Pharmaceutical Technology

FEBRUARY 13, 2023

Gene therapies are an attractive way of targeting the underlying genetic mutations, but traditional approaches cannot be used while targeting mutations for Dravet Syndrome, forcing researchers to develop new ways. Meanwhile, others are researching the link between gene variation and different responses to treatments.

Research 264

Research Gene Gene Therapy Genomics 264

Pharmaceutical Technology

JULY 12, 2022

A protein-based vaccine, NVX-CoV2373 is created from the genetic sequence of the SARS-CoV-2 virus’ initial strain. It is formulated with the company’s saponin-based Matrix-M and also comprises a purified protein antigen.

Vaccination 162

Vaccination Vaccine Protein Pharmacy 162

XTalks

DECEMBER 2, 2021

Natreve, a premium wellness company based in Vancouver, has partnered with Perfect Day to give consumers the first animal-free whey protein powder. The powder is called Mooless and it offers the same taste, texture and nutritional benefits of whey protein, just without the animal components. lactoglobulin protein.

Protein 59

Protein Marketing Production Genetics 59

XTalks

MARCH 28, 2024

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.

FDA Approval 105

FDA Approval Genetics Gene Therapy Gene 105

XTalks

APRIL 1, 2025

Hemophilia A and B are rare genetic bleeding disorders caused by a deficiency or dysfunction of clotting factors VIII (FVIII) and IX (FIX), respectively. Through RNA silencing, it targets the expression of antithrombin, a protein that inhibits blood clotting. Qfitlia will be entering a small but competitive market.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

Pharmaceutical Technology

FEBRUARY 14, 2023

It will also support smaller dose vials development, selection of strain as per the US Food and Drug Administration (FDA) recommendations, and transition to the commercial market. The company stated that the deal would maintain the access of the US public to its vaccine.

Vaccination 162

Vaccination Vaccine Protein Engineer 162

Pharmaceutical Technology

JULY 7, 2022

Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. But scientists have struggled to find effective treatments for many of these diseases since the dawn of modern medicine. A new frontier in cancer research.

Gene Therapy 245

Gene Therapy Gene Medicine Genetics 245

Roots Analysis

JUNE 1, 2022

DNA Cloning Kit Providers – Current Market Landscape. The market is highly fragmented, featuring the presence of both new entrants and established players, based in different geographical regions. DNA Cloning Kits Market. DNA Cloning Reagent Providers – Current Market Landscape. DNA Cloning Reagents Market.

DNA 52

DNA Genetics Reagent Marketing 52

Pharmaceutical Technology

JANUARY 19, 2023

Nuvaxovid is a protein-based vaccine created from the genetic sequence of the initial SARS-CoV-2 virus strain. Novavax president and CEO Stanley Erck said: “We are pleased to collaborate with SK bioscience to offer our protein-based vaccine, Nuvaxovid, for use as a booster in adults regardless of previous vaccine history.

Vaccination 147

Vaccination Vaccine Immune Response Protein 147

Pharmaceutical Technology

SEPTEMBER 22, 2022

AVR-RD-04 works by genetically modify a patient's hematopoietic stem cells (HSCs) to express the gene that encodes the cystinosin protein. Cystinosis patients have a high deficiency of this protein. A rare and fatal ailment, cystinosis is characterised by cystine buildup in cellular organelles called lysosomes.

Gene Therapy 147

Gene Therapy Gene Protein Clinical Trials 147

pharmaphorum

SEPTEMBER 14, 2020

Merck & Co has signed a multi-billion cancer drug development deal, which will see it investing $1 billion in partner Seattle Genetics. US-based Merck, known as MSD outside North America, will globally develop and market Seattle’s investigational antibody-drug conjugate (ladiratuzumab vedotin). billion equity investment in 5.0

Genetics 52

Genetics Development Drugs Protein 52

Pharmaceutical Technology

JUNE 13, 2023

Ziftomenib targets the menin-KMT2a protein-protein interaction that may occur in patients with KMT2ar or NPM1m genetic alterations. The aberrant protein complex is essential for regulating leukaemia-promoting gene expression. Thus, targeting menin may be an effective strategy for patients with high unmet needs.

Gene Expression 130

Gene Expression Protein Sales Genetics 130

Roots Analysis

MAY 2, 2022

In the past few years, plants have been gaining a rapid traction, involving genetic modification and expression of new genes in plant host cells. Currently, a large number of plant-based proteins are being evaluated in clinical phases of development, including vaccine candidates against COVID 19 and multiple rare therapeutic disorders.

