Drug Discovery World

FEBRUARY 27, 2024

Arvinas has commenced dosing in the Phase I clinical trial of ARV-102, its first oral PROTAC (PROteolysis-TArgeting Chimera) protein degrader in development to treat neurodegenerative diseases. The post First PROTAC degrader for neurodegenerative disease enters trials appeared first on Drug Discovery World (DDW).

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Drug Discovery World

FEBRUARY 28, 2024

The first 20 participants have been enrolled in a multicentre trial to test whether ImmunityBio’s Nant Cancer Vaccine (NCV) could potentially prevent colon and other cancers in individuals with Lynch syndrome. Each of the three vaccines in Tri-Ad5 targets different proteins associated with precancer and cancer cells.

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pharmaphorum

MARCH 26, 2021

There’s a new kid on the block among companies using artificial intelligence in drug discovery, after 1910 Genetics launched today with $26 million in financing. 1910 Genetics is named after the year in which sickle cell disease (SCD) was first discovered in the US by James Herrick. Dr Jen Nwankwo.

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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.

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Pharmaceutical Technology

JUNE 24, 2022

The protein-based vaccine is engineered from the genetic sequence of the SARS-CoV-2 virus’ initial strain. Developed utilising Novavax’s recombinant nanoparticle technology to generate antigen obtained from the coronavirus spike (S) protein, the vaccine is also formulated with the company’s saponin-based Matrix-M adjuvant.

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XTalks

DECEMBER 12, 2023

The world of neuroscience trials is rapidly evolving, presenting new challenges and opportunities for researchers and clinicians. The worldwide market for neurology clinical trials stood at a valuation of $5.24 There is currently a heightened emphasis on imaging and liquid biomarkers in neuroscience clinical trials.

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Drug Discovery World

AUGUST 16, 2023

A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy. The presence of these biomarkers could be used to accelerate the development of more personalised treatments for triple negative breast cancers.

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Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . The disease is caused by anomalous repeating mutations in the huntingtin gene leading to abnormal protein aggregates in nerve cells.

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XTalks

JULY 19, 2022

The two-dose protein-based, adjuvanted vaccine has been authorized for use in adults 18 years of age and older for the prevention of COVID-19 caused by SARS-CoV-2. In a press release outlining the authorization, the FDA noted that the clinical trial for Novavax’s vaccine was conducted before the appearance of the omicron and delta variants.

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Scienmag

JUNE 1, 2021

Researchers world-wide are focused on clearing the toxic mutant Huntingtin protein that leads to neuronal cell death and systemic dysfunction in Huntington’s disease (HD), a devastating, incurable, progressive neurodegenerative genetic disorder.

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FDA Law Blog

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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Pharmaceutical Technology

JULY 6, 2022

A protein-based vaccine, NVX-CoV2373 is made from the genetic sequence of the SARS-CoV-2 virus’ first strain. The CMA was based on findings from the paediatric expansion of Phase III PREVENT-19 clinical trial of 2,247 adolescents of the age 12 to 17 years at 73 sites in the US.

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Drug Discovery World

JULY 6, 2023

The drug candidate, a monoclonal antibody (mAb) targeting the protein Semaphorin-3A (Sema3A), is being developed as a potential first-to-market treatment for Alport syndrome, a rare genetic kidney disease. Semaphorin-3A is an extracellular guidance protein and a well-known regulator of the actin cytoskeleton.

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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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Pharmaceutical Technology

FEBRUARY 22, 2023

Taldefgrobep is a complete human anti-myostatin recombinant protein that is developed for lowering free myostatin. Currently, the company is enrolling participants in a Phase III clinical trial of taldefgrobep to treat SMA. It also works as a receptor antagonist to block myostatin signalling in skeletal muscles.

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pharmaphorum

AUGUST 23, 2022

It is a different approach to that taken by uniQure with its AAV-based gene therapy AMT-130, which is designed to silence the huntingtin (Htt) gene, which when mutated produces a protein thought to be toxic to neurons. “Importantly, BV-101 does not affect the levels of normal huntingtin protein in cells,” she added. .

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XTalks

FEBRUARY 19, 2024

The team used the data and patient samples from a gB/MF59 Phase II trial in adolescent girls as a benchmark to assess the new mRNA-based vaccine. The comparison is particularly important as gB/MF59 demonstrated promise in Phase II clinical trials but did not advance to Phase III.

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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.

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The Pharma Data

MARCH 23, 2022

Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. The researchers discovered that Ebola virus polymerase hijacks a cellular protein called GSPT1. To treat Ebola virus infections, researchers are taking a close look at a key piece of the virus: polymerase.

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Pharmaceutical Technology

APRIL 26, 2023

Qalsody is an antisense oligonucleotide (ASO) that has been designed for binding to SOD1 mRNA to reduce the production of SOD1 protein. It is reported to be the first treatment targeting a genetic cause of ALS to receive approval. Continued approval for this indication may depend on the verification of clinical benefits in trials.

