 is a stable, prefusion protein made using Novavax’ nanoparticle technology and includes its proprietary MatrixM adjuvant. NVX-CoV2373 is a vaccine candidate engineered from the genetic sequence of SARS-CoV-2, the virus that causes COVID-19 disease.

Vaccination 52

Vaccination Vaccine Clinical Trials Clinical Trials 52

The Pharma Data

MAY 31, 2022

More than 5,000 patients have now been treated worldwide with Evrysdi in clinical trials, compassionate use or real-world settings. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. Food and Drug Administration in 2017.

FDA Approval 52

FDA Approval Protein In-Vitro Medicine 52

The Pharma Data

APRIL 29, 2022

Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. About Evrysdi® (risdiplam) Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency.

Protein 52

Protein In-Vitro Medicine Development 52

The Pharma Data

OCTOBER 27, 2020

AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. The company’s lead program is aimed at Friedrich’s Ataxia, a rare inherited genetic disease that causes cardiac and central nervous system dysfunction. Be Biopharma .

Gene 52

Gene Gene Therapy Medicine Development 52

XTalks

JUNE 9, 2022

What happens when the liver is unable to process bad cholesterol due to genetic factors? It is one of the common genetic causes of premature coronary heart disease. When a child born from parents with FH genes, inherits one FH gene from each parent, they might develop homozygous familial hypercholesterolemia (HoFH).

Trials 52

Trials Antibody Genetics Gene 52

pharmaphorum

NOVEMBER 23, 2021

GSK’s head of research, John Lepore, said there is “compelling genetic evidence” showing that reduced HSD17B13 function confers protection to the liver from inflammatory injury that is a characteristic of NASH.

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Sales Drugs Trials RNA 52

XTalks

FEBRUARY 24, 2023

Lunsumio is a CD20xCD3 T-cell engaging bispecific antibody that works by targeting the protein CD20 on the surface of B cells and CD3 on the surface of T cells. Every year, over 100,000 people are diagnosed with FL worldwide and around 13,000 of these new cases occur in the US.

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Drugs FDA Approval Antibody Medicine 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. Through its Shielded Living Therapeutics platform, the company is developing functional cures for chronic diseases.

RNA 52

RNA Antibody Life Science Protein 52

The Pharma Data

OCTOBER 26, 2020

AR-711 exhibits high affinity for SARS-CoV-2 spike protein, approximately 10-fold or higher than mAb candidates currently in late stage clinical testing. AR-301 is a fully human IgG1 mAb currently in Phase 3 clinical development targeting gram-positive Staphylococcus aureus ( S. AR-301 (VAP). AR-101 (HAP).

Antibody 52

Antibody Genetic Engineering Development Clinical Trials 52

Delveinsight

AUGUST 20, 2020

The team successfully protected HILOs from the immune system without genetic manipulation. Nipocalimab is in clinical development in myasthenia gravis , warm autoimmune hemolytic anemia (wAIHA), and hemolytic disease of the fetus and newborn indications. J&J has agreed to pay USD 52.50

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Gene Therapy Research Gene Antibody 52

The Pharma Data

JANUARY 27, 2021

Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. We are excited to investigate the potential of PBFT02 as a treatment for FTD-GRN as we initiate our clinical development program in the coming months.”.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

JANUARY 20, 2021

” “Dynacure is a leader in rare disease drug development, as evidenced by its lead product candidate DYN101, which is in clinical development for the treatment of Myotubular and Centronuclear Myopathies for which there are no FDA or EMA approved therapeutics,” said Ms. ” Ms. Prior to Merrimack, Ms.

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Life Science Development Genetics Production 40

The Pharma Data

APRIL 8, 2021

Roche will present data from five studies from the EVRYSDI clinical development programme, which was designed to represent a broad spectrum of people living with SMA. The full range of data from Roche’s clinical development programme in neuroscience being presented at 2021 AAN include: Medicine and/or Therapeutic Area.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Antibody 52

The Pharma Data

OCTOBER 30, 2020

To develop REGN-COV2, Regeneron scientists evaluated thousands of fully-human antibodies produced by the company’s VelocImmune ® mice, which have been genetically modified to have a human immune system, as well as antibodies identified from humans who have recovered from COVID-19.

Production 52

Production Antibody Manufacturing Trials 52

Roots Analysis

OCTOBER 30, 2023

These RNA based therapeutics play a crucial role in protein production and regulation of gene functions. Key players engaged in the development of self amplifying RNA based therapeutics include ( established before 2010, in alphabetical order ) Alphavax, GlaxoSmithKline, BioNTech and Pfizer.

RNA 40

RNA Vaccination Vaccine Development 40

Delveinsight

JULY 30, 2020

Adrenoleukodystrophy (ALD/ X-ALD) is a rare, X-linked disorder secondary to a mutation in the ABCD1 gene, which encodes for a peroxisomal membrane protein, resulting in an excessive accumulation of very-long-chain fatty acids (VLCFAs) in the brain and adrenal glands. Adrenoleukodystrophy Pipeline.

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Hormones Marketing Gene Trials 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.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

XTalks

NOVEMBER 26, 2024

This progressive disease occurs when misfolded transthyretin (TTR) proteins form amyloid deposits in the heart, leading to cardiac dysfunction. BridgeBio has been highly active in the genetic diseases space.

Genetic Disease 105

Genetic Disease Genetics Protein Clinical Development 105

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

The Pharma Data

SEPTEMBER 20, 2020

Amgen’s clinical development team and clinical investigators, will discuss Phase 1 data being presented on the Company’s investigational KRAS G12C ?inhibitor 1,2 Targeting the KRAS protein, the most commonly altered family member in solid tumors, has been one of the toughest challenges in cancer research.

Medicine 52

Medicine Production Sales Clinical Trials 52

The Pharma Data

DECEMBER 17, 2020

Senior Vice President and Head of Global Clinical Development at Regeneron and lead author of the publication. patients under an Emergency Use Authorization, and we also continue a robust clinical development program.” “The investigational cocktail is now available to indicated high-risk U.S.

Antibody 52

Antibody Medicine Production Trials 52

The Pharma Data

JUNE 7, 2022

1-2 Lebrikizumab is a novel, monoclonal antibody (mAb) that binds to the interleukin-13 (IL-13) protein with high affinity to specifically prevent the formation of IL-13R?1/IL-4R? These studies are part of the comprehensive clinical development program for lebrikizumab in AD evaluating more than 2,000 patients.

Trials 40

Trials Clinical Trials Clinical Trials Medicine 40

The Pharma Data

OCTOBER 28, 2020

All patients experienced numerical increases in the sub-scale growth scores of expressive and/or receptive communication of the Bayley Scales of Infant and Toddler Development (Bayley-4) domains, and three patients showed improvements in the Observed Reported Communication Ability (ORCA) measure of expressive, receptive, and pragmatic communication.

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Protein Production Development Drugs 40

The Pharma Data

DECEMBER 5, 2020

BCMA (B-cell maturation antigen) is a protein that is typically over-expressed on multiple myeloma cells. REGN5458 and odronextamab are currently under clinical development, and their safety and efficacy have not been evaluated by any regulatory authority. and the world, respectively, in 2020.

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Antibody Production Trials Medicine 40