Bioavailability and Gene - Clinical Research Informer

Orphagen’s ACC therapy receives FDA rare pediatric disease status

Pharmaceutical Technology

JANUARY 17, 2023

OR-449 is a first-in-class, orally bioavailable, potent and selective small molecule antagonist of the orphan nuclear receptor steroidogenic factor-1 (SF-1, NR5A1). In paediatric ACC, the transcription factor is commonly amplified at the gene level. SF-1 is an important transcription factor for adrenal gland growth and development.

Bioavailability

Bioavailability Clinical Trials Clinical Trials Clinical Development

Erasca receives fast track designation for glioblastoma therapy

Pharmaceutical Technology

MAY 2, 2023

Erasca has received the US Food and Drug Administration’s (FDA) fast track designation (FTD) for ERAS-801 to treat adults with glioblastoma (GBM) with epidermal growth factor receptor (EGFR) gene alterations.

Bioavailability

Bioavailability Clinical Development Gene Trials

Vevye: A New Cyclosporine Solution for Dry Eye Disease

XTalks

JUNE 14, 2023

Furthermore, the bioavailability of the active pharmaceutical ingredient (API) into the cornea is up to four times stronger with EyeSol technology. Tivanisiran is a preservative-free eye drop that uses RNAi technology, specifically gene silencing, to target and control the signs and symptoms of dry eye disease.

Gene Silencing

Gene Silencing Drug Delivery FDA Approval RNA

Cell-penetrating peptides as a delivery system for oligonucleotides

Drug Discovery World

AUGUST 3, 2022

Oligonucleotides act on the RNA level through different molecular pathways, but there is a major drawback when using them as drugs: their poor bioavailability and cellular uptake. Ensuring that the CPP can escape this endosomal compartment is crucial for its bioavailability and bioactivity properties. cells, such as diabetes.

Bioavailability

Bioavailability In-Vivo RNA In-Vitro

In the News: June 2021 Regulatory and Development Updates

Camargo

JULY 8, 2021

The UCLA researchers measured monoamine oxidase A (MAOA) gene expression in tumors from cancer patients and found that high MAOA expression was associated with shorter survival times and turned to a mouse model to investigate these observations with promising results.

Development

Development Bioequivalency Gene Expression Drugs

Orserdu (elacestrant) Approved for Patients with ESR1 mutations in ER+/HER2- Advanced or Metastatic Breast Cancers

XTalks

FEBRUARY 3, 2023

In addition, about 40 percent of these cases involve mutations in the ESR1 gene. Unlike fulvestrant, elacestrant is not only orally bioavailable, but it has also demonstrated longer steady-states and works as a better combination partner with inhibitor agents like CDK4/6.

Clinical Trials

Clinical Trials Clinical Trials FDA Approval Trials

Nanoparticle Formulation: A Paradigm Shift towards Novel Drug Delivery

Roots Analysis

OCTOBER 2, 2023

Owing to their unique size and physicochemical properties ( surface roughness, surface area, surface energy, crystal structure and shape ), nanoparticles can be widely used as a contrasting agent in medical imaging, a vesicle to cross the blood-brain barrier and a carrier for targeted delivery of genes / drugs, proteins, vaccines and antibiotics.

Drug Delivery

Drug Delivery Drugs Bioavailability Marketing

The Power of Lipids: Enhancing Drug Delivery with Nanoparticles

Roots Analysis

MAY 21, 2024

In fact, it has been observed that around 40% of the pharmaceutical drugs approved by regulatory organizations exhibit poor bioavailability / solubility. Further, every year, poor bioavailability is one of the main causes of drug failure in obtaining approval authorization.

Drug Delivery

Drug Delivery Drugs Gene Therapy Bioavailability

Biopharmaceutical Excipient Manufacturing – Current Scenario and Future Trends

Roots Analysis

MARCH 13, 2022

In the last decade alone, the annual number of approvals of biopharmaceuticals (including monoclonal antibodies, recombinant proteins, vaccines and gene therapies), by the US FDA, have steadily risen. Over the years, the rising popularity of biologics has led to a paradigm shift in the healthcare industry.

Manufacturing

Manufacturing Drug Delivery Marketing Drug Delivery Systems

A Complex Chromosomal Abberation Called Adrenoleukodystrophy

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. A novel, brain penetrant, orally bioavailable and selective PPAR?

Hormones

Hormones Marketing Gene Trials

Considerations for Neuroscience Trials with Intrathecal Delivery and Other Methods of Direct CNS Administration

XTalks

AUGUST 30, 2022

We know that a lot of different pathologies, especially as we’ve entered the era of molecular medicine and gene therapies, cause many of the same symptoms and work through final common pathways. Viral Vectors for Gene Delivery to the CNS. Reporter genes to understand dose and distribution. The patients are waiting!

Trials

Trials Drug Delivery Drugs Gene

Lilly adds to pain pipeline with Asahi Kasei licensing deal

pharmaphorum

JANUARY 29, 2021

While tanezumab is an antibody and has to be delivered by subcutaneous injection, AK1780 is orally bioavailable. Lilly and Asahi Kaesi’s interest in developing AK1780 for pain stems from animal studies showing that knocking out the gene for P2X7 receptors in animal models seemed to abolish the pain response.

Licensing

Licensing Licensing Bioavailability Antibody

Fibrosis research: Advances and challenges

Drug Discovery World

JANUARY 24, 2024

PW: Through our exclusive access to one of the world’s largest fibrosis tissue biobanks, we have correlated disease progression to gene expression data, enabling us to discover novel targets on the pathway that drives the advanced stages of fibrotic disease. The gene expression data were generated by RNAseq on biopsies stored at -78°C.

Research

Research Gene Expression Drugs Development

Monday 17 April at AACR Annual Meeting 2023

Drug Discovery World

APRIL 17, 2023

AACR-Cancer Research Institute Lloyd J. Old Award in Cancer Immunology Another award highlight was the AACR-Cancer Research Institute Lloyd J. Old Award in Cancer Immunology.

Research

Research Antibody Bioavailability Genetics

Clinical Research Informer

Orphagen’s ACC therapy receives FDA rare pediatric disease status

Erasca receives fast track designation for glioblastoma therapy

Trending Sources

Vevye: A New Cyclosporine Solution for Dry Eye Disease

Cell-penetrating peptides as a delivery system for oligonucleotides

In the News: June 2021 Regulatory and Development Updates

Orserdu (elacestrant) Approved for Patients with ESR1 mutations in ER+/HER2- Advanced or Metastatic Breast Cancers

Nanoparticle Formulation: A Paradigm Shift towards Novel Drug Delivery

The Power of Lipids: Enhancing Drug Delivery with Nanoparticles

Biopharmaceutical Excipient Manufacturing – Current Scenario and Future Trends

A Complex Chromosomal Abberation Called Adrenoleukodystrophy

Considerations for Neuroscience Trials with Intrathecal Delivery and Other Methods of Direct CNS Administration

Lilly adds to pain pipeline with Asahi Kasei licensing deal

Fibrosis research: Advances and challenges

Monday 17 April at AACR Annual Meeting 2023

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