Protein 52

Protein Development Marketing Manufacturing 52

Outsourcing Pharma

JUNE 7, 2023

Duchenne Muscular Dystrophy (DMD) is a genetic disorder that primarily affects males, caused by a mutation in the gene that encodes for a protein called dystrophin. This protein is crucial for the normal functioning of muscles, and its absence leads to progressive muscle weakness and wasting.

Protein 82

Protein Genetics Gene Research 82

Roots Analysis

SEPTEMBER 9, 2024

Over the years, pharmaceutical companies have shifted their priorities from traditional interventions towards more advanced pharmacological strategies, such as protein therapeutics. Further, $400 billion is the anticipated sales of protein-based therapeutics in 2023. The mRNA is then translated into to form functional proteins.

Protein 40

Protein Gene Production DNA 40

The Pharma Data

JULY 8, 2021

Today, access to the world’s largest browsable resource linking rare protein-coding genetic variants to human health and disease was launched through a genetic exome sequence analysis collaboration between AbbVie (NYSE: ABBV), Biogen Inc. Nasdaq: BIIB) and Pfizer (NYSE: PFE). About the UK Biobank.

Genetics 52

Genetics Biobanking Protein Containment 52

pharmaphorum

APRIL 23, 2021

billion in milestone payments in the deal, which will focus on using small-molecule compounds to “fine tune” the effects of genetic medicines in the body. The approach relies on the use of drug responsive domains (DRDs) on the gene-editing medicine, which can be used to either increase or decrease protein expression.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

The Pharma Data

JULY 15, 2021

The access to the world’s largest browsable resource linking rare protein-coding genetic variants to human health and disease was launched through a genetic exome sequence analysis collaboration between AbbVie (NYSE: ABBV), Biogen Inc. Nasdaq: BIIB) and Pfizer (NYSE: PFE).

Biobanking 52

Biobanking Genetics Protein Medicine 52

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.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

pharmaphorum

NOVEMBER 7, 2022

This month, we highlight new appointments from Lumeris, Lineage Cell Therapeutics, 1910 Genetics, and ADC Therapeutics. . As we continue to enter new markets, grow within our existing markets and reach a broader population, we continue to invest in our operations, talent and technology capabilities to support that growth.”

Life Science 105

Life Science Genetics Business Development Marketing 105

Pharmaceutical Technology

MAY 25, 2023

DMD is a rare genetic disorder caused by changes to the dystrophin protein, leading to progressive muscular degeneration. The FDA has told Sarepta that in this additional timeframe, it will complete final label negotiations and work out post-marketing commitments.

Gene Therapy 130

Gene Therapy Gene Clinical Trials Clinical Trials 130

pharmaphorum

OCTOBER 2, 2020

The rolling review, which is designed to speed up the assessment of a promising vaccine or drug by the EMA’s CHMP scientific committee, will continue until there is enough evidence available to support a marketing authorisation. But this does not mean that the CHMP is ready to make a conclusion on the safety and efficacy of the vaccine.

Regulation 105

Regulation Vaccination Vaccine Protein 105

Worldwide Clinical Trials

MAY 30, 2024

Since marketing authorization for the first breakthrough treatment in 1994, the steady increase in clinical trials reflects the community’s commitment to finding effective ALS treatments despite the numerous hurdles associated with clinical trial design, from proof-of-concept to pivotal trials. Director, Therapeutic Area Medical Lead.

Gene 204

Gene Clinical Trials Clinical Trials Gene Therapy 204

Pharmaceutical Technology

MARCH 1, 2023

“By combining Tevard’s ability to restore the production of critical proteins with Vertex’s clinical, regulatory, and manufacturing expertise, we hope to make an important difference for patients and their families.” The company is pioneering tRNA-based therapeutics for modulating mRNA function and curing several genetic diseases.

Development 130

Development Gene Therapy Genetics Genetic Disease 130

pharmaphorum

FEBRUARY 16, 2021

Inflammasomes are a group of intracellular proteins associated with the inflammatory response, and prior research has linked the NLRP3 inflammasome to the aggregation of amyloid beta and tau protein – which together form the characteristic plaques and tangles in the brains of AD patients.