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pharmaphorum

SEPTEMBER 14, 2020

AstraZeneca has resumed UK trials for its coronavirus vaccine, after the country’s medicines regulator gave the all-clear following a safety scare. A UK safety committee has concluded its investigations and recommended to the country’s Medicines and Healthcare products Regulatory Agency (MHRA) that trials are safe to resume.

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pharmaphorum

JUNE 16, 2022

The litany of failed trials of amyloid-targeting drugs for Alzheimer’s disease has added another verse, as Roche concedes that its crenezumab candidate was unable to slow cognitive decline in patients with an inherited form of the disease. It was sponsored by Roche, the National Institute on Aging and the Banner Alzheimer’s Foundation.

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Pharmaceutical Technology

MAY 9, 2023

Koselugo is a mitogen-activated protein kinase (MEK) inhibitor that works by blocking the enzymes MEK1 and MEK2 that are involved in stimulating cell growth. The regulatory approval was based on data obtained from the SPRINT Stratum 1 trial, backed by the National Cancer Institute’s (NCI) cancer therapy evaluation programme (CTEP).

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Drug Discovery World

DECEMBER 9, 2022

A new type of targeted medicine has shown ‘remarkable’ benefits for patients with advanced breast cancer in a major Phase III clinical trial. . Capivasertib is a potential first-in-class drug that blocks activity of the cancer-driving protein molecule AKT. The CAPItello-291 trial. Genetic alterations of the AKT pathway.

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Camargo

JULY 27, 2021

The technology of gene therapy is possible due to extensive DNA research and our resulting understanding of many diseases on the genetic level, and it encompasses several mechanisms such as introducing new genes (gene addition) and inactivating or replacing mutated genes (gene editing). Formulation Considerations.

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Drug Discovery World

JUNE 23, 2023

Elevidys addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as Elevidys micro-dystrophin.

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Drug Discovery World

FEBRUARY 21, 2024

In October 2023, pharmaceutical company NKGen Biotech released data from its Phase I clinical trial on the use of its investigational natural killer therapy, SNK01, to treat patients with Alzheimer’s disease. This is done without any genetic modification. RA: What are some of the unmet needs in current treatments for Alzheimer’s?

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The Pharma Data

SEPTEMBER 14, 2020

. AstraZeneca has confirmed that clinical trials assessing its Oxford University partnered coronavirus vaccine AZD1222 have resumed in the UK, following a green light by the Medicines Health Regulatory Authority (MHRA). Source link.

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Pharmaceutical Technology

AUGUST 11, 2022

In a Pompe mouse disease model, ABX1100 was demonstrated to greatly reduce the Gys1 mRNA and GYS1 protein, causing a meaningful decline in glycogen levels in the skeletal muscle. The company plans to advance ABX1100 into clinical trials in the middle of next year.

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Advarra

JANUARY 11, 2024

Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials. One strategy proposed the use of synthetic mRNA or modified mRNA analogs, which can enhance the stability and protein expression of mRNA.

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XTalks

NOVEMBER 6, 2020

Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinical trials this week. The first healthy volunteer was dosed with the vaccine in Australia as part of the bacTRL-Spike COVID-19 Phase I clinical trial. COVID-19 Clinical Trials.

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Pharmaceutical Technology

JULY 27, 2022

It is the first protein-based vaccine to be offered in Australia for adolescents aged 12 to 17 years. The approval is based on findings from the paediatric expansion of the Phase III PREVENT-19 clinical trial of 2,247 adolescents of this age group at 73 sites in the US. Biocelect is the sponsor of Novavax in Australia.

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The Pharma Data

DECEMBER 3, 2020

New Tau Protein is Abnormal in Very Early Alzheimer’s Disease. Investigators with the University of Gothenburg identified new forms of tau protein that become abnormal in the very early stages of Alzheimer’s disease, even before the development of cognitive problems. “The Every week there are numerous scientific studies published.

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Bioengineer

JANUARY 16, 2024

This mutation hinders the drug’s binding to the mutant EGFR protein. Osimertinib, however, can covalently bind to the T790M and cysteine-797 (C797) residue at the protein’s ATP binding site, overcoming resistance mechanisms. Real-world cohorts report a prevalence of C797 mutations between 11% and 29%.

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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. Additionally, no adverse events (AEs) linked to the drug have been reported so far. .

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Pharmaceutical Technology

JULY 5, 2022

Researchers at the UNC School of Medicine have discovered that hepatitis A virus (HAV) replication needs particular interactions between the human protein ZCCHC14 and TENT4 poly(A) polymerases, a group of enzymes. In addition, the scientists later found that the HAV needs TENT4A/B for its replication.

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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 also said that a non-age-restricted expansion of the delandistrogene moxeparvovec label could be possible, depending on meeting objectives in the Phase III Embark clinical trial (NCT05096221).

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