Research 119

Research Drugs Protein Development 119

XTalks

FEBRUARY 19, 2025

It occurs when a genetic error causes the overproduction of a protein called CSF1, which attracts inflammatory cells to the joint, often causing pain, swelling and reduced mobility. In 2024, Deciphera was acquired by ONO Pharmaceutical for $2.4 TGCT is a rare, non-cancerous tumor that develops inside or near joints.

FDA Approval 59

FDA Approval Drugs Trials Clinical Trials 59

Roots Analysis

JANUARY 19, 2024

Introduction In a strategic move to accelerate the development and launch of genetic medicines, BridgeBio Pharma has successfully secured up to USD 1.25 ATTR is an inherited disease causing abnormal transthyretin protein and the accumulation of amyloid protein in various tissues, including the heart.

Genetics 40

Genetics Medicine Development Protein 40

XTalks

MARCH 7, 2024

Industry professionals will explore the market, innovative formulations and nutritional solutions tailored to empower women toward optimal health and well-being. The test accurately identified a range of genetic abnormalities, including trisomies and microdeletions.

Genetics 115

Genetics Hormones Genomics Genome 115

pharmaphorum

OCTOBER 4, 2020

CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein. ALD protein is needed to clear toxic molecules called very long-chain fatty acids (VLCFAs) in the brain, and if mutated causes the VLCFAs to accumulate and damage the myelin sheath.

Gene Therapy 98

Gene Therapy Gene Genetic Disease Protein 98

XTalks

JANUARY 4, 2024

Hemophilia B, also known as congenital Factor IX (FIX) deficiency or Christmas disease, is a rare genetic bleeding disorder caused by a deficiency or defect in the FIX clotting protein. Treatment involves replacing the missing FIX clotting protein, traditionally through regular infusions.

Gene Therapy 111

Gene Therapy Gene Gene Editing Sales 111

XTalks

JULY 22, 2021

Impossible Foods announced that they would enter the vegan chicken market later this year by introducing their own plant-based Impossible Chicken Nuggets. This already crowded market will now include more competition with the entry of Impossible Foods. In 2021, the global poultry market is predicted to grow at a CAGR of 3.8

Marketing 98

Marketing Production Genetics Branding 98

Pharmaceutical Technology

FEBRUARY 15, 2023

Innovation S-curve for the pharmaceutical industry Gene splicing using nucleases is a key innovation area in the pharmaceutical industry Nucleases play a fundamental role in the field of recombinant DNA technology, or genetic engineering. Gene splicing using nucleases is used to design gene therapeutics for various genetic disorders.

Gene Splicing 130

Gene Splicing Gene DNA Recombinant DNA Technology 130

Delveinsight

JULY 23, 2020

The current Familial chylomicronemia syndrome market is mainly dominated by traditional TG-lowering agents, and as per DelveInsight analysis, the market worth was estimated to be USD 1.0 Familial Chylomicronemia Syndrome Market. Similar is the case with Japan FCS market. million in 2017. 2017, Stroes et al.

Marketing 61

Marketing Gene Therapy Drugs Genomics 61

XTalks

JUNE 5, 2023

Abrysvo is an unadjuvanted vaccine and is composed of two preF proteins selected to optimize protection against RSV A and B strains. “A In February 2023, Pfizer revealed that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for Abrysvo. How Does Abrysvo Work? billion by 2028.

Vaccination 98

Vaccination Vaccine FDA Approval Protein 98

STAT News

NOVEMBER 23, 2022

The one-time treatment, called Hemgenix, was developed by the Dutch biotech company UniQure and will be marketed by CSL Behring, an Australian pharmaceutical company. Hemgenix will cost $3.5 million, making it the most expensive drug approved to date. Continue to STAT+ to read the full story…

Gene Therapy 97

Gene Therapy FDA Approval Gene Genetics 97

pharmaphorum

JULY 22, 2021

The EMA has cleared Skysona (elivaldogene autotemcel) for use in CALD patients aged under 18 who have an ABCD1 genetic mutation and no matched sibling donor who could provide a stem cell transplant. The modified cells are reinfused and can produce a functional version of the ALD protein that is lacking in CALD.

Gene Therapy 98

Gene Therapy Gene Trials Marketing 98

Roots Analysis

MARCH 16, 2023

It is important in the process of protein synthesis because mRNA is responsible for transferring genetic information from DNA to ribosomes, which then decodes the genetic information into a protein. What are the key trends that are shaping up the mRNA therapeutics and vaccines market?

Vaccination 52

Vaccination Vaccine DNA Protein